Tenapanor for Pediatric IBS with Constipation

Recruiting at 18 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Ardelyx

Disqualifiers: Surgery on stomach, Pregnant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 6

Duration: 40 weeks

150 Participants Needed

Trial Summary

What is the purpose of this trial?

Open-label long-term safety study of tenapanor in pediatric patients with IBS-C.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

What data supports the effectiveness of the drug Tenapanor for treating pediatric IBS with constipation?

Research shows that Tenapanor is effective in treating adults with irritable bowel syndrome with constipation (IBS-C), as demonstrated in multiple clinical trials. It works by targeting a specific part of the gut to help relieve constipation, and its approval for adult IBS-C suggests potential benefits for pediatric use. ^{1 2 3 4 5}

Is Tenapanor safe for humans?

Tenapanor has been tested in clinical trials for adults with irritable bowel syndrome with constipation (IBS-C) and has been approved by the FDA for this use, indicating it is generally considered safe for humans. It is minimally absorbed in the body, which may reduce the risk of side effects. ^{1 2 4 5 6}

How is the drug Tenapanor unique for treating pediatric IBS with constipation?

Tenapanor is unique because it works by reducing the absorption of sodium in the intestines, which helps to increase water in the gut and soften stools, making it easier to pass. This mechanism is different from other treatments that typically focus on fiber supplements or laxatives to relieve constipation. ^{7 8 9 10 11}

Research Team

David Rosenbaum

Principal Investigator

Ardelyx

Eligibility Criteria

This trial is for children and teenagers aged between 6 and under 18 who have Irritable Bowel Syndrome with Constipation (IBS-C). They must have completed a previous study on this condition, followed the procedures well, and be able to walk. Girls who can have babies need a negative pregnancy test and must use birth control.

Inclusion Criteria

I am between 6 and 17 years old.

I can walk on my own.

Patient completed the parent study immediately with adequate compliance with study procedures in the opinion of the Investigator Females of child-bearing potential must have negative pregnancy test at the last visit of the parent study and confirm the use of appropriate contraception (including abstinence).

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue tenapanor at the same dose assigned in the parent study, with possible titration to 50 mg BID or 25 mg BID after the first week

40 weeks

Visits approximately every 6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Tenapanor

Trial Overview The trial is testing Tenapanor, which is being considered as a long-term treatment option for young patients with IBS-C. It's an open-label study, meaning everyone knows they're getting Tenapanor without any placebo or comparison drug involved.

Participant Groups

Group I: Tenapanor Experimental Treatment 1 Intervention

Eligible patients from the parent study will continue tenapanor at the same dose assigned in the parent study which can be titrated to either 50 mg BID or 25 mg BID per Investigator guidance after a patient's first week on the assigned dose.

Tenapanor is already approved in United States for the following indications:

Approved in United States as Ibsrela for:

• Irritable Bowel Syndrome with Constipation (IBS-C)
• Chronic Kidney Disease

Who Is Running the Clinical Trial?

Ardelyx

Lead Sponsor

Trials

31

Recruited

6,100+

Findings from Research

In a phase 3 trial involving 620 patients with irritable bowel syndrome with constipation (IBS-C), tenapanor 50 mg twice daily significantly improved abdominal pain and bowel movement frequency compared to placebo, with 36.5% of patients responding positively versus 23.7% in the placebo group.

Tenapanor was generally well tolerated, with diarrhea being the most common side effect, but it was usually mild to moderate and led to discontinuation in only 6.5% of patients on tenapanor.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of Tenapanor in Treating Patients With Irritable Bowel Syndrome With Constipation: A 26-Week, Placebo-Controlled Phase 3 Trial (T3MPO-2). Chey, WD., Lembo, AJ., Yang, Y., et al.[2023]

Tenapanor, a selective NHE3 inhibitor, has been approved in the USA for treating constipation-predominant irritable bowel syndrome (IBS-C) in adults, based on positive results from the phase III T3MPO trial program.

In addition to IBS-C, tenapanor is also being developed for managing hyperphosphataemia in patients with chronic kidney disease, indicating its potential for multiple therapeutic uses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tenapanor: First Approval. Markham, A.[2020]

In a phase 2 study involving 356 patients with constipation-predominant irritable bowel syndrome (IBS-C), tenapanor 50 mg taken twice daily significantly improved bowel movement frequency, with a complete spontaneous bowel movement (CSBM) responder rate of 60.7% compared to 33.7% in the placebo group.

