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Short Bowel SyndromePhase 3NCT03663582

Study of Teduglutide in Japanese Participants With Short Bowel Syndrome

A Phase 3 study testing Teduglutide, Syringe and Needle in people with Short Bowel Syndrome, run by Shire at 5 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The objectives of this clinical study are to evaluate the safety, efficacy, and pharmacokinetics (PK) of teduglutide in Japanese participants with short bowel syndrome (SBS) who are dependent on parenteral nutrition/intravenous (PN/IV) over a 24-week treatment period. Read more about this study

Who is it for?
Ages 16 years and older
Conditions the study is for
Short Bowel Syndrome
What is being tested?
Teduglutide, Syringe and Needle
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
5 sitesJapan
Study dates
6 Jul 2018to 6 Aug 2019
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 16 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Ability to voluntarily provide written, signed, and informed consent to participate in the study.
  • Male or female 16 years of age or older at the time of signing informed consent.
  • Intestinal failure due to short bowel syndrome (SBS) as a result of major intestinal resection (example, due to injury, volvulus, vascular disease, cancer, Crohn's disease) that resulted in at least 12 continuous months of parenteral nutrition/intravenous (PN/IV) dependence at the time of informed consent.
  • Parenteral nutrition requirement of at least 3 times per week during the week before the screening visit and during the 2 weeks prior to the baseline visit.
  • Stable PN/IV requirement for at least 4 consecutive weeks immediately prior to the start of teduglutide treatment. Stability is defined as: a. Actual PN/IV usage is similar to prescribed PN/IV; b. Baseline (Visit 2) 48-hour oral fluid intake and urine output (I/O) volumes fall within +/- 25 percent (%) of the respective 48-hour I/O volumes at the last optimization visit; c. Urine output volume should NOT fall below 2 liter (L) and should not exceed 4 L per 48 hours at the last optimization visit, the stabilization visit, and the baseline visit.
  • For participants with a history of Crohn's disease, clinical remission for at least 12 weeks prior to the baseline visit as demonstrated by clinical assessment, which may include procedure-based evidence of remission.
  • Females of childbearing potential must agree to comply with the contraceptive requirements of the protocol.
  • An understanding, ability, and willingness to fully comply with study procedures and restrictions.

Who cannot join

  • Participation in a clinical study using an experimental drug within 30 days or 5.5 halflives, whichever is longer, prior to screening, or concurrent participation in any other clinical study.
  • Use of glucagon-like peptide (GLP)-2 or human growth hormone or analogs of these hormones within the past 6 months.
  • Use of octreotide, GLP-1 analogs, dipeptidyl peptidase-IV inhibitors, or enteral glutamine within 30 days.
  • Previous use of teduglutide.
  • Participants with active inflammatory bowel disease (IBD) or participants with IBD who received a change in immunosuppressant therapy (example, azathioprine, anti- tumor necrosis factor (TNFs)) within the past 6 months.
  • Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, familial adenomatous polyposis, etc.
Show all 18Show fewer
  • Chronic intestinal pseudo-obstruction or severe dysmotility.
  • Clinically significant intestinal stenosis or obstruction, or evidence of such on upper gastrointestinal (GI) series with small bowel follow-through, within the past 6 months.
  • Major GI surgical intervention, including bowel lengthening procedures, within the past 3 months (insertion of feeding tube or endoscopic procedure is allowed).
  • Unstable cardiac disease, (example, congestive heart failure, cyanotic disease, or congenital heart disease).
  • Moderate or severe renal impairment, defined as creatinine clearance less than (<) 50 millilitre (ml)/ minute (min).
  • Currently diagnosed with cancer or a history of any cancer except surgically curative skin cancer within the past 5 years.
  • Severe hepatobiliary disease including: a. Total bilirubin level greater than or equal to (>=) 2 times the upper limit of normal (ULN); b. Aspartate aminotransferase (AST) >=5 times ULN; c. Alanine aminotransferase (ALT) >=5 times ULN.
  • Active clinically significant pancreatic disease, including clinical signs of pancreatitis associated with elevations in serum amylase or lipase >=2 times ULN.
  • More than 4 SBS-related or PN/IV-related hospital admissions (example, central line associated bloodstream infection, bowel obstruction, severe fluid/electrolyte disturbances) within the past 12 months.
  • Unscheduled hospitalization within 30 days prior to screening.
  • Pregnant or lactating female.
  • Any condition or circumstance that in the investigator's opinion put the participant at any undue risk, prevent completion of the study, or interfere with analysis of the study results.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

