Study of Teduglutide Effectiveness in Parenteral Nutrition (PN)-Dependent Short Bowel Syndrome (SBS) Subjects

NCT00798967 | Completed Phase 3 Results DMC

• 2 other identifiers: CL0600-0202008-006193-15
• Study Type: interventional
• Target: 86
• Locations: 10 countries
• Sites: 35

Brief Summary

Teduglutide is an investigative medicine being evaluated as a possible treatment for people with parenteral nutrition (PN) dependent Short Bowel Syndrome (SBS). Teduglutide is similar to a protein the body makes. When people have SBS, their bodies do not make enough of the protein and they have trouble getting nutrients and fluids from the food they eat and drink. This study was designed to provide evidence of efficacy, safety, and tolerability of teduglutide 0.05 mg/kg daily in SBS subjects.

Conditions

Short Bowel Syndrome

Keywords

SBS; short bowel syndrome; TPN; parenteral nutrition; PN

Outcome Measures

Primary Outcomes (1)

• Responder

  Comparison of subjects treated with teduglutide to placebo who achieve a 20 to 100% reduction from baseline in weekly parenteral nutrition/intravenous fluid (PN/I.V.) volume at weeks 20 and 24.

  Weeks 20 and 24

Secondary Outcomes (1)

• Absolute Change in PN/I.V. Volume From Baseline to Last Time Point

  Week 0 to last visit when data was collected.

Study Arms (2)

Teduglutide

EXPERIMENTAL

0.05 mg/kg/day sc dose of teduglutide

Drug: teduglutide

Placebo

PLACEBO COMPARATOR

Matching subcutaneous dose of placebo to teduglutide

Drug: placebo

Interventions

teduglutide DRUG

0.05 mg/kg/day sc injection for 24 weeks

Also known as: GATTEX, ALX-0600

Teduglutide

placebo DRUG

Matching daily subcutaneous dose of placebo to teduglutide for 24 weeks

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed and dated informed consent prior to any study-related procedures are performed
• Men and women 18 years of age or older at the time of informed consent signing
• Intestinal failure resulting in Short Bowel Syndrome
• At least 12 months of continuous PN dependency
• weeks of clinical remission of Crohn's disease (CD) prior to dosing
• PN required at least 3 times weekly
• A stable PN volume for four weeks prior to dosing

You may not qualify if:

• History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state
• Participation in clinical study within 30 days for drug or 90 days for antibody
• Use of native GLP-2 or human growth hormone (HGH) within 6 months of screening
• Use of iv glutamine within 30 days prior to screening
• Use of teduglutide
• CD patients who have been treated with biological therapy within 6 months of screening
• IBD patients who require chronic systemic immunosuppressant therapy
• More than 4 SBS- or PN-related hospitalizations within 12 months of screening
• Unplanned hospitalization within one month of screening
• Pregnant or lactating women
• Body weight \> 88kg
• Body mass index (BMI) \< 15 kg/m2
• Severe hepatic impairment or disturbed renal function
• Female subjects who are not surgically sterile or postmenopausal or who are not using medically acceptable methods of birth control during and for 30 days after the treatment period
• Not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements

Sponsors & Collaborators

• Shire: lead
• Nycomed: collaborator

Study Sites (35)

Scripps Clinic & Research Foundation

La Jolla, California, 92037, United States

Location

Washington Hospital Center

Washington D.C., District of Columbia, 20010, United States

Location

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66160, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

University of Pennslyvania

Philadelphia, Pennsylvania, 19104, United States

Location

University Of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Critical Care Research, Royal Alexandra Hospital

Edmonton, Alberta, T5H 3V9, Canada

Location

Hamilton Health Sciences Centre

Hamilton, Ontario, L8N 3Z5, Canada

Location

Polyclinic Family and Specialty Medicine Facility

North York, Ontario, M3H 5S4, Canada

Location

University Health Network - Toronto General Hospital

Toronto, Ontario, M5G 2N2, Canada

Location

Rigshospitalet - Abdominalcentret, Hepatologisk Kilinik A

Copenhagen, 2100, Denmark

Location

Hôpital Beaujon

Clichy, 92110, France

Location

Hôpital Croix Rousse Unité de Nutrition Clinique Intensive

Lyon, France

Location

Hôpital de l'Archet Pôle Digestif

Nice, 06100, France

Location

Universitaetsklinikum Tuebingen

Tübingen, Baden-Wurttemberg, 72076, Germany

Location

Israelitisches Krankenhaus

Hamburg, Hamburg, 22297, Germany

Location

Med. Klinik m.S. Hepatologie und Gastroenterologie

Berlin, State of Berlin, 13353, Germany

Location

Azienda Ospedaliera Universitaria S. Giovanni Battista - Le Molinette

Torino, TO, 10126, Italy

Location

Policlinico Sant'Orsola - Malpighi centro insufficienza intestinale

Bologna, 40138, Italy

Location

Azienda Universitaria Policlinico Federico II

Napoli, 80131, Italy

Location

Universitair Medisch Centrum St. Radboud

Nijmegen, 6525 GA, Netherlands

Location

Wojewodzki Szpital Specjalistyczny im. M. Pirogowa, Pracownia Leczenia Zywieniowego

