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Rifaximin to Modify the Disease Course in Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT03719729
Recruitment Status : Recruiting
First Posted : October 25, 2018
Last Update Posted : March 14, 2019
Sponsor:
Information provided by (Responsible Party):
New York Medical College

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).

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Antibiotics Drug: Rifaximin Phase 2

Detailed Description:
In this single-arm Phase II study, the investigators will accrue 20 SCD patients who had at least two hospital admissions in the previous 12 months to receive rifaximin 550 mg twice a day for a total of 12 months. The investigators will measure changes in the annual rate of hospital admissions due to vaso-occlusive crisis and the annual rate of hospital days. The investigators will also determine the annual rates of uncomplicated crises and acute chest syndrome. Quality of life due to the disease and to treatment will be determined using a questionnaire. This study will be complemented with exploratory laboratory studies to determine changes in the number of circulating activated neutrophils, intestinal microbiome diversity, urinary 3-indoxyl sulfate levels and serum biomarkers of intestinal permeability (lipopolysaccharides; zonulin, citrulline, and fatty acid binding proteins).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Rifaximin (Xifaxan) for Patients With Sickle Cell Disease (SCD)
Actual Study Start Date : August 22, 2018
Estimated Primary Completion Date : February 22, 2020
Estimated Study Completion Date : July 22, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Rifaximin

Arm Intervention/treatment
Experimental: Single
Each subject will receive rifaximin 550 mg twice a day for up to one year.
Drug: Rifaximin
Administer daily rifaximin to modify intestinal microbiome to alter the course of the disease.
Other Name: Xifaxan




Primary Outcome Measures :
  1. Toxicity profile [ Time Frame: 24 months ]
    Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.


Secondary Outcome Measures :
  1. Changes in the annual rate of hospital admission for painful crisis [ Time Frame: 12 months ]
    Changes in the frequency of hospitalization for painful crisis

  2. Changes in the annual days of hospitalization for painful crisis [ Time Frame: 12 months ]
    Changes in the total number of days in hospital due to painful crisis

  3. Changes in the annual number of units of blood transfusion [ Time Frame: 12 months ]
    Changes in the number of units of blood transfused

  4. Changes in the quality of life as measured by the FANLTC questionnaire [ Time Frame: 24 months ]
    Changes in the quality of life due to treatment with rifaximin



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with HbSS, HbSC, or HbS beta thal.
  2. Age 18-70 years.
  3. 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.
  4. Ability to comprehend and sign an informed consent. -

Exclusion Criteria:

  1. 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).
  2. Life expectancy of < 12 months.
  3. History of allergy to rifaximin.
  4. Patients with newly developed abnormal vital signs or abnormal physical examination (outside the signs that are expected in patients with SCD).
  5. Patients in active VOC.
  6. Patients with a baseline prothrombin time International Normalized ratio (INR) >2.0.
  7. Patients who receive any blood products within three weeks of the screening visit.
  8. Patients with uncontrolled liver disease or renal insufficiency, colitis, or inflammatory bowel disease.
  9. Patients with HIV, or other concomitant immunodeficiency.
  10. Patients on penicillin prophylaxis or antibiotics for treatment of infection.
  11. Patients with significant medical condition that require hospitalization (other than sickle cell VOC) within two months of the screening visit.
  12. Patients currently taking or has been treated with an investigational drug within 30 days of the screening visit.

    -


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03719729


Contacts
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Contact: Seah Lim, MD PhD 4126946980 seah.lim@wmchealth.org
Contact: Judy Moore 4126946980 judy.moore@wmchealth.org

Locations
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United States, New York
Westchester Medical Cancer Cancer Institute Recruiting
Valhalla, New York, United States, 10532
Contact: Seah Lim, MD PhD    914-246-6600    seahhlim@yahoo.com   
Contact: Bettina Knoll, MD PhD       bettina.knoll@wmchealth.org   
Principal Investigator: Seah Lim, MD PhD         
Sub-Investigator: Bettina Knoll, MD PhD         
Sponsors and Collaborators
New York Medical College

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Responsible Party: New York Medical College
ClinicalTrials.gov Identifier: NCT03719729     History of Changes
Other Study ID Numbers: L-12648
First Posted: October 25, 2018    Key Record Dates
Last Update Posted: March 14, 2019
Last Verified: August 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Rifaximin
Anti-Bacterial Agents
Anti-Infective Agents
Gastrointestinal Agents