Safety and Efficacy Study of Teduglutide in Subjects With Short Bowel Syndrome

NCT00081458 | Completed Phase 3 Results DMC

• 2 other identifiers: CL0600-0042004-000438-35
• Study Type: interventional
• Target: 84
• Locations: 9 countries
• Sites: 32

Brief Summary

The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of teduglutide compared with placebo in subjects with parenteral nutrition (PN)-dependent short bowel syndrome (SBS).

Conditions

Short Bowel Syndrome

Keywords

Short Bowel Syndrome; Parenteral Nutrition; SBS

Outcome Measures

Primary Outcomes (1)

• A Graded Response Score in Parenteral Nutrition (PN) Reduction

  The intensity of the response relied on a reduction from Baseline in weekly parenteral nutrition (PN) volume (minimum reduction of 20% and a maximum of 100%). Duration of the response incorporated responses at Weeks(Wk) 16-20 and at Wk20-24. Zero (0 - lowest) assigned if \<20% reduction at Wk20-24 and reduction at Wk16-20 of \< 20%, 20-39%, or \>=40%. One (1) assigned if reduction of 20-39% at Wk20-24 but \< 20% at Wk16-20. Two(2) assigned if reductions of 40-99% at Wk20-24 AND \<20% at Wk16-20 OR 20-39% at Wk20-24 AND 20-39% at Wk16-20. Three (3) assigned if reductions of 100% at Wk20-24 AND \<20% at Wk16-20 OR 40-99% at Wk20-24 AND 20-39% at Wk16-20 OR 20-39% at Wk20-24 AND \>=40% at Wk16-20. Four (4) assigned if reductions of 100% at Wk20-24 AND 20-39% at Wk16-20 OR 40-99% at Wk20-24 AND \>=40% at Wk16-20.

  6 months

Secondary Outcomes (1)

• Number of Subjects Achieving Binary Response at Week 20, Maintained at Week 24

  6 months of treatment

Study Arms (3)

placebo

PLACEBO COMPARATOR

Placebo injectable subcutaneously daily into the thigh or abdomen

Drug: Placebo

2

EXPERIMENTAL

teduglutide 0.05 mg/kg/d

Drug: Teduglutide 0.05 mg/kg/d

3

EXPERIMENTAL

teduglutide 0.1 mg/kg/d

Drug: Teduglutide 0.1 mg/kg/d

Interventions

Placebo DRUG

placebo injectable subcutaneously daily into thigh or abdomen

placebo

Teduglutide 0.05 mg/kg/d DRUG

Teduglutide 0.05 mg/kg/d daily injectable subcutaneously into the thigh or abdomen

Also known as: GATTEX

2

Teduglutide 0.1 mg/kg/d DRUG

Teduglutide 0.1 /g/kg/d daily injection subcutaneously into thigh or abdomen

Also known as: GATTEX

3

Eligibility Criteria

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

You may qualify if:

• Men and women, aged 18 years of age or older at the time of signing the informed consent form (ICF)
• SBS as a result of major intestinal resection resulting in at least 12 months intravenous feeding
• Body weight must be less than 90 kg
• At baseline, subjects must require PN treatment to meet their caloric or electrolyte needs due to ongoing malabsorption at least 3 times weekly and to be on a stable PN regimen for 4 weeks before dosing
• Body mass index (BMI) 18 to 27 kg/m2
• Adequate hepatic and renal function

You may not qualify if:

• History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state
• History of alcohol or drug abuse (within previous year)
• Participation in a clinical study within 30 days prior to signing the ICF, or concurrent participation in any clinical study
• Clinically significant laboratory abnormalities at the time of randomization
• Previous use of teduglutide (ALX-0600)
• Prior use of native GLP-2 within 3 months of screening visit
• Hospital admission within 1 month prior to screening visit
• Pregnant or lactating women
• Any condition or circumstance, which in the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with analysis of the study results.
• Presence of excluded disease: Radiation enteritis, Scleroderma, Celiac disease, Refractory/Tropical sprue, Pseudo-obstruction, Active inflammatory bowel disease (IBD), Pre-malignant/malignant change in colonoscopy biopsy or polypectomy, Surgery scheduled within the time frame of the study, Human immunodeficiency virus (HIV) positive test, Immunological disorders, Possible allergies to teduglutide or its constituents, Significant, active, uncontrolled, untreated systemic diseases

