STEPS3: A One-Year, Open-Label Study With Teduglutide for Subjects Who Completed Study CL0600-021

• Sponsor: Shire (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01560403
• Collaborator: (none)
• Enrollment: 14
• Locations: 5
• Arm: 1
• Actual Duration (Months): 14.1
• Patients Per Site: 2.8
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

This study is a 1-year open label extension study to collect long term efficacy and safety data from patients who have completed approximately 2 years of dosing in Study CL0600-021.

Condition or Disease	Intervention/Treatment	Phase
Short Bowel Syndrome	Drug: Teduglutide	Phase 3

Detailed Description

This study served as a 1-year extension study for those US patients who have completed the CL0600-021 study.

Study Design

• Study Type: Interventional
• Actual Enrollment: 14 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A One-Year, Open-Label Study With Teduglutide for Subjects With Parenteral Nutrition-dependent Short Bowel Syndrome Who Completed Study CL0600-021
• Actual Study Start Date: May 21, 2012
• Actual Primary Completion Date: Jul 23, 2013
• Actual Study Completion Date: Jul 23, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Teduglutide 0.05 mg/kg/day	Drug: Teduglutide; 0.05 mg/kg/day subcutaneously taken once per day for 12 months; Other Names: Gattex

Outcome Measures

Primary Outcome Measures

1. Summary of Treatment-emergent Adverse Events [12 months]

   As the primary intent of this study was to collect additional safety data, this outcomes measure will provide a summary of the treatment emergent adverse events. Based on the start date of each subject in this study and the study end date, not all subjects reached 12 months.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• At completion of Study CL0600-021, subjects will be invited to participate in this trial if they meet the following criteria:

1. Completion of the 24-month study, CL0600-021, regardless if fully weaned from PN/I.V. support

2. Signed and dated informed consent form (ICF) to participate before any study-related procedures of Study TED-C11-001 are performed

Exclusion Criteria:

• None

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Scripps Clinic & Research Foundation	La Jolla	California	United States	92037
2	Emory University School of Medicine	Atlanta	Georgia	United States	30322
3	Mount Sinai Medical Center	New York	New York	United States	10029
4	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
5	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232

Sponsors and Collaborators

• Shire

Investigators

• Study Director: Study Director, Takeda

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Shire
• ClinicalTrials.gov Identifier: NCT01560403
• Other Study ID Numbers: TED-C11-001
• First Posted: Mar 22, 2012
• Last Update Posted: Jun 10, 2021
• Last Verified: May 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Keywords provided by Shire: Parenteral nutrition; TPN; HPN; PN; SBS; Short bowel syndrome
• Additional relevant MeSH terms: Short Bowel Syndrome; Syndrome; Teduglutide

Study Results

Participant Flow

Recruitment Details	This was a 1-year, open-label, multi-center study in subjects who completed Study CL0600-021 (NCT00930644) at any US site.

Arm/Group Title	TED/TED	NT/PBO,TED
Arm/Group Description	This group represents those subjects exposed to active treatment with teduglutide for 24 weeks in Study CL0600-020 (NCT00798967) and an additional 24 months in Study CL0600-021 (NCT00930644)	This group represents those subjects who either participated in Study CL0600-020 and received placebo, or who were eligible for randomization in Study CL0600-020 (NCT00798967), but qualified after the enrollment number was already satisfied and therefore entered the open-label extension Study CL0600-021 (NCT00930644) directly
Period Title: Overall Study
STARTED	5	9
COMPLETED	5	8
NOT COMPLETED	0	1

Baseline Characteristics

Arm/Group Title	TED/TED	NT/PBO,TED	Total
Arm/Group Description	This group represents those subjects exposed to active treatment with teduglutide for 24 weeks in Study CL0600-020 (NCT00798967) and an additional 24 months in Study CL0600 021 (NCT00930644)	This group represents those subjects who either participated in Study CL0600-020 and received placebo, or who were eligible for randomization in Study CL0600-020 (NCT00798967), but qualified after the enrollment number was already satisfied and therefore entered the open-label extension Study CL0600-021 (NCT00930644) directly	Total of all reporting groups
Overall Participants	5	9	14
Age, Customized (Count of Participants)
< 45 years	1 20%	2 22.2%	3 21.4%
45 - < 65 years	3 60%	6 66.7%	9 64.3%
>=65 years	1 20%	1 11.1%	2 14.3%
Sex: Female, Male (Count of Participants)
Female	4 80%	6 66.7%	10 71.4%
Male	1 20%	3 33.3%	4 28.6%

Outcome Measures

1. Primary Outcome

Title	Summary of Treatment-emergent Adverse Events
Description	As the primary intent of this study was to collect additional safety data, this outcomes measure will provide a summary of the treatment emergent adverse events. Based on the start date of each subject in this study and the study end date, not all subjects reached 12 months.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

