Short Bowel Syndrome
Conditions
Keywords
short bowel syndrome, parenteral nutrition, PN, HPN, TPN, SBS
Brief summary
This study is a 2-year open label extension study to collect long term efficacy and safety data from patients who have completed the 24-weeks of study drug dosing in CL0600-020.
Detailed description
Some people with SBS need to infuse parenteral nutrition (PN) in order to live. Although PN can save lives, it can lead to some serious side effects such as infection or liver damage. The risk for those effects increases the longer people are on PN. Teduglutide is an investigative medicine being evaluated as a possible treatment for people with PN-dependent 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. We want to see if teduglutide can help decrease the amount of PN needed because the body is doing a better job of taking up what it eats and drinks. We also want to find out how safe teduglutide is and record any side effects.
Interventions
DRUGteduglutide
0.05 mg/kg/day subcutaneously taken once per day for 24 months
Sponsors
Nycomed Germany GmbH
Shire
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* must have completed 24 weeks of dosing of the CL0600-020 study
Exclusion criteria
* none
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in PN/IV Volume by Visit | 24 months | The mean change from baseline in weekly PN.IV volume in percent change is shown by visit. |
| Absolute Change in PN/IV Volume by Visit | 24 months | The mean change from baseline in weekly PN.IV volume in Liters is shown by visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects Achieving PN/IV Reduction | 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. |
Countries
Canada, Denmark, France, Germany, Italy, Poland, Spain, United Kingdom, United States
Participant flow
Recruitment details
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
Participants by arm
| Arm | Count |
|---|---|
| Teduglutide 0.05 mg/kg/Day teduglutide: 0.05 mg/kg/day subcutaneously taken once per day for 24 months | 88 |
| Total | 88 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 16 |
| Overall Study | Death | 1 |
| Overall Study | Physician Decision | 2 |
| Overall Study | Withdrawal by Subject | 4 |
Baseline characteristics
| Characteristic | Teduglutide 0.05 mg/kg/Day |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 15 Participants |
| Age, Categorical Between 18 and 65 years | 73 Participants |
| Prescribed weekly number of days on PN/IV | 5.6 days STANDARD_DEVIATION 1.68 |
| Prescribed weekly PN/IV volume | 12.07 Liters STANDARD_DEVIATION 7.56 |
| Sex: Female, Male Female | 47 Participants |
| Sex: Female, Male Male | 41 Participants |
| Years since start of PN/IV dependecy | 6.41 years STANDARD_DEVIATION 6.272 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 49 / 51 | 35 / 37 |
| serious Total, serious adverse events | 32 / 51 | 24 / 37 |
Outcome results
Primary
Absolute Change in PN/IV Volume by Visit
The mean change from baseline in weekly PN.IV volume in Liters is shown by visit.
Time frame: 24 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 1 | -1.05 Liters | Standard Deviation 1.315 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 2 | -1.88 Liters | Standard Deviation 2.057 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 3 | -2.55 Liters | Standard Deviation 3.123 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 6 | -3.85 Liters | Standard Deviation 2.761 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 9 | -3.69 Liters | Standard Deviation 2.916 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 12 | -2.90 Liters | Standard Deviation 2.762 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 15 | -3.58 Liters | Standard Deviation 2.811 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 18 | -3.63 Liters | Standard Deviation 2.834 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 21 | -4.01 Liters | Standard Deviation 2.91 |
| NT/TED | Absolute Change in PN/IV Volume by Visit | Month 24 | -4.01 Liters | Standard Deviation 2.91 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 21 | -3.00 Liters | Standard Deviation 3.571 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 1 | -0.95 Liters | Standard Deviation 1.938 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 12 | -2.72 Liters | Standard Deviation 4.31 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 9 | -2.61 Liters | Standard Deviation 4.071 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 2 | -1.38 Liters | Standard Deviation 2.228 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 24 | -3.11 Liters | Standard Deviation 3.88 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 18 | -2.90 Liters | Standard Deviation 4.394 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 3 | -1.65 Liters | Standard Deviation 2.456 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 15 | -2.48 Liters | Standard Deviation 4.398 |
| PBO/TED | Absolute Change in PN/IV Volume by Visit | Month 6 | -1.90 Liters | Standard Deviation 3.309 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 18 | -6.99 Liters | Standard Deviation 4.048 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 6 | -5.20 Liters | Standard Deviation 4.65 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 9 | -5.59 Liters | Standard Deviation 4.899 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 12 | -6.36 Liters | Standard Deviation 4.633 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 21 | -7.34 Liters | Standard Deviation 4.412 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 15 | -6.37 Liters | Standard Deviation 4.261 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 1 | -5.28 Liters | Standard Deviation 3.819 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 24 | -7.55 Liters | Standard Deviation 4.93 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 2 | -5.62 Liters | Standard Deviation 3.873 |
| TED/TED | Absolute Change in PN/IV Volume by Visit | Month 3 | -5.72 Liters | Standard Deviation 3.771 |
Primary
Percent Change in PN/IV Volume by Visit
The mean change from baseline in weekly PN.IV volume in percent change is shown by visit.
