Trials / Completed
CompletedNCT03406962
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
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)
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 8 (actual)
- Sponsor
- Magenta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 0 Years – 17 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | MGTA-456 | Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456. |
Timeline
- Start date
- 2018-02-09
- Primary completion
- 2020-02-10
- Completion
- 2021-01-15
- First posted
- 2018-01-23
- Last updated
- 2021-11-03
- Results posted
- 2021-10-28
Locations
2 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03406962. Inclusion in this directory is not an endorsement.