Safety Population

Arm/Group Title	NT,PBO/TED	TED/TED
Arm/Group Description	This group represents those subjects who either participated in Study CL0600-020 (NCT00798967) and received placebo, or who were eligible for randomization in Study CL0600-020 (NCT00798967), but qualified after the enrollment number was already satisfied and therefore entered the open-label extension study CL0600-021 (NCT00930644) directly.	This group represents those subjects exposed to active treatment with teduglutide for 24 weeks in Study CL0600-020 (NCT00798967) and an additional 24 months in Study CL0600 021 (NCT00930644).
Measure Participants	9	5
Number [participants]	4 80%	5 55.6%

Adverse Events

Time Frame	1 year, 2 months
Adverse Event Reporting Description	Date of Study Initiation (first subject, first visit) 21 May 2012 Date of Study Completion (last subject, last visit) 23 July 2013. Due to the different start dates of each subject in this study, not all subjects completed the 12 month visits by the study completion date as the study was terminated on this date by the sponsor.
Arm/Group Title	NT/PBO,TED		TED/TED
Arm/Group Description	This group represents those subjects who either participated in Study CL0600-020 and received placebo, or who were eligible for randomization in Study CL0600-020 (NCT00798967), but qualified after the enrollment number was already satisfied and therefore entered the open-label extension Study CL0600-021 (NCT00930644) directly		This group represents those subjects exposed to active treatment with teduglutide for 24 weeks in Study CL0600-020 (NCT00798967) and an additional 24 months in Study CL0600-021 (NCT00930644)
All Cause Mortality
	NT/PBO,TED		TED/TED
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	NT/PBO,TED		TED/TED
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/9 (11.1%)		5/5 (100%)
Cardiac disorders
Atrial fibrillation	0/9 (0%)	0	1/5 (20%)	1
Atrial tachycardia	0/9 (0%)	0	1/5 (20%)	1
Cardiac failure acute	0/9 (0%)	0	1/5 (20%)	1
Cardiac failure congestive	0/9 (0%)	0	1/5 (20%)	1
Cardiomyopathy	0/9 (0%)	0	1/5 (20%)	1
Ischaemic cardiomyopathy	0/9 (0%)	0	1/5 (20%)	1
Infections and infestations
Central line infection	0/9 (0%)	0	1/5 (20%)	1
Pneumonia	0/9 (0%)	0	1/5 (20%)	2
Urinary tract infection	0/9 (0%)	0	1/5 (20%)	1
Injury, poisoning and procedural complications
In-stent arterial restenosis	0/9 (0%)	0	1/5 (20%)	1
Upper limb fracture	0/9 (0%)	0	1/5 (20%)	1
Metabolism and nutrition disorders
Lactic acidosis	1/9 (11.1%)	1	0/5 (0%)	0
Respiratory, thoracic and mediastinal disorders
Dyspnoea	0/9 (0%)	0	1/5 (20%)	1
Other (Not Including Serious) Adverse Events
	NT/PBO,TED		TED/TED
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	4/9 (44.4%)		4/5 (80%)
Gastrointestinal disorders
Diarrhoea	1/9 (11.1%)	1	2/5 (40%)	3
Abdominal pain	2/9 (22.2%)	2	0/5 (0%)	0
General disorders
Asthenic conditions	2/9 (22.2%)	2	1/5 (20%)	1
Immune system disorders
Hypersensitivity	2/9 (22.2%)	2	0/5 (0%)	0
Seasonal allergy	1/9 (11.1%)	1	1/5 (20%)	1
Infections and infestations
Catheter sepsis	0/9 (0%)	0	2/5 (40%)	2
Lower respiratory tract infection	1/9 (11.1%)	1	1/5 (20%)	2
Upper respiratory tract infection	1/9 (11.1%)	1	1/5 (20%)	1
Viral infection	2/9 (22.2%)	2	0/5 (0%)	0
Injury, poisoning and procedural complications
Catheter site related reaction	1/9 (11.1%)	2	1/5 (20%)	1
Investigations
Hepatic enzyme increased	1/9 (11.1%)	3	1/5 (20%)	1
Weight decreased	0/9 (0%)	0	2/5 (40%)	3
Metabolism and nutrition disorders
Fluid overload	1/9 (11.1%)	2	1/5 (20%)	2
Musculoskeletal and connective tissue disorders
Musculoskeletal pain	1/9 (11.1%)	1	1/5 (20%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign neoplasms gastrointestinal	2/9 (22.2%)	2	0/5 (0%)	0
Psychiatric disorders
Anxiety symptoms	1/9 (11.1%)	1	1/5 (20%)	1
Cognition and attention disorders and disturbances	2/9 (22.2%)	3	0/5 (0%)	0
Respiratory, thoracic and mediastinal disorders
Dyspnoea	0/9 (0%)	0	2/5 (40%)	2
Vascular disorders
Hypertension	1/9 (11.1%)	1	1/5 (20%)	1

Limitations/Caveats

This study provided further long-term safety and efficacy data on the 14 US subjects who enrolled in the CL0600-020 and the CL0600-021 study. Given the small number of subjects in this study, only descriptive statistics were employed and presented.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.