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

NCT04958642 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: VTS301 (Part C)2015-002548-15
• Study Type: interventional
• Target: 66
• Locations: 9 countries
• Sites: 30

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.

Conditions

Niemann-Pick Disease, Type C

Keywords

Niemann-Pick Type C1 (NPC1) Disease; neurologic disease; gross motor dysfunction; fine motor dysfunction; dysphagia; swallowing problems; cognitive dysfunction; gait abnormalities; pediatrics

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  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.

  Baseline up to 5 years

Study Arms (1)

Adrabetadex

EXPERIMENTAL

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.

Drug: Adrabetadex

Interventions

Adrabetadex DRUG

Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.

Also known as: VTS-270, 2-hydroxypropyl-β-cyclodextrin, Cyclodextrin

Adrabetadex

Eligibility Criteria

• Age: 4 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Has agreed to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 weeks dosing regimen
• The investigator has received prior written authorization from the sponsor for the participant to enter VTS301 Part C on an amended dose and/or regimen
• Has received prior written authorization from Vtesse to enroll directly into Part C

Sponsors & Collaborators

• Mandos LLC: lead

Study Sites (30)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Children's Hospital of Orange County: CHOC Children's

Orange, California, 92867, United States

Location

University of California San Francisco

San Francisco, California, 94143-0780, United States

Location

University of Colorado Denver

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Shands Children's Hospital

Gainesville, Florida, 32608, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Eunice Kennedy Shriver National Institute of Child Health and Human Development

Bethesda, Maryland, 20892-2425, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27514, United States

Location

Lehigh Valley Health Network

Allentown, Pennsylvania, 18101, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States

Location

Multicare Institute for Research and Innovation

Tacoma, Washington, 98405, United States

Location

The Prince of Wales Hospital

Randwick, New South Wales, 2031, Australia

Location

Monash Medical Centre

Clayton, Victoria, 3168, Australia

Location

Royal Melbourne Hospital

Melbourne, Victria, Australia

Location

Royal Perth Hospital

Perth, Western Australia, 6000, Australia

Location

CHU Paris Est - Hopital d'Enfants Armand-Trousseau

Paris, Cedex 12, 75 571, France

Location

Katholisches Klinikim Bochum gGmbH

Bochum, 44791, Germany

Location

Universitaetsklinikum Mainz

Mainz, 55131, Germany

Location

Universitaetsklinikum Muenster

Münster, 48149, Germany

Location

Waikato Hospital

Hamilton, 3204, New Zealand

Location

National University Hospital (Singapore) Pte, Ltd

Singapore, 119074, Singapore

Location

INSELSPITAL, Universitätsspital Bern

Bern, 3010, Switzerland

Location

Gazi University Medical Faculty

Ankara, Turkey (Türkiye)

Location

Hacettepe University Medical Faculty

Ankara, Turkey (Türkiye)

Location

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, B4 6NH, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Niemann-Pick Disease, Type C; Disease; Deglutition Disorders; Cognitive Dysfunction; Mobility Limitation

Interventions

2-Hydroxypropyl-beta-cyclodextrin; Cyclodextrins

Condition Hierarchy (Ancestors)

Niemann-Pick Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Pharyngeal Diseases; Otorhinolaryngologic Diseases; Cognition Disorders; Neurocognitive Disorders; Mental Disorders; Signs and Symptoms

Intervention Hierarchy (Ancestors)

beta-Cyclodextrins; Macrocyclic Compounds; Polycyclic Compounds; Dextrins; Starch; Dietary Carbohydrates; Carbohydrates; Glucans; Polysaccharides

Limitations and Caveats

The development program for adrabetadex was discontinued and Part C of the study was terminated by the Sponsor.

Results Point of Contact

• Title: Executive Vice President, Regulatory Affairs
• Organization: Mandos, LLC

jspinella@mandoshealth.com

619-905-0489

Study Officials

• Clinical Study Lead

  Mandos LLC

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: In Part C, all participants receive adrabetadex

Study Record Dates

• First Submitted: June 29, 2021
• First Posted: July 12, 2021
• Study Start: December 23, 2015
• Primary Completion: April 11, 2022
• Study Completion: April 11, 2022
• Last Updated: August 29, 2023
• Results First Posted: August 29, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1); FR (1); GB (2); SG (1); US (15); TU (2); AU (4); DE (3); NZ (1)