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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MGTA-456 | Drug | Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Engraftment | Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days. | 42 days |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Infusion Toxicities | Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration | 48 hours |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Magenta Study Coordinator | Magenta Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States | ||
| Cincinnati Children's Hospital Medical Center |
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Before an enrolled patient was infused with MGTA-456, the patient had to undergo a protocol-defined conditioning regimen and the proper expansion and manufacture of MGTA-456 investigational cell therapy product was confirmed.
Patients were referred to clinical sites.
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| ID | Title | Description |
|---|---|---|
| FG000 | MGTA-456 | MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | MGTA-456 | MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Engraftment | Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days. | Patients who received MGTA-456 investigational product | Posted | Count of Participants | Participants | 42 days |
|
Adverse event data was collected at the start of the patient's conditioning regimen (Day -9) up until the patient's 1 year follow-up visit.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | MGTA-456 | MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. . |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Autoimmune hemolytic anemia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
Magenta discontinued the enrollment of the study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Magenta Therapeutics | (857) 242-0170 | clinicaltrials@magentatx.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 10, 2020 | Sep 29, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 15, 2021 | Sep 29, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| D007965 | Leukodystrophy, Globoid Cell |
| D007966 | Leukodystrophy, Metachromatic |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| Cincinnati |
| Ohio |
| 45229 |
| United States |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Height | Mean | Standard Deviation | cm |
|
| Weight | Mean | Standard Deviation | kg |
|
|
|
| Secondary | Number of Participants With Infusion Toxicities | Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration | All patients who received MGTA-456 investigational product. | Posted | Count of Participants | Participants | 48 hours |
|
|
|
| 2 |
| 8 |
| 8 |
| 8 |
| 8 |
| 8 |
|
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Pyrexia | General disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Pneumonia | Infections and infestations | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Sepsis | Infections and infestations | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Staphylococcal bacteremia | Infections and infestations | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Autoimmune pancytopenia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
|
| Neutropenia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Hypertension | Vascular disorders | MedDRA v23.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Nausea | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Hypoalbuminemia | Metabolism and nutrition disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA v23.1 | Systematic Assessment | Not related to MGTA-456 |
|
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| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D052439 | Lipid Metabolism Disorders |
| D052516 | Sulfatidosis |
| Anemia |
|
| Hypertension |
|
| Hypocalcemia |
|
| Leukopenia |
|