Phase 2 N=23 Randomized Treatment

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

Skin Ulcers · Sickle Cell Anemia

Bottom Line

View on ClinicalTrials.gov: NCT00004412 ↗

Enrolled (actual)

Serious AEs

53.8%

Results posted

Mar 2015

Primary outcome: Primary: Healing Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area — 24; 78 percentage of healed ulcers — p=0.05

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Arginine Butyrate (Drug); Standard local care dressing (Other)
Age: Pediatric, Adult · 16+ yrs
Sex: All
Sponsor: Susan P. Perrine
Primary completion: Dec 2002

Outcome Measures

Outcome	Result	p-value
PRIMARY Healing Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area	24; 78	0.05
SECONDARY % Ulcers Which Completely Healed in Each Group, After 3 Months	8; 31	—

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.

Eligibility Criteria

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

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Data sourced from ClinicalTrials.gov (NCT00004412). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.