A Phase 2 interventional study of MGTA-456 in Inherited Metabolic Disorders (IMD), sponsored by Magenta Therapeutics, Inc.. Completed at 2 sites in United States. Open to participants aged 0 Years to 17 Years. Per ClinicalTrials.gov, last updated 2021-11-03.
Sponsored by Magenta Therapeutics, Inc. · Phase 2, Interventional, and Treatment
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.
74 studies on the registry are indexed under Mucopolysaccharidosis I; 7 are open to participants now.
This study's enrollment of 8 is below the median of 14 across 47 interventional studies indexed under Mucopolysaccharidosis I.
Browse Mucopolysaccharidosis I studies →Magenta Therapeutics, Inc. is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Drug: MGTA-456
Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.
Also known as: HSC835
Number of Participants With Engraftment
Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
Time frame: 42 days
Number of Participants With Infusion Toxicities
Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration
Time frame: 48 hours
Patients were referred to clinical sites.
| Milestone | MGTA-456 |
|---|---|
| Started | 8 |
| Completed | 5 |
| Not completed | 3 |
| Withdrew: Death | 2 |
| Withdrew: Adverse event | 1 |
Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
| Participants | MGTA-456 Cohort 1 | MGTA-456 Cohort 2 |
|---|---|---|
| Number of Participants With Engraftment | 6 | 2 |
Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration
| Participants | MGTA-456 Cohort 1 | MGTA-456 Cohort 2 |
|---|---|---|
| Nausea | 5 | 1 |
| Hypoalbuminemia | 4 | 1 |
| Anemia | 4 | 1 |
| Hypertension | 4 | 1 |
| Hypocalcemia | 3 | 1 |
| Leukopenia | 4 | 0 |
Collected over 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.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| MGTA-456 | 2/8 (25%) | 8/8 (100%) | 8/8 (100%) |
| Event | MGTA-456 |
|---|---|
| Autoimmune hemolytic anemiaBlood and lymphatic system disorders | 3/8 |
| Immune thrombocytopeniaBlood and lymphatic system disorders | 3/8 |
| PyrexiaGeneral disorders | 3/8 |
| PneumoniaInfections and infestations | 2/8 |
| SepsisInfections and infestations | 2/8 |
| Staphylococcal bacteremiaInfections and infestations | 2/8 |
| Autoimmune pancytopeniaBlood and lymphatic system disorders | 2/8 |
| Event | MGTA-456 |
|---|---|
| AnemiaBlood and lymphatic system disorders | 7/8 |
| NeutropeniaBlood and lymphatic system disorders | 7/8 |
| ThrombocytopeniaBlood and lymphatic system disorders | 7/8 |
| HypertensionVascular disorders | 7/8 |
| ConstipationGastrointestinal disorders | 6/8 |
| NauseaGastrointestinal disorders | 6/8 |
| PyrexiaGeneral disorders | 6/8 |
| Immune thrombocytopeniaBlood and lymphatic system disorders | 5/8 |
| HypoalbuminemiaMetabolism and nutrition disorders | 5/8 |
| LeukopeniaBlood and lymphatic system disorders | 5/8 |
| Age, Continuous(years) | MGTA-456 |
|---|---|
| Mean | 2.49 ± 2.765 |
| Sex: Female, Male(Participants) | MGTA-456 |
|---|---|
| Female | 3 |
| Male | 5 |
| Ethnicity (NIH/OMB)(Participants) | MGTA-456 |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 7 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | MGTA-456 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 7 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | MGTA-456 |
|---|---|
| United States | 8 |
| Height(cm) | MGTA-456 |
|---|---|
| Mean | 84.44 ± 23.419 |
| Weight(kg) | MGTA-456 |
|---|---|
| Mean | 13.74 ± 7.863 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Feb 2018 to primary completion in Feb 2020, 24 months.
Results were first posted in Oct 2021, 1 year 8 months after primary completion.
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Studies of the drug tested here: Spanlecortemlocel
Magenta Therapeutics, Inc.