Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

• Register: ClinicalTrials.gov
• Last refreshed on: 17 October 2022
• Main ID: NCT03643562
• Date of registration: 19/08/2018
• Prospective Registration: No
• Primary sponsor: Mandos LLC
• Public title: Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
• Scientific title: Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
• Date of first enrolment: June 18, 2018
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03643562
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Clinical Study Lead
• Affiliation: Mandos LLC

Key inclusion & exclusion criteria

Inclusion Criteria:

To be included in the study, a participant must meet the following criteria:

- Is male or female and at least 4 years of age at time of screening.

- Has a confirmed diagnosis of NPC and exhibits neurologic symptoms.

- Has written informed consent/assent to participate.

- Has the ability to undergo LP and IT drug administration.

- If taking miglustat (Zavesca®), must have been on a stable dose for the past 6 weeks
and be willing to remain on a stable dose for the duration of participation in the
study, or discontinue miglustat use at least 6 weeks before entry into the study (Day
1).

- If has a history of seizures, the condition is adequately controlled as per protocol
requirements.

- Agrees to discontinue any investigational treatments (other than adrabetadex) for at
least 1 month before first dose on Day 1.

- If engaging in heterosexual sex, agrees to use a protocol-defined method of
contraception throughout the study, and until 30 days after completing the study.

- Has a responsible adult who the investigator determines is able and willing to comply
with study requirements and a parent/guardian who will accompany the participant to
study visits.

Exclusion Criteria:

Participants will be excluded from the study if they meet any of the following criteria:

- Weighs less than 15 kg.

- Has a history of hypersensitivity reactions to any product containing
2-hydroxypropyl-ß-cyclodextrin (HP-ß-CD) or has a history of hypersensitivity
reactions or allergy to anesthesia/sedation.

- Has received treatment with any investigational product (other than adrabetadex)
within 1 month prior to Day 1 of treatment.

- Is pregnant or nursing.

- Has systemic infection or uncontrolled psychosis.

- Has known history of a bleeding disorder.

- Has used anticoagulants within 2 months of entry into the study.

- Per protocol, or in the opinion of the investigator:

1. has laboratory values that would preclude participation

2. has suspected infection of the central nervous system (CNS)

3. has a spinal deformity that could impact performance of repeated LPs

4. has a serious skin infection in the lumbar region or evidence of obstructive or
normal pressure hydrocephalus

5. is unable to comply with the study requirements

6. has a medical condition that might increase the risk of participation



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Niemann-Pick Type C Disease

Intervention(s)

Drug: Adrabetadex

Primary Outcome(s)

Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time Frame: Up to 134 Weeks]

Secondary ID(s)

VTS-270-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors