Stem Cell Factor Medication for Aplastic Anemia

A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia

NCT00001398•National Heart, Lung, and Blood Institute (NHLBI)

View on ClinicalTrials.gov

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

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion 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.
+13 more criteria

Locations (1)

National Heart, Lung and Blood Institute (NHLBI)

Bethesda, Maryland, United States

Key Dates

Start Date

October 1, 1993

Completion Date

June 1, 2002

Last Updated

March 4, 2008

Enrollment

Estimated participants

Conditions

Aplastic AnemiaPancytopenia

Interventions

Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)

DRUG

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05012111

Severe Aplastic AnemiaTelomere Biology Disorders+1 more

View study

RECRUITINGPHASE2

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 4, 2008Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Stem Cell Factor Medication for Aplastic Anemia

Inclusion Criteria

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Revolade Tablets Specified Drug-use Survey

National Longitudinal Cohort of Hematological Diseases

A Prediction Model in Pregnant Women With Aplastic Anemia

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia