Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission

NCT00011830 | Completed Phase 1

• 2 other identifiers: 01008301-H-0083
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This study will examine 1) whether it is possible to collect enough stem cells (cells produced by the bone marrow that mature into white and red blood cells and platelets) from patients with aplastic anemia to use for future treatment, and 2) whether patients who have been treated successfully and relapse will benefit from autologous stem cell transfusion (transfusion of their own stem cells). Patients 12 years of age or older with aplastic anemia who have been successfully treated with immunosuppressive drugs and are now in remission may be eligible for this study. Participants will undergo a complete history and physical examination, bone marrow biopsy (removal of a small sample of bone marrow from the hip bone) and blood tests, plus procedures to collect stem cells, as follows:

• G-CSF (Filgrastim) administration - G-CSF will be given by injection under the skin daily for up to 10 days. This drug causes stem cells to move from the marrow into the blood where they can be collected more easily.
• Apheresis - Stem cells will be collected through apheresis, usually starting the 5th to 6th day of Filgrastim injections. For this procedure, whole blood is collected through a needle in an arm vein. The blood circulates through a cell separator machine where the white cells and stem cells are removed. The red cells, platelets and plasma are returned to the body through a second needle in the other arm. The procedure takes about 5 hours. Up to five procedures, done on consecutive days, may be required to collect enough cells for transplantation. If enough cells are collected, they will be purified (treated to remove the white blood cells) using an experimental device. Removing the lymphocytes may reduce the chance of relapse of aplastic anemia following the stem cell transplant. The stem cells will be frozen for later use, if needed.
• Follow-up - Participants are followed at NIH at 6-month intervals.

Conditions

Aplastic Anemia

Keywords

Stem Cell Cryopreservation; Leukapheresis

Interventions

G-CSF DRUG

Apheresis PROCEDURE

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• History of severe AA as defined by a hypocellular bone marrow and depression of two out of three peripheral counts as indicated below:
• ANC less than 0.5 x 10 (9)/L;
• platelet count less than 20 x10 (9)/L,
• reticulocyte count less than 60 x 10 (9)/L.
• Demonstrated hematologic response to first or second course of immunosuppression or growth factors or exhibit a spontaneous remission as defined by all peripheral counts as indicated below (must be at least 3 months following the initial course of immunosupressive or growth factor therapy and must be sustained for at least 3 week)
• ANC greater than 1.5 x 10 (9)/L
• platelet count greater than 80 x10 (9)/L
• hemoglobin greater than 10 g/dl (not transfused)
• Weight \> 18 kg
• Age greater than or equal to 2 years
• Able to comprehend the investigational nature of the protocol and be willing to sign an informed consent/assent.

You may not qualify if:

• Current diagnosis or past history of myelodysplastic syndrome, Fanconis anemia, dyskeratosis congenita or other congenital forms of aplastic anemia.
• Evidence of uncontrolled infection
• ECOG performance status of 2 or more
• Inadequate organ function as defined:
• bilirubin greater than 4.0 mg/dl and
• transaminases greater than than 2 x ULN.
• Current therapy for malignancy
• HIV infection
• Unfit to receive G-CSF and undergo apheresis (uncontrolled hypertension, currently active ischemic heart disease, unstable arrhythmia, history of chest pain, myocardial infarction, peripheral vascular disease, transient ischemic attack, or stroke).
• Psychiatric, affective or any other disorder that would compromise ability to give informed consent
• Moribund or patients with concurrent hepatic, renal, cardiac, metabolic disease of such severity that death within 1-4 weeks from the initiation of the therapy is likely.
• An enlarged spleen by physical exam.
• Pregnant or lactating.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Heart, Lung and Blood Institute (NHLBI)

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Young NS, Barrett AJ. The treatment of severe acquired aplastic anemia. Blood. 1995 Jun 15;85(12):3367-77. No abstract available.

  PMID: 7780125 BACKGROUND

• Zoumbos NC, Gascon P, Djeu JY, Trost SR, Young NS. Circulating activated suppressor T lymphocytes in aplastic anemia. N Engl J Med. 1985 Jan 31;312(5):257-65. doi: 10.1056/NEJM198501313120501.

  PMID: 2981406 BACKGROUND

• Young NS, Maciejewski J. The pathophysiology of acquired aplastic anemia. N Engl J Med. 1997 May 8;336(19):1365-72. doi: 10.1056/NEJM199705083361906. No abstract available.

  PMID: 9134878 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Granulocyte Colony-Stimulating Factor; Blood Component Removal

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: February 28, 2001
• First Posted: March 1, 2001
• Study Start: February 1, 2001
• Study Completion: February 1, 2006
• Last Updated: March 4, 2008

Record last verified: 2006-02

Locations

US (1)