Clinical trial NCT03406962: MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Official title: A Phase 2, Single-arm, Open-label Study to Evaluate the Safety and Efficacy of MGTA-456 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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
Lead sponsor: Magenta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-01-23
Enrollment: 8
Sex: ALL
Minimum age (years): 0
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Detailed description: This phase 2 study is designed to evaluate the safety and efficacy of MGTA-456 in patients with IMD after receiving myeloablative conditioning and HSCT. MGTA-456 is an expanded CD34+ cell therapy product candidate given to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein and preserve neurodevelopment. Since MGTA-456 offers increased numbers of HSCs over standard umbilical cord blood, it is expected to reduce the risks of prolonged neutropenia and thrombocytopenia and graft failure, and potentially transplant-related mortality (TRM). Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) (also referred to as Krabbe disease) are eligible for this study.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Number of Participants With Engraftment
timeFrame: 42 days
Secondary outcomes:
measure: Number of Participants With Infusion Toxicities
timeFrame: 48 hours
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Magenta Therapeutics
name: Magenta Study Coordinator
role: STUDY_DIRECTOR
References: