Brief Title
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
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.
Study Phase
Phase 1
Study Type
Interventional
Condition
Aplastic Anemia
Intervention
Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
40
Start Date
October 1993
Completion Date
June 2002
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.
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00001398
Organization ID
940016
Secondary IDs
94-H-0016
Study Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study Sponsor
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Verification Date
June 2002