Tenapanor also effectively reduced abdominal symptoms, with a composite responder rate of 50.0% in the 50 mg group versus 23.6% in the placebo group, although diarrhea was noted as a common side effect.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tenapanor Treatment of Patients With Constipation-Predominant Irritable Bowel Syndrome: A Phase 2, Randomized, Placebo-Controlled Efficacy and Safety Trial. Chey, WD., Lembo, AJ., Rosenbaum, DP.[2018]

In a phase 3 trial involving 629 patients with constipation-predominant irritable bowel syndrome (IBS-C), tenapanor 50 mg twice daily significantly improved symptoms, with 27% of patients achieving the primary endpoint compared to 18.7% in the placebo group.

While tenapanor was generally well tolerated, diarrhea was the most common side effect, leading to discontinuation in 6.5% of patients, indicating a need for monitoring in clinical use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of Tenapanor in Treating Patients With Irritable Bowel Syndrome With Constipation: A 12-Week, Placebo-Controlled Phase 3 Trial (T3MPO-1). Chey, WD., Lembo, AJ., Rosenbaum, DP.[2022]

Tenapanor is a first-in-class oral medication for treating irritable bowel syndrome with constipation (IBS-C) that works locally in the gut by inhibiting the NHE3 transporter, which may lead to fewer systemic side effects and drug interactions.

Clinical trials (Phase 2b and 3) have demonstrated that tenapanor effectively meets both primary and secondary treatment goals, suggesting it could be a promising new option for patients with IBS-C who find conventional laxatives unsatisfactory.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tenapanor for the treatment of irritable bowel syndrome with constipation. Sinagra, E., Rossi, F., Raimondo, D., et al.[2022]

Tenapanor is a newly FDA-approved medication for treating irritable bowel syndrome with constipation (IBS-C) that works by inhibiting the sodium/hydrogen exchanger isoform 3 (NHE3), leading to increased fluid retention and softer stools.

In addition to improving bowel symptoms, tenapanor also helps reduce abdominal pain and visceral hypersensitivity, making it a promising option for IBS-C patients who do not respond to first-line treatments like soluble fiber.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tenapanor in the Treatment of Irritable Bowel Syndrome with Constipation: Discovery, Efficacy, and Role in Management. Herekar, A., Shimoga, D., Jehangir, A., et al.[2023]

In a pilot study of 14 children aged 8-16 with obstructive sleep apnea syndrome (OSAS), nasal expiratory positive airway pressure (NEPAP) significantly improved the obstructive apnea index compared to a placebo, indicating its potential efficacy as an alternative therapy.

Despite the overall positive results, individual responses to NEPAP varied, with some children showing no improvement or worsening symptoms, highlighting the need for further evaluation of its effectiveness through polysomnography.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pilot study of nasal expiratory positive airway pressure devices for the treatment of childhood obstructive sleep apnea syndrome. Kureshi, SA., Gallagher, PR., McDonough, JM., et al.[2021]

References

1. United States pubmed.ncbi.nlm.nih.gov

Efficacy of Tenapanor in Treating Patients With Irritable Bowel Syndrome With Constipation: A 26-Week, Placebo-Controlled Phase 3 Trial (T3MPO-2). [2023]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Tenapanor: First Approval. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Tenapanor Treatment of Patients With Constipation-Predominant Irritable Bowel Syndrome: A Phase 2, Randomized, Placebo-Controlled Efficacy and Safety Trial. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Efficacy of Tenapanor in Treating Patients With Irritable Bowel Syndrome With Constipation: A 12-Week, Placebo-Controlled Phase 3 Trial (T3MPO-1). [2022]

5. England pubmed.ncbi.nlm.nih.gov

Tenapanor for the treatment of irritable bowel syndrome with constipation. [2022]

6. New Zealand pubmed.ncbi.nlm.nih.gov

Tenapanor in the Treatment of Irritable Bowel Syndrome with Constipation: Discovery, Efficacy, and Role in Management. [2023]

7. China pubmed.ncbi.nlm.nih.gov

[Treatment of pediatric obstructive sleep apnea hypopnea syndrome]. [2004]

8. China pubmed.ncbi.nlm.nih.gov

[Observation the clinical curative effect of children's laryngopharyngeal reflux and sleep apnea hypopnea syndrome]. [2015]

9. Netherlands pubmed.ncbi.nlm.nih.gov

Positional device therapy for the treatment of positional obstructive sleep apnea in children: a pilot study. [2021]

10. United States pubmed.ncbi.nlm.nih.gov

Pilot study of nasal expiratory positive airway pressure devices for the treatment of childhood obstructive sleep apnea syndrome. [2021]

11. United States pubmed.ncbi.nlm.nih.gov

Updates on management of pediatric obstructive sleep apnea. [2020]