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

This study is currently recruiting participants.

Verified by FDA Office of Orphan Products Development, May 1999

First Received: October 18, 1999 Last Updated: June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	Boston University
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004412

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 II

Study Type:	Interventional
Study Design:	Treatment, Randomized

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Anemia Sickle Cell Anemia

Drug Information available for: Arginine butyrate Arginine Arginine hydrochloride

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

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

Skin Diseases
Hematologic Diseases
Antineoplastic Agents
Ulcer
Anemia
Anemia, Hemolytic
Pharmacologic Actions
Anemia, Hemolytic, Congenital

Pathologic Processes
Genetic Diseases, Inborn
Therapeutic Uses
Hemoglobinopathies
Skin Ulcer
Arginine butyrate
Anemia, Sickle Cell

ClinicalTrials.gov processed this record on November 25, 2009