Anemia Clinical Trial
Official 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
Verified date | June 2016 |
Source | Children's Hospital Los Angeles |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Institutional Review Board |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 25 |
Est. completion date | August 2015 |
Est. primary completion date | August 2015 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 21 Years |
Eligibility |
Inclusion Criteria: - 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). Exclusion Criteria: - 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). |
Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Children Hospital Los Angeles | Los Angeles | California |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital Los Angeles |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | toxicities | 3 years | Yes | |
Primary | adverse events | 3 years | Yes | |
Primary | engraftment | 1 year | Yes | |
Primary | immune reconstitution | 3 years | Yes | |
Primary | overall and event free survival survival | 3 years | Yes |
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