Mobilize Trial

TEN-01-202

Dose-ranging study to assess safety and efficacy of tenapanor for treatment of irritable bowel syndrome with constipation (IBS-C) in pediatric patients six to less than 12 years old.

About This Study

This is a randomized, double-blind, placebo-controlled dose-ranging study to assess the safety and efficacy of tenapanor for treatment of the IBS-C in pediatric patients six to less than 12 years old.

Actively Enrolling

4-Week, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Assess the Safety & Efficacy of Tenapanor for the Treatment of IBS-C in Patients 6 to < 12 Yrs. Old

ClinicalTrials.gov ID: NCT06553547

ClinicalTrials.gov

Who May Participate

Individuals interested in participating in the study must meet certain criteria to qualify for screening and enrollment into the study.

Prior to enrollment, and after signing the informed consent, the patient will enter a 2-week screening period to ensure that the patient has active IBS-C.  During this time, patients will be required to self-report symptoms of IBS-C daily in an electronic diary (eDiary). Information in the eDiary will also be used to determine eligibility prior to enrollment.

Patient compliance with the eDiary is critical, as those who complete fewer than 71% of the daily entries will not be eligible to participate in the study.  Once the screening period is complete, the site staff will review the data to determine eligibility.

Inclusion criteria

  • Six and <12 years old at the screening visit
  • Females of child-bearing potential must have negative pregnancy test and confirm the use of appropriate contraception (including abstinence)
  • Patient meets the Modified Diagnostic Rome IV criteria for child/adolescent diagnosis of IBS-C
  • Patient is willing to discontinue any laxatives used in favor of the protocol-permitted rescue medicine
  • Meet the entry criteria assessed during the two-week screening period, including daily compliance with the ediary entries (a minimum of 71%).
  • Ability of both the patient and guardian to communicate with the Investigator and to comply with the requirements of the entire study, including an understanding of the assessments in the eDiary and how to use the eDiary device
  • Patient must provide verbal assent, and the guardian must provide written informed consent before the initiation of any study-specific procedures

Joining a clinical trial is an important and personal decision. We thank you for considering participation as an option that may be right for you.

Study Procedures

During the four-week randomized treatment period (RTP), subjects will be randomized to receive tenapanor or matching placebo for four weeks. During the RTP, patients will continue recording daily assessments in the eDiary system as instructed, and compliance with eDiary entries will be monitored. Patients will return for study visits every week for four weeks and will undergo safety assessments as per the protocol.

At the end of this four-week period, subjects will complete a two-week treatment free period, with safety assessments conducted following the two-week period.

The study plans to enroll up to six cohorts of eligible patients sequentially, starting from Cohort 1 with 12 patients randomized.  The dose for Cohort 1 will be tenapanor 2 mg BID or matching placebo for four weeks. Subsequent cohorts will receive increasing tenapanor doses, following a dose escalation order with the maximum dose of 25mg BID for Cohort 6 The study will proceed to the next dosing cohort if the current cohort completes the four-week RTP and does not meet any of the dose escalation stopping criteria pre-specified in the protocol.

4 weeks

Length of study treatment

Up to 7

Number of study visits

2 week

Treatment-free follow-up

Find a Trial Location

Search by state to find a trial location near you. Trial locations may change over time.

Frequently asked questions

Will my travel expense be reimbursed for participation in Mobilize?

The patient will be eligible for compensation for their time and travel with a pre-specified stipend for each completed visit.

What is the time commitment for participation?

Six in-person visits over eight weeks and daily eDiary entries (five minutes or less).

What is a randomized, double-blind, placebo-controlled, dose-ranging study?

A randomized, double-blind, placebo-controlled study is a rigorous research design commonly used in clinical trials to evaluate the effectiveness of new treatments or interventions. Here’s a breakdown of what each term means:

Randomized: Participants are randomly assigned to different groups—usually an experimental group that receives the treatment and a control group that receives a placebo or standard treatment. This helps ensure that the groups are comparable and reduces bias. Any benefit that is shown should be greater in the treatment group than the placebo group to show that the treatment is effective.

Double-blind: Both the participants and the researchers involved in administering the treatment and assessing the outcomes do not know which group participants are in (i.e., which group is receiving the treatment and which is receiving the placebo). This helps prevent bias in treatment administration and outcome assessment.

Placebo-controlled: The study includes a control group that receives a placebo, which is an inactive substance designed to look like the treatment but has no therapeutic effect. This helps determine whether the treatment has a genuine effect or if improvements are due to participants’ expectations or other factors.

Overall, this study design helps ensure the validity and reliability of the results by minimizing biases and controlling for various factors that could influence the outcome.

Dose-ranging: In order to determine the best and safest dose, the starting dose is generally a dose

What is the study measuring?

The purpose of the study is to assess the safety and efficacy of tenapanor at increasing doses for pediatric IBS-C patients ages six to 11. The primary measurements include the number of daily spontaneous bowel movements (those that occur without any other medications used to treat constipation) and the decrease in abdominal pain associated with IBS-C.

Are there benefits to participating in Mobilize?

Your symptoms of irritable bowel syndrome (IBS) may or may not improve and could even worsen if you take part in this study. There is no expected direct medical benefit to you from participating in this study. Your participation in the study will contribute important information about the study drug and may benefit pediatric IBS-C patients in the future.

Are there risks to participating in Mobilize?

There are potential harms or risks if you take part in this study. The drugs you would take can cause side effects, sometimes severe. A side effect is an unintended result of the treatment that is not related to the reason the treatment is being used. Some are common and some are rare. Please tell the study doctor or study staff right away if you have any side effects or any other problems with your health whether or not you think these problems are related to the study drug.

Tenapanor contains a warning for risk of serious dehydration in pediatric patients. In the most recent 26-week placebo-controlled study in about 600 people with IBS-C, the most common side effects associated with taking tenapanor that were reported in more than 2% of tenapanor-treated people and were greater than placebo include:

  • Diarrhea (16%)
  • Abdominal distension (swelling or bloating) (3%)
  • Flatulence (having gas) (3%)
  • Dizziness (2%)

Please see Medication Guide within the full Prescribing Information for additional risk information.