TerminatedNot Applicable

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Stopped: Due to low enrollment

United States3 participantsStarted 2019-05-21

Plain-language 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

Who can participate

Age range16 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 Exclusion Criteria: •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.

What they're measuring

Incidence of related adverse events

Timeframe: 2 years

Incidence of serious adverse events

Timeframe: 2 years

Incidence of late hematological graft failure

Timeframe: 2 years

Incidence of chronic graft versus host disease

Timeframe: 2 years

Overall survival

Timeframe: 2 years

Event-free survival

Timeframe: 2 years

Change in cALD Neurologic Function Score over time

Timeframe: 2 years

Proportion of subjects without gadolinium enhancement on MRI over time

Timeframe: 2 years

Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients

Trial details

NCT IDNCT04008849

SponsorMagenta Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2020-10-08

View on ClinicalTrials.gov More Inherited Metabolic Disorders (IMD) trials