Assessing Tenapanor as a Treatment of CF-related Constipation.
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-75
SponsorMassachusetts General Hospital
About this trial
Tenapanor is the newest FDA-approved drug for IBS with constipation (IBS-C). This study seeks to understand tenapanor as a treatment for cystic fibrosis-related constipation (CFrC) in CF patients. Participants will ingest one 50 mg tablet of tenapanor, twice daily, for a 4-week treatment period. They will also complete three questionnaires, the PAC-SYM, PAC-QoL, and IBS-SSS, and daily diaries to characterize GI symptom burden and spontaneous bowel movement (SBM) frequency.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Having confirmed cystic fibrosis (either by sweat chloride or genetic testing)
Meeting criteria for CFrC
Straining in at least 25% of defecations
Bristol Stool Scale 1-2 more than 25% of defecations (change in stool form)
Disqualifiers
Use of any antibiotic to treat infection within the 4-weeks prior to study initiation (stable azithromycin dosed 3-times weekly for lung function is to be allowed)
Inability to discontinue standing bowel regimen (including fiber, stool softener, as well as either osmotic or stimulant laxative, pro-kinetic serotonergic agents, or other therapy) 2-weeks prior to study drug initiation (with ability to use osmotic laxative therapy as rescue therapy only).
Severe CFrC as determined by study team
Prior tenapanor usage
Trial design
Design model
Single group
Treatments tested in this trial
Tenapanor
DrugCF patients with CFrC will ingest one 50 mg tablet of tenapanor twice daily for 4 weeks.
Treatment groups
25 Participants
are divided into 1 treatment groupGroup A: Cystic fibrosis patients with CF-related constipationExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Increase in SBM frequency
There will be a statistically significant increase in spontaneous bowel movement frequency (SBM) in cystic fibrosis patients with CF-related constipation receiving tenapanor.
Time frame
Patients will record SBM frequency over the 4-week treatment period.Secondary outcomes
1
Change in PAC-SYM questionnaire score
There will be a change (with use of descriptive statistics) in bowel symptoms characterized by the Patient Assessment of Constipation Symptoms (PAC-SYM) questionnaire with tenapanor administration. PAC-SYM is a PRO previously used to examine constipation in pwCF. A decreased score corresponds to improvement of bowel symptoms.
Time frame
Patients will complete the PAC-SYM at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.2
Change in PAC-QOL questionnaire score
There will be a change (with use of descriptive statistics) in bowel symptoms characterized by the Patient Assessment of Constipation Quality of Life (PAC-QOL) questionnaire with tenapanor administration. PAC-QOL is a PRO previously used to examine constipation in pwCF. A decreased score corresponds to improvement of bowel symptoms.
Time frame
Patients will complete the PAC-QOL at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.3
Change in IBS-SSS questionnaire score
There will be a 50-point change in the Irritable Bowel Syndrome Scoring System - IBS-SSS (in line with recent bowel distress related literature Ford AC, Wright-Hughes A, Alderson SLet al Lancet 2023) in pwCF and CFrC receiving tenapanor.
Time frame
Patients will complete the IBS-SSS at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Massachusetts General Hospital
Lead sponsor
Ardelyx
Collaborator
Source ClinicalTrials.gov