Stem Cell Factor Medication for Aplastic Anemia

This study has been completed.
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00001398
First received: November 3, 1999
Last updated: March 3, 2008
Last verified: June 2002
  Purpose

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.


Condition Intervention Phase
Aplastic Anemia
Pancytopenia
Drug: Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Primary Purpose: Treatment
Official Title: A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 40
Study Start Date: October 1993
Estimated Study Completion Date: June 2002
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

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00001398

Locations
United States, Maryland
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00001398     History of Changes
Other Study ID Numbers: 940016, 94-H-0016
Study First Received: November 3, 1999
Last Updated: March 3, 2008
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Hematopoiesis
Stem Cell Cycling
Bone Marrow Failure
Acquired Aplastic Anemia

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Pancytopenia
Bone Marrow Diseases
Hematologic Diseases

ClinicalTrials.gov processed this record on October 21, 2014