Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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TERMINATED
PHASE1/PHASE2
9 participants
INTERVENTIONAL
2007-01-02
2016-08-01
Brief Summary
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Detailed Description
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* overall survival
* event-free survival (events are defined as: death,non-engraftment/2nd transplant, immune reconstitution failure)
* acute toxicity of the conditioning regimen
* engraftment frequency immune reconstitution frequency and tempo acute and chronic graft-versus-host disease (GVHD), frequency and severity.
The outcome from this protocol will be compared to the retrospective cohort consisting of all patients who have undergone haplo-identical HSCT for SCID at CHLA from 1984-2006 based on the assessment of the above-listed endpoints.
The CliniMACS device will be used for CD34+ selection in place of the Isolex 300i. The CliniMACS CD34 Reagent System is an investigational medical device that has not yet been approved by the FDA. This device is used in vitro to select and enrich specific cell populations. When using the CliniMACS CD34 Reagent, the system selects CD34+ cells from heterogenous hematological cell populations for transplantation in cases where this is clinically indicated.
Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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unrelated BM with T cell depletion
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion
Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
unrelated cord blood
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood
Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
haplo BM with T cell depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion
haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
unrelated PBSC with T cell depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion
peripheral blood stem cell will be processed for CD34+ cell isolation.
Interventions
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unrelated BM with T cell depletion
Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
unrelated cord blood
Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
haplo BM with T cell depletion
haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
unrelated PBSC with T cell depletion
peripheral blood stem cell will be processed for CD34+ cell isolation.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Eligible patients must have adequate physical function to tolerate the chemotherapy conditioning regimen and the HSCT, as measure by:
1. Renal: creatinine clearance or GFR ≥50 ml/min/1.73m2, and not requiring dialysis
2. Pulmonary: Because patients with SCID frequently present with infectious pneumonia causing ventilatory failure, patients will be considered for enrollment in the study even if respiratory failure requiring mechanical ventilatory support is present. In patients recently diagnosed with pneumonia, efforts to stabilize the respiratory status will be made prior to enrollment in the study.
3. Infectious disease status. The presence of infection per se will not be a reason for exclusion from the study. Patients with SCID are frequently infected with both routine pathogens as well as opportunistic infections. Antibiotic, antifungal and antiviral prophylaxis and therapy will be instituted as clinically indicated. Despite the use of antimicrobial therapy, the ability to control infections will not be achieved unless HSCT is performed. Therefore, subjects may be enrolled in the study, even though infection is present, because control of infection may depend on engraftment of a donor immune system.
4. Patients will be 0-21 years of age.
Exclusion Criteria
* End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning.
* Renal failure requiring dialysis
* Congenital heart disease resulting in congestive heart failure
* Severe CNS disease, e.g., coma or intractable seizures
* Ventilatory failure due to non-infectious etiology
* Major congenital anomalies that adversely affect survival, eg CNS malformations
* Metabolic diseases that would affect transplant survival, eg urea cycle disorders
* HIV infection
Since the only chance of survival for patients with SCID is successful transplantation, all patients with SCID will be considered to be potential subjects for the study, regardless of end-organ dysfunction.
21 Years
ALL
No
Sponsors
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Neena Kapoor, M.D.
OTHER
Responsible Party
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Neena Kapoor, M.D.
Professor of Pediatrics, Keck School of Medicine; Division Head, Division of Research Immunology/BMT
Principal Investigators
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Neena Kapoor, M.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Los Angeles, University of Southern California
Locations
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Children's Hospital Los Angeles
Los Angeles, California, United States
Countries
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Other Identifiers
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CCI-06-00243
Identifier Type: -
Identifier Source: org_study_id