A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol Cyclo (HP-Beta-CD) in Patients With Niemann-Pick ... View Homepage


Ontology type: schema:MedicalStudy     


Clinical Trial Info

YEARS

2017-2019

ABSTRACT

This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 2 different dose levels and what effects it has on people who have this condition. NPC-1 is caused by a defect in the protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in which this experimental treatment works is not fully understood but laboratory experiments have shown that it can potentially remove cholesterol build up from the cells in people who have NPC-1. Approximately 12 patients will be asked to take part in this research study for up to 20 weeks (w) in total (including screening. treatment and follow-up). Recruitment is expected to take 6- 9 months.Patients who take part will receive treatment by an intravenous infusion every two weeks. The study will look at what the body does to the drug as well as what the drug does to the body by taking and examining blood and urine samples. Samples of cerebrospinal fluid (CSF) will be taken by lumbar puncture during and following the first and subsequent treatment doses. Liver and skin biopsy specimens will be taken to assess filipin staining. Cholesterol metabolism will be investigated in liver samples and splenic and hepatic elasticity will be assessed by ultrasound..Patients will also have their hearing tested, be asked questions by their doctor as well completing questionnaires to help assess any changes in their condition during treatment.This study is being sponsored and funded by CTD holdings Inc. It is planned to be run in the USA,. Detailed Description The planned study has been designed as a Phase I, double-blind, randomised, single-centre, parallel group study based on information and data available from the administration of Trappsol Cyclo via compassionate/named patient use in patients with NPC-1, and data on other cyclodextrin products in the scientific literature. The study is comprised of a screening phase of up to 4w a treatment phase of 12w and a 4w follow-up The primary objective is to compare the plasma pharmacokinetics of single and multiple doses of two different levels of IV Trappsol Cyclo .Secondary objectives include investigation of the HP-β-CD effect of different doses of IV Trappsol Cyclo upon serum and lymphocytic markers of cholesterol metabolism and evaluation of Trappsol concentrations in the cerebrospinal fluid (CSF) following IV administration , evaluation of the impact of treatment upon measures of neurological function including ataxia, aphasia and saccadic eye movements, and the impact of treatment upon behavioral aspects of NPC-1. It is planned to recruit a total of 12 patients to the study. Patients will be randomised 1:1 to one of the two dose levels (1500 mg/kg or 2500 mg/kg; six patients per dose level). Treatment will be administered every two weeks by slow IV infusion over 8 hours at different concentrations to achieve the proscribed dose levels. Patients will receive treatment for a total of 12 weeks. Patients who withdraw prior to completion of the initial pharmacokinetic and pharmacodynamic assessments will be replaced. The design of the proposed study thus enables early assessment of potential biochemical markers of response but allows for a sufficient dosing duration to enable the short-term effectiveness of Trappsol in NPC to be assessed. The maximum dose proposed for this study is below the maximum dose for which long term clinical data is available in 2 patients (2800 mg/kg weekly for 3-5 years). Although individual clinicians have not always utilized an escalating rate of infusion, the reports of infusion related reactions in three patients suggest that this is an appropriate clinical strategy to mitigate the risk of such events and is consistent with dosing administration for other therapeutic agents. In the proposed study, treatment will be administered less frequently than has been undertaken in compassionate use. This longer dosing interval is supported by nonclinical data comparing the metabolism of cholesterol in non-human species with that in man; although a once weekly dosing interval was initially studied in man based on data in the mouse, HP-β-CD cholesterol metabolism/turnover in the mouse is 13-fold higher than in man which, in NPC, likely translates into a 13-fold slower accumulation of cholesterol in human cells compared with those of the mouse.Therefore, it is theorized that, given the slower cholesterol metabolism in humans, the dosing interval could be much less frequent in man than in mouse; however, based on what is known about cholesterol metabolism in humans and the pharmacokinetic and pharmacodynamic effect of HP-β-CD in the mouse, a dosing interval of 2 weeks in man is likely to be well within the therapeutic dosing interval and also minimizes the amount of infusions required to be administered. More... »

URL

https://clinicaltrials.gov/show/NCT02939547

Related SciGraph Publications

JSON-LD is the canonical representation for SciGraph data.

