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Status unknownNCT03719729Updated Mar 14, 2019

Rifaximin to Modify the Disease Course in Sickle Cell Disease

A Phase 2 interventional study of Rifaximin in Sickle Cell Disease and Antibiotics, sponsored by New York Medical College. Status unknown at 1 site in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2019-03-14.

Sponsored by New York Medical College · Phase 2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Aug 2018), so the status shown — last known as Recruiting — may be out of date.
Phase
Phase 2
Study type
Interventional
Enrollment
20
Allocation
Not applicable
Ages
18 Years to 70 Years
Sex
All
01

Study 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).

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).

02

Conditions studied

  • Sickle Cell Disease
  • Antibiotics

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03

In context

Anemia, Sickle Cell

1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.

This study's planned enrollment of 20 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.

Browse Anemia, Sickle Cell studies →

Lead sponsor

New York Medical College is the lead sponsor of 69 studies on the registry; 23 are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.

    -

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
20 participants (estimated)

Study arms

  • Experimental
    Single

    Each subject will receive rifaximin 550 mg twice a day for up to one year.

    Drug: Rifaximin

Interventions

  • DrugRifaximin

    Administer daily rifaximin to modify intestinal microbiome to alter the course of the disease.

    Also known as: Xifaxan

06

What researchers measure

Primary outcomes

  1. Toxicity profile

    Incidence of nausea, vomiting, diarrhea, abdominal discomfort, worsening anemia.

    Time frame: 24 months

Secondary outcomes

  1. Changes in the annual rate of hospital admission for painful crisis

    Changes in the frequency of hospitalization for painful crisis

    Time frame: 12 months

  2. Changes in the annual days of hospitalization for painful crisis

    Changes in the total number of days in hospital due to painful crisis

    Time frame: 12 months

  3. Changes in the annual number of units of blood transfusion

    Changes in the number of units of blood transfused

    Time frame: 12 months

  4. Changes in the quality of life as measured by the FANLTC questionnaire

    Changes in the quality of life due to treatment with rifaximin

    Time frame: 24 months

07

Study locations

1 of 1 sites recruiting
  • Westchester Medical Cancer Cancer Institute
    Valhalla, New York 10532, United States
    Recruiting
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 14, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT03719729
Lead sponsor
New York Medical College
Responsible party
Sponsor
First posted
Oct 25, 2018
Start date
Aug 22, 2018
Primary completion
Feb 22, 2020 (estimated)
Completion
Jul 22, 2020 (estimated)
Last update
Mar 14, 2019

Study contacts

Seah Lim, MD PhD
Contact
seah.lim@wmchealth.org
4126946980
Judy Moore
Contact
judy.moore@wmchealth.org
4126946980

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Aug 2018. You cannot join it, but the record below documents what was studied.

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