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出境医 / 临床实验 / 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 in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 (IMD-002)

Study Description
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

Condition or disease Intervention/treatment
Inherited Metabolic Disorders (IMD) Other: Safety and efficacy assessments

Detailed Description:
This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.
Study Design
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Study Type : Observational
Actual Enrollment : 3 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: 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
Actual Study Start Date : May 21, 2019
Actual Primary Completion Date : October 8, 2020
Actual Study Completion Date : October 8, 2020
Arms and Interventions
Group/Cohort Intervention/treatment
Subjects treated with MGTA-456
MGTA-456 is an investigational expanded CD34+ cell therapy
 Other: Safety and efficacy assessments
Long term safety and clinical outcomes
Outcome Measures
Primary Outcome Measures :
  1. Incidence of related adverse events [ Time Frame: 2 years ]
  2. Incidence of serious adverse events [ Time Frame: 2 years ]
  3. Incidence of late hematological graft failure [ Time Frame: 2 years ]
  4. Incidence of chronic graft versus host disease [ Time Frame: 2 years ]
  5. Overall survival [ Time Frame: 2 years ]
  6. Event-free survival [ Time Frame: 2 years ]
  7. Change in cALD Neurologic Function Score over time [ Time Frame: 2 years ]
  8. Proportion of subjects without gadolinium enhancement on MRI over time [ Time Frame: 2 years ]
  9. Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients [ Time Frame: 2 years ]
  10. Very long chain fatty acid blood level (ug/mL) in cALD patients [ Time Frame: 2 years ]

Biospecimen Retention:   Samples With DNA
Peripheral blood mononuclear cells & plasma

Eligibility Criteria
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Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

•Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.

Contacts and Locations

Locations
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United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Magenta Therapeutics, Inc.
Investigators
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Principal Investigator: Paul J Orchard, MD University of Minnesota
Tracking Information
First Submitted Date May 23, 2019
First Posted Date July 5, 2019
Last Update Posted Date March 15, 2021
Actual Study Start Date May 21, 2019
Actual Primary Completion Date October 8, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 22, 2020)
  • Incidence of related adverse events [ Time Frame: 2 years ]
  • Incidence of serious adverse events [ Time Frame: 2 years ]
  • Incidence of late hematological graft failure [ Time Frame: 2 years ]
  • Incidence of chronic graft versus host disease [ Time Frame: 2 years ]
  • Overall survival [ Time Frame: 2 years ]
  • Event-free survival [ Time Frame: 2 years ]
  • Change in cALD Neurologic Function Score over time [ Time Frame: 2 years ]
  • Proportion of subjects without gadolinium enhancement on MRI over time [ Time Frame: 2 years ]
  • Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients [ Time Frame: 2 years ]
  • Very long chain fatty acid blood level (ug/mL) in cALD patients [ Time Frame: 2 years ]
Original Primary Outcome Measures
 (submitted: July 3, 2019)
  • Incidence of related adverse events [ Time Frame: 5 years ]
  • Incidence of serious adverse events [ Time Frame: 5 years ]
  • Incidence of late hematological graft failure [ Time Frame: 5 years ]
  • Incidence of chronic graft versus host disease [ Time Frame: 5 years ]
  • Overall survival [ Time Frame: 5 years ]
  • Event-free survival [ Time Frame: 5 years ]
  • cALD Neurologic Function Scale Score over time [ Time Frame: 5 years ]
  • Proportion of subjects without gadolinium enhancement on MRI over time [ Time Frame: 5 years ]
  • Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients [ Time Frame: 5 years ]
  • Very long chain fatty acid blood level (ug/mL) in cALD patients [ Time Frame: 5 years ]
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
Official Title 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
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
Detailed Description This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Peripheral blood mononuclear cells & plasma
Sampling Method Non-Probability Sample
Study Population Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456
Condition Inherited Metabolic Disorders (IMD)
Intervention Other: Safety and efficacy assessments
Long term safety and clinical outcomes
Study Groups/Cohorts Subjects treated with MGTA-456
MGTA-456 is an investigational expanded CD34+ cell therapy
Intervention: Other: Safety and efficacy assessments
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Terminated
Actual Enrollment
 (submitted: June 22, 2020) 		3
Original Estimated Enrollment
 (submitted: July 3, 2019) 		12
Actual Study Completion Date October 8, 2020
Actual Primary Completion Date October 8, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

•Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.
Sex/Gender
Sexes Eligible for Study: All
Ages up to 16 Years   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04008849
Other Study ID Numbers IMD-002
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Magenta Therapeutics, Inc.
Study Sponsor Magenta Therapeutics, Inc.
Collaborators Not Provided
Investigators
Principal Investigator: Paul J Orchard, MD University of Minnesota
PRS Account Magenta Therapeutics, Inc.
Verification Date March 2021

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