Japan5 sites
  • Hiroshima University Hospital

    Hiroshima, Hiroshima, 734-8551

  • Hyogo College of Medicine Hospital

    Hyōgo, 663-8501

  • Tohoku University Hospital

    Miyagi-Ken, 980-8574

  • Osaka University Hospital

    Osaka, 565-0871

  • Yokohama Municipal Citizen's Hospital

    Yokohama, 240-8555

Other trials in Japan

About this study

Official title: A 24-Week Safety, Efficacy, Pharmacokinetic Study of Teduglutide in Japanese Subjects With Short Bowel Syndrome Who Are Dependent on Parenteral Support

The objectives of this clinical study are to evaluate the safety, efficacy, and pharmacokinetics (PK) of teduglutide in Japanese participants with short bowel syndrome (SBS) who are dependent on parenteral nutrition/intravenous (PN/IV) over a 24-week treatment period.

Being tested
Teduglutide, Syringe, Needle, Vial Adapter for Device
Medical subject headings
Short Bowel Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TeduglutideDrug

    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.

  • SyringeDevice

    Teduglutide will be administered using syringe. Syringe is approved for use in Japan by Pharmaceuticals and Medical Devices Agency (PMDA).

  • NeedleDevice

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

  • Vial Adapter for DeviceDevice

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

Study groups in NCT03663582
Study groupRoleWhat they receive
Teduglutide 0.05 mgExperimentalParticipants 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 weeks.
Receives: Drug: Teduglutide; Device: Syringe; Device: Needle; Device: Vial Adapter for Device

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in weekly PS volume at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  2. Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

    Percent change from baseline in weekly PS volume at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  3. Percentage of Participants Who Achieved at Least 20 Percent (%) Reduction From Baseline in Weekly Parenteral Support (PS) Volume at Week 20

    Percentage of participants who achieved at least 20% reduction from baseline in weekly PS volume at Week 20 was reported.

    Measured: Baseline, Week 20

  4. Percentage of Participants Who Achieved at Least 20 Percent (%) Reduction From Baseline in Weekly Parenteral Support (PS) Volume at Week 24

    Percentage of participants who achieved at least 20% reduction from baseline in weekly PS volume at Week 24 was reported.

    Measured: Baseline, Week 24

  5. Percentage of Participants Who Achieved at Least 20 Percent (%) Reduction From Baseline in Weekly Parenteral Support (PS) at End of Treatment/Early Termination (EOT/ET)

    Percentage of participants who achieve at least 20% reduction from baseline in weekly PS at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  6. Change From Baseline in Days Per Week of Parenteral Support (PS) at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in days per week of PS at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  7. Change From Baseline in Plasma Citrulline Levels at End of Treatment/Early Termination (EOT/ET)

    Plasma citrulline levels were measured as a biomarker of enterocyte mass. Change from baseline in plasma citrulline levels up to EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  8. Number of Participants Who Were Completely Weaned Off Parenteral Support (PS) at Week 24/End of Treatment (EOT)

    Number of participants who were completely weaned off PS at Week 24/EOT was reported.

    Measured: Week 24/EOT

  9. Area Under the Plasma Concentration-Time Curve From Zero to the Last Measurable Concentration (AUC0-t) of Teduglutide

    AUC0-t of teduglutide was reported.

    Measured: Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours Post-dose on Day 1; Pre-dose, 1, 2 hours Post-dose on Week 4 or Week 12

  10. Maximum Plasma Concentration (Cmax) of Teduglutide

    Cmax of teduglutide was reported.

