Study Stopped
Due to low enrollment
A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
IMD-002
A Follow-Up Study to Evaluate the Safety and Clinical Outcomes of Patients With Non-Malignant Disease Who Have Undergone Hematopoietic Stem Cell Transplantation With MGTA-456
1 other identifier
observational
3
1 country
1
Brief Summary
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started May 2019
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 21, 2019
CompletedFirst Submitted
Initial submission to the registry
May 23, 2019
CompletedFirst Posted
Study publicly available on registry
July 5, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 8, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
October 8, 2020
CompletedMarch 15, 2021
March 1, 2021
1.4 years
May 23, 2019
March 10, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (10)
Incidence of related adverse events
2 years
Incidence of serious adverse events
2 years
Incidence of late hematological graft failure
2 years
Incidence of chronic graft versus host disease
2 years
Overall survival
2 years
Event-free survival
2 years
Change in cALD Neurologic Function Score over time
2 years
Proportion of subjects without gadolinium enhancement on MRI over time
2 years
Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients
2 years
Very long chain fatty acid blood level (ug/mL) in cALD patients
2 years
Study Arms (1)
Subjects treated with MGTA-456
MGTA-456 is an investigational expanded CD34+ cell therapy
Interventions
Long term safety and clinical outcomes
Eligibility Criteria
Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456
You may qualify if:
- An Institutional Review Board (IRB)/Independent Ethics Committee-approved written informed consent form must be signed and dated by the patient or legal guardian. Study assents will also be prepared for children and adolescents to review when applicable.
- Patient completed an IMD study in the MGTA-456 program and was administered MGTA-456 for HSCT.
You may not qualify if:
- Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Biospecimen
Peripheral blood mononuclear cells \& plasma
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Paul J Orchard, MD
University of Minnesota
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 23, 2019
First Posted
July 5, 2019
Study Start
May 21, 2019
Primary Completion
October 8, 2020
Study Completion
October 8, 2020
Last Updated
March 15, 2021
Record last verified: 2021-03
Data Sharing
- IPD Sharing
- Will not share