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Studies

Interventional Recruiting Phase 2

NCT06904872 on ClinicalTrials.gov

Safety and Efficacy of Crofelemer in Adult Patients With Short Bowel Syndrome and Intestinal Failure (SBS-IF) Without Colon-in-continuity (CIC)

Official title: A Phase 2, Placebo-Controlled, Randomized, Double-Blind Study of 2 Doses of Crofelemer for the Treatment of Adult Patients With Short Bowel Syndrome and Intestinal Failure (SBS-IF) Without Colon-in-continuity (CIC)

  • Sponsor: Napo Therapeutics, S.p.A.
  • Enrollment: 18 (Estimated)
  • Study start: 2025-05-29 (Actual)
  • Primary completion: 2027-03-01 (Estimated)
  • Study completion: 2027-03-01 (Estimated)
  • Allocation: Randomized
  • InterventionModel: Parallel
  • InterventionModelDescription: Eligible patients who have met the stabilization criteria above will be randomized to one of the following treatment groups and entered into the 24-week double-blind treatment period: * Crofelemer 3 mg/kg/dose TID, morning, midday and evening; * Crofelemer 10 mg/kg/dose TID, morning, midday and evening; * Matched placebo TID, morning, midday and evening.
  • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • PrimaryPurpose: Treatment

Brief Summary

A 24-week, randomized, placebo-controlled, double-blind study to evaluate the efficacy, safety and tolerability of crofelemer in patients with Short Bowel Syndrome and Intestinal Failure (SBS-IF) without colon-in-continuity (CIC) requiring parenteral support (PS).

Blinded study drug will be administered orally (or enterally) three times daily (TID) as a novel crofelemer formulation, Crofelemer Powder for Oral Solution, or a matching placebo powder formulation for oral solution.

Patients will be randomized in a 1:1:1 ratio to crofelemer 3 mg/kg/dose TID, crofelemer 10 mg/kg/dose TID or placebo.

Conditions

  • Short Bowel Syndrome
  • Malabsorption Syndromes
  • Short Gut Syndrome
  • Post-Op Complication
  • Functional Gastrointestinal Disorders

Intervention / Treatment

  • Drug: Crofelemer Powder for Oral Solution
  • Drug: Matched Placebo Powder for Oral Solution

Primary Outcome Measures

  • Safety and Tolerability (24 weeks)
    Frequency of Treatment-Emergent-Adverse Events
  • Safety and Tolerability (24 weeks)
    Frequency of IP interruption and/or discontinuation considered related to the study drug
  • Preliminary Efficacy (24 weeks)
    Change in weekly volume of parenteral support (PS: parenteral nutrition (PN) with or without intravenous (IV) fluid volume) from baseline, by recording PS volume in the patient daily diary
  • Preliminary Efficacy (24 weeks)
    Change in weekly stool volume from baseline, by measuring and recording daily stool volume in the patient daily diary

Countries

Germany, Italy

Locations and Investigators

  • Universitäatsklinik RWTH, Aachen, Germany (Recruiting)
    • Martin von Websky, MD (Principal Investigator)
  • Charité Universitätsmedizin, Berlin, Germany (Recruiting)
    • Elisabeth Blüthner, MD (Principal Investigator)
  • Universitätsklinikum, Essen, Germany (Recruiting)
    • Christoph Schramm, MD (Principal Investigator)
  • Asklepios Klinik St. Georg, Hamburg, Germany (Recruiting)
    • Ulrich-Frank Pape, MD (Principal Investigator)
  • Universitätsmedizin, Rostock, Germany (Recruiting)
    • Georg Lamprecht, MD (Principal Investigator)
  • Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi, Bologna, Italy (Recruiting)
    • Anna Simona Sasdelli, MD (Principal Investigator)
  • Azienda Ospedaliera Universitaria Federico II, Naples, Italy (Recruiting)
  • Ospedale Università di Padova, Padova, Italy (Recruiting)
    • Edoardo Savarino, MD (Principal Investigator)