Stem Cell Factor Medication for Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00001398
• Date of registration: 03/11/1999
• Prospective Registration: No
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Stem Cell Factor Medication for Aplastic Anemia
• Scientific title: A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia
• Date of first enrolment: October 1993
• Target sample size: 40
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00001398
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion 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.



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Pancytopenia

Intervention(s)

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

Secondary ID(s)

940016

94-H-0016

Source(s) of Monetary Support

Please refer to primary and secondary sponsors