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NCT00730314COMPLETEDanonymous

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

Sponsor

Source record

Children's Hospital Los Angeles

Phase

Source record

PHASE1

Modality

AI-normalized

cell therapy

Target

AI-normalized

Hematopoietic stem cell transplantation

Indication / condition

AI-normalized

Sickle Cell Disease

Intervention

Source record

Hematopoietic stem cell transplantation

Source & freshness

Source record

NCT ID

NCT00730314

Original source

ClinicalTrials.gov

Source last updated

Jun 23, 2016

Ingested at

Jun 19, 2026

Internal sync

Jun 19, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

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NCT ID

NCT00730314

Title

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

Sponsor

Children's Hospital Los Angeles

Status

COMPLETED

Phase

PHASE1

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

Interventions

Hematopoietic stem cell transplantation

Public preview

Source record

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-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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