TIP: You can open this SciGraph record using an external JSON-LD service: JSON-LD Playground Google SDTT

[
  {
    "@context": "https://springernature.github.io/scigraph/jsonld/sgcontext.json", 
    "about": [
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/3164", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "type": "DefinedTerm"
      }, 
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/3053", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "type": "DefinedTerm"
      }
    ], 
    "description": "This research study is being conducted to find out whether Trappsol\u00ae Cyclo\u2122, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 2 different dose levels and what effects it has on people who have this condition. NPC-1 is caused by a defect in the protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in which this experimental treatment works is not fully understood but laboratory experiments have shown that it can potentially remove cholesterol build up from the cells in people who have NPC-1. Approximately 12 patients will be asked to take part in this research study for up to 20 weeks (w) in total (including screening. treatment and follow-up). Recruitment is expected to take 6- 9 months.Patients who take part will receive treatment by an intravenous infusion every two weeks. The study will look at what the body does to the drug as well as what the drug does to the body by taking and examining blood and urine samples. Samples of cerebrospinal fluid (CSF) will be taken by lumbar puncture during and following the first and subsequent treatment doses. Liver and skin biopsy specimens will be taken to assess filipin staining. Cholesterol metabolism will be investigated in liver samples and splenic and hepatic elasticity will be assessed by ultrasound..Patients will also have their hearing tested, be asked questions by their doctor as well completing questionnaires to help assess any changes in their condition during treatment.This study is being sponsored and funded by CTD holdings Inc. It is planned to be run in the USA,.\n\nDetailed Description\nThe planned study has been designed as a Phase I, double-blind, randomised, single-centre, parallel group study based on information and data available from the administration of Trappsol Cyclo via compassionate/named patient use in patients with NPC-1, and data on other cyclodextrin products in the scientific literature. The study is comprised of a screening phase of up to 4w a treatment phase of 12w and a 4w follow-up The primary objective is to compare the plasma pharmacokinetics of single and multiple doses of two different levels of IV Trappsol Cyclo .Secondary objectives include investigation of the HP-\u03b2-CD effect of different doses of IV Trappsol Cyclo upon serum and lymphocytic markers of cholesterol metabolism and evaluation of Trappsol concentrations in the cerebrospinal fluid (CSF) following IV administration , evaluation of the impact of treatment upon measures of neurological function including ataxia, aphasia and saccadic eye movements, and the impact of treatment upon behavioral aspects of NPC-1. It is planned to recruit a total of 12 patients to the study. Patients will be randomised 1:1 to one of the two dose levels (1500 mg/kg or 2500 mg/kg; six patients per dose level). Treatment will be administered every two weeks by slow IV infusion over 8 hours at different concentrations to achieve the proscribed dose levels. Patients will receive treatment for a total of 12 weeks. Patients who withdraw prior to completion of the initial pharmacokinetic and pharmacodynamic assessments will be replaced. The design of the proposed study thus enables early assessment of potential biochemical markers of response but allows for a sufficient dosing duration to enable the short-term effectiveness of Trappsol in NPC to be assessed. The maximum dose proposed for this study is below the maximum dose for which long term clinical data is available in 2 patients (2800 mg/kg weekly for 3-5 years). Although individual clinicians have not always utilized an escalating rate of infusion, the reports of infusion related reactions in three patients suggest that this is an appropriate clinical strategy to mitigate the risk of such events and is consistent with dosing administration for other therapeutic agents. In the proposed study, treatment will be administered less frequently than has been undertaken in compassionate use. This longer dosing interval is supported by nonclinical data comparing the metabolism of cholesterol in non-human species with that in man; although a once weekly dosing interval was initially studied in man based on data in the mouse, HP-\u03b2-CD cholesterol metabolism/turnover in the mouse is 13-fold higher than in man which, in NPC, likely translates into a 13-fold slower accumulation of cholesterol in human cells compared with those of the mouse.Therefore, it is theorized that, given the slower cholesterol metabolism in humans, the dosing interval could be much less frequent in man than in mouse; however, based on what is known about cholesterol metabolism in humans and the pharmacokinetic and pharmacodynamic effect of HP-\u03b2-CD in the mouse, a dosing interval of 2 weeks in man is likely to be well within the therapeutic dosing interval and also minimizes the amount of infusions required to be administered.", 
    "endDate": "2019-02-01T00:00:00Z", 
    "id": "sg:clinicaltrial.NCT02939547", 
    "keywords": [
      "Phase I study", 
      "Evaluate", 
      "multiple dose", 
      "cyclo", 
      "beta", 
      "Niemann-Pick disease", 
      "dosing", 
      "NPC disease", 
      "research study", 
      "experimental treatment", 
      "Niemann Pick", 
      "disease type", 
      "different dose level", 
      "condition", 
      "defect", 
      "protein", 
      "transport", 
      "substance", 
      "cholesterol", 
      "cell", 
      "organ damage", 
      "laboratory experiment", 
      "patient", 
      "Mass Screening", 
      "recruitment", 
      "intravenous infusion", 
      "body", 
      "drug", 
      "blood", 
      "urine sample", 
      "sample", 
      "cerebrospinal fluid", 
      "Spinal Puncture", 
      "subsequent treatment", 
      "liver", 
      "biopsy specimen", 
      "cholesterol metabolism", 
      "liver sample", 
      "elasticity", 
      "ultrasound", 
      "hearing", 
      "doctor", 
      "questionnaire", 
      "holding", 
      "USA", 
      "planned study", 
      "parallel group study", 
      "Organization and Administration", 
      "patient use", 
      "cyclodextrins", 
      "scientific literature", 
      "treatment phase", 
      "primary objective", 
      "plasma pharmacokinetics", 
      "different level", 
      "secondary objective", 
      "HP", 
      "different dose", 
      "serum", 
      "marker", 
      "Evaluation Study as Topic", 
      "neurological function", 
      "ataxia", 
      "aphasia", 
      "saccade", 
      "behavioral aspect", 
      "dose level", 
      "infusion", 
      "different concentration", 
      "completion", 
      "pharmacokinetics", 
      "early assessment", 
      "duration", 
      "short-term effectiveness", 
      "NPC", 
      "maximum dose", 
      "clinical data", 
      "individual", 
      "escalating", 
      "related reaction", 
      "clinical strategy", 
      "risk", 
      "therapeutic agent", 
      "trial", 
      "interval", 
      "metabolism", 
      "non-human specie", 
      "human", 
      "mouse", 
      "translates", 
      "accumulation", 
      "human cell", 
      "pharmacodynamic effect", 
      "therapeutic", 
      "minimizes"
    ], 
    "name": "A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol Cyclo (HP-Beta-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease", 
    "sameAs": [
      "https://app.dimensions.ai/details/clinical_trial/NCT02939547"
    ], 
    "sdDataset": "clinical_trials", 
    "sdDatePublished": "2019-03-07T15:26", 
    "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
    "sdPublisher": {
      "name": "Springer Nature - SN SciGraph project", 
      "type": "Organization"
    }, 
    "sdSource": "file:///pack/app/us_ct_data_00022.json", 
    "sponsor": [
      {
        "id": "https://www.grid.ac/institutes/grid.414016.6", 
        "type": "Organization"
      }
    ], 
    "startDate": "2017-10-01T00:00:00Z", 
    "subjectOf": [
      {
        "id": "https://doi.org/10.1179/jmt.2009.17.3.163", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1002816074"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1186/1750-1172-5-16", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1005623887", 
          "https://doi.org/10.1186/1750-1172-5-16"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1186/s13023-015-0274-1", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1005979361", 
          "https://doi.org/10.1186/s13023-015-0274-1"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/humu.10255", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1006616197"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mds.10541", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1006635563"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1194/jlr.m700525-jlr200", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012167280"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1074/jbc.271.35.21604", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012367026"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/scitranslmed.3010101", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012551405"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1194/jlr.m013789", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1014481242"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.7863/ultra.33.2.197", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1018909132"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1242/dmm.010124", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1019513430"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s1474-4422(07)70194-1", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1021955671"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1007/s10545-012-9583-x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1022369447", 
          "https://doi.org/10.1007/s10545-012-9583-x"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1007/s10545-012-9583-x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1022369447", 
          "https://doi.org/10.1007/s10545-012-9583-x"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1172/jci72835", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1023308017"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/j.ymgme.2012.03.012", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1027225530"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/0022-3956(75)90026-6", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1027523106"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1007/8904_2011_47", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1035654877", 
          "https://doi.org/10.1007/8904_2011_47"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1194/jlr.p013524", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037061331"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1371/journal.pone.0114669", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037474842"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1136/jnnp.2005.086785", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037485737"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1073/pnas.0810895106", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1039042817"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1097/aud.0b013e3182a362b8", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1044245566"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1074/jbc.r100057200", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1048401077"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1001/jama.282.12.1157", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1050564301"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1371/journal.pone.0006951", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1050731206"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/j.fct.2005.03.007", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1052037868"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1093/brain/awl260", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1052588466"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1086/302620", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1058610189"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1126/science.290.5500.2298", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1062572556"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/01.wnl.0000219042.60538.92", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064348165"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e31828869f9", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064357619"
        ], 
        "type": "CreativeWork"
      }
    ], 
    "type": "MedicalStudy", 
    "url": "https://clinicaltrials.gov/show/NCT02939547"
  }
]
 