    Measured: Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours Post-dose on Day 1; Pre-dose, 1, 2 hours Post-dose on Week 4 or Week 12

  11. Time to Maximum Plasma Concentration (Tmax) of Teduglutide

    Tmax of teduglutide was reported.

    Measured: Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours Post-dose on Day 1; Pre-dose, 1, 2 hours Post-dose on Week 4 or Week 12

  12. Terminal-phase Half-life (T1/2) of Teduglutide

    T1/2 of teduglutide was reported.

    Measured: Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours Post-dose on Day 1; Pre-dose, 1, 2 hours Post-dose on Week 4 or Week 12

  13. Apparent Clearance (CL/F) of Teduglutide

    CL/F of teduglutide was reported.

    Measured: Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours Post-dose on Day 1; Pre-dose, 1, 2 hours Post-dose on Week 4 or Week 12

  14. Apparent Volume of Distribution (Vz/F) of Teduglutide

    Vz/F of teduglutide was reported.

    Measured: Pre-dose, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours Post-dose on Day 1; Pre-dose, 1, 2 hours Post-dose on Week 4 or Week 12

  15. Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    An adverse event (AE) was 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. TEAEs were defined as AEs whose onset occurred, severity worsened, or intensity increased after receiving the study medication.

    Measured: From start of study drug administration up to EOT/ET (up to Week 28)

  16. Number of Participants With Clinically Significant Abnormalities in 12-Lead Electrocardiogram (ECG)

    12-lead ECG was performed at the study center after the participant has been resting for at least 5 minutes. Number of participants with clinically significant abnormalities in 12-Lead ECG was reported.

    Measured: From start of study drug administration up to EOT/ET (up to Week 28)

  17. Change From Baseline in Blood Pressure at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in systolic and diastolic blood pressure at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  18. Change From Baseline in Pulse Rate at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in pulse rate at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  19. Change From Baseline in Body Temperature at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in body temperature at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  20. Change From Baseline in Hemoglobin at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in hemoglobin at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  21. Change From Baseline in Hematocrit at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in hematocrit at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  22. Change From Baseline in Serum Blood Urea Nitrogen (BUN) at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in serum blood urea nitrogen at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  23. Change From Baseline in Creatinine at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in creatinine at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  24. Change From Baseline in Urine Sodium at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in urine sodium at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  25. Number of Participants Who Reported Positive Specific Antibodies to Teduglutide at End of Treatment/Early Termination (EOT/ET)

    Number of participants who reported positive specific antibodies to teduglutide at EOT/ET was reported.

    Measured: EOT/ET (up to Week 28)

  26. Change From Baseline in 48-Hour Urine Output at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in 48-hour urine output at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  27. Change From Baseline in Body Weight at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in body weight at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  28. Change From Baseline in Body Mass Index (BMI) at End of Treatment/Early Termination (EOT/ET)

    Change from baseline in BMI at EOT/ET was reported.

    Measured: Baseline, EOT/ET (up to Week 28)

  29. Number of Participants With Abnormal Clinically Significant Changes in Gastrointestinal (GI) Specific Tests at Week 24/ET (Early Termination)

    GI specific tests included colonoscopy or sigmoidoscopy, abdominal ultrasound, upper GI series with small bowel follow-through (UGI/SBFT). Number of participants with abnormal clinically significant changes in gastrointestinal specific tests at Week 24/ET was reported.

    Measured: Week 24/ET

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Sep 2018
  2. Study start6 Jul 2018
  3. Primary completion6 Aug 2019
  4. Study completion6 Aug 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03663582
Sponsor's protocol ID
SHP633-306
Status last verified
Jul 2020
First submitted
28 Aug 2018
Last updated
4 Aug 2020
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites, …).

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Teduglutide, Syringe and Needle in people with Short Bowel Syndrome, run by Shire at 5 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) (time frame: Baseline, EOT/ET (up to Week 28)). The study lists 29 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Aug 2020.

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