Studie om de veiligheid en werkzaamheid van Tenapanor voor de behandeling van IBS-C te beoordelen
Een 12 weken durend, gerandomiseerd, dubbelblind, placebogecontroleerd, fase 3-onderzoek om de veiligheid en werkzaamheid van Tenapanor te beoordelen voor de behandeling van het prikkelbaredarmsyndroom met constipatie (PDS-C) bij pediatrische patiënten van 12 tot jonger dan 18 jaar
This is a randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of tenapanor (25 mg and 50 mg) in pediatric patients (≥12 and <18 years old) with IBS-C when administered twice daily (BID) for 12 consecutive weeks.
Studie Overzicht
Toestand
Interventie / Behandeling
Gedetailleerde beschrijving
This study consists of 2-week screening period followed by 12 week randomized treatment period (RTP) and a 2-week treatment-free Follow-Up period (only for patients who will not enter the 40-week Long Term Safety Extension Study [TEN-01-306]). At the beginning of the 2-week Screening period, patients who provide written assent will be fully assessed for eligibility into the study and will be asked to self-report daily information about the status of their IBS symptoms via an electronic diary (eDiary) device. Patient compliance with the eDiary will be monitored actively by the site staff and will be reviewed to determine eligibility at the end of screening. Eligible patients will be randomized to receive one of the study medications: tenapanor 25 mg BID, tenapanor 50 mg BID, or placebo.
During the 12-week double-blind RTP, patients will continue recording daily assessments via the eDiary system as instructed and compliance with eDiary entries will be monitored on an ongoing basis. Patients will return for study visit every two or four weeks (Visits 3-6) and will undergo safety assessments at these visits.
Patients who do not enter the 40-week Long Term Safety Extension Study [TEN-01-306] including those who complete the RTP but do not enter study TEN-01-306 and those who prematurely discontinue from the RTP, a Follow-Up Visit will be scheduled approximately 2 weeks after the completion of the RTP (Visit 6) or the Early Termination Visit at which safety assessments will be performed
Studietype
Inschrijving (Werkelijk)
Fase
- Fase 3
Contacten en locaties
Studie Locaties
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Alabama
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Foley, Alabama, Verenigde Staten, 36535
- G & L Research, LLC
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Arizona
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Tucson, Arizona, Verenigde Staten, 85745
- Eclipse Clinical Research
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Arkansas
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Little Rock, Arkansas, Verenigde Staten, 72205
- Applied Research Center of Arkansas
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California
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Anaheim, California, Verenigde Staten, 92805
- Advanced Research Center, Inc.
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Connecticut
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Hartford, Connecticut, Verenigde Staten, 06106
- Connecticut Children's Medical Center
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Florida
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Doral, Florida, Verenigde Staten, 33166
- Prohealth Research Center
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Kissimmee, Florida, Verenigde Staten, 34741
- I.H.S. Health, LLC
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Miami, Florida, Verenigde Staten, 33165
- Valencia Medical and Research Center
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Miami, Florida, Verenigde Staten, 33155
- Waterway Research & Associates Corp
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Orlando, Florida, Verenigde Staten, 32806
- Orlando Health, Inc.- APH Center for Digestive Health and Nutrition
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South Miami, Florida, Verenigde Staten, 33143
- Florida Pharmaceutical Research and Associates, Inc.
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Georgia
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Stockbridge, Georgia, Verenigde Staten, 30281
- Clinical Research Institute
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Illinois
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Peoria, Illinois, Verenigde Staten, 61637
- OSF Saint Francis Medical Center
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Indiana
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Indianapolis, Indiana, Verenigde Staten, 46202
- Riley Children's Health IUH
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Maine
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Portland, Maine, Verenigde Staten, 04102
- Maine Health
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Massachusetts
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Boston, Massachusetts, Verenigde Staten, 02115
- Boston Children's Hospital
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Minnesota
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Mankato, Minnesota, Verenigde Staten, 56001
- Mankato Clinic/ Javara Research Ltd
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Nebraska
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Boys Town, Nebraska, Verenigde Staten, 68010
- Boys Town National Research Hospital
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New Jersey
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Irvington, New Jersey, Verenigde Staten, 07111
- Med Clinical Research Partners, LLC
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New Mexico
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Albuquerque, New Mexico, Verenigde Staten, 87131
- University of New Mexico Health Sciences Center
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New York
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Brooklyn, New York, Verenigde Staten, 11203
- SUNY Downstate Medical Center
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Rochester, New York, Verenigde Staten, 14642
- University of Rochester
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The Bronx, New York, Verenigde Staten, 10467
- Advantage Clinical Trials
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North Carolina
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Charlotte, North Carolina, Verenigde Staten, 28203
- Atrium Health
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Durham, North Carolina, Verenigde Staten, 27705
- Duke University School of Medicine
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Wilmington, North Carolina, Verenigde Staten, 28401
- Wilmington Health
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Pennsylvania
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Scottdale, Pennsylvania, Verenigde Staten, 15683
- Frontier Clinical Research, LLC
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Smithfield, Pennsylvania, Verenigde Staten, 15478
- Frontier Clinical Research, LLC
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South Carolina
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Greenville, South Carolina, Verenigde Staten, 29615
- Prisma Health Children's Hospital
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Texas
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Abilene, Texas, Verenigde Staten, 79606
- Advance Clinical Trial PLLC
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El Paso, Texas, Verenigde Staten, 79902
- Maspons Pediatric Gastro
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El Paso, Texas, Verenigde Staten, 79902
- Proactive El Paso, LLC
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Harlingen, Texas, Verenigde Staten, 78550
- Texas Digestive Specialists
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Plano, Texas, Verenigde Staten, 75093
- AIM Trials, LLC
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San Antonio, Texas, Verenigde Staten, 78215
- Sun Research Institute
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Sugar Land, Texas, Verenigde Staten, 77479
- Pioneer Research Solutions Inc
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Waxahachie, Texas, Verenigde Staten, 75165
- ClinPoint Trials
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West Virginia
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Huntington, West Virginia, Verenigde Staten, 25701
- University Physicians and Surgeons, Inc.
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Kingwood, West Virginia, Verenigde Staten, 26537
- Frontier Clinical Research
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
Inclusion Criteria:
- ≥12 and <18 years old
- Patient weighs ≥18 kg at the time the patient provides written assent
- Females of child-bearing potential must have negative pregnancy test at Visit 1 (serum) and Visit 2 (urine) and confirm the use of appropriate contraception (including abstinence).
- Patient meets the 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 (which will only be allowed after 72 hours with no bowel movement)
- Patient meets the entry criteria assessed during the 2-week Screening period.
- Ability of both the patient and parent/guardian/LAR 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 written assent and the parent/guardian/LAR must provide written informed consent before the initiation of any study-specific procedures
Exclusion Criteria:
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Functional diarrhea as defined by Rome IV child/adolescent criteria
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IBS with diarrhea (IBS-D), mixed IBS (IBS-M), or unsubtyped IBS as defined by Rome IV child/adolescent criteria
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History of non-retentive fecal incontinence.
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Required manual disimpaction any time prior to randomization (after consent);
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Has both unexplained and clinically significant alarm symptoms (lower gastrointestinal [GI] bleeding [rectal bleeding or heme-positive stool], iron-deficiency anemia, or any unexplained anemia, or weight loss) and systemic signs of infection or colitis, or any neoplastic process
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Patient has any of the following conditions:
- Celiac disease, or positive serological test for celiac disease
- Cystic fibrosis
- Hypothyroidism that is untreated or treated with thyroid hormone
- Down's syndrome or any other chromosomal disorder
- Active anal fissure
- Anatomic malformations (eg, imperforate anus)
- Intestinal nerve or muscle disorders (eg, Hirschprung disease)
- Neuropathic conditions (eg, spinal cord abnormalities)
- Lead toxicity, hypercalcemia
- Neurodevelopmental disabilities producing a cognitive delay that precludes comprehension and completion of the daily eDiary (Electronic handheld device)
- Inflammatory bowel disease
- Childhood functional abdominal pain syndrome
- Childhood functional abdominal pain;
- Poorly treated or poorly controlled psychiatric disorders that might influence his or her ability to participate in the study;
- Lactose intolerance that is associated with abdominal pain or discomfort
- History of cancer other than treated basal cell carcinoma of the skin; (Note: Patients with a history of cancer are allowed provided that the malignancy has been in a complete remission for at least 5 years before the Randomization Visit.)
