Trials / Terminated
TerminatedNCT03643562
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
- Status
- Terminated
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 27 (actual)
- Sponsor
- Mandos LLC · Industry
- Sex
- All
- Age
- 4 Years
- Healthy volunteers
- Not accepted
Summary
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks. Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg. Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Adrabetadex | Administered via lumbar puncture (LP) and IT infusion |
Timeline
- Start date
- 2018-06-18
- Primary completion
- 2021-11-02
- Completion
- 2021-11-02
- First posted
- 2018-08-23
- Last updated
- 2024-01-17
- Results posted
- 2024-01-17
Locations
17 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03643562. Inclusion in this directory is not an endorsement.