Back to resultsRifaximin to Modify the Disease Course in Sickle Cell Disease
Primary Purpose
Sickle Cell Disease, Antibiotics
Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Rifaximin
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria:
- 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. -
Exclusion Criteria:
- 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.
-
Sites / Locations
- Westchester Medical Cancer Cancer InstituteRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Single
Arm Description
Each subject will receive rifaximin 550 mg twice a day for up to one year.
Outcomes
Primary Outcome Measures
Toxicity profile
Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.
Secondary Outcome Measures
Changes in the annual rate of hospital admission for painful crisis
Changes in the frequency of hospitalization for painful crisis
Changes in the annual days of hospitalization for painful crisis
Changes in the total number of days in hospital due to painful crisis
Changes in the annual number of units of blood transfusion
Changes in the number of units of blood transfused
Changes in the quality of life as measured by the FANLTC questionnaire
Changes in the quality of life due to treatment with rifaximin
Full Information
NCT ID
NCT03719729
First Posted
September 4, 2018
Last Updated
March 12, 2019
Sponsor
New York Medical College
1. Study Identification
Unique Protocol Identification Number
NCT03719729
Brief Title
Rifaximin to Modify the Disease Course in Sickle Cell Disease
Official Title
A Phase II Study of Rifaximin (Xifaxan) for Patients With Sickle Cell Disease (SCD)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2018
Overall Recruitment Status
Unknown status
Study Start Date
August 22, 2018 (Actual)
Primary Completion Date
February 22, 2020 (Anticipated)
Study Completion Date
July 22, 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
New York Medical College
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Antibiotics
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Single
Arm Type
Experimental
Arm Description
Each subject will receive rifaximin 550 mg twice a day for up to one year.
Intervention Type
Drug
Intervention Name(s)
Rifaximin
Other Intervention Name(s)
Xifaxan
Intervention Description
Administer daily rifaximin to modify intestinal microbiome to alter the course of the disease.
Primary Outcome Measure Information:
Title
Toxicity profile
Description
Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Changes in the annual rate of hospital admission for painful crisis
Description
Changes in the frequency of hospitalization for painful crisis
Time Frame
12 months
Title
Changes in the annual days of hospitalization for painful crisis
Description
Changes in the total number of days in hospital due to painful crisis
Time Frame
12 months
Title
Changes in the annual number of units of blood transfusion
Description
Changes in the number of units of blood transfused
Time Frame
12 months
Title
Changes in the quality of life as measured by the FANLTC questionnaire
Description
Changes in the quality of life due to treatment with rifaximin
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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. -
Exclusion Criteria:
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.
-
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Seah Lim, MD PhD
Phone
4126946980
Email
seah.lim@wmchealth.org
First Name & Middle Initial & Last Name or Official Title & Degree
Judy Moore
Phone
4126946980
Email
judy.moore@wmchealth.org
Facility Information:
Facility Name
Westchester Medical Cancer Cancer Institute
City
Valhalla
State/Province
New York
ZIP/Postal Code
10532
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Seah Lim, MD PhD
Phone
914-246-6600
Email
seahhlim@yahoo.com
First Name & Middle Initial & Last Name & Degree
Bettina Knoll, MD PhD
Email
bettina.knoll@wmchealth.org
First Name & Middle Initial & Last Name & Degree
Seah Lim, MD PhD
First Name & Middle Initial & Last Name & Degree
Bettina Knoll, MD PhD
12. IPD Sharing Statement
Learn more about this trial
Rifaximin to Modify the Disease Course in Sickle Cell Disease
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