Download the RDF metadata as:  json-ld nt turtle xml License info

HOW TO GET THIS DATA PROGRAMMATICALLY:

JSON-LD is a popular format for linked data which is fully compatible with JSON.

curl -H 'Accept: application/ld+json' 'https://scigraph.springernature.com/clinicaltrial.NCT02939547'

N-Triples is a line-based linked data format ideal for batch operations.

curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/clinicaltrial.NCT02939547'

Turtle is a human-readable linked data format.

curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/clinicaltrial.NCT02939547'

RDF/XML is a standard XML format for linked data.

curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/clinicaltrial.NCT02939547'


 

This table displays all metadata directly associated to this object as RDF triples.

214 TRIPLES      16 PREDICATES      142 URIs      104 LITERALS      1 BLANK NODES

Subject Predicate Object
1 sg:clinicaltrial.NCT02939547 schema:about anzsrc-for:3053
2 anzsrc-for:3164
3 schema:description This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 2 different dose levels and what effects it has on people who have this condition. NPC-1 is caused by a defect in the protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in which this experimental treatment works is not fully understood but laboratory experiments have shown that it can potentially remove cholesterol build up from the cells in people who have NPC-1. Approximately 12 patients will be asked to take part in this research study for up to 20 weeks (w) in total (including screening. treatment and follow-up). Recruitment is expected to take 6- 9 months.Patients who take part will receive treatment by an intravenous infusion every two weeks. The study will look at what the body does to the drug as well as what the drug does to the body by taking and examining blood and urine samples. Samples of cerebrospinal fluid (CSF) will be taken by lumbar puncture during and following the first and subsequent treatment doses. Liver and skin biopsy specimens will be taken to assess filipin staining. Cholesterol metabolism will be investigated in liver samples and splenic and hepatic elasticity will be assessed by ultrasound..Patients will also have their hearing tested, be asked questions by their doctor as well completing questionnaires to help assess any changes in their condition during treatment.This study is being sponsored and funded by CTD holdings Inc. It is planned to be run in the USA,. Detailed Description The planned study has been designed as a Phase I, double-blind, randomised, single-centre, parallel group study based on information and data available from the administration of Trappsol Cyclo via compassionate/named patient use in patients with NPC-1, and data on other cyclodextrin products in the scientific literature. The study is comprised of a screening phase of up to 4w a treatment phase of 12w and a 4w follow-up The primary objective is to compare the plasma pharmacokinetics of single and multiple doses of two different levels of IV Trappsol Cyclo .Secondary objectives include investigation of the HP-β-CD effect of different doses of IV Trappsol Cyclo upon serum and lymphocytic markers of cholesterol metabolism and evaluation of Trappsol concentrations in the cerebrospinal fluid (CSF) following IV administration , evaluation of the impact of treatment upon measures of neurological function including ataxia, aphasia and saccadic eye movements, and the impact of treatment upon behavioral aspects of NPC-1. It is planned to recruit a total of 12 patients to the study. Patients will be randomised 1:1 to one of the two