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	Treatment or Intervention	Phase
Skin Ulcers Sickle Cell Anemia	Drug: arginine butyrate	Phase II

Further Study Details: 

Expected Total Enrollment:  30

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.

Ages Eligible for Study:  16 Years   -   60 Years,  Genders Eligible for Study:  Both

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

Illinois
      University of Illinois College of Medicine, Chicago,  Illinois,  60612,  United States; Recruiting
Massachusetts
      Boston University School of Medicine, Boston,  Massachusetts,  02118,  United States; Recruiting
New York
      Mount Sinai School of Medicine, New York,  New York,  10029,  United States; Recruiting
Tennessee
      University of Tennessee, Memphis Cancer Center, Memphis,  Tennessee,  38103,  United States; Recruiting

Study chairs or principal investigators

Douglas V. Faller,  Study Chair,  Boston University School of Medicine   

Study ID Numbers:  199/13302; BUSM-FDR001376; BUSM-3889
Record last reviewed:  May 1999
Last Updated:  October 13, 2004
Record first received:  October 18, 1999
ClinicalTrials.gov Identifier:  NCT00004412
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08