Trial Outcomes & Findings for Open-Label Study of Teduglutide for Subjects With PN-Dependent Short Bowel Syndrome (SBS) (NCT NCT00930644)

NCT ID: NCT00930644

Last Updated: 2021-06-11

Results Overview

The mean change from baseline in weekly PN.IV volume in percent change is shown by visit.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

88 participants

Primary outcome timeframe

24 months

Results posted on

2021-06-11

Participant Flow

Subjects who met any of the following could be enrolled: Completed 24 weeks of treatment in Study CL0600-020; based on PI and sponsor decision, subjects who were required to stop treatment prematurely due to a non drug related AE; or successfully completed Stage I (optimization/stabilization) in Study CL0600-020 after \~86 subjects were randomized

Participant milestones

Participant milestones
Measure	Teduglutide 0.05 mg/kg/Day teduglutide: 0.05 mg/kg/day subcutaneously taken once per day for 24 months
Overall Study STARTED	88
Overall Study COMPLETED	65
Overall Study NOT COMPLETED	23

Reasons for withdrawal

Reasons for withdrawal
Measure	Teduglutide 0.05 mg/kg/Day teduglutide: 0.05 mg/kg/day subcutaneously taken once per day for 24 months
Overall Study Withdrawal by Subject	4
Overall Study Physician Decision	2
Overall Study Death	1
Overall Study Adverse Event	16

Baseline Characteristics

Open-Label Study of Teduglutide for Subjects With PN-Dependent Short Bowel Syndrome (SBS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Teduglutide 0.05 mg/kg/Day n=88 Participants teduglutide: 0.05 mg/kg/day subcutaneously taken once per day for 24 months
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	73 Participants n=99 Participants
Age, Categorical >=65 years	15 Participants n=99 Participants
Sex: Female, Male Female	47 Participants n=99 Participants
Sex: Female, Male Male	41 Participants n=99 Participants
Years since start of PN/IV dependecy	6.41 years STANDARD_DEVIATION 6.272 • n=99 Participants
Prescribed weekly PN/IV volume	12.07 Liters STANDARD_DEVIATION 7.56 • n=99 Participants
Prescribed weekly number of days on PN/IV	5.6 days STANDARD_DEVIATION 1.68 • n=99 Participants

PRIMARY outcome

Timeframe: 24 months

The mean change from baseline in weekly PN.IV volume in percent change is shown by visit.

Outcome measures

Outcome measures
Measure	NT/TED n=12 Participants No treatment in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	PBO/TED n=39 Participants Placebo in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	TED/TED n=37 Participants 0.05 mg/kg/day subcutaneously taken once per day for 6 months CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial
Percent Change in PN/IV Volume by Visit Month 1	-5.35 percent change Standard Deviation 11.287	-6.44 percent change Standard Deviation 11.241	-40.65 percent change Standard Deviation 21.546
Percent Change in PN/IV Volume by Visit Month 2	-12.88 percent change Standard Deviation 22.789	-10.27 percent change Standard Deviation 14.027	-44.24 percent change Standard Deviation 21.823
Percent Change in PN/IV Volume by Visit Month 3	-10.83 percent change Standard Deviation 47.396	-14.18 percent change Standard Deviation 16.440	-45.80 percent change Standard Deviation 22.681
Percent Change in PN/IV Volume by Visit Month 6	-24.13 percent change Standard Deviation 17.496	-16.48 percent change Standard Deviation 20.979	-34.70 percent change Standard Deviation 55.319
Percent Change in PN/IV Volume by Visit Month 9	-25.35 percent change Standard Deviation 17.855	-20.95 percent change Standard Deviation 27.500	-37.91 percent change Standard Deviation 57.460
Percent Change in PN/IV Volume by Visit Month 12	-23.33 percent change Standard Deviation 20.296	-22.00 percent change Standard Deviation 33.858	-51.33 percent change Standard Deviation 28.299
Percent Change in PN/IV Volume by Visit Month 15	-31.62 percent change Standard Deviation 25.901	-18.89 percent change Standard Deviation 33.165	-54.24 percent change Standard Deviation 27.871
Percent Change in PN/IV Volume by Visit Month 18	-35.11 percent change Standard Deviation 35.545	-24.18 percent change Standard Deviation 32.183	-63.01 percent change Standard Deviation 26.587
Percent Change in PN/IV Volume by Visit Month 21	-39.40 percent change Standard Deviation 36.504	-27.67 percent change Standard Deviation 33.064	-64.99 percent change Standard Deviation 27.920
Percent Change in PN/IV Volume by Visit Month 24	-39.40 percent change Standard Deviation 36.504	-28.33 percent change Standard Deviation 35.169	-65.61 percent change Standard Deviation 33.606

PRIMARY outcome

Timeframe: 24 months

The mean change from baseline in weekly PN.IV volume in Liters is shown by visit.

