Open‐Label Study of Long‐Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann‐Pick Disease Type C
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|ClinicalTrials.gov Identifier: NCT03893071|
Recruitment Status : Recruiting
First Posted : March 28, 2019
Last Update Posted : March 28, 2019
|Condition or disease||Intervention/treatment||Phase|
|Niemann-Pick Disease, Type C1||Drug: Hydroxypropyl-β-cyclodextrin||Phase 1|
This is an open‐label extension study of intravenous HP‐β‐CD (administered as Trappsol® Cyclo(TM)) in patients with NPC‐1 to evaluate long‐term safety and efficacy by providing continued access to treatment following completion of study CTD‐TCNPC‐101. Patients will receive medication at their home or local site under the supervision of a home nurse professional or at the parent site under the supervision of the site principal investigator. Patients will visit the parent site for safety and efficacy evaluations after receiving treatment for 3 months in the extension protocol.
Thereafter, visits to the parent site will be every 6 months until cessation. The study will continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient discontinuation. All patients who complete study CTD‐TCNCP‐101 and pass the screening criteria will be eligible for this study. The study will be directed from parent site in the US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.
For logistical reasons home infusions will be permitted as long as AEs, infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines. A Safety Review Committee (SRC) consisting of an independent advisor, the medical monitor, and the principal investigator will be established to review AEs and laboratory data throughout the study.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open‐Label Extension Study of the Long‐Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP‐β‐CD) in Patients With Niemann‐Pick Disease Type C (NPC‐1)|
|Estimated Study Start Date :||March 2019|
|Estimated Primary Completion Date :||March 2021|
|Estimated Study Completion Date :||March 2021|
Experimental: Hydroxypropyl-β-cyclodextrin IV
Hydroxypropyl-β-cyclodextrin will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.
HP-β-CD will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.
- Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product [ Time Frame: 1-104 weeks ]Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
- Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product [ Time Frame: 1-104 weeks ]Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product
- Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation [ Time Frame: 1-104 weeks ]Discontinuation, Study and Site Discontinuation
- Auditory capacity will be measured by behavioral auditory assessment [ Time Frame: 1-104 weeks ]Auditory capacity will be measured by behavioral auditory assessment
- Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS) [ Time Frame: 1-104 weeks ]Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03893071
|Contact: Sharon H Hrynkow, PhDfirstname.lastname@example.org|
|Contact: Karen Rabb||+12144763754||Karen.Rabb@synteract.com|
|United States, California|
|UCSF Benioff Children's Hospital Oakland||Recruiting|
|Oakland, California, United States, 94609|
|Contact: Dr Caroline Hastings|
|Principal Investigator:||Caroline Hastings, MD||UCSF Benioff Children's Hospital Oakland|