MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

NCT03406962 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: IMD-001
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 2

Brief Summary

This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.

Conditions

Inherited Metabolic Disorders (IMD)

Keywords

inherited metabolic disorders; cerebral adrenoleukodystrophy; Hurler syndrome; globoid cell leukodystrophy; Krabbe disease; metachromatic leukodystrophy; hematopoietic stem cells; hematopoietic stem cell transplant; umbilical cord blood; umbilical cord blood transplant; MGTA-456; myeloablative conditioning regimen; bone marrow transplant; mucopolysaccharidosis-1H

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Engraftment

  Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.

  42 days

Secondary Outcomes (1)

• Number of Participants With Infusion Toxicities

  48 hours

Study Arms (1)

MGTA-456

EXPERIMENTAL

MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.

Drug: MGTA-456

Interventions

MGTA-456 DRUG

Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.

Also known as: HSC835

MGTA-456

Eligibility Criteria

• Age: 0 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age \< 2.5 years with Hurler syndrome, age 2-17 years with cerebral adrenoleukodystrophy (cALD), age \< 16 years with metachromatic leukodystrophy (MLD) and age ≤ 10 years with globoid cell leukodystrophy (GLD) (also referred to as Krabbe)
• Cord blood grafts require genetic testing and/or demonstration of enzyme activity for patients with Hurler syndrome, MLD or GLD and are tested for very long chain fatty acids (VLCFA) to confirm there is no evidence of VLCFA consistent with ALD
• Adequate organ function
• Availability of eligible donor material

You may not qualify if:

• Availability of a matched-related donor who is not a carrier of the same genetic defect
• Active infection at screening
• Prior myeloablative conditioning
• History of human immunodeficiency virus (HIV) infection

Sponsors & Collaborators

• Magenta Therapeutics, Inc.: lead

Study Sites (2)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I; Leukodystrophy, Globoid Cell; Leukodystrophy, Metachromatic

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Leukoencephalopathies; Demyelinating Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Sulfatidosis

Limitations and Caveats

Magenta discontinued the enrollment of the study.

Results Point of Contact

• Title: Medical Director
• Organization: Magenta Therapeutics

clinicaltrials@magentatx.com

(857) 242-0170

Study Officials

• Magenta Study Coordinator

  Magenta Therapeutics

  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

Study Record Dates

• First Submitted: January 16, 2018
• First Posted: January 23, 2018
• Study Start: February 9, 2018
• Primary Completion: February 10, 2020
• Study Completion: January 15, 2021
• Last Updated: November 3, 2021
• Results First Posted: October 28, 2021

Record last verified: 2020-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)