Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affect…

Organic AcidemiaMethylmalonic AcidemiaInborn Errors of Metabolism

~Estimated

~$10 – $675

National Human Genome Research Institute (NHGRI)NCT00078078

Phase 1

Phase 1/2 Study: CD45RA Depleted Peripheral Stem Cell Addback to Prevent Viral and Fungal Infections Following Alternative Donor TCRab/CD19 Depleted Hematopoietic Stem Cell Transplant

The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graf…

LeukemiaHigh Risk Acute Lymphoblastic LeukemiaHigh Risk Acute Myeloid Leukemia+11 more

~Estimated

~$10 – $6,250

Children's Hospital of PhiladelphiaNCT06839456

Phase 1

Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells

The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allo…

LeukemiaInborn Errors of MetabolismBone Marrow Failure Syndromes+2 more

~Estimated

~$10 – $6,250

Children's Hospital of PhiladelphiaNCT02356653