Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Completed · Phase 1/Phase 2

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

In brief

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.

Key facts

Study ID: NCT00730314
Run by: Children's Hospital Los Angeles
People needed: 25
Starts: 2008-08-01
Expected to finish: 2015-08-01
Last updated by the study team: 2016-06-23

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Lethal or sublethal genetic disease of blood cells, who lack a fully histocompatible sibling or other family donor
Genetic diseases that would be candidates for this protocol includes those that have been shown to benefit from allogeneic HSCT: Red blood cell defects, Leukocyte defects/ Primary immune deficiencies, Platelets defects, Metabolic/storage disorders and Stem cell defects.
Renal: creatinine clearance or glomerular filtration rate (GFR) ≥50 ml/min/1.73m2 and not requiring dialysis.
Pulmonary: FEV1, FVC and DLCO (corrected for hemoglobin) ≥ 50% predicted. if unable to perform pulmonary function tests, then O2 saturation ≥ 92% in room air.
Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction ≥ 26%
Hepatic: Bilirubin ≤3x upper limit of normal (ULN) and ALT and AST ≤ 5x for age (with the exception of isolated hyperbilirubinemia due to Gilbert's syndrome).
Patients will be 0-21 years of age.
Disease specific inclusion criteria (as applicable per protocol).

You may not qualify if…

Recipients should not have any of the general exclusion criteria, and disease specific exclusion criteria when applicable.
Patient with histocompatible sibling
End-organ failure that precludes the ability to tolerate the transplant procedure, including the conditioning regimen.
Creatinine clearance or GFR < 50 ml/min/1.73m2 or renal failure requiring dialysis.
Congenital heart disease resulting in congestive heart failure.
Severe residual CNS disease/impairment [(other than hemiplegia alone) e.g. coma or intractable seizures]
Ventilatory failure
Major congenital anomalies that adversely affect survival, e.g. CNS malformations
Lansky score < 40% or Karnofsky score < 60%
HIV seropositivity
Diagnosis of Fanconi's anemia, Severe Combined Immunodeficiency (SCID)
Positive pregnancy test (For female patients in child bearing period)
Uncontrolled bacterial, viral, or fungal infections (currently taking medication yet clinical symptoms progress)
Disease specific exclusion criteria (as applicable per protocol).

Where it is running

Children Hospital Los Angeles — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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