Studio per valutare la sicurezza e l'efficacia di Tenapanor per il trattamento dell'IBS-C
Uno studio di fase 3 di 12 settimane, randomizzato, in doppio cieco, controllato con placebo per valutare la sicurezza e l'efficacia del tenapanor per il trattamento della sindrome dell'intestino irritabile con costipazione (IBS-C) in pazienti pediatrici da 12 a meno di 18 anni
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.
Panoramica dello studio
Stato
Intervento / Trattamento
Descrizione dettagliata
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
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 3
Contatti e Sedi
Luoghi di studio
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Alabama
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Foley, Alabama, Stati Uniti, 36535
- G & L Research, LLC
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Arizona
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Tucson, Arizona, Stati Uniti, 85745
- Eclipse Clinical Research
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Arkansas
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Little Rock, Arkansas, Stati Uniti, 72205
- Applied Research Center of Arkansas
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California
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Anaheim, California, Stati Uniti, 92805
- Advanced Research Center, Inc.
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Connecticut
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Hartford, Connecticut, Stati Uniti, 06106
- Connecticut Children's Medical Center
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Florida
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Doral, Florida, Stati Uniti, 33166
- Prohealth Research Center
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Kissimmee, Florida, Stati Uniti, 34741
- I.H.S. Health, LLC
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Miami, Florida, Stati Uniti, 33165
- Valencia Medical and Research Center
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Miami, Florida, Stati Uniti, 33155
- Waterway Research & Associates Corp
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Orlando, Florida, Stati Uniti, 32806
- Orlando Health, Inc.- APH Center for Digestive Health and Nutrition
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South Miami, Florida, Stati Uniti, 33143
- Florida Pharmaceutical Research and Associates, Inc.
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Georgia
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Stockbridge, Georgia, Stati Uniti, 30281
- Clinical Research Institute
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Illinois
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Peoria, Illinois, Stati Uniti, 61637
- OSF Saint Francis Medical Center
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Indiana
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Indianapolis, Indiana, Stati Uniti, 46202
- Riley Children's Health IUH
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Maine
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Portland, Maine, Stati Uniti, 04102
- Maine Health
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Massachusetts
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Boston, Massachusetts, Stati Uniti, 02115
- Boston Children's Hospital
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Minnesota
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Mankato, Minnesota, Stati Uniti, 56001
- Mankato Clinic/ Javara Research Ltd
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Nebraska
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Boys Town, Nebraska, Stati Uniti, 68010
- Boys Town National Research Hospital
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New Jersey
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Irvington, New Jersey, Stati Uniti, 07111
- Med Clinical Research Partners, LLC
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New Mexico
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Albuquerque, New Mexico, Stati Uniti, 87131
- University of New Mexico Health Sciences Center
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New York
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Brooklyn, New York, Stati Uniti, 11203
- SUNY Downstate Medical Center
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Rochester, New York, Stati Uniti, 14642
- University of Rochester
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The Bronx, New York, Stati Uniti, 10467
- Advantage Clinical Trials
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North Carolina
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Charlotte, North Carolina, Stati Uniti, 28203
- Atrium Health
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Durham, North Carolina, Stati Uniti, 27705
- Duke University School of Medicine
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Wilmington, North Carolina, Stati Uniti, 28401
- Wilmington Health
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Pennsylvania
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Scottdale, Pennsylvania, Stati Uniti, 15683
- Frontier Clinical Research, LLC
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Smithfield, Pennsylvania, Stati Uniti, 15478
- Frontier Clinical Research, LLC
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South Carolina
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Greenville, South Carolina, Stati Uniti, 29615
- Prisma Health Children's Hospital
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Texas
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Abilene, Texas, Stati Uniti, 79606
- Advance Clinical Trial PLLC
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El Paso, Texas, Stati Uniti, 79902
- Maspons Pediatric Gastro
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El Paso, Texas, Stati Uniti, 79902
- Proactive El Paso, LLC
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Harlingen, Texas, Stati Uniti, 78550
- Texas Digestive Specialists
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Plano, Texas, Stati Uniti, 75093
- AIM Trials, LLC
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San Antonio, Texas, Stati Uniti, 78215
- Sun Research Institute
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Sugar Land, Texas, Stati Uniti, 77479
- Pioneer Research Solutions Inc
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Waxahachie, Texas, Stati Uniti, 75165
- ClinPoint Trials
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West Virginia
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Huntington, West Virginia, Stati Uniti, 25701
- University Physicians and Surgeons, Inc.
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Kingwood, West Virginia, Stati Uniti, 26537
- Frontier Clinical Research
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Doppio
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: 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) |
|
Sperimentale: 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) |
|
Comparatore placebo: Placebo Comparator
Patients will be randomized to receive matching placebo twice daily |
Participants will be randomized to receive matching placebo |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
6/12-week APS (abdominal pain and SBM) +2 response
Lasso di tempo: 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.
|
12 weeks
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
6/12-week SBM +2 response
Lasso di tempo: 12 weeks
|
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
Lasso di tempo: 12 weeks
|
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
Lasso di tempo: 12 weeks
|
Change from baseline in average weekly SBM frequency |
12 weeks
|
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Change from baseline in average weekly stool consistency score
Lasso di tempo: 12 weeks
|
Change from baseline in average weekly stool consistency score |
12 weeks
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Change from baseline in average weekly abdominal pain score
Lasso di tempo: 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
Lasso di tempo: 12 weeks
|
Overall use of rescue medication |
12 weeks
|
Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: Jocelyn Tabora, Ardelyx
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Inizio studio (Effettivo)
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- TEN-01-304
Piano per i dati dei singoli partecipanti (IPD)
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Informazioni su farmaci e dispositivi, documenti di studio
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