Stem Cell Factor Medication for Aplastic Anemia - Tabular View

Tracking Information
First Received Date ^ICMJE	November 3, 1999
Last Updated Date	March 3, 2008
Start Date ^ICMJE	October 1993
Primary Completion Date
Current Primary Outcome Measures ^ICMJE
Original Primary Outcome Measures ^ICMJE
Change History	Complete list of historical versions of study NCT00001398 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE
Original Secondary Outcome Measures ^ICMJE

Descriptive Information
Brief Title ^ICMJE	Stem Cell Factor Medication for Aplastic Anemia
Official Title ^ICMJE	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 I
Study Type ^ICMJE	Interventional
Study Design ^ICMJE	Treatment, Safety Study
Condition ^ICMJE	Aplastic Anemia Pancytopenia
Intervention ^ICMJE	Drug: Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
Study Arms / Comparison Groups
Publications *	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. 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. No abstract available. 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.
* 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 ^ICMJE	Completed
Enrollment ^ICMJE	40
Completion Date	June 2002
Primary Completion Date
Eligibility Criteria ^ICMJE	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	Both
Ages
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT ID ^ICMJE	NCT00001398
Responsible Party
Study ID Numbers ^ICMJE	940016, 94-H-0016
Study Sponsor ^ICMJE	National Heart, Lung, and Blood Institute (NHLBI)
Collaborators ^ICMJE
Investigators ^ICMJE
Information Provided By	National Institutes of Health Clinical Center (CC)
Verification Date	June 2002
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP