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

Bausch Health Americas, Inc.8 sites in 3 countries44 target enrollmentMarch 22, 2022

ConditionsSickle Cell Disease

InterventionsLow Dose Rifaximin ER Low Dose Rifaximin DER High Dose Rifaximin ER High Dose Rifaximin DER Placebo

DrugsLow Dose Rifaximin ER Low Dose Rifaximin DER High Dose Rifaximin ER High Dose Rifaximin DER Placebo

Overview

Phase: Phase 2
Intervention: Low Dose Rifaximin ER
Conditions: Sickle Cell Disease
Sponsor: Bausch Health Americas, Inc.
Enrollment: 44
Locations: 8
Primary Endpoint: Maximum Plasma Concentration
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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 participants in each of 2 placebo groups.

Registry

clinicaltrials.gov

Start Date

March 22, 2022

End Date

September 4, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Bausch Health Americas, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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:
•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

Exclusion Criteria

•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.
•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.