Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04860960 | Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 | PHASE3 | ACTIVE NOT_RECRUITING | 94 | — | — | Jul 20, 2021 | Jun 1, 2026 | Jun 4, 2024 | 35 | United States, Argentina +11 |
| NCT02939547 | Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) | PHASE1 | COMPLETED | 13 | — | — | Oct 11, 2017 | Feb 10, 2020 | Feb 21, 2021 | 2 | United States |
| NCT02912793 | Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients | PHASE1 | COMPLETED | 12 | — | — | Mar 20, 2017 | Mar 3, 2021 | Aug 10, 2021 | 5 | Israel, Sweden +1 |
Ambulation, Fine Motor, Speech, Swallow
Ambulation, Fine Motor, Speech, Swallow, Cognition
To compare the C max of Trappsol following 2 different dose levels of intravenous Trappsol in patients with NPC-1 disease following single and multiple doses
To compare the T max of Trappsol following 2 different dose levels of intravenous Trappsol in patients with NPC-1 disease following single and multiple doses
To compare the Volume of Distribution of Trappsol following 2 different dose levels of intravenous Trappsol in patients with NPC-1 disease following single and multiple doses
To compare the T1/2 of Trappsol following 2 different doses.
To evaluate plasma PK of Trappsol by comparison of Maximum Concentration (Cmax ) of the three doses
To evaluate the plasma PK of Trappsol by comparison of the Tmax of three doses
To evaluate the plasma PK of Trappsol by comparison of the Volume of Distribution of three doses
To evaluate the PK of Trappsol by comparison of the Elimination half-lives of three doses
| Arm | Type | Description |
|---|---|---|
| Experimental | EXPERIMENTAL | Intravenous administration of 2000 mg/kg hydroxypropyl betacyclodextrin (Trappsol Cyclo) (based on body weight) diluted with 0.5N saline over at least 6.5 hours every 2 weeks |
| Placebo comparator | PLACEBO_COMPARATOR | Intravenous administration of 0.5N saline over at least 6.5 hours every 2 weeks |
| Open Label sub-study for Infants up to age 3 | EXPERIMENTAL | Up to 12 patients age 0 - 3 yrs in countries following EMA guidance may be enrolled in this open label sub-study. All patients will receive 2000 mg/kg hydroxypropyl betacyclodextrin (Trappsol Cyclo) diluted with 0.5N saline at the clinician's discretion over 6.5 hours every 2 weeks. Outcome measures are safety, clinician and caregiver impressions. |
| Hydroxypropyl-beta-cyclodextrin IV 1500 mg/kg | ACTIVE_COMPARATOR | Hydroxypropyl-beta-cyclodextrin administered by slow IV infusion for 8 - 9h every 2 weeks |
| Hydroxypropyl-beta-cyclodextrin IV 2500 mg/kg | ACTIVE_COMPARATOR | Hydroxypropyl-beta-cyclodextrin administered by slow IV infusion for 8 - 9h every 2 weeks |
| Hydroxy-propyl-beta-cyclodextrin IV 2000 mg/kg | ACTIVE_COMPARATOR | Hydroxypropyl-beta-cyclodextrin administered by slow IV infusion for 8h every 2 weeks |
| Name | Type | Description |
|---|---|---|
| Hydroxypropyl-beta-cyclodextrin | DRUG | Dose is 2000 mg/kg body weight provided every 2 weeks intravenously |
| Placebo | DRUG | 0.5N saline provided every 2 weeks intravenously |
Inclusion Criteria: 1. Confirmed diagnosis of NPC1 2. Annual Severity Increment Score between 0.5 and 2.0 using the 17-domain NPC Severity Scale 3. 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 Miglu...