MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03406962|
Recruitment Status : Active, not recruiting
First Posted : January 23, 2018
Last Update Posted : June 24, 2020
|Condition or disease||Intervention/treatment||Phase|
|Inherited Metabolic Disorders (IMD)||Drug: MGTA-456||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||8 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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)|
|Actual Study Start Date :||February 9, 2018|
|Actual Primary Completion Date :||February 10, 2020|
|Estimated Study Completion Date :||January 2021|
MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.
Other Name: HSC835
- Incidence of engraftment [ Time Frame: 42 days ]Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
- Incidence of infusion toxicities [ Time Frame: 48 hours ]Incidence of MGTA-456-related adverse events (AEs) that limit MGTA-456 administration
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03406962
|United States, Minnesota|
|University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Study Director:||Magenta Study Coordinator||Magenta Therapeutics|