Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
Recruitment status was Recruiting
|First Received Date ICMJE||October 18, 1999|
|Last Updated Date||June 23, 2005|
|Start Date ICMJE||September 1997|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE||Not Provided|
|Original Primary Outcome Measures ICMJE||Not Provided|
|Change History||Complete list of historical versions of study NCT00004412 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE||Not Provided|
|Original Secondary Outcome Measures ICMJE||Not Provided|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers|
|Official Title ICMJE||Not Provided|
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.
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.
|Study Type ICMJE||Interventional|
|Study Phase||Phase 2|
|Study Design ICMJE||Allocation: Randomized
Primary Purpose: Treatment
|Intervention ICMJE||Drug: arginine butyrate|
|Study Arm (s)||Not Provided|
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Recruiting|
|Completion Date||Not Provided|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
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
|Ages||16 Years to 60 Years|
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Not Provided|
|Location Countries ICMJE||United States|
|NCT Number ICMJE||NCT00004412|
|Other Study ID Numbers ICMJE||199/13302, BUSM-FDR001376, BUSM-3889|
|Has Data Monitoring Committee||Not Provided|
|Responsible Party||Not Provided|
|Study Sponsor ICMJE||FDA Office of Orphan Products Development|
|Collaborators ICMJE||Boston University|
|Information Provided By||FDA Office of Orphan Products Development|
|Verification Date||May 1999|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP