Assessing Tenapanor as a Treatment of CF-related Constipation.

Official title

An Open-label, Single Center Study Assessing Tenapanor as a Non-CFTR-mediated Treatment of CF-related Constipation in People With CF.

Overview

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.

Interventions

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

Primary outcome measures

• Increase in SBM frequency [Time frame: Patients will record SBM frequency over the 4-week treatment period.]

Secondary outcome measures (3)

• Change in PAC-SYM questionnaire score [Time frame: Patients will complete the PAC-SYM at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.]
• Change in PAC-QOL questionnaire score [Time frame: Patients will complete the PAC-QOL at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.]
• Change in IBS-SSS questionnaire score [Time frame: Patients will complete the IBS-SSS at -2 weeks, 0 weeks, 2 weeks, and 4 weeks.]

Eligibility criteria

Inclusion criteria

• 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.

Exclusion criteria

• 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 1 center

• Massachusetts General Hospital — Boston

Identifiers

NCT: NCT06810167 · 2024P002058

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