dose levels (1500 mg/kg or 2500 mg/kg; six patients per dose level). Treatment will be administered every two weeks by slow IV infusion over 8 hours at different concentrations to achieve the proscribed dose levels. Patients will receive treatment for a total of 12 weeks. Patients who withdraw prior to completion of the initial pharmacokinetic and pharmacodynamic assessments will be replaced. The design of the proposed study thus enables early assessment of potential biochemical markers of response but allows for a sufficient dosing duration to enable the short-term effectiveness of Trappsol in NPC to be assessed. The maximum dose proposed for this study is below the maximum dose for which long term clinical data is available in 2 patients (2800 mg/kg weekly for 3-5 years). Although individual clinicians have not always utilized an escalating rate of infusion, the reports of infusion related reactions in three patients suggest that this is an appropriate clinical strategy to mitigate the risk of such events and is consistent with dosing administration for other therapeutic agents. In the proposed study, treatment will be administered less frequently than has been undertaken in compassionate use. This longer dosing interval is supported by nonclinical data comparing the metabolism of cholesterol in non-human species with that in man; although a once weekly dosing interval was initially studied in man based on data in the mouse, HP-β-CD cholesterol metabolism/turnover in the mouse is 13-fold higher than in man which, in NPC, likely translates into a 13-fold slower accumulation of cholesterol in human cells compared with those of the mouse.Therefore, it is theorized that, given the slower cholesterol metabolism in humans, the dosing interval could be much less frequent in man than in mouse; however, based on what is known about cholesterol metabolism in humans and the pharmacokinetic and pharmacodynamic effect of HP-β-CD in the mouse, a dosing interval of 2 weeks in man is likely to be well within the therapeutic dosing interval and also minimizes the amount of infusions required to be administered.
4 schema:endDate 2019-02-01T00:00:00Z
5 schema:keywords Evaluate
6 Evaluation Study as Topic
7 HP
8 Mass Screening
9 NPC
10 NPC disease
11 Niemann Pick
12 Niemann-Pick disease
13 Organization and Administration
14 Phase I study
15 Spinal Puncture
16 USA
17 accumulation
18 aphasia
19 ataxia
20 behavioral aspect
21 beta
22 biopsy specimen
23 blood
24 body
25 cell
26 cerebrospinal fluid
27 cholesterol
28 cholesterol metabolism
29 clinical data
30 clinical strategy
31 completion
32 condition
33 cyclo
34 cyclodextrins
35 defect
36 different concentration
37 different dose
38 different dose level
39 different level
40 disease type
41 doctor
42 dose level
43 dosing
44 drug
45 duration
46 early assessment
47 elasticity
48 escalating
49 experimental treatment
50 hearing
51 holding
52 human
53 human cell
54 individual
55 infusion
56 interval
57 intravenous infusion
58 laboratory experiment
59 liver
60 liver sample
61 marker
62 maximum dose
63 metabolism
64 minimizes
65 mouse
66 multiple dose
67 neurological function
68 non-human specie
69 organ damage
70 parallel group study
71 patient
72 patient use
73 pharmacodynamic effect
74 pharmacokinetics
75 planned study
76 plasma pharmacokinetics
77 primary objective
78 protein
79 questionnaire
80 recruitment
81 related reaction
82 research study
83 risk
84 saccade
85 sample
86 scientific literature
87 secondary objective
88 serum
89 short-term effectiveness
90 subsequent treatment
91 substance
92 therapeutic
93 therapeutic agent
94 translates
95 transport