- History of diabetic neuropathy.
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Use of medications that are known to affect stool consistency (Prohibited Medications), including fiber supplements, anti-diarrheals, cathartics, antacids, opiates, prokinetic drugs, laxatives, enemas, antibiotics during the Screening period; unless specified as rescue medication, and used accordingly as directed by the Investigator.
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Patient has had surgery that meets any of the following criteria:
- Bariatric surgery for treatment of obesity, or surgery to remove a segment of the GI tract at any time before the Screening Visit;
- Surgery of the abdomen, pelvis, or retroperitoneal structures during the 6 months before the Screening Visit;
- An appendectomy or cholecystectomy during the 60 days before the Screening Visit;
- Other major surgery during the 30 days before the Screening Visit
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History of alcohol or substance abuse
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Participation in other clinical trials within 1 month prior to Screening
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Patient and/or parent/guardian/LAR is involved in the conduct and/or administration of this trial as an investigator, sub-investigator, trial coordinator, or other staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved in the trial
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If, in the opinion of the Investigator, the patient is unable or unwilling to fulfill the requirements of the protocol or has a condition, which would render the results uninterpretable
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Gerandomiseerd
- Interventioneel model: Parallelle opdracht
- Masker: Dubbele
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: Tenpanor 50 mg BID
Patients will be randomized to receive 50 mg tenapanor twice daily |
Participants will receive tenapanor 50 mg BID (total of 100 mg daily) |
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Experimenteel: Tenpanor 25 mg BID
Patients will be randomized to receive 25 mg tenapanor twice daily |
Participants will receive tenapanor 25 mg BID (total of 50 mg daily) |
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Placebo-vergelijker: Placebo Comparator
Patients will be randomized to receive matching placebo twice daily |
Participants will be randomized to receive matching placebo |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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6/12-week APS (abdominal pain and SBM) +2 response
Tijdsspanne: 12 weeks
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6/12-week APS (abdominal pain and SBM (spontaneous bowel movement)) +2 response, defined as achieving the weekly APS +2 response criteria (i.e., achieving both weekly SBM +2 response and weekly abdominal pain response during the same week) for ≥6 out of the 12 weeks of the RTP.
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12 weeks
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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6/12-week SBM +2 response
Tijdsspanne: 12 weeks
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6/12-week SBM +2 response: defined as achieving the weekly SBM +2 response for ≥6 out of the 12 weeks of the RTP |
12 weeks
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6/12-week abdominal pain response
Tijdsspanne: 12 weeks
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6/12-week abdominal pain response: defined as achieving the weekly abdominal pain response for ≥6 out of the 12 weeks of the RTP |
12 weeks
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Change from baseline in average weekly SBM frequency
Tijdsspanne: 12 weeks
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Change from baseline in average weekly SBM frequency |
12 weeks
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Change from baseline in average weekly stool consistency score
Tijdsspanne: 12 weeks
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Change from baseline in average weekly stool consistency score |
12 weeks
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Change from baseline in average weekly abdominal pain score
Tijdsspanne: 12 weeks
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Change from baseline in average weekly abdominal pain score |
12 weeks
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Overall use of rescue medication
Tijdsspanne: 12 weeks
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Overall use of rescue medication |
12 weeks
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Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Studie directeur: Jocelyn Tabora, Ardelyx
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- TEN-01-304
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
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