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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 February 2015
Main ID:  NCT00011505
Date of registration: 22/02/2001
Prospective Registration: Yes
Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Public title: Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia
Scientific title: Investigation of G-CSF-Induced Stem Cell Mobilization Potential in Patients With Diamond-Blackfan Anemia
Date of first enrolment: February 2001
Target sample size: 15
Recruitment status: Completed
URL:  http://clinicaltrials.gov/show/NCT00011505
Study type:  Interventional
Study design:  Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Key inclusion & exclusion criteria

INCLUSION CRITERIA:

Male or female patients greater than or equal to 4 years of age and weighing at least 12
kg (27 lbs) will be included.

Diagnosis of DBA as defined by the onset of anemia before age 2; or, if after age 2, a
positive family history of classical DBA or typical physical anomalies

Low reticulocyte counts (less than 30,000/ul)

Absent or blocked maturation of erythroid precursors in the bone marrow.

Red-cell transfusion dependence

Adequate birth control while on protocol

EXCLUSION CRITERIA:

Female patients that are pregnant or lactating

Neutrophil count less than 1000/uL or platelet count less than 100,000/uL

Positive DEB stimulation test

Evidence of transformation to MDS or AML on pre-study marrow aspiration and biopsy, or
abnormal marrow cytogenetics.

History or evidence of clinically significant autoimmune inflammatory disease or
significant cardiopulmonary disease by history or physical exam

Uncorrected coagulopathy

Renal disease (GFR less than 100mL/min/1/73 M(2)) as estimated for children by Schwartz
formula. Adults (age greater than 21) excluded for serum Cr less than 2.5 mg/dL.

Hepatic disease (bilirubin greater than 2 mg/dL or transaminases greater than 7 times the
upper limit of normal).

Seropositivity for hepatitis B (HbsAg), hepatitis C, or HIV will be excluded.

Other medical illness or condition which, in the opinion of the Investigators, may
contraindicate participation in this study due to risk to patient or comprise of study
integrity.



Age minimum: N/A
Age maximum: N/A
Gender: Both
Health Condition(s) or Problem(s) studied
Diamond Blackfan Anemia
Intervention(s)
Drug: G-CSF
Procedure: Leukapheresis
Primary Outcome(s)
Secondary Outcome(s)
Secondary ID(s)
01-H-0097
010097
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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