Trial Outcomes & Findings for Efficacy and Safety of Liraglutide in Subjects With Type 1 Diabetes Undergoing Islet Cell Transplantation (NCT NCT01206101)

NCT ID: NCT01206101

Last Updated: 2017-03-30

Results Overview

Proportion Of Insulin-independent Subjects After Receiving Only One (Single-Donor) Islet Cell Transplant.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

At week 52 after initial transplantation

Results posted on

2017-03-30

Participant Flow

The trial was conducted at 3 sites in 3 countries: Canada: 1 site; Switzerland: 1 site; US: 1 site.

All the subjects were on pre-trial insulin at screening.

Participant milestones

Participant milestones
Measure	Liraglutide Liraglutide was injected subcutaneously once-daily. The dose of liraglutide was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.	Liraglutide Placebo Liraglutide placebo was injected subcutaneously once-daily. The dose of liraglutide placebo was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.
Overall Study STARTED	2	1
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Liraglutide Liraglutide was injected subcutaneously once-daily. The dose of liraglutide was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.	Liraglutide Placebo Liraglutide placebo was injected subcutaneously once-daily. The dose of liraglutide placebo was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.
Overall Study Adverse Event	1	0
Overall Study Physician Decision	0	1
Overall Study Unclassified	1	0

Baseline Characteristics

Efficacy and Safety of Liraglutide in Subjects With Type 1 Diabetes Undergoing Islet Cell Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Liraglutide n=2 Participants Liraglutide was injected subcutaneously once-daily. The dose of liraglutide was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.	Liraglutide Placebo n=1 Participants Liraglutide placebo was injected subcutaneously once-daily. The dose of liraglutide placebo was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=99 Participants	1 Participants n=107 Participants	3 Participants n=206 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Sex: Female, Male Female	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants
Sex: Female, Male Male	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants

PRIMARY outcome

Timeframe: At week 52 after initial transplantation

Population: Full analysis set (FAS) - all randomised subjects who underwent one or more transplantations after randomisation. Due to the premature termination of the trial prior to islet cell transplantation in any randomised subject, no formal statistical analyses were performed.

Proportion Of Insulin-independent Subjects After Receiving Only One (Single-Donor) Islet Cell Transplant.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: During week 0 to week 52

A hypoglycaemic episode was defined as treatment emergent if the onset of the episode was on or after the first day of exposure to randomised treatment and until the last day on randomised treatment. Confirmed hypoglycaemic episodes were categorised either as minor (PG\<3.1 mmol/L \[56 mg/dL\]) or severe (subject unable to treat himself/herself).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From week 0 to week 52 after initial transplantation

Proportion of subjects with HbA1c below or equal to 6.5% at week 52 that were free from severe hypoglycaemic events

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At 52 weeks after initial transplantation

Proportion of insulin-independent subjects among all randomised subjects who had one or more transplantations after randomisation

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From 0 hours pre-culture to 24 hours to 72 hours

Change in islet cell yield from pre-culture to post-culture

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At 12 weeks pre-transplant, at 24 weeks post-transplant, 52 weeks post-transplant and 56 weeks (4 weeks after withdrawal of liraglutide or liraglutide placebo)

Change from baseline in glucose level variability and hypoglycaemia at baseline, weekly during liraglutide dose escalation, at 12 weeks pre-transplant, at 24 weeks post-transplant, 52 weeks post-transplant and 56 weeks (4 weeks after withdrawal of liraglutide or liraglutide placebo)

Outcome measures

Outcome data not reported

Adverse Events

Liraglutide

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Liraglutide Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Liraglutide n=2 participants at risk Liraglutide was injected subcutaneously once-daily. The dose of liraglutide was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.	Liraglutide Placebo n=1 participants at risk Liraglutide placebo was injected subcutaneously once-daily. The dose of liraglutide placebo was escalated up to 1.8 mg (or 1.2 mg if 1.8 mg not tolerated) prior to islet cell transplant and continued until one year after the first transplant in each subject.
Gastrointestinal disorders Gastric ulcer	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Gastrointestinal disorders Nausea	100.0% 2/2 • Number of events 2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Gastrointestinal disorders Vomiting	50.0% 1/2 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	0.00% 0/1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
General disorders Fatigue	50.0% 1/2 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	0.00% 0/1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
General disorders Pyrexia	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Infections and infestations Pharyngitis	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Investigations Computerised tomogram abnormal	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Metabolism and nutrition disorders Decreased appetite	50.0% 1/2 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	0.00% 0/1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Metabolism and nutrition disorders Hypoglycaemia	50.0% 1/2 • Number of events 3 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	0.00% 0/1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Nervous system disorders Headache	50.0% 1/2 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Psychiatric disorders Depression	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/2 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.	100.0% 1/1 • Number of events 1 • Adverse events were collected from week 0 to week 52. Safety analysis set includes all subjects who received at least one dose of the trial product or its comparator.

Additional Information

Public Access to Clinical Trials

Novo Nordisk A/S

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Novo Nordisk maintains the right to be informed of any Investigator plans for publication and to review any scientific paper, presentation, communication or other information concerning the investigation described in this protocol. Any such communication must be submitted in writing to the Novo Nordisk trial manager prior to submission for comments. Comments will be given within four weeks from receipt of the planned communication.
Publication