Time frame: 24 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 1 | -5.35 percent change | Standard Deviation 11.287 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 2 | -12.88 percent change | Standard Deviation 22.789 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 3 | -10.83 percent change | Standard Deviation 47.396 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 6 | -24.13 percent change | Standard Deviation 17.496 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 9 | -25.35 percent change | Standard Deviation 17.855 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 12 | -23.33 percent change | Standard Deviation 20.296 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 15 | -31.62 percent change | Standard Deviation 25.901 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 18 | -35.11 percent change | Standard Deviation 35.545 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 21 | -39.40 percent change | Standard Deviation 36.504 |
| NT/TED | Percent Change in PN/IV Volume by Visit | Month 24 | -39.40 percent change | Standard Deviation 36.504 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 21 | -27.67 percent change | Standard Deviation 33.064 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 1 | -6.44 percent change | Standard Deviation 11.241 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 12 | -22.00 percent change | Standard Deviation 33.858 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 9 | -20.95 percent change | Standard Deviation 27.5 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 2 | -10.27 percent change | Standard Deviation 14.027 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 24 | -28.33 percent change | Standard Deviation 35.169 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 18 | -24.18 percent change | Standard Deviation 32.183 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 3 | -14.18 percent change | Standard Deviation 16.44 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 15 | -18.89 percent change | Standard Deviation 33.165 |
| PBO/TED | Percent Change in PN/IV Volume by Visit | Month 6 | -16.48 percent change | Standard Deviation 20.979 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 18 | -63.01 percent change | Standard Deviation 26.587 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 6 | -34.70 percent change | Standard Deviation 55.319 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 9 | -37.91 percent change | Standard Deviation 57.46 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 12 | -51.33 percent change | Standard Deviation 28.299 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 21 | -64.99 percent change | Standard Deviation 27.92 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 15 | -54.24 percent change | Standard Deviation 27.871 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 1 | -40.65 percent change | Standard Deviation 21.546 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 24 | -65.61 percent change | Standard Deviation 33.606 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 2 | -44.24 percent change | Standard Deviation 21.823 |
| TED/TED | Percent Change in PN/IV Volume by Visit | Month 3 | -45.80 percent change | Standard Deviation 22.681 |
Secondary
Number of Subjects Achieving PN/IV Reduction
The number of subjects who achieve at least 1-, 2-, and 3-day reductions in PN/IV per Week.
Time frame: 24 Months or Last Dosing Visit
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| NT/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 2 Day of PN/IV Reduction | 2 participants |
| NT/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 1 Day of PN/IV Reduction | 3 participants |
| NT/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 3 Day of PN/IV Reduction | 2 participants |
| PBO/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 2 Day of PN/IV Reduction | 7 participants |
| PBO/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 1 Day of PN/IV Reduction | 14 participants |
| PBO/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 3 Day of PN/IV Reduction | 5 participants |
| TED/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 1 Day of PN/IV Reduction | 21 participants |
| TED/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 3 Day of PN/IV Reduction | 18 participants |
| TED/TED | Number of Subjects Achieving PN/IV Reduction | Achieving >= 2 Day of PN/IV Reduction | 18 participants |