Stem Cell Factor Medication for Aplastic Anemia

Aplastic Anemia Clinical Trial

Official title:

A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00001398
• Other study ID #: 940016
• Secondary ID: 94-H-0016
• Status: Completed
• Phase: Phase 1
• First received: November 3, 1999
• Last updated: March 3, 2008
• Start date: October 1993
• Est. completion date: June 2002

Study information

• Verified date: June 2002
• Source: National Institutes of Health Clinical Center (CC)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the effect of r-metHuSCF on peripheral blood counts. Potential effects of r-metHuSCF on frequency of need for red cell or platelet transfusions and on bone marrow morphology/cellularity will also be evaluated.

Description:

This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the effect of r-metHuSCF on peripheral blood counts. Potential effects of r-metHuSCF on frequency of need for red cell or platelet transfusions and on bone marrow morphology/cellularity will also be evaluated.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: June 2002
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Acquired moderate or severe aplastic anemia.

Intolerant to, or failure to durably respond to, ATG/ALG therapy with or without cyclosporin.

Patients may not have received ATG/ALG, therapy for 12 weeks prior to enrollment or cyclosporin for 4 weeks prior to enrollment.

Two years of age or older.

Karnofsky Performance Status greater than or equal to 60 percent.

Adequate organ function as defined by serum creatine less than 2.0 mg/dl and a bilirubin less than 2.0 mg/dl.

Patients (or their parent[s]/responsible guardian[s]) must be able to comprehend and be willing to sign an informed consent prior to starting r-metHuSCF therapy.

No current diagnosis or past history of myelodysplastic syndromes.

No diagnosis of Fanconi's anemia, dyskeratosis congenita, or other congenital forms of aplastic anemia.

No current diagnosis of clinically active paroxysmal nocturnal hemoglobinuria (PNH) defined as patients with clinically significant thrombosis or hemolysis.

No diagnosis of eosinophilic fasciitis.

No treatment with ATG, ALG, or other immunosuppresive agents within 12 weeks of enrollment or treatment with cyclosporine A or IL-3 within 4 weeks of enrollment.

No treatment with hematopoietic growth factors within 2 weeks of enrollment.

No evidence of active uncontrolled infection.

No known allergy to Ecoli-derived products.

No current or recent symptoms of asthma occurring within the past 10 years (including allergic asthma, or asthma induced by cold temperature, infection, or exercise).

No history of anaphylactic/anaphylactoid-type event manifested by disseminated urticaria, laryngeal edema, and/or bronchospasm (or for example: food, insect bites, etc). Patients with drug allergies, manifested solely by rash and/or urticaria, are not excluded. An isolated episode of urticaria occuring more than 3 years earlier is not a contraindication.

No significant nonmalignant disease including previously documented HIV infection, uncontrolled hypertension (diastolic blood pressure greater than 115 mmHg), unstable angina, congestive heart failure (greater than NY Class II), poorly controlled diabetes, coronary angioplasty within 6 months, or uncontrolled atrial or ventricular cardiac arrhythmias.

No pregnancy or breast feeding. Those of childbearing potential must observe adequate birth control measures.

No treatment with an investigational agent (other than hematopoietic growth factors) within 4 weeks of study entry.

No concurrent use of beta adrenergic blocking agents.

No concurrent use, or use within the past 2 weeks, of Monoamine Oxidase Inhibitors (MAO Inhibitors).

No psychiatric, addictive, or any disorder which compromises ability to give truly informed consent for participation in this study.

Study Design

Endpoint Classification: Safety Study, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Pancytopenia

Intervention

Drug:
• Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)

Locations

Country	Name	City	State
United States	National Heart, Lung and Blood Institute (NHLBI)	Bethesda	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

References & Publications (3)

Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982 Mar 18;306(11):645-52. Review. — View Citation

Camitta BM, Thomas ED, Nathan DG, Gale RP, Kopecky KJ, Rappeport JM, Santos G, Gordon-Smith EC, Storb R. A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia. Blood. 1979 Mar;53(3):504-14. — View Citation

Szklo M, Sensenbrenner L, Markowitz J, Weida S, Warm S, Linet M. Incidence of aplastic anemia in metropolitan Baltimore: a population-based study. Blood. 1985 Jul;66(1):115-9. — View Citation