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Clinical Trials/EUCTR2015-005761-23-SE
EUCTR2015-005761-23-SE
Active, not recruiting
Phase 1

A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes

Cyclo Therapeutics, Inc.0 sites11 target enrollmentSeptember 9, 2016
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Conditionsiemann-Pick disease type CMedDRA version: 20.0Level: PTClassification code 10029403Term: Niemann-Pick diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Overview

Phase
Phase 1
Intervention
Not specified
Conditions
iemann-Pick disease type C
Sponsor
Cyclo Therapeutics, Inc.
Enrollment
11
Status
Active, not recruiting
Last Updated
4 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
September 9, 2016
End Date
TBD
Last Updated
4 years ago
Study Type
Interventional clinical trial of medicinal product
Sex
All

Investigators

Eligibility Criteria

Inclusion Criteria

  • •1\. Confirmed diagnosis of NPC\-1 defined as one of the following:
  • •a) Two NPC\-1 mutations on genotyping
  • •b) One NPC\-1 mutation and positive filipin staining (current or prior)
  • •c) Vertical supranuclear gaze palsy \[VSNGP] plus either \= one NPC\-1 mutation or positive filipin staining and no NPC\-2 mutations
  • •2\. NIH NPC Severity Score \<30 and with no more than 4 individual domains with a score \= 3\.
  • •3\. Age range: 2 years upwards
  • •a) Inclusion of the first six patients will be restricted to individuals aged \= 5 years. In the event that three patients aged \>18 (i.e. adults) are recruited before the total recruitment has reached n\=6, no more adults will be randomised to treatment until the first cohort of 6 patients is complete. Once the first six are recruited and randomised, study entry will be open to all ages \=2 years as per the protocol.
  • •4\. Negative pregnancy test for females of child bearing potential
  • •5\. Written, informed consent
  • •Are the trial subjects under 18? yes

Exclusion Criteria

  • •1\. The presence of NPC\-2 mutations on genotyping
  • •2\. Previous receipt of cyclodextrin therapy
  • •3\. Lanksy score \< 50 if aged \=16 or Karnofsky score \< 40 if aged \> 16\.
  • •4\. Inability to comply with the proposed protocol assessments
  • •5\. Concurrent treatment with any type of cholesterol lowering agents such as statins, fibrates, ezetimibe
  • •6\. Concurrent medical conditions representing a contraindication to any of the study medications
  • •7\. Stage 3 chronic kidney disease (CKD) or worse as indicated by an eGFR \< 60 mL/min/1\.73 m2\. In patients aged \= 18 years, eGFR is calculated according to the Schwartz equation (ref: Schwartz GJ \& Work DF) and in patients aged \> 18 years eGFR is calculated using the MDRD equation.
  • •8\. Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalised ratio (INR) \>1\. 8
  • •9\. Involvement in another interventional clinical trial within the previous 6 months from screening
  • •10\. Weight \>100 kg

Outcomes

Primary Outcomes

Not specified

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