Rifaximin to Modify the Disease Course in Sickle Cell Disease

NCT03719729 | Unknown Phase 2 DMC FDA Drug

• 1 other identifier: L-12648
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

In this single-arm, one-stage Phase II study, the investigators hypothesize that gut decontamination with rifaximin will reduce the frequency of hospital admission due to painful crisis in patients with SCD. The study will accrue 20 SCD patients who had at least two hospital admissions in the previous 12 months. These patients will receive rifaximin 550 mg twice a day for a total of 12 months. This following clinical parameters will be measured: 1. Changes in the annual rate of hospital admissions due to painful crisis; 2. Changes in the annual rate of days hospitalized; 3. Annual rates of uncomplicated crises; 4. Annual rate of acute chest syndrome; 5. Changes in the quality of life; and 6). Toxicities. The following laboratory parameters will be measured: 1. Changes in the number of circulating activated neutrophils; 2. Changes in the intestinal microbiome diversity; 3. Changes in the urinary 3-indoxyl sulfate levels; 4. Changes in the serum biomarkers of intestinal permeability (lipopolysaccharides; zonulin, citrulline, and fatty acid binding proteins).

Conditions

Sickle Cell Disease; Antibiotics

Outcome Measures

Primary Outcomes (1)

• Toxicity profile

  Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.

  24 months

Secondary Outcomes (4)

• Changes in the annual rate of hospital admission for painful crisis

  12 months

• Changes in the annual days of hospitalization for painful crisis

  12 months

• Changes in the annual number of units of blood transfusion

  12 months

• Changes in the quality of life as measured by the FANLTC questionnaire

  24 months

Study Arms (1)

Single

EXPERIMENTAL

Each subject will receive rifaximin 550 mg twice a day for up to one year.

Drug: Rifaximin

Interventions

Rifaximin DRUG

Administer daily rifaximin to modify intestinal microbiome to alter the course of the disease.

Also known as: Xifaxan

Single

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with HbSS, HbSC, or HbS beta thal.
• Age 18-70 years.
• More than two hospital admissions for painful VOC in the prior 12 months, whether on any anti-sickling agents (e.g. hydroxyurea, L-glutamine, or transfusion therapy) or not. These agents may be continued during the study period. However, subjects are not allowed to be started on any of these agents during the study period.
• Ability to comprehend and sign an informed consent. -

You may not qualify if:

• Pregnant or lactating. For female subjects of child-bearing potential, the subject must agree to avoid pregnancy during the rifaximin study period and to practice a recognized form of birth control during this period (e.g. barrier, birth control pills, abstinence).
• Life expectancy of \< 12 months.
• History of allergy to rifaximin.
• Patients with newly developed abnormal vital signs or abnormal physical examination (outside the signs that are expected in patients with SCD).
• Patients in active VOC.
• Patients with a baseline prothrombin time International Normalized ratio (INR) \>2.0.
• Patients who receive any blood products within three weeks of the screening visit.
• Patients with uncontrolled liver disease or renal insufficiency, colitis, or inflammatory bowel disease.
• Patients with HIV, or other concomitant immunodeficiency.
• Patients on penicillin prophylaxis or antibiotics for treatment of infection.
• Patients with significant medical condition that require hospitalization (other than sickle cell VOC) within two months of the screening visit.
• Patients currently taking or has been treated with an investigational drug within 30 days of the screening visit.

Sponsors & Collaborators

• New York Medical College: lead

Study Sites (1)

Westchester Medical Cancer Cancer Institute

Valhalla, New York, 10532, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Rifaximin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Rifamycins; Heterocyclic Compounds, 4 or More Rings; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Lactams, Macrocyclic; Macrocyclic Compounds; Polycyclic Compounds

Central Study Contacts

Seah Lim, MD PhD

CONTACT

4126946980; seah.lim@wmchealth.org

Judy Moore

CONTACT

4126946980; judy.moore@wmchealth.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 4, 2018
• First Posted: October 25, 2018
• Study Start: August 22, 2018
• Primary Completion: February 22, 2020
• Study Completion: July 22, 2020
• Last Updated: March 14, 2019

Record last verified: 2018-08

Locations

US (1)