Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00023192
• Date of registration: 29/08/2001
• Prospective Registration: No
• Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Public title: Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care
• Scientific title: Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care
• Date of first enrolment: August 2001
• Target sample size: 60
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00023192
• Study type: Interventional
• Study design: Endpoint Classification: Efficacy Study, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

United States

Key inclusion & exclusion criteria

INCLUSION CRITERIA

The following conditions must be met before a patient may be enrolled in the study:

Patients ages 2 to 17 years; minimum weight of 12 kg.

DHR proven chronic granulomatous disease with gp91 phox or p22 phox deficiency.

History of at least one life-threatening infection (defined as any infection requiring
treatment with intravenous antibiotic therapy) or a family member with CGD and a history
of life-threatening infection.

Free of active infection.

Patients with consenting HLA-matched related donors that meet donor selection criteria
(test patients), or patients without an eligible HLA-matched related donor (control
patients). Patients with eligible donors who choose not to undergo stem cell
transplantation or patients with eligible but non-consenting donors may be enrolled in the
control arm of the study. Patients enrolled in the control arm who complete a 24 month
follow-up period may enroll in the transplant arm should an eligible, consenting donor
become available.

Patients with adequate organ function as measured by:

Cardiac: Asymptomatic or, if symptomatic, then left ventricular ejection fraction at rest
must be greater than 35%.

Hepatic: SGOT within 4 times normal range and total bilirubin less than 2mg/dL.

Renal: Creatinine clearance greater than or equal to 50 mL/min/ 1.73m(2). A maximum age
adjusted serum creatinine will be used for patients who are unable to provide an accurate
24 hour urine collection. For children less than or equal to 5 years of age, the maximum
serum creatinine (mg/dl) is 0.8; for children whose age is greater than 5 to less than or
equal to 10, the maximum serum creatinine (mg/dl) is 1.0; for children whose age is
greater than 10 to less than or equal to age 15, the maximum serum creatinine (mg/dl) is
1.2; and for children whose age is greater than 15, the maximum serum creatinine (mg/dl)
is 1.5.

Pulmonary: DLCO (diffusion capacity) and FEV1 greater than 45 percent of predicted
(corrected for hemoglobin). Minors in whom pulmonary function tests are not possible will
be evaluated for significant pulmonary dysfunction by a pulmonary consultant.

Written informed consent/assent conforming to institutional guidelines obtained from
patient and parent.

Absence of co-existing medical problems that would significantly increase the risk of a
transplant procedure in the judgment of the principal investigator.

EXCLUSION CRITERIA:

Any one of the following conditions eliminates a patient from participating in this
protocol:

Female patients who are pregnant or lactating.

ECOG performance status of 2 or more or less than 50 percent on the Lansky scale for ages
0-10.

Seropositivity for HIV due to excessive risk of infection and neurotoxicity of
antiretroviral medications.

Evidence of rapid deterioration due to progressive infection and/or organ damage.

Malignant diseases liable to relapse or progress within 5 years.

DONOR SELECTION:

Donors must be fit to receive G-CSF and give peripheral blood stem cells (normal blood
count, normotensive, no history of stroke, no history of severe heart disease, greater
than 12kg).

Related to the patient and HLA-phenotypically identical with the patient for HLA-A, B and
DRB1 alleles. Matching assessed minimally by serology for Class I and DNA typing for
Class II antigens.

Female x-linked CGD carriers must have greater than 30 percent normal neutrophils.

If donor is a sibling who is a minor, he/ she is the oldest eligible sibling and no adult
siblings are eligible donors.

Written informed consent from donor. Donors who are minors will be evaluated by a social
worker, psychologist or psychiatrist prior to the assent process to determine willingness
to participate. If willingness to participate has been confirmed, informed consent will
be obtained from adult parent or legal guardian. Informed assent will be obtained from
minor donor in the presence of a third party who will assess comprehension and voluntary
participation.



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Chronic Granulomatous Disease

Intervention(s)

Drug: T-Cell Depleted & CD34+Select/w/StemCell Enriched Product

Secondary ID(s)

010242

01-I-0242

Source(s) of Monetary Support

Please refer to primary and secondary sponsors