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

Niemann-Pick Type C Disease Clinical Trial

Official title:

Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03643562
• Other study ID #: VTS-270-001
• Status: Terminated
• Phase: Phase 3
• Start date: June 18, 2018
• Est. completion date: November 2, 2021

Study information

• Verified date: December 2023
• Source: Mandos LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 27
• Est. completion date: November 2, 2021
• Est. primary completion date: November 2, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• Niemann-Pick Type C Disease

Intervention

Drug:
• Adrabetadex
Administered via lumbar puncture (LP) and IT infusion

Locations

Country	Name	City	State
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Dell Children's Medical Center of Central Texas	Austin	Texas
United States	Rush University Medical Center	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Children's Specialty Center of Nevada	Las Vegas	Nevada
United States	University of Arkansas System	Little Rock	Arkansas
United States	Loma Linda University Health System	Loma Linda	California
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	The Children's Hospital at TriStar Centennial	Nashville	Tennessee
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	NYU Langone Medical Center	New York	New York
United States	Children's Hospital of Orange County	Orange	California
United States	Oregon Health & Science University	Portland	Oregon
United States	Carilion Medical Center, Carilion Clinic	Roanoke	Virginia
United States	Gillette Children's Specialty Healthcare	Saint Paul	Minnesota
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Mandos LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.	Baseline up to Week 134