Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03406962 | MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) | PHASE2 | COMPLETED | 8 | — | — | Feb 9, 2018 | Jan 15, 2021 | Nov 3, 2021 | 2 | United States |
Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
| Arm | Type | Description |
|---|---|---|
| MGTA-456 | EXPERIMENTAL | MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. |
| Name | Type | Description |
|---|---|---|
| MGTA-456 | DRUG | Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456. |
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 t...