Sponsors & Collaborators

• Shire: lead

Study Sites (32)

Mayo Clinic Scottsdale

Scottsdale, Arizona, 85259, United States

Location

Georgetown University

Washington D.C., District of Columbia, 20007, United States

Location

Emory University Hospital

Atlanta, Georgia, 30322, United States

Location

Northwestern Center for Clinical Research

Chicago, Illinois, 60611, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Albany Medical Center

Albany, New York, 12208, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

University of Pennsylvania - Penn Nursing

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

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

l'Hôpital Erasme

Brussels, 1070, Belgium

Location

Royal Alexandra Hospital

Edmonton, Alberta, T5H4B9, Canada

Location

St. Paul's Hospital

Vancouver, British Columbia, V6Z1Y6, Canada

Location

St. Michael's Hospital

Toronto, Ontario, M5B1W8, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G2N2, Canada

Location

Rigshospitalet, University of Copenhagen

Copenhagen, 2100, Denmark

Location

Hôpital Claude-Huriez

Lille, 59037, France

Location

Hôpital de la Croix-Rousse

Lyon, 69317, France

Location

Hôpital Edouard Herriot

Lyon, 69437, France

Location

Hôpital Beaujon

Paris, 92110, France

Location

Charité University Hospital

Berlin, 10117, Germany

Location

Charité-Universitätsmedizin Berlin

Berlin, 13353, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt, 60590, Germany

Location

Academic Medical Center

Amsterdam, 1100 DD, Netherlands

Location

Pracownia Żywienia Klinicznego

Olsztyn, 10-651, Poland

Location

Samodzielny Publiczny Szpital Kliniczny-Im.prof W.Orłowskiego CMKP

Warsaw, 00-416, Poland

Location

Hope Hospital

Salford, Greater Manchester, M68HD, United Kingdom

Location

St. Mark's Hospital

Harrow, HA13UJ, United Kingdom

Location

Related Publications (6)

• Dudrick SJ, Latifi R, Fosnocht DE. Management of the short-bowel syndrome. Surg Clin North Am. 1991 Jun;71(3):625-43. doi: 10.1016/s0039-6109(16)45438-1.

  PMID: 1904648 BACKGROUND

• Rombeau JL, Rolandelli RH. Enteral and parenteral nutrition in patients with enteric fistulas and short bowel syndrome. Surg Clin North Am. 1987 Jun;67(3):551-71. doi: 10.1016/s0039-6109(16)44232-5.

  PMID: 3109044 BACKGROUND

• Shanbhogue LK, Molenaar JC. Short bowel syndrome: metabolic and surgical management. Br J Surg. 1994 Apr;81(4):486-99. doi: 10.1002/bjs.1800810404.

  PMID: 8205419 BACKGROUND

• Vanderhoof JA, Langnas AN. Short-bowel syndrome in children and adults. Gastroenterology. 1997 Nov;113(5):1767-78. doi: 10.1053/gast.1997.v113.pm9352883.

  PMID: 9352883 BACKGROUND

• Jeppesen PB, Gilroy R, Pertkiewicz M, Allard JP, Messing B, O'Keefe SJ. Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition and/or intravenous fluid requirements in patients with short bowel syndrome. Gut. 2011 Jul;60(7):902-14. doi: 10.1136/gut.2010.218271. Epub 2011 Feb 11.

  PMID: 21317170 RESULT

• 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

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: LTE60
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: April 13, 2004
• First Posted: April 14, 2004
• Study Start: May 25, 2004
• Primary Completion: July 6, 2007
• Study Completion: July 6, 2007
• Last Updated: June 9, 2021
• Results First Posted: July 15, 2013

Record last verified: 2021-05

Locations

BE (1); DE (3); GB (2); FR (4); NL (1); CA (4); US (14); DK (1); PL (2)