Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Rifaximin to Modify the Disease Course in Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03719729
Recruitment Status : Unknown
Verified August 2018 by New York Medical College.
Recruitment status was:  Recruiting
First Posted : October 25, 2018
Last Update Posted : March 14, 2019
Sponsor:
Information provided by (Responsible Party):
New York Medical College

Tracking Information
First Submitted Date  ICMJE September 4, 2018
First Posted Date  ICMJE October 25, 2018
Last Update Posted Date March 14, 2019
Actual Study Start Date  ICMJE August 22, 2018
Estimated Primary Completion Date February 22, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
Toxicity profile [ Time Frame: 24 months ]
Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 12, 2019)
  • Changes in the annual rate of hospital admission for painful crisis [ Time Frame: 12 months ]
    Changes in the frequency of hospitalization for painful crisis
  • 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
  • Changes in the annual number of units of blood transfusion [ Time Frame: 12 months ]
    Changes in the number of units of blood transfused
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
  • Reduction of annual rate of hospital admission for painful crisis [ Time Frame: 12 months ]
  • Reduction in the annual days of hospitalization for painful crisis [ Time Frame: 12 months ]
  • Changes in the annual number of units of blood transfusion [ Time Frame: 12 months ]
  • Changes in the quality of life as measured by the FANLTC questionnaire [ Time Frame: 24 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Rifaximin to Modify the Disease Course in Sickle Cell Disease
Official Title  ICMJE A Phase II Study of Rifaximin (Xifaxan) for Patients With Sickle Cell Disease (SCD)
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).
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).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Disease
  • Antibiotics
Intervention  ICMJE Drug: Rifaximin
Administer daily rifaximin to modify intestinal microbiome to alter the course of the disease.
Other Name: Xifaxan
Study Arms  ICMJE Experimental: Single
Each subject will receive rifaximin 550 mg twice a day for up to one year.
Intervention: Drug: Rifaximin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: October 23, 2018)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 22, 2020
Estimated Primary Completion Date February 22, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

    -

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03719729
Other Study ID Numbers  ICMJE L-12648
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party New York Medical College
Study Sponsor  ICMJE New York Medical College
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account New York Medical College
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP