Clinical trial NCT00001398: Stem Cell Factor Medication for Aplastic Anemia
Official title: A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Aplastic Anemia
Pancytopenia
Keywords: Hematopoiesis
Stem Cell Cycling
Bone Marrow Failure
Acquired Aplastic Anemia
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 1999-11-04
Enrollment: 40
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Heart, Lung and Blood Institute (NHLBI)
state: Maryland
Study officials:
References:
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. No abstract available.
citation: 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. doi: 10.1056/NEJM198203183061105. No abstract available.
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.