A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Inherited Metabolic Disorders (IMD) Clinical Trial

— IMD-002  

Official title:

A Follow-Up Study to Evaluate the Safety and Clinical Outcomes of Patients With Non-Malignant Disease Who Have Undergone Hematopoietic Stem Cell Transplantation With MGTA-456

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04008849
• Other study ID #: IMD-002
• Status: Terminated
• Start date: May 21, 2019
• Est. completion date: October 8, 2020

Study information

• Verified date: March 2021
• Source: Magenta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456

Description:

This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 3
• Est. completion date: October 8, 2020
• Est. primary completion date: October 8, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 16 Years

Eligibility

Inclusion Criteria:

• An Institutional Review Board (IRB)/Independent Ethics Committee-approved written informed consent form must be signed and dated by the patient or legal guardian. Study assents will also be prepared for children and adolescents to review when applicable.
• Patient completed an IMD study in the MGTA-456 program and was administered MGTA-456 for HSCT. Exclusion Criteria: •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.

Related Conditions & MeSH terms

• Inherited Metabolic Disorders (IMD)
• Metabolic Diseases

Intervention

Other:
• Safety and efficacy assessments
Long term safety and clinical outcomes

Locations

Country	Name	City	State
United States	University of Minnesota	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Magenta Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of related adverse events		2 years
Primary	Incidence of serious adverse events		2 years
Primary	Incidence of late hematological graft failure		2 years
Primary	Incidence of chronic graft versus host disease		2 years
Primary	Overall survival		2 years
Primary	Event-free survival		2 years
Primary	Change in cALD Neurologic Function Score over time		2 years
Primary	Proportion of subjects without gadolinium enhancement on MRI over time		2 years
Primary	Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients		2 years
Primary	Very long chain fatty acid blood level (ug/mL) in cALD patients		2 years