Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00011505
• Date of registration: 22/02/2001
• Prospective Registration: No
• 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

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)

Procedure: Leukapheresis

Drug: G-CSF

Secondary ID(s)

01-H-0097

010097

Source(s) of Monetary Support

Please refer to primary and secondary sponsors