Lodz, 90-531, Poland

Location

Samodzielny Publiczny Szpital Kliniczny nr 4, Klinika Chirurgii Ogolnej i Transplantacyjnej

Lublin, 20-954, Poland

Location

Wojewodzki Szpital Specjalistyczny,

Olsztyn, 10-561, Poland

Location

Samodzielny Publiczny Szpital Kliniczny im. prof W. Orlowskiego CMKP, Oddzial Kliniczny Zywienia i Chirurgii

Warsaw, 00-416, Poland

Location

Hospital Universitario de Bellvitge

Barcelona, 08907, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

St. Mark's Hospital Northwick Park

Harrow, GT LON, HA1 3UJ, United Kingdom

Location

University College Hospital

London, GT LON, NW1 2BU, United Kingdom

Location

Green Area, Main Hospital Salford Royal Hospitals NHS Foundation Trust

Manchester, GT MAN, M6 8HD, United Kingdom

Location

Related Publications (8)

• Jeppesen PB, Sanguinetti EL, Buchman A, Howard L, Scolapio JS, Ziegler TR, Gregory J, Tappenden KA, Holst J, Mortensen PB. Teduglutide (ALX-0600), a dipeptidyl peptidase IV resistant glucagon-like peptide 2 analogue, improves intestinal function in short bowel syndrome patients. Gut. 2005 Sep;54(9):1224-31. doi: 10.1136/gut.2004.061440.

  PMID: 16099790 RESULT

• Jeppesen PB, Gabe SM, Seidner DL, Lee HM, Olivier C. Citrulline correlations in short bowel syndrome-intestinal failure by patient stratification: Analysis of 24 weeks of teduglutide treatment from a randomized controlled study. Clin Nutr. 2020 Aug;39(8):2479-2486. doi: 10.1016/j.clnu.2019.11.001. Epub 2019 Nov 9.

  PMID: 31784300 DERIVED

• Jeppesen PB, Gabe SM, Seidner DL, Lee HM, Olivier C. Factors Associated With Response to Teduglutide in Patients With Short-Bowel Syndrome and Intestinal Failure. Gastroenterology. 2018 Mar;154(4):874-885. doi: 10.1053/j.gastro.2017.11.023. Epub 2017 Nov 22.

  PMID: 29174926 DERIVED

• Fujioka K, Jeejeebhoy K, Pape UF, Li B, Youssef NN, Schneider SM. Patients With Short Bowel on Narcotics During 2 Randomized Trials Have Abdominal Complaints Independent of Teduglutide. JPEN J Parenter Enteral Nutr. 2017 Nov;41(8):1419-1422. doi: 10.1177/0148607116663481. Epub 2016 Aug 9.

  PMID: 27507402 DERIVED

• Vipperla K, O'Keefe SJ. Study of teduglutide effectiveness in parenteral nutrition-dependent short-bowel syndrome subjects. Expert Rev Gastroenterol Hepatol. 2013 Nov;7(8):683-7. doi: 10.1586/17474124.2013.842894. Epub 2013 Oct 17.

  PMID: 24134154 DERIVED

• Jeppesen PB, Pertkiewicz M, Forbes A, Pironi L, Gabe SM, Joly F, Messing B, Loth S, Youssef NN, Heinze H, Berghofer P. Quality of life in patients with short bowel syndrome treated with the new glucagon-like peptide-2 analogue teduglutide--analyses from a randomised, placebo-controlled study. Clin Nutr. 2013 Oct;32(5):713-21. doi: 10.1016/j.clnu.2013.03.016. Epub 2013 Mar 28.

  PMID: 23587733 DERIVED

• Berghofer P, Fragkos KC, Baxter JP, Forbes A, Joly F, Heinze H, Loth S, Pertkiewicz M, Messing B, Jeppesen PB. Development and validation of the disease-specific Short Bowel Syndrome-Quality of Life (SBS-QoL) scale. Clin Nutr. 2013 Oct;32(5):789-96. doi: 10.1016/j.clnu.2012.12.001. Epub 2012 Dec 12.

  PMID: 23274148 DERIVED

• Jeppesen PB, Pertkiewicz M, Messing B, Iyer K, Seidner DL, O'keefe SJ, Forbes A, Heinze H, Joelsson B. Teduglutide reduces need for parenteral support among patients with short bowel syndrome with intestinal failure. Gastroenterology. 2012 Dec;143(6):1473-1481.e3. doi: 10.1053/j.gastro.2012.09.007. Epub 2012 Sep 11.

  PMID: 22982184 DERIVED

Related Links

• HPN support

MeSH Terms

Conditions

Short Bowel Syndrome; Hyperphagia

Interventions

teduglutide; ALX-0600

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Digestive; Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 25, 2008
• First Posted: November 27, 2008
• Study Start: November 25, 2008
• Primary Completion: January 25, 2011
• Study Completion: January 25, 2011
• Last Updated: June 3, 2021
• Results First Posted: February 1, 2012

Record last verified: 2021-05

Locations

NL (1); CA (4); FR (3); ES (2); US (11); PL (4); IT (3); GB (3); DE (3); DK (1)