Phase 2
Completed N=8
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Inherited Metabolic Disorders (IMD)
Source: ClinicalTrials.gov NCT03406962 ↗
Enrolled (actual)
8
Serious AEs
100.0%
Results posted
Oct 2021
Primary outcomePrimary: Number of Participants With Engraftment — 6; 2 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Engraftment |
6; 2 | — |
| SECONDARY Number of Participants With Infusion Toxicities |
5; 1; 4; 1; 4; 1 | — |
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
Data sourced from ClinicalTrials.gov (NCT03406962). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.