Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

NCT00004464 | Completed Phase 2 FDA Drug

• 5 other identifiers: J961196-01-17-02JHOC-96011702JHOC-9611199/13895
• Study Type: interventional
• Target: 69
• Locations: 1 country
• Sites: 1

Brief Summary

OBJECTIVES: I. Confirm the efficacy demonstrated in a pilot study using high dose cyclophosphamide in patients with severe aplastic anemia. II. Determine whether the addition of filgrastim (G-CSF) to high dose cyclophosphamide shortens the time to recovery in these patients. III. Determine whether this regimen is efficacious in treating paroxysmal nocturnal hemoglobinuria.

Conditions

Aplastic Anemia; Paroxysmal Hemoglobinuria, Nocturnal

Keywords

aplastic anemia; hematologic disorders; paroxysmal nocturnal hemoglobinuria; rare disease

Interventions

cyclophosphamide DRUG

filgrastim DRUG

Eligibility Criteria

• Age: Up to 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Acquired severe aplastic anemia or paroxysmal nocturnal hemoglobinuria * Not a candidate for allogeneic bone marrow transplantation * Must meet one of the following criteria: * Severe aplastic anemia * Less than 25% bone marrow cellularity and depression in two of three blood counts (reticulocytes less than 40,000/mm3 * platelet count less than 20,000/mm3 and granulocytes less than 500/mm3) * Life-threatening paroxysmal nocturnal hemoglobinuria * Absolute neutrophil count less than 500/mm3 * platelet transfusion dependent * thrombotic disease * No Fanconi anemia * No abnormal cytogenetics --Patient Characteristics-- * Renal: Creatinine no greater than 2.0 mg/dL * Cardiovascular: Cardiac ejection fraction at least 45% * Other: Not preterminal or moribund Not pregnant

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead

Study Sites (1)

Johns Hopkins Oncology Center

Baltimore, Maryland, 21231, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic; Hemoglobinuria, Paroxysmal; Hematologic Diseases; Rare Diseases

Interventions

Cyclophosphamide; Filgrastim

Condition Hierarchy (Ancestors)

Anemia; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Anemia, Hemolytic; Myelodysplastic Syndromes; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Robert A. Brodsky

  Johns Hopkins University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Purpose: TREATMENT
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: February 1, 1996
• Primary Completion: March 1, 2007
• Study Completion: November 11, 2008
• Last Updated: January 18, 2019

Record last verified: 2018-08

Locations

US (1)