Sickle Cell Anemia Clinical Trial
Official title:
Phase II Trial of Arginine Butyrate for Treatment of Refractory Leg Ulcers in Sickle Cell Disease or Beta Thalassemia
Verified date | March 2015 |
Source | Boston University |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 23 |
Est. completion date | February 2005 |
Est. primary completion date | December 2002 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 16 Years to 60 Years |
Eligibility |
PROTOCOL CRITERIA INCLUSION: --Disease Characteristics-- Significant sickle cell syndrome including Hemoglobin SS (HbSS), Hemoglobin S-beta thalassemia, and hemoglobin variants. Lower extremity or ankle ulcer (or ulcers) present for at least 6 months without healing --Patient Characteristics-- Age: 16-60 yrs 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 - Prior/Concurrent Therapy Other: Must be treated with antibiotics prior to entry for complicating cellulitis or secondary infections EXCLUSION : --Prior/Concurrent Therapy-- Biologic therapy: chronic transfusion therapy Chemotherapy: prior or concurrent cancer chemotherapy concurrent butyrate derivatives Endocrine therapy: concurrent corticosteroid therapy Radiotherapy: Not specified Surgery: Not specified --Patient Characteristics-- Hepatic: hepatic compromise Transaminases greater than 250 IU Renal: renal compromise Creatinine greater than 1.2 mg/mL (adults) Creatinine greater than 0.9 mg/mL (teenagers) Other: pregnant; Fertile patients with no effective contraception Poorly controlled seizure disorders other secondary conditions that might inhibit immune function |
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Boston University School of Medicine | Boston | Massachusetts |
United States | University of Illinois College of Medicine | Chicago | Illinois |
United States | University of Tennessee, Memphis Cancer Center | Memphis | Tennessee |
United States | Mount Sinai School of Medicine | New York | New York |
Lead Sponsor | Collaborator |
---|---|
Susan P. Perrine | Boston Medical Center, Icahn School of Medicine at Mount Sinai, Schneider Children's Medical Center, Israel, University of Illinois at Chicago, University of Tennessee Health Science Center |
United States,
McMahon L, Tamary H, Askin M, Adams-Graves P, Eberhardt RT, Sutton M, Wright EC, Castaneda SA, Faller DV, Perrine SP. A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers. Br J Haematol. 2010 De — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Healing Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area | Treatment Arm: The AB is given as an IV infusion at 500 mg/kg over 6-9 hrs. 5 days per week for 12 weeks. After 12 weeks of therapy, if he ulcer has decreased by 25% , the AB may be continued for additional 8 weeks (twice) or, until ulcer closes plus 2 weeks, additionally. Ulcers photographed, traced, and ulcer areas calculated by computerized planimetry. |
participants were followed for an average of 3 months | No |
Secondary | % Ulcers Which Completely Healed in Each Group, After 3 Months | Control Arm: given option to crossover to Treatment Arm if, ulcers have not closed after 12 weeks standard local care alone. | two additional courses of 8 week cycles | No |
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