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Long-term Safety and Efficacy Study of Teduglutide in Pediatric Participants With Short Bowel Syndrome (SBS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02954458
Recruitment Status : Active, not recruiting
First Posted : November 3, 2016
Last Update Posted : April 1, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
This study will follow participants who completed the TED-C14-006 study. The purpose of this study is to evaluate the long-term safety and efficacy of teduglutide in pediatric participants with Short Bowel Syndrome (SBS). This study will also offer teduglutide treatment to eligible participants, regardless of treatment received in TED-C14-006 or SHP633-301.

Condition or disease Intervention/treatment Phase
Short Bowel Syndrome Drug: TED Other: SOC Device: Syringe Device: Needle Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 65 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Pediatric Patients With Short Bowel Syndrome Who Completed TED-C14-006 or SHP633-301
Actual Study Start Date : January 9, 2017
Estimated Primary Completion Date : September 30, 2019
Estimated Study Completion Date : September 30, 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Teduglutide

Arm Intervention/treatment
Experimental: Standard of care (SOC) treatment +/- teduglutide (TED)
Participants will receive 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injections once daily into 1 of the 4 quadrants of the abdomen or into either the thigh or arm as needed in addition to SOC treatment.
Drug: TED
0.05 mg/kg SC injection once daily.

Other: SOC
Standard safety assessments and adjustments in nutritional support.

Device: Syringe
Teduglutide will be administered using syringe.

Device: Needle
Teduglutide will be administered using needle.




Primary Outcome Measures :
  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Baseline, each study visit for approximately 3 years ]
    An adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.

  2. Change From Baseline in Urine Output [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Urine output will be recorded in the output diary over a 48-hour period of nutritional stability before every clinic visit, and within 1 week of implementing a change in the PN prescription.

  3. Change From Baseline in Stool Output [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Stool output will be recorded in the output diary over a 48-hour period of nutritional stability before every clinic visit, and within 1 week of implementing a change in the PN prescription.

  4. Antibodies to teduglutide [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Number of participants classified as having positive specific antibodies to teduglutide will be used to summarize the presence of antibodies.

  5. Change From Baseline in Body Weight Z-Score [ Time Frame: Baseline, each study visit for approximately 3 years ]
    Change from baseline in body weight Z-Score will be assessed.

  6. Change From Baseline in Length Z-Score [ Time Frame: Baseline, each study visit for approximately 3 years ]
    Change from baseline in length Z-Score will be assessed.

  7. Change From Baseline in Head Circumference Z-Score [ Time Frame: Baseline, each study visit for approximately 3 years ]
    Change from baseline in head circumference Z-Score will be assessed.

  8. Change From Baseline in Body Mass Index Z-Score [ Time Frame: Baseline, each study visit for approximately 3 years ]
    Change from baseline in body mass index Z-Score will be assessed.


Secondary Outcome Measures :
  1. Number of Participants who Achieve At least a 20 Percent (%) Reduction From Baseline in Parenteral Support (PS) Volume [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Number of participants who achieve at least a 20% reduction from baseline in PS volume will be assessed.

  2. Change in Parenteral Support (PS) Volume [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Change in PS volume will be assessed.

  3. Number of Participants who Were Completely Weaned off Parenteral Support (PS) [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Number of participants who were completely weaned off parenteral support (PS) will be assessed.

  4. Change From Baseline in Days per Week of Parenteral Support (PS) [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Change from baseline in days per week of parenteral support (PS) will be assessed.


Other Outcome Measures:
  1. Change in Pediatric Quality of Life Inventory (PedsQL) Score [ Time Frame: Approximately every 12 weeks for 3 years ]
    The Child and Parent Reports of the PedsQL Generic Core Scale for Young Pediatric subjects (ages 5-7), Pediatric subjects (ages 8-12), and Teens (ages 13-18) are composed of 23 items comprising 4 dimensions as follows: 1) Physical Functioning (8 items), 2) Emotional Functioning (5 items), 3) Social Functioning (5 items), 4) School Functioning (5 items).

  2. Change in Pediatric Quality of Life inventory (PedsQL) in Family Impact Module Score [ Time Frame: Approximately every 12 weeks for 3 years ]
    The 36-item PedsQL Family Impact Module consists of 6 scales measuring parent self-reported functioning as follows: 1) Physical Functioning (6 items), 2) Emotional Functioning (5 items), 3) Social Functioning (4 items), 4) Cognitive Functioning (5 items; worries about treatment and disease), 5) Communication (3 items), 6) Worry (5 items).

  3. Change in Pediatric Quality of Life inventory (PedsQL) Gastrointestinal Symptoms Sub-scales for Food and Drink Limits and Diarrhea [ Time Frame: Approximately every 12 weeks for 3 years ]
    The PedsQL Gastrointestinal Symptom Module is a disease-specific 58-item module, comprised of 10 different symptom scales that assess gastrointestinal symptom-related quality of life: food and drink limits, trouble swallowing, heartburn and reflux, nausea and vomiting, gas and bloating, constipation, blood in poop, and diarrhea.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Participant provides written informed consent (participant, parent or legal guardian and, as appropriate, informed assent) to participate in the study before completing any study-related procedures.
  2. Participant completed the TED-C14-006 or SHP633-301 studies (including participants in the standard of care treatment arms). Participants are considered to have completed SHP633-301 if they completed study assessments through week 24.
  3. Participant understands and is willing and able to fully adhere to study requirements as defined in this protocol.

Exclusion Criteria:

1. There are no exclusion criteria for this study.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02954458


  Show 22 Study Locations
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02954458     History of Changes
Other Study ID Numbers: SHP633-304
2016-000849-30 ( EudraCT Number )
First Posted: November 3, 2016    Key Record Dates
Last Update Posted: April 1, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No

Additional relevant MeSH terms:
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Syndrome
Short Bowel Syndrome
Disease
Pathologic Processes
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Postoperative Complications
Teduglutide
Gastrointestinal Agents
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs