Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With … (NCT04860960) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
United States94 participantsStarted 2021-07-20
Plain-language summary
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.
Who can participate
Age range3 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Exclusion criteria
✕. Recipient of a liver transplant within \<12 months or planned liver transplantation
✕
What they're measuring
1
Change from Baseline in 4-Domain NPC Severity Score (US only)
Timeframe: Interim Analysis at Week 48
2
Change from Baseline in 4-Domain NPC Severity Score (US only)
Timeframe: End of Study at Week 96
3
Change from Baseline in 5-Domain NPC Severity Score (ex-US)
Timeframe: Interim Analysis at Week 48
4
Change from Baseline in 5-Domain NPC Severity Score (ex-US)
. Patients with active liver disease from any cause other than NPC1
✕. Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalized ratio \> 1.8
✕. Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate \<60ml/min/1.73m2.
✕. Use of curcumin or fish oil within 12 weeks prior to enrollment
✕. Known or suspected allergy or intolerance to the study treatment
✕. In the opinion of the Investigator, the patient's clinical condition does not allow for the blood collection required as per protocol specific procedures.
✕. Treatment with any investigational drug during the 3 months prior to entering the study. If the investigational drug has a short half-life (\<8 hours) and would be expected to be cleared from the body within 1 month, then the wash-out period is 1 month. Treatment with any form of leucine, whether as an investigational drug or other formulation is not allowed