96 treatment phase
97 trial
98 ultrasound
99 urine sample
100 schema:name A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol Cyclo (HP-Beta-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease
101 schema:sameAs https://app.dimensions.ai/details/clinical_trial/NCT02939547
102 schema:sdDatePublished 2019-03-07T15:26
103 schema:sdLicense https://scigraph.springernature.com/explorer/license/
104 schema:sdPublisher Nb8eb7e812d034fb8aebb4383e07c8964
105 schema:sponsor https://www.grid.ac/institutes/grid.414016.6
106 schema:startDate 2017-10-01T00:00:00Z
107 schema:subjectOf sg:pub.10.1007/8904_2011_47
108 sg:pub.10.1007/s10545-012-9583-x
109 sg:pub.10.1186/1750-1172-5-16
110 sg:pub.10.1186/s13023-015-0274-1
111 https://doi.org/10.1001/jama.282.12.1157
112 https://doi.org/10.1002/humu.10255
113 https://doi.org/10.1002/mds.10541
114 https://doi.org/10.1016/0022-3956(75)90026-6
115 https://doi.org/10.1016/j.fct.2005.03.007
116 https://doi.org/10.1016/j.ymgme.2012.03.012
117 https://doi.org/10.1016/s1474-4422(07)70194-1
118 https://doi.org/10.1073/pnas.0810895106
119 https://doi.org/10.1074/jbc.271.35.21604
120 https://doi.org/10.1074/jbc.r100057200
121 https://doi.org/10.1086/302620
122 https://doi.org/10.1093/brain/awl260
123 https://doi.org/10.1097/aud.0b013e3182a362b8
124 https://doi.org/10.1126/science.290.5500.2298
125 https://doi.org/10.1126/scitranslmed.3010101
126 https://doi.org/10.1136/jnnp.2005.086785
127 https://doi.org/10.1172/jci72835
128 https://doi.org/10.1179/jmt.2009.17.3.163
129 https://doi.org/10.1194/jlr.m013789
130 https://doi.org/10.1194/jlr.m700525-jlr200
131 https://doi.org/10.1194/jlr.p013524
132 https://doi.org/10.1212/01.wnl.0000219042.60538.92
133 https://doi.org/10.1212/wnl.0b013e31828869f9
134 https://doi.org/10.1242/dmm.010124
135 https://doi.org/10.1371/journal.pone.0006951
136 https://doi.org/10.1371/journal.pone.0114669
137 https://doi.org/10.7863/ultra.33.2.197
138 schema:url https://clinicaltrials.gov/show/NCT02939547
139 sgo:license sg:explorer/license/
140 sgo:sdDataset clinical_trials
141 rdf:type schema:MedicalStudy
142 Nb8eb7e812d034fb8aebb4383e07c8964 schema:name Springer Nature - SN SciGraph project
143 rdf:type schema:Organization
144 anzsrc-for:3053 schema:inDefinedTermSet anzsrc-for:
145 rdf:type schema:DefinedTerm
146 anzsrc-for:3164 schema:inDefinedTermSet anzsrc-for:
147 rdf:type schema:DefinedTerm
148 sg:pub.10.1007/8904_2011_47 schema:sameAs https://app.dimensions.ai/details/publication/pub.1035654877
149 https://doi.org/10.1007/8904_2011_47
150 rdf:type schema:CreativeWork
151 sg:pub.10.1007/s10545-012-9583-x schema:sameAs https://app.dimensions.ai/details/publication/pub.1022369447
152 https://doi.org/10.1007/s10545-012-9583-x
153 rdf:type schema:CreativeWork
154 sg:pub.10.1186/1750-1172-5-16 schema:sameAs https://app.dimensions.ai/details/publication/pub.1005623887
155 https://doi.org/10.1186/1750-1172-5-16
156 rdf:type schema:CreativeWork
157 sg:pub.10.1186/s13023-015-0274-1 schema:sameAs https://app.dimensions.ai/details/publication/pub.1005979361
158 https://doi.org/10.1186/s13023-015-0274-1
159 rdf:type schema:CreativeWork
160 https://doi.org/10.1001/jama.282.12.1157 schema:sameAs https://app.dimensions.ai/details/publication/pub.1050564301
161 rdf:type schema:CreativeWork
162 https://doi.org/10.1002/humu.10255 schema:sameAs https://app.dimensions.ai/details/publication/pub.1006616197
163 rdf:type schema:CreativeWork
164 https://doi.org/10.1002/mds.10541 schema:sameAs https://app.dimensions.ai/details/publication/pub.1006635563
165 rdf:type schema:CreativeWork
166 https://doi.org/10.1016/0022-3956(75)90026-6 schema:sameAs https://app.dimensions.ai/details/publication/pub.1027523106
