Sickle Cell DiseasePhase 2NCT05098028
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
A Phase 2 study testing Low Dose Rifaximin ER, Low Dose Rifaximin DER and High Dose Rifaximin ER in people with Sickle Cell Disease, run by Bausch Health Americas, Inc. at 8 sites in 3 countries.
Trial summary
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs). Approximately 60 participants with sickle cell disease will be enrolled and randomized: 12 participants in each of four active novel formulation rifaximin groups and 6… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Low Dose Rifaximin ER, Low Dose Rifaximin DER and High Dose Rifaximin ER
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 8 sitesin 3 countries
- Study dates
- 22 Mar 2022to 4 Sep 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- ability and willingness to sign a written informed consent form.
- between the ages of 18 to 70 years old (inclusive) at the time of consent.
- SCD of any genotype (HbSS, HbSC, HbS β-thalassemia). If the subject's genotype has not been previously documented, genotyping will be performed during Screening using high-performance liquid chromatography (HPLC)/electrophoresis.
- least 2 VOCs within the 12 months prior to Screening.
- if receiving hydroxyurea (HU)/hydroxycarbamide (HC), subject must have been receiving the treatment for at least 6 months prior to Screening and must agree to maintain the same dose and schedule for the duration of the study.
- must have laboratory values at Screening as follows:
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- Absolute Neutrophil Count ≥1.0 x 109/L
- Platelets ≥ 75 x 109/L
- Hemoglobin (Hgb) ≥ 6.0 g/dL
- Glomerular filtration rate (GFR) ≥ 45 mL/min/1.73 m2 using the CKD-EPI formula
- Total bilirubin ≤ 15 mg/dL
- Alanine transaminase (ALT) ≤ 3.0 x ULN
- International Normalized Ratio (INR) ≤ 2.0
- Eastern Cooperate Oncology Group (ECOG) performance status ≤ 2
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, must have a negative serum pregnancy test at Screening and agree to use standard prevention methods for the duration of the study.
Who cannot join
- receiving concomitant treatment with voxelotor, crizanlizumab, or L-glutamine.
- any history of stem cell transplant, is planning to begin or has received in past 30 days.
- acute VOC, requiring a visit to a medical facility and/or healthcare professional, ending within 7 days prior to Day 1 dosing.
- has received any blood products within 30 days prior to Day 1 dosing.
- uncontrolled liver disease or renal impairment, ulcerative colitis, Crohn's disease, or other chronic GI disorder.
- has received active treatment in another investigational trial within 30 days or 5 half-lives of the last dose of the investigational agent, whichever is greater, prior to Screening.
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- has received penicillin prophylaxis or antibiotics for treatment of infection within 30 days or 5 half-lives of the treatment, whichever is greater, prior to Screening.
- significant medical condition that required hospitalization (other than for a VOC) within 2 months prior to Screening.
- planning on undergoing an exchange transfusion during the duration of the study or has completed one within 4 weeks prior to Day 1 dosing.
- hypersensitivity to rifaximin, rifampin, rifamycin antimicrobial agents, or any components of rifaximin ER and DER.
- pregnant or a nursing woman.
- history of illicit drug use or abuse, either documented or in the opinion of the Investigator.
- using any medication that is known to inhibit or induce CYP3A4, or P-gp and OATP1B1/B3 within 30 days or 5 half-lives, whichever is longer, prior to Day 1 dosing, or in the opinion of the Investigator, may affect the evaluation of the study product or place the subject at undue risk.
- has had any prior gastrointestinal surgery which has altered the anatomy of the esophagus, stomach, or small/large intestine (with the exception of appendectomy, cholecystectomy, and fundoplication).
- has had a colonoscopy or sigmoidoscopy within 30 days prior to Day 1 or plans to undergo such a procedure during the duration of the study.
- has used bowel prep, laxative, or enema within 30 days prior to Day 1.
- bleeding disorder including, but not limited to, acquired or congenital platelet function defects, disseminated intravascular coagulation (DIC), bleeding factor deficiencies, hemophilia, idiopathic thrombocytopenia purpura (ITP), or von Willebrand's disease.
- planning to undergo a major surgical procedure during the duration of the study.
- positive test for human immunodeficiency virus (HIV)1 or HIV2.
- active Hepatitis B infection (HBsAg positive). Prior infection that is not active (i.e., HBsAg negative, HBcAb positive, and HBsAb positive) is permitted.
- positive test for Hepatitis C (HCV RNA). Prior infection with spontaneous resolution or sustained resolution for ≥ 24 weeks after cessation of antivirals is permitted.
- active COVID-19 infection or complication(s) related to COVID 19 infection that are unresolved or, in the opinion of the Investigator, may affect evaluation of the study drug or place the subject at undue risk.
- received a vaccine (including COVID-19 vaccine) within 2 weeks prior to Screening. If subject has received their first of two COVID-19 vaccination doses, as applicable, they must wait for at least 2 weeks after receiving the second dose, and be symptom-free, prior to beginning Screening. Subject must not be planning for COVID-19 or other vaccinations while on study.
- malignant disease. Exceptions include malignancies that were treated curatively and have not recurred within 2 years prior to study treatment, completely resected basal cell and squamous cell skin cancers, and any completely resected carcinoma in situ.
- prolonged QT interval as assessed by ECG history within the past 3 months. For subjects with no historical ECG information, subject has a resting QTcF ≥ 460 msec for males and ≥ 470 msec for females at Screening.
- any unstable cardiac condition that, in the opinion of the Investigator, may worsen during the study or interfere with successful evaluation of the study treatment.
- serious mental or physical illness which, in the opinion of the Investigator, would compromise participation in the study.
- any condition which, in the opinion of the Investigator, is likely to interfere with the successful collection of the measurements required for the study.
