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hematopoietic stem cell transplantation clinical trial intelligence

Trials are included when the normalized target field matches this target directory. Full protocol, result, eligibility and IP analysis is available inside the premium workspace.

Validated reports

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target

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normalized field

NCT00730314
Source recordAI-normalized

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

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.

AI analysis

Indication: Sickle Cell Disease

Modality: cell therapy

Target: Hematopoietic stem cell transplantation

Sponsor: Children's Hospital Los Angeles

Source URL: ClinicalTrials.gov

Source updated: Jun 23, 2016

Ingested: Jun 19, 2026

Model: trialsignal-ai-v1

Validation: validated

Matched by target_normalized: Hematopoietic stem cell transplantation

View original source fields

Condition raw: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis

Condition normalized: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis

Modality raw: cell therapy

Modality normalized: cell therapy

Target raw: Hematopoietic stem cell transplantation

Target normalized: Hematopoietic stem cell transplantation

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