Outcome measures

Outcome measures
Measure	NT/TED n=12 Participants No treatment in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	PBO/TED n=39 Participants Placebo in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	TED/TED n=37 Participants 0.05 mg/kg/day subcutaneously taken once per day for 6 months CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial
Absolute Change in PN/IV Volume by Visit Month 1	-1.05 Liters Standard Deviation 1.315	-0.95 Liters Standard Deviation 1.938	-5.28 Liters Standard Deviation 3.819
Absolute Change in PN/IV Volume by Visit Month 2	-1.88 Liters Standard Deviation 2.057	-1.38 Liters Standard Deviation 2.228	-5.62 Liters Standard Deviation 3.873
Absolute Change in PN/IV Volume by Visit Month 3	-2.55 Liters Standard Deviation 3.123	-1.65 Liters Standard Deviation 2.456	-5.72 Liters Standard Deviation 3.771
Absolute Change in PN/IV Volume by Visit Month 6	-3.85 Liters Standard Deviation 2.761	-1.90 Liters Standard Deviation 3.309	-5.20 Liters Standard Deviation 4.650
Absolute Change in PN/IV Volume by Visit Month 9	-3.69 Liters Standard Deviation 2.916	-2.61 Liters Standard Deviation 4.071	-5.59 Liters Standard Deviation 4.899
Absolute Change in PN/IV Volume by Visit Month 12	-2.90 Liters Standard Deviation 2.762	-2.72 Liters Standard Deviation 4.310	-6.36 Liters Standard Deviation 4.633
Absolute Change in PN/IV Volume by Visit Month 15	-3.58 Liters Standard Deviation 2.811	-2.48 Liters Standard Deviation 4.398	-6.37 Liters Standard Deviation 4.261
Absolute Change in PN/IV Volume by Visit Month 18	-3.63 Liters Standard Deviation 2.834	-2.90 Liters Standard Deviation 4.394	-6.99 Liters Standard Deviation 4.048
Absolute Change in PN/IV Volume by Visit Month 21	-4.01 Liters Standard Deviation 2.910	-3.00 Liters Standard Deviation 3.571	-7.34 Liters Standard Deviation 4.412
Absolute Change in PN/IV Volume by Visit Month 24	-4.01 Liters Standard Deviation 2.910	-3.11 Liters Standard Deviation 3.880	-7.55 Liters Standard Deviation 4.930

SECONDARY outcome

Timeframe: 24 Months or Last Dosing Visit

The number of subjects who achieve at least 1-, 2-, and 3-day reductions in PN/IV per Week.

Outcome measures

Outcome measures
Measure	NT/TED n=12 Participants No treatment in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	PBO/TED n=39 Participants Placebo in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	TED/TED n=37 Participants 0.05 mg/kg/day subcutaneously taken once per day for 6 months CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial
Number of Subjects Achieving PN/IV Reduction Achieving >= 1 Day of PN/IV Reduction	3 participants	14 participants	21 participants
Number of Subjects Achieving PN/IV Reduction Achieving >= 2 Day of PN/IV Reduction	2 participants	7 participants	18 participants
Number of Subjects Achieving PN/IV Reduction Achieving >= 3 Day of PN/IV Reduction	2 participants	5 participants	18 participants

Adverse Events

NT,PBO/TED

Serious events: 32 serious events

Other events: 49 other events

Deaths: 0 deaths

TED/TED

Serious events: 24 serious events

Other events: 35 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	NT,PBO/TED n=51 participants at risk No treatment or placebo in CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial	TED/TED n=37 participants at risk 0.05 mg/kg/day subcutaneously taken once per day for 6 months CL0600-020 trial and teduglutide 0.05 mg/kg/day subcutaneously taken once per day for 24 months in CL0600-021 trial
Blood and lymphatic system disorders Blood and Lymphatic System Disorders	9.8% 5/51 • Number of events 10 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	8.1% 3/37 • Number of events 5 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Gastrointestinal disorders Gastrointestinal Disorders	66.7% 34/51 • Number of events 148 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	54.1% 20/37 • Number of events 68 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
General disorders General Disorders, and Administration Site Conditions	52.9% 27/51 • Number of events 100 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	40.5% 15/37 • Number of events 40 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Hepatobiliary disorders Hepatobiliary Disorders	15.7% 8/51 • Number of events 10 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	5.4% 2/37 • Number of events 2 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Infections and infestations Infections and Infestations	60.8% 31/51 • Number of events 97 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	70.3% 26/37 • Number of events 62 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Injury, poisoning and procedural complications Injury, Poisoning, and Procedural Complications	37.3% 19/51 • Number of events 39 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	35.1% 13/37 • Number of events 39 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Investigations Investigations	43.1% 22/51 • Number of events 62 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	51.4% 19/37 • Number of events 31 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Metabolism and nutrition disorders Metabolism and Nutrition Disorder	27.5% 14/51 • Number of events 44 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	37.8% 14/37 • Number of events 33 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Musculoskeletal and connective tissue disorders Musculoskeletal and Connective Tissue Disorders	21.6% 11/51 • Number of events 37 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	35.1% 13/37 • Number of events 23 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Nervous system disorders Nervous System Disorders	19.6% 10/51 • Number of events 60 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	27.0% 10/37 • Number of events 18 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Psychiatric disorders Psychiatric Disorders	15.7% 8/51 • Number of events 11 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	5.4% 2/37 • Number of events 2 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Renal and urinary disorders Renal and Urinary Disorders	11.8% 6/51 • Number of events 17 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	10.8% 4/37 • Number of events 17 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Respiratory, thoracic and mediastinal disorders Respiratory, Thoracic, and Mediastinal Disorders	19.6% 10/51 • Number of events 20 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	18.9% 7/37 • Number of events 9 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.
Vascular disorders Vascular Disorder	27.5% 14/51 • Number of events 18 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.	16.2% 6/37 • Number of events 16 • Adverse event data were collected for each subject from the time informed consent was signed to the end of the study. The most commonly reported treatment emergent adverse events (>= 3% grouped at PT level) are listed. Adverse events monitoring was performed through investigator assessment and safety laboratory testing at each visit.

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of NPS Pharmaceuticals agreements with its investigators may vary. However, NPS does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e. data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER