Inherited Metabolic Disorders (IMD)

2

Review clinical trials related to Inherited Metabolic Disorders (IMD). Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.

Participants needed: 50
Trial details
Age: 2-60Biological sex: AllType: ObservationalSponsor: Paul SzabolcsUpdated: Jan 13, 2026Locations: 1
Eligibility criteria

Patient, parent, or legal guardian must have given written informed consent. [+36]

Status: Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.

Participants needed: 100
Trial details
Phase: Phase 2Age: 2-55Biological sex: AllType: InterventionalSponsor: Paul SzabolcsUpdated: Dec 15, 2025Locations: 1
Eligibility criteria

A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will... [+55]