Trial Outcomes & Findings for MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) (NCT NCT03406962)

NCT ID: NCT03406962

Last Updated: 2021-11-03

Results Overview

Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 8 participants
• Primary outcome timeframe: 42 days
• Results posted on: 2021-11-03

Participant Flow

Patients were referred to clinical sites.

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.

Participant milestones

Measure	MGTA-456 MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Overall Study STARTED	8
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	MGTA-456 MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Overall Study Death	2
Overall Study Adverse Event	1

Baseline Characteristics

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Baseline characteristics by cohort

Measure	MGTA-456 n=8 Participants MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Age, Continuous	2.49 years STANDARD_DEVIATION 2.765 • n=93 Participants
Sex: Female, Male Female	3 Participants n=93 Participants
Sex: Female, Male Male	5 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	1 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	7 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	8 Participants n=93 Participants
Height	84.44 cm STANDARD_DEVIATION 23.419 • n=93 Participants
Weight	13.74 kg STANDARD_DEVIATION 7.863 • n=93 Participants

PRIMARY outcome

Timeframe: 42 days

Population: Patients who received MGTA-456 investigational product

Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.

Outcome measures

Measure	MGTA-456 Cohort 1 n=6 Participants Patients receiving freshly-prepared MGTA-456 CD34+ cell therapy investigational product in replacement of single umbilical cord blood transplantation	MGTA-456 Cohort 2 n=2 Participants Patients receiving cryopreserved MGTA-456 CD34+ cell therapy investigational product in replacement of single umbilical cord blood transplantation
Number of Participants With Engraftment	6 Participants	2 Participants

SECONDARY outcome

Timeframe: 48 hours

Population: All patients who received MGTA-456 investigational product.

Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration

Outcome measures

Measure	MGTA-456 Cohort 1 n=6 Participants Patients receiving freshly-prepared MGTA-456 CD34+ cell therapy investigational product in replacement of single umbilical cord blood transplantation	MGTA-456 Cohort 2 n=2 Participants Patients receiving cryopreserved MGTA-456 CD34+ cell therapy investigational product in replacement of single umbilical cord blood transplantation
Number of Participants With Infusion Toxicities Nausea	5 Participants	1 Participants
Number of Participants With Infusion Toxicities Hypoalbuminemia	4 Participants	1 Participants
Number of Participants With Infusion Toxicities Anemia	4 Participants	1 Participants
Number of Participants With Infusion Toxicities Hypertension	4 Participants	1 Participants
Number of Participants With Infusion Toxicities Hypocalcemia	3 Participants	1 Participants
Number of Participants With Infusion Toxicities Leukopenia	4 Participants	0 Participants

Adverse Events

MGTA-456

Serious events: 8 serious events

Other events: 8 other events

Deaths: 2 deaths

Serious adverse events

Measure	MGTA-456 n=8 participants at risk MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. .
Blood and lymphatic system disorders Autoimmune hemolytic anemia	37.5% 3/8 • 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.
Blood and lymphatic system disorders Immune thrombocytopenia	37.5% 3/8 • 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.
General disorders Pyrexia	37.5% 3/8 • 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.
Infections and infestations Pneumonia	25.0% 2/8 • 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.
Infections and infestations Sepsis	25.0% 2/8 • 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.
Infections and infestations Staphylococcal bacteremia	25.0% 2/8 • 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.
Blood and lymphatic system disorders Autoimmune pancytopenia	25.0% 2/8 • 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.

Other adverse events

Measure	MGTA-456 n=8 participants at risk MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. .
Blood and lymphatic system disorders Anemia	87.5% 7/8 • 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.
Blood and lymphatic system disorders Neutropenia	87.5% 7/8 • 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.
Blood and lymphatic system disorders Thrombocytopenia	87.5% 7/8 • 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.
Vascular disorders Hypertension	87.5% 7/8 • 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.
Gastrointestinal disorders Constipation	75.0% 6/8 • 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.
Gastrointestinal disorders Nausea	75.0% 6/8 • 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.
General disorders Pyrexia	75.0% 6/8 • 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.
Blood and lymphatic system disorders Immune thrombocytopenia	62.5% 5/8 • 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.
Metabolism and nutrition disorders Hypoalbuminemia	62.5% 5/8 • 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.
Blood and lymphatic system disorders Leukopenia	62.5% 5/8 • 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.

Additional Information

Medical Director

Magenta Therapeutics

Phone: (857) 242-0170

Email: clinicaltrials@magentatx.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place