- unable to understand or comply with study instructions and requirements.
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
United States5 sites
Bausch Site 104
Atlanta, Georgia, 30329
Bausch Site 103
Denver, Colorado, 80220
Bausch Site 102
Greenville, North Carolina, 27834
Bausch Site 105
Orange, California, 92868
Bausch Site 101
Syracuse, New York, 13210
About this study
Official title: A Phase 2a Randomized, Double-Blind, Placebo-Controlled Study to Characterize the Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs). Approximately 60 participants with sickle cell disease will be enrolled and randomized: 12 participants in each of four active novel formulation rifaximin groups and 6 participants in each of 2 placebo groups.
- Conditions
- Sickle Cell Disease
- Being tested
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER, High Dose Rifaximin DER, Placebo
- 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.
Low Dose Rifaximin ERDrug
Low Dose Rifaximin Extended Release Twice Daily
Low Dose Rifaximin DERDrug
Low Dose Rifaximin Delayed Extended Release Twice Daily
High Dose Rifaximin ERDrug
High Dose Rifaximin Extended Release Twice Daily
High Dose Rifaximin DERDrug
High Dose Rifaximin Delayed Extended Release Twice Daily
PlaceboDrug
Placebo Twice Daily
| Study group | Role | What they receive |
|---|---|---|
| Low Dose Rifaximin ER | Experimental | twice daily Receives: Drug: Low Dose Rifaximin ER |
| Low Dose Rifaximin DER | Experimental | twice daily Receives: Drug: Low Dose Rifaximin DER |
| High Dose Rifaximin ER | Experimental | twice daily Receives: Drug: High Dose Rifaximin ER |
| High Dose Rifaximin DER | Experimental | twice daily Receives: Drug: High Dose Rifaximin DER |
| Placebo | Placebo comparator | twice daily Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Plasma Concentration
Maximum observed plasma concentration
Measured: Day 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2021
- Study start22 Mar 2022
- Primary completion4 Sep 2023
- Study completion4 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Bausch Health Americas, Inc.The sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bausch Health Americas, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Varsha Bhatt, Study director, Bausch Health Americas, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05098028
- Sponsor's protocol ID
- RBSC2161
- Status last verified
- Aug 2024
- First submitted
- 18 Oct 2021
- Last updated
- 19 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Randomized, Double-Blind, Placebo-Controlled Study to Characterize the Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT05098028
- Acronym / sponsor ID
- RBSC2161
- Conditions
- Sickle Cell Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Bausch Health Americas, Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Mar 2022 (actual)
- Primary completion
- 4 Sep 2023 (actual)
- Study completion
- 4 Sep 2023 (actual)
- First posted
- 28 Oct 2021 (actual)
- Results
- Posted 19 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs). Approximately 60 participants with sickle cell disease will be enrolled and randomized: 12 participants in each of four active novel formulation rifaximin groups and 6 participants in each of 2 placebo groups.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Low Dose Rifaximin ER | Experimental | Drug: Low Dose Rifaximin ERtwice daily |
| Low Dose Rifaximin DER | Experimental | Drug: Low Dose Rifaximin DERtwice daily |
| High Dose Rifaximin ER | Experimental | Drug: High Dose Rifaximin ERtwice daily |
| High Dose Rifaximin DER | Experimental | Drug: High Dose Rifaximin DERtwice daily |
| Placebo | Placebo comparator | Drug: Placebotwice daily |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- ability and willingness to sign a written informed consent form.
- between the ages of 18 to 70 years old (inclusive) at the time of consent.
- SCD of any genotype (HbSS, HbSC, HbS β-thalassemia). If the subject's genotype has not been previously documented, genotyping will be performed during Screening using high-performance liquid chromatography (HPLC)/electrophoresis.
- least 2 VOCs within the 12 months prior to Screening.
- if receiving hydroxyurea (HU)/hydroxycarbamide (HC), subject must have been receiving the treatment for at least 6 months prior to Screening and must agree to maintain the same dose and schedule for the duration of the study.
Key exclusion criteria (29 listed)
- receiving concomitant treatment with voxelotor, crizanlizumab, or L-glutamine.
- any history of stem cell transplant, is planning to begin or has received in past 30 days.
- acute VOC, requiring a visit to a medical facility and/or healthcare professional, ending within 7 days prior to Day 1 dosing.
- has received any blood products within 30 days prior to Day 1 dosing.
- uncontrolled liver disease or renal impairment, ulcerative colitis, Crohn's disease, or other chronic GI disorder.
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 |
|---|---|---|
| Low Dose Rifaximin ERLow Dose Rifaximin Extended Release Twice Daily | Drug | Low Dose Rifaximin ER |
| Low Dose Rifaximin DERLow Dose Rifaximin Delayed Extended Release Twice Daily | Drug | Low Dose Rifaximin DER |
| High Dose Rifaximin ERHigh Dose Rifaximin Extended Release Twice Daily | Drug | High Dose Rifaximin ER |
| High Dose Rifaximin DERHigh Dose Rifaximin Delayed Extended Release Twice Daily | Drug | High Dose Rifaximin DER |
| PlaceboPlacebo Twice Daily | Drug | Placebo |
Primary outcome
Maximum Plasma Concentration
Time frame: Day 29
Maximum observed plasma concentration
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Varsha Bhatt study director
Bausch Health Americas, Inc.
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Low Dose Rifaximin ER, Low Dose Rifaximin DER and High Dose Rifaximin ER in people with Sickle Cell Disease, run by Bausch Health Americas, Inc. at 8 sites in 3 countries.
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 15 inclusion and 29 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: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Kenya, Canada. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 19 Sep 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Plasma Concentration (time frame: Day 29). 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 19 Sep 2024.