167 rdf:type schema:CreativeWork
168 https://doi.org/10.1016/j.fct.2005.03.007 schema:sameAs https://app.dimensions.ai/details/publication/pub.1052037868
169 rdf:type schema:CreativeWork
170 https://doi.org/10.1016/j.ymgme.2012.03.012 schema:sameAs https://app.dimensions.ai/details/publication/pub.1027225530
171 rdf:type schema:CreativeWork
172 https://doi.org/10.1016/s1474-4422(07)70194-1 schema:sameAs https://app.dimensions.ai/details/publication/pub.1021955671
173 rdf:type schema:CreativeWork
174 https://doi.org/10.1073/pnas.0810895106 schema:sameAs https://app.dimensions.ai/details/publication/pub.1039042817
175 rdf:type schema:CreativeWork
176 https://doi.org/10.1074/jbc.271.35.21604 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012367026
177 rdf:type schema:CreativeWork
178 https://doi.org/10.1074/jbc.r100057200 schema:sameAs https://app.dimensions.ai/details/publication/pub.1048401077
179 rdf:type schema:CreativeWork
180 https://doi.org/10.1086/302620 schema:sameAs https://app.dimensions.ai/details/publication/pub.1058610189
181 rdf:type schema:CreativeWork
182 https://doi.org/10.1093/brain/awl260 schema:sameAs https://app.dimensions.ai/details/publication/pub.1052588466
183 rdf:type schema:CreativeWork
184 https://doi.org/10.1097/aud.0b013e3182a362b8 schema:sameAs https://app.dimensions.ai/details/publication/pub.1044245566
185 rdf:type schema:CreativeWork
186 https://doi.org/10.1126/science.290.5500.2298 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062572556
187 rdf:type schema:CreativeWork
188 https://doi.org/10.1126/scitranslmed.3010101 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012551405
189 rdf:type schema:CreativeWork
190 https://doi.org/10.1136/jnnp.2005.086785 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037485737
191 rdf:type schema:CreativeWork
192 https://doi.org/10.1172/jci72835 schema:sameAs https://app.dimensions.ai/details/publication/pub.1023308017
193 rdf:type schema:CreativeWork
194 https://doi.org/10.1179/jmt.2009.17.3.163 schema:sameAs https://app.dimensions.ai/details/publication/pub.1002816074
195 rdf:type schema:CreativeWork
196 https://doi.org/10.1194/jlr.m013789 schema:sameAs https://app.dimensions.ai/details/publication/pub.1014481242
197 rdf:type schema:CreativeWork
198 https://doi.org/10.1194/jlr.m700525-jlr200 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012167280
199 rdf:type schema:CreativeWork
200 https://doi.org/10.1194/jlr.p013524 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037061331
201 rdf:type schema:CreativeWork
202 https://doi.org/10.1212/01.wnl.0000219042.60538.92 schema:sameAs https://app.dimensions.ai/details/publication/pub.1064348165
203 rdf:type schema:CreativeWork
204 https://doi.org/10.1212/wnl.0b013e31828869f9 schema:sameAs https://app.dimensions.ai/details/publication/pub.1064357619
205 rdf:type schema:CreativeWork
206 https://doi.org/10.1242/dmm.010124 schema:sameAs https://app.dimensions.ai/details/publication/pub.1019513430
207 rdf:type schema:CreativeWork
208 https://doi.org/10.1371/journal.pone.0006951 schema:sameAs https://app.dimensions.ai/details/publication/pub.1050731206
209 rdf:type schema:CreativeWork
210 https://doi.org/10.1371/journal.pone.0114669 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037474842
211 rdf:type schema:CreativeWork
212 https://doi.org/10.7863/ultra.33.2.197 schema:sameAs https://app.dimensions.ai/details/publication/pub.1018909132
213 rdf:type schema:CreativeWork
214 https://www.grid.ac/institutes/grid.414016.6 schema:Organization
 




Preview window. Press ESC to close (or click here)


...