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A Study in Japanese Children With Short Bowel Syndrome Who Completed SHP633-302

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ClinicalTrials.gov Identifier: NCT03268811
Recruitment Status : Recruiting
First Posted : August 31, 2017
Last Update Posted : October 8, 2018
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
The purpose of this clinical study is to evaluate the long-term safety and tolerability of teduglutide treatment in Japanese pediatric participants with short bowel syndrome (SBS) who completed Study SHP633-302 (NCT02980666).

Condition or disease Intervention/treatment Phase
Short Bowel Syndrome Drug: Teduglutide Device: Syringe Device: Needle Device: Vial Adapter for Device Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 7 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 Japanese Pediatric Subjects With Short Bowel Syndrome Who Completed SHP633-302
Actual Study Start Date : August 23, 2017
Estimated Primary Completion Date : June 1, 2020
Estimated Study Completion Date : June 1, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Teduglutide

Arm Intervention/treatment
Experimental: Teduglutide
Participants will receive teduglutide 0.05 milligram per kilogram (mg/kg) subcutaneous (SC) injection once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm for 24-week intervals. If a participant deteriorates during a follow-up period, the participant may be evaluated immediately for additional teduglutide treatment (24-week interval) until teduglutide is commercially available for each participant, the participant's participation in this study is discontinued, or the study is discontinued.
Drug: Teduglutide
Teduglutide 0.05 mg/kg SC injection will be administered once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm.

Device: Syringe
Teduglutide will be administered using a syringe. Syringe is approved for use in Japan by pharmaceuticals and medical devices agency (PMDA).

Device: Needle
Teduglutide will be administered using needle. Needle is approved for use in Japan by PMDA.

Device: Vial Adapter for Device
Vial adapter for device is approved for use in Japan by PMDA.




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

  2. Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.

  3. Number of Participants With Clinically Significant Laboratory Safety Data Reported as an Adverse Event [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Laboratory safety data includes biochemistry, hematology and urinalysis.

  4. Number of Participants With Significant Change in Urine Output Reported as an Adverse Event [ 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 parenteral support (PS) stability.

  5. Number of Participants With Significant Change in Stool Output Reported as an Adverse Event [ 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 PS stability.

  6. Number of Participants With Positive Specific or Neutralizing Antibodies to Teduglutide [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Blood samples will be drawn for the analysis of positive/specific antibodies to teduglutide.

  7. Number of Participants With a Positive Fecal Occult Blood Testing [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Gastrointestinal-specific assessment will be analysed by fecal occult blood testing. Participants with newly positive fecal occult blood testing results at the pre-treatment visit for which a readily detectable cause cannot be identified (example: anal fissure ) will undergo a colonoscopy prior to receiving teduglutide.

  8. Number of Participants With Colonoscopy/Sigmoidoscopy Results [ Time Frame: Baseline to each study visit for approximately 3 years ]
    Participants who have received the equivalent of 2 treatment cycles (48 weeks of investigational product exposure) will undergo colonoscopy/sigmoidoscopy.

  9. Change From Baseline in Z-Score Body Weight [ Time Frame: Baseline to each study visit for approximately 3 years ]
    The Z-score indicates the number of standard deviations away from the mean.

  10. Change From Baseline in Z-Score Height [ Time Frame: Baseline to each study visit for approximately 3 years ]
    The Z-score indicates the number of standard deviations away from the mean.

  11. Change From Baseline in Z-Score Head Circumference [ Time Frame: Baseline to each study visit for approximately 3 years ]
    The Z-score indicates the number of standard deviations away from the mean.

  12. Change From Baseline in Z-Score Body Mass Index (BMI) [ Time Frame: Baseline to each study visit for approximately 3 years ]
    The Z-score indicates the number of standard deviations away from the mean.


Secondary Outcome Measures :
  1. Number of Participants who Demonstrate at least 20 Percent (%) Reduction From Baseline in Parenteral Support (PS) Volume [ Time Frame: Baseline to each study visit for approximately 3 years ]
    PS (parenteral nutrition or intravenous fluids) will be considered for managing nutritional support in terms of volume and calories during the treatment period.

  2. Absolute Change From Baseline in Parenteral Support (PS) Volume [ Time Frame: Baseline to each study visit for approximately 3 years ]
    PS (parenteral nutrition or intravenous fluids) will be considered for managing nutritional support in terms of volume and calories during the treatment period.

  3. Relative Change From Baseline in Parenteral Support (PS) Volume [ Time Frame: Baseline to each study visit for approximately 3 years ]
    PS (parenteral nutrition or intravenous fluids) will be considered for managing nutritional support in terms of volume and calories during the treatment period.

  4. Number of Participants who Completely Wean off Parenteral Support (PS) [ Time Frame: Baseline to each study visit for approximately 3 years ]
    PS (parenteral nutrition or intravenous fluids) will be considered for managing nutritional support in terms of volume and calories during the treatment period.

  5. Change in Days per Week of Parenteral Support (PS) [ Time Frame: Baseline to each study visit for approximately 3 years ]
    PS (parenteral nutrition or intravenous fluids) will be considered for managing nutritional support in terms of volume and calories during the treatment period.



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

Inclusion Criteria:

  • Informed consent by a parent or guardian prior to any study-related procedures.
  • When applicable, informed assent (as deemed appropriate by the Institutional Review Board [IRB]) by the participant prior to any study-related procedures.
  • Participant completed Study SHP633-302 (NCT02980666).
  • Participant (and/or parent/guardian) understands and is willing and able to fully adhere to study requirements as defined in this protocol.

Exclusion Criteria:

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): NCT03268811


Contacts
Contact: Shire Contact 1 866-842-5335 ClinicalTransparency@shire.com

Locations
Japan
Kyushu University Hospital Not yet recruiting
Fukuoka-shi, Fukuoka-ken, Japan, 812-8582
Contact: Site Contact    +819264111151    matsuura@pedsurg.med.kyushu-u.ac.jp   
Principal Investigator: Toshiharu Matsuura, MD         
Tsukuba University Hospital Not yet recruiting
Tsukuba, Ibaraki-Ken, Japan, 305-8576
Contact: Site Contact    +181298533900    kmasu@md.tsukuba.ac.jp   
Principal Investigator: Kouji Masumoto, MD         
Kagoshima University Hospital Recruiting
Kagoshima, Kagoshima-Ken, Japan, 890-8520
Contact: Site Contact    +81992755111    tatu@m2.kufm.kagoshima-u.ac.jp   
Principal Investigator: Tatsuru Kaji         
Tohoku University Hospital Not yet recruiting
Sendai-shi, Miyagi-Ken, Japan, 980-8574
Contact: Site Contact    +81227177000      
Principal Investigator: Motoshi Wada, MD         
Showa University Not yet recruiting
Shinagawa-ku, Tokyo-To, Japan, 142-8555
Contact: Site Contact    +81337848000      
Principal Investigator: Masahiro Chiba, MD         
Sponsors and Collaborators
Shire
Investigators
Study Director: Study Director Shire

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03268811     History of Changes
Other Study ID Numbers: SHP633-305
First Posted: August 31, 2017    Key Record Dates
Last Update Posted: October 8, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Short Bowel Syndrome
Disease
Pathologic Processes
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Postoperative Complications