Assessing Tenapanor as a Treatment of CF-related Constipation.

NCT06810167 | Recruiting Phase 3 FDA Drug

• 1 other identifier: 2024P002058
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis; Constipation

Keywords

Constipation; Cystic Fibrosis; CFrC

Outcome Measures

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.

  Patients will record SBM frequency over the 4-week treatment period.

Secondary Outcomes (3)

• Change in PAC-SYM questionnaire score

  Patients will complete the PAC-SYM at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.

• Change in PAC-QOL questionnaire score

  Patients will complete the PAC-QOL at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.

• Change in IBS-SSS questionnaire score

  Patients will complete the IBS-SSS at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.

Study Arms (1)

Cystic fibrosis patients with CF-related constipation

EXPERIMENTAL

Drug: Tenapanor

Interventions

Tenapanor DRUG

CF patients with CFrC will ingest one 50 mg tablet of tenapanor twice daily for 4 weeks.

Cystic fibrosis patients with CF-related constipation

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Having confirmed cystic fibrosis (either by sweat chloride or genetic testing)
• Meeting criteria for CFrC
• Must include 2 of the following, with or without abdominal pain for at least 3 months, with symptom onset at least 6-months prior:
• Straining in at least 25% of defecations
• Bristol Stool Scale 1-2 more than 25% of defecations (change in stool form)
• Sensation of incomplete evacuation more than 25% of defecations
• Sensation of anorectal obstruction/blockage more than 25% of defecations
• Manual disimpaction/manipulation of pelvic floor to facilitate more than 25% of defecations
• Fewer than 3 spontaneous bowel movements weekly (change in stool frequency)
• Loose stools rarely present without the use of laxatives
• Willingness to avoid major dietary or lifestyle changes during study.

You may not qualify if:

• 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
• Hospitalization within 4-weeks prior to study initiation.
• DIOS within 4-weeks prior to study initiation.
• Other known/suspected mechanical obstruction

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Ardelyx: collaborator

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis; Constipation

Interventions

tenapanor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Christoher D Velez, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chloe Butzel, BA

CONTACT

6176437088; cbutzel@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: January 23, 2025
• First Posted: February 5, 2025
• Study Start: June 1, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: March 18, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)