4 recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Inherited BMF Syndrome: new trials by email Literature Review of Inherited BMF Syndrome trials Research Report on Inherited BMF Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3 - Leukemia, High Risk Acute Lymphoblastic Leukemia | Phase 1 / Phase 2 | Recruiting | Children's Hospital of Philadelphia | 100 | 2025-03-21 | 2026-04-15 |
| Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform Shwachman-Diamond Syndrome, SDS | Recruiting | Shwachman-Diamond Syndrome Alliance Inc | 8000 | 2024-02-07 | 2025-05-31 | |
| Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study) whole genome and transcriptome sequencing - Inherited BMF Syndrome, Inherited Platelet Disorder | Recruiting | Peter MacCallum Cancer Centre, Australia | 350 | 2022-03-18 | 2024-11-07 | |
| Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes The whole-exome sequencing - Inherited BMF Syndrome | Recruiting | Sohag University | 250 | 2022-01-10 | 2022-07-01 |
Congenital Bone Marrow Failure Syndromes (4) · Bone Marrow Failure Disorders (2) · Immunologic Deficiency Syndromes (2) · Pancytopenia (2) · Anemia, Refractory, with Excess of Blasts (1) · Exocrine Pancreatic Insufficiency (1) · Hematologic Diseases (1) · Hemoglobinopathies (1) · Leukemia (1) · Lymphoma, Follicular (1) · Lymphoma, Non-Hodgkin (1) · Metabolism, Inborn Errors (1) · Myelodysplastic Syndromes (1) · Neoplastic Syndromes, Hereditary (1) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (1)
Bone Marrow Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Hemic and Lymphatic Diseases (4) · Infant, Newborn, Diseases (4) · Hematologic Diseases (3) · Bone Marrow Failure Disorders (2) · Cytopenia (2) · Genetic Diseases, Inborn (2) · Immune System Diseases (2) · Immunoproliferative Disorders (2) · Lymphatic Diseases (2) · Lymphoma (2) · Lymphoproliferative Disorders (2) · Metabolic Diseases (2) · Neoplasms (2)
Base Sequence (1) · Clinical Trials, Phase I as Topic (1)
Children's Hospital of Philadelphia (1) · Peter MacCallum Cancer Centre, Australia (1) · Shwachman-Diamond Syndrome Alliance Inc (1) · Sohag University (1)
United States (2) · Australia (1) · Egypt (1)
Mahmoud I Yousef, PhD (1) · Piers Blombery, MBBS(Hons) (1) · Timothy Olson, MD, PhD (1)