SCID Bu/Flu/ATG Study With T Cell Depletion

Stopped early · Phase 1/Phase 2

Conditions studied: Severe Combined Immunodeficiency

In brief

This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD), 2) unrelated cord blood donor, or 3) a haplo-identical (parental) donor (in descending order of preference).Patients will receive a novel conditioning regimen with Busulfan, Fludarabine and Anti-thymocyte globulin (ATG) followed by an unrelated donor hematopoietic stem cell transplant (HSCT) with T-cell depletion using the CliniMACS device.

Key facts

Study ID

NCT02127892

Run by

Neena Kapoor, M.D.

People needed

9

Starts

2007-01-02

Expected to finish

2016-08-01

Last updated by the study team

2017-09-18

Who can join

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

You may qualify if…

• All patients with SCID who lack a histocompatible sibling or HLA-matched related donor will be considered as candidates for this study protocol.
• Eligible patients must have adequate physical function to tolerate the chemotherapy conditioning regimen and the HSCT, as measure by:
• Renal: creatinine clearance or GFR ≥50 ml/min/1.73m2, and not requiring dialysis
• 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.
• 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.
• Patients will be 0-21 years of age.

You may not qualify if…

• Patient with histocompatible sibling or other related donor
• 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.

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.