CTD-TCNPC-301
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StatusAccepting Candidates
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Age3 Years - N/A
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SexesAll
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Healthy VolunteersNo
Objective
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.
Description
The TransportNPC study is a prospective, randomized, double-blind, placebo controlled therapeutic study for 93 patients age 3 and older with confirmed diagnosis of NPC1. The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously by slow infusion every two weeks in addition to standard of care as compared to placebo and standard of care. Standard of care may include Miglustat or leucine products that are not currently under investigation as a therapeutic. Patients will be randomized to receive Trappsol Cyclo or placebo at a 2:1 ratio. The study duration is 96 weeks, with an unblinded interim analysis at 48 weeks. An open-label extension of up to 96 weeks follows the interventional study. Patients whose disease progression worsens by two levels in the Clinical Global Impression of Severity scale over 12 weeks, starting at week 36, may be moved to open label treatment. Efficacy will be measured at week 48 and week 96 by a composite score of major disease features. A sub-study will be conducted in countries following EMA guidance for up to 12 patients age 0 * 3 years who may be asymptomatic. Outcomes for the sub-study are safety, clinical and caregiver impression of disease.
Details
Full study title | A Phase 3, Double-blind, Randomized, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol? Cyclo (Hydroxypropyl-β-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann-Pick Disease Type C1 |
Protocol number | OCR41901 |
ClinicalTrials.gov ID | NCT04860960 |
Phase | Phase 3 |
Eligibility
Inclusion Criteria:
Confirmed diagnosis of NPC1
Annual Severity Increment Score between 0. 5 and 2. 0 using the 17-domain NPC Severity Scale
Treated or Not Treated with Miglustat (patients must be on a stable dose for at least 3 months prior to the Screening Visit, or have discontinued Miglustat for at least 3 months prior to Screening Visit).
Body weight greater than 4. 5 kg and less than or equal to 125 kg
Presenting at least 1 neurological symptom of the disease
Written informed consent
Willing and capable to participate in all aspects of trial design
Ability to travel to the trial site at scheduled times
Contraception requirements per protocol
Caregiver consent as appropriate to participate in all protocol-specified assessments
for duration of trial
- Inclusion criteria for Open Label Extension are 1) Received double-blind treatment for
at least 48 weeks with CGI-S deterioration by at least 2 levels for 2 consecutive
assessment visits 12 weeks apart, or 2) completion of double-blind treatment and
completed all assessments through week 96, or 3) Discontinued early from double-blind
treatment but completed all assessments through week 96
- Inclusion criteria for patients age 0 to 3 years in open-label sub-study in countries
Following Ema guidance only: Confirmed diagnosis of Npc1; treated or not with
Miglustat per main study; body weight greater than 4.5kg; patient may be asymptomatic;
written assent for child to participate in safety assessments; caregiver consent to
participate in caregiver assessments; ability to travel to the trial site for all
scheduled visits.
Exclusion Criteria:
Recipient of a liver transplant within 1. 8
Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate
Participate in a study
Here are some general steps to consider when participating in a research study:
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Step1
Contact the research team
Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.
Primary contact
Amanda Prince -
Step2
Get screened to confirm eligibility
You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.
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Step3
Provide your consent to participate
If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.
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Step4
Participate
If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.