Sickle Cell DiseasePhase 2NCT03719729
Rifaximin to Modify the Disease Course in Sickle Cell Disease
A Phase 2 study testing Rifaximin in people with Sickle Cell Disease and Antibiotics, run by New York Medical College at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Sickle Cell Disease, Antibiotics
- What is being tested?
- Rifaximin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Aug 2018to 22 Jul 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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. -
Who cannot join
- 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.
Show all 13Show fewer
- 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.
- -
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Westchester Medical Cancer Cancer Institute
Valhalla, New York, 10532
Seah Lim, MD PhD, 914-246-6600, seahhlim@yahoo.com
Bettina Knoll, MD PhD, bettina.knoll@wmchealth.org
About this study
Official title: A Phase II Study of Rifaximin (Xifaxan) for Patients With Sickle Cell Disease (SCD)
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).
Read the 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).
- Conditions
- Sickle Cell Disease, Antibiotics
- Being tested
- Rifaximin
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RifaximinDrug
Also called Xifaxan
Administer daily rifaximin to modify intestinal microbiome to alter the course of the disease.
| Study group | Role | What they receive |
|---|---|---|
| Single | Experimental | Each subject will receive rifaximin 550 mg twice a day for up to one year. Receives: Drug: Rifaximin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity profile
Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.
Measured: 24 months
Secondary outcomes (4)
Changes in the annual rate of hospital admission for painful crisis
Changes in the frequency of hospitalization for painful crisis
Measured: 12 months
Changes in the annual days of hospitalization for painful crisis
Changes in the total number of days in hospital due to painful crisis
Measured: 12 months
Changes in the annual number of units of blood transfusion
Changes in the number of units of blood transfused
Measured: 12 months
Changes in the quality of life as measured by the FANLTC questionnaire
Changes in the quality of life due to treatment with rifaximin
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2018
- Study start22 Aug 2018
- Primary completion22 Feb 2020 (estimated)
- Study completion22 Jul 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03719729
- Sponsor's protocol ID
- L-12648
- Status last verified
- Aug 2018
- First submitted
- 4 Sep 2018
- Last updated
- 14 Mar 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Rifaximin (Xifaxan) for Patients With Sickle Cell Disease (SCD)
- ClinicalTrials.gov ID
- NCT03719729
- Acronym / sponsor ID
- L-12648
- Conditions
- Sickle Cell Disease, Antibiotics
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Study start
- 22 Aug 2018 (actual)
- Primary completion
- 22 Feb 2020 (estimated)
- Study completion
- 22 Jul 2020 (estimated)
- First posted
- 25 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single | Experimental | Drug: RifaximinEach subject will receive rifaximin 550 mg twice a day for up to one year. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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. -
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RifaximinAlso: XifaxanAdminister daily rifaximin to modify intestinal microbiome to alter the course of the disease. | Drug | Single |
Primary outcome
Toxicity profile
Time frame: 24 months
Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.
4 secondary outcomes
Changes in the annual rate of hospital admission for painful crisis
Time frame: 12 months
Changes 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Mar 2019.
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rifaximin in people with Sickle Cell Disease and Antibiotics, run by New York Medical College at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 14 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Seah Lim, MD PhD (4126946980, seah.lim@wmchealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Toxicity profile (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Mar 2019.