Clinical trial NCT04008849: A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
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
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Inherited Metabolic Disorders (IMD)
Keywords: Inherited Metabolic Disorders
Hematopoietic Stem Cell Transplant
MGTA-456
Umbilical Cord Blood Transplant
Hurler Syndrome
Cerebral Adrenoleukodystrophy
Globoid Cell Leukodystrophy
Metachromatic Leukodystrophy
Lead sponsor: Magenta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-07-05
Enrollment: 3
Sex: ALL
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456

Brief 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

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of related adverse events
timeFrame: 2 years
measure: Incidence of serious adverse events
timeFrame: 2 years
measure: Incidence of late hematological graft failure
timeFrame: 2 years
measure: Incidence of chronic graft versus host disease
timeFrame: 2 years
measure: Overall survival
timeFrame: 2 years
measure: Event-free survival
timeFrame: 2 years
measure: Change in cALD Neurologic Function Score over time
timeFrame: 2 years
measure: Proportion of subjects without gadolinium enhancement on MRI over time
timeFrame: 2 years
measure: Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients
timeFrame: 2 years
measure: Very long chain fatty acid blood level (ug/mL) in cALD patients
timeFrame: 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
Study officials:
affiliation: University of Minnesota
name: Paul J Orchard, MD
role: PRINCIPAL_INVESTIGATOR
References: