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Trial record 7 of 11 for:    hlhs cord blood

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

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ClinicalTrials.gov Identifier: NCT00730314
Recruitment Status : Completed
First Posted : August 8, 2008
Last Update Posted : June 23, 2016
Sponsor:
Information provided by (Responsible Party):
Hisham Abdel-Azim, Children's Hospital Los Angeles

Tracking Information
First Submitted Date  ICMJE August 6, 2008
First Posted Date  ICMJE August 8, 2008
Last Update Posted Date June 23, 2016
Study Start Date  ICMJE August 2008
Actual Primary Completion Date August 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 27, 2010)
  • toxicities [ Time Frame: 3 years ]
  • adverse events [ Time Frame: 3 years ]
  • engraftment [ Time Frame: 1 year ]
  • immune reconstitution [ Time Frame: 3 years ]
  • overall and event free survival survival [ Time Frame: 3 years ]
Original Primary Outcome Measures  ICMJE
 (submitted: August 7, 2008)
toxicities, adverse events, engraftment,immune reconstitution,overall and event free survival survival. [ Time Frame: 1 year ]
Change History Complete list of historical versions of study NCT00730314 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Official Title  ICMJE 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
Brief Summary

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.

Detailed Description

This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD) or 2) unrelated umbilical cord blood (UCB). Patients will receive a novel conditioning regimen with Busulfan, Cytoxan and Fludarabine (Bu/Cy/Flu) and either Alemtuzumab (Campath 1H) for recipients of a MUD or rabbit Antithymocyte Globulin (rATG) for recipients of unrelated UCB prior to hematopoietic stem cell transplant (HSCT).

It is hypothesized that reduced dosages of Cytoxan will decrease the acute toxicities associated with the standard chemotherapies of Busulfan and Cytoxan (i.e. sinusoidal obstructive syndrome (SOS), hemorrhagic cystitis and mucositis). And the addition of fludarabine to a conditioning regimen with myeloablative doses of Busulfan and reduced dosages of Cytoxan prior to HSCT will overcome the engraftment barrier posed by an intact immune system, which is seen in patients with a genetic disease.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Disease
  • Thalassemia
  • Anemia
  • Granuloma
  • Wiskott-Aldrich Syndrome
  • Chediak Higashi Syndrome
  • Osteopetrosis
  • Neutropenia
  • Thrombocytopenia
  • Hurler Disease
  • Niemann-Pick Disease
  • Fucosidosis
Intervention  ICMJE Procedure: Hematopoietic stem cell transplantation
hematopoietic stem cell transplantation conditioning regimen depending on graft source
Study Arms  ICMJE
  • Experimental: 1
    Unrelated donor
    Intervention: Procedure: Hematopoietic stem cell transplantation
  • Experimental: 2
    Cord Blood
    Intervention: Procedure: Hematopoietic stem cell transplantation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 7, 2008)
25
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE August 2015
Actual Primary Completion Date August 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00730314
Other Study ID Numbers  ICMJE CCI #07-00119
CHLA-#07-00119
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Hisham Abdel-Azim, Children's Hospital Los Angeles
Study Sponsor  ICMJE Children's Hospital Los Angeles
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Hisham Abdel-Azim, MD Childrens Hospital Los Angeles, University of Southern California
PRS Account Children's Hospital Los Angeles
Verification Date June 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP