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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00004412
Other study ID # 199/13302
Secondary ID BUSM-FDR001376IN
Status Completed
Phase Phase 2
First received October 18, 1999
Last updated March 18, 2015
Start date September 1997
Est. completion date February 2005

Study information

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

Clinical Trial Summary

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.


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 alone 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 cycle may receive 2 additional 8-week cycles of arginine butyrate therapy and are followed for 2 months after healing is completed.


Recruitment information / eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

Drug:
Arginine Butyrate
To determine if Arginine Butyrate accelerates healing of refractory leg ulcers over Standard Local Care alone.
Other:
Standard local care dressing
To heal leg ulcers.

Locations

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

Sponsors (6)

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

Country where clinical trial is conducted

United States, 

References & Publications (1)

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

Outcome

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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