Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Hydroxypropyl-beta-cyclodextrin · 2 trials · 1 indication
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 |
|---|---|---|
| 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 | Used in the treatment of Niemann-Pick Disease C1 ( NPC1) |
Inclusion Criteria: 1. Confirmed diagnosis of NPC-1 defined as one of the following 1. Two NPC-1 mutations on exome gene sequencing 2. One NPC-1 mutation and positive filipin staining (current or prior) 3. Vertical supranuclear gaze palsy \[VSGP\] plus either ≥ one NPC-1 mutation or posit...
Hydroxypropyl-beta-cyclodextrin is an investigational small molecule being developed for Niemann-Pick Disease, Type C1, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied for its potential to treat this condition.
Hydroxypropyl-beta-cyclodextrin is being developed by Cyclo Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol CYTH. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Niemann-Pick Disease, Type C1.
Hydroxypropyl-beta-cyclodextrin is in Phase 1 clinical development. It is an investigational drug and has not yet received FDA approval. The drug is being studied for the treatment of Niemann-Pick Disease, Type C1, and is currently in early-stage clinical trials to assess its safety and tolerability.
Hydroxypropyl-beta-cyclodextrin has been studied in two completed Phase 1 clinical trials. The first trial, NCT02912793, evaluated the safety and efficacy of intravenous Trappsol Cyclo in patients with Niemann-Pick Type C across Israel, Sweden, and the United Kingdom. The second trial, NCT02939547, studied the pharmacokinetics of Trappsol and its effects on potential biomarkers in the United States.
Yes, Hydroxypropyl-beta-cyclodextrin is also known as Trappsol Cyclo. Clinical trials for the drug, such as NCT02912793 and NCT02939547, refer to it as Trappsol Cyclo. This alternative name is used in the context of studying the drug for Niemann-Pick Disease, Type C1.