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Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 1999 by FDA Office of Orphan Products Development.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
Boston University
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004412
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: May 1999
History of Changes
  Purpose

OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers.

II. Determine the effect of arginine butyrate therapy on tissue factors related to promotion or inhibition of wound healing in these patients.

III. Determine whether the regimen used in this study is appropriate for testing in pivotal trials.


Condition Intervention Phase
Skin Ulcers
Sickle Cell Anemia
 Drug: arginine butyrate
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Primary Purpose: Treatment

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 30
Study Start Date: September 1997
Detailed Description:

PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to one of two treatment arms. Arm I: Patients receive arginine butyrate IV over 6-9 hours at night 5 days a week for 12 weeks, plus concurrent standard local therapy consisting of cleaning, saline irrigation, and dressing changes as prescribed by each patient's physician. Patients who experience progressive healing receive arginine butyrate 3-4 times a week. Arginine butyrate treatment may be discontinued and reinstated following a single 2 week medical complication.

Arm II: Patients receive standard local therapy only for 12 weeks. Patients randomized to arm II may cross over to receive arginine butyrate if no or less than 25% healing is observed after 12 weeks.

Patients whose ulcers have closed by at least 15% per course may receive 2 additional 8-week courses of arginine butyrate therapy and are followed for 2 months after healing is completed.

  Eligibility

Ages Eligible for Study:   16 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Significant sickle cell syndrome including HbSS, S-beta thalassemia, and hemoglobin variants Lower extremity or ankle ulcer (or ulcers) present for at least 6 months without healing --Prior/Concurrent Therapy-- Biologic therapy: No chronic transfusion therapy Chemotherapy: No prior or concurrent cancer chemotherapy No concurrent butyrate derivatives Prior treatment with hydroxyurea allowed if on a stable dose for at least 1 year Endocrine therapy: No concurrent corticosteroid therapy Radiotherapy: Not specified Surgery: Not specified Other: Must be treated with antibiotics prior to entry for complicating cellulitis or secondary infections --Patient Characteristics-- Age: 16-60 Performance status: Not specified Hematopoietic: Not specified Hepatic: No hepatic compromise Transaminases no greater than 250 IU Renal: No renal compromise Creatinine no greater than 1.2 mg/mL (adults) Creatinine no greater than 0.9 mg/mL (teenagers) Other: Not pregnant Fertile patients must use effective contraception No poorly controlled seizure disorders No other secondary conditions that might inhibit immune function

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004412

Locations
United States, Illinois
University of Illinois College of Medicine Recruiting
Chicago, Illinois, United States, 60612
Contact: Mabel Koshy     312-996-5680        
United States, Massachusetts
Boston University School of Medicine Recruiting
Boston, Massachusetts, United States, 02118
Contact: Susan Park Perrine     617-638-4173        
United States, New York
Mount Sinai School of Medicine Recruiting
New York, New York, United States, 10029
Contact: George F. Atweh     212-785-6662        
United States, Tennessee
University of Tennessee, Memphis Cancer Center Recruiting
Memphis, Tennessee, United States, 38103
Contact: Patricia E. Adams-Graves     901-448-5798        
Sponsors and Collaborators
FDA Office of Orphan Products Development
Boston University
Investigators
Study Chair: Douglas V. Faller Boston University
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004412     History of Changes
Other Study ID Numbers: 199/13302, BUSM-FDR001376, BUSM-3889
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
dermatologic disorders
genetic diseases and dysmorphic syndromes
hematologic disorders
rare disease
 sickle cell anemia
skin ulcers
thalassemia major

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Skin Ulcer
Ulcer
Hematologic Diseases
Hemoglobinopathies
 Genetic Diseases, Inborn
Skin Diseases
Pathologic Processes
Arginine butyrate
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013