Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Phase 2

Terminated

Conditions: Niemann-Pick Disease, Type C

Interventions: Drug: Adrabetadex

Registration Number: NCT04958642

Lead Sponsor: Mandos LLC

Brief Summary: Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here.

This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords).

In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control).

In Part C, all participants will receive study drug.

Detailed Description: Participants in Part C will receive adrabetadex until the investigator considers adrabetadex to no longer be beneficial to the participant, or the development program is discontinued.

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 66

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Adrabetadex	Adrabetadex	All participants receive their prescribed dose of adrabetadex. Dose is allowed to be adjusted down to a minimum of 400 milligrams (mg) or up to a maximum of 900 mg, at the investigator's discretion.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Baseline up to 5 years	An 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.

Secondary Outcome Measures

Name	Time	Method

Trial Locations

Locations (30): Gazi University Medical Faculty
🇹🇷
Ankara, Turkey
Universitaetsklinikum Mainz
🇩🇪
Mainz, Germany
Monash Medical Centre
🇦🇺
Clayton, Victoria, Australia
Great Ormond Street Hospital
🇬🇧
London, United Kingdom
Katholisches Klinikim Bochum gGmbH
🇩🇪
Bochum, Germany
Waikato Hospital
🇳🇿
Hamilton, New Zealand
Birmingham Children's Hospital NHS Foundation Trust
🇬🇧
Birmingham, United Kingdom
Universitaetsklinikum Muenster
🇩🇪
Münster, Germany
Multicare Institute for Research and Innovation
🇺🇸
Tacoma, Washington, United States
CHU Paris Est - Hopital d'Enfants Armand-Trousseau
🇫🇷
Paris, Cedex 12, France
The Prince of Wales Hospital
🇦🇺
Randwick, New South Wales, Australia
Hacettepe University Medical Faculty
🇹🇷
Ankara, Turkey
Royal Perth Hospital
🇦🇺
Perth, Western Australia, Australia
INSELSPITAL, Universitätsspital Bern
🇨🇭
Bern, Switzerland
Children's National Medical Center
🇺🇸
Washington, District of Columbia, United States
National University Hospital (Singapore) Pte, Ltd
🇸🇬
Singapore, Singapore
University of Alabama at Birmingham
🇺🇸
Birmingham, Alabama, United States
Lehigh Valley Health Network
🇺🇸
Allentown, Pennsylvania, United States
The Children's Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
Rush University Medical Center
🇺🇸
Chicago, Illinois, United States
University of California San Francisco
🇺🇸
San Francisco, California, United States
Boston Children's Hospital
🇺🇸
Boston, Massachusetts, United States
Children's Hospital of Orange County: CHOC Children's
🇺🇸
Orange, California, United States
Shands Children's Hospital
🇺🇸
Gainesville, Florida, United States
University of Colorado Denver
🇺🇸
Aurora, Colorado, United States
Eunice Kennedy Shriver National Institute of Child Health and Human Development
🇺🇸
Bethesda, Maryland, United States
University of North Carolina
🇺🇸
Chapel Hill, North Carolina, United States
Dell Children's Medical Center of Central Texas
🇺🇸
Austin, Texas, United States
Royal Melbourne Hospital
🇦🇺
Melbourne, Victria, Australia
Montefiore Medical Center
🇺🇸
Bronx, New York, United States