TrialSignal
Clinical trial intelligence report
Phase I/II Trial Of Hematopoietic Stem Cell Transplant (HSCT) For Children With A Genetic Disease Of Blood Cells Without An HLA-Matched Sibling Donor
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jun 19, 2026
NCT ID
NCT00730314
Status
COMPLETED
Phase
PHASE1
Sponsor
Children's Hospital Los Angeles
Executive brief
Investment-Ready Snapshot
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease, Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia), Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar conditions. The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT00730314
Indication
Sickle Cell Disease
Modality
cell therapy
Target
Hematopoietic stem cell transplantation
Intervention
Hematopoietic stem cell transplantation
Source record
Protocol Description
Detailed source ingestion pending.
Source record
Outcome Measures
Detailed source ingestion pending.
Source record
Eligibility
Detailed source ingestion pending.
AI analysis
Known Results And Readout Context
Detailed source ingestion pending.
IP intelligence
Patent And IP Landscape
Detailed source ingestion pending.
Source record
Contacts
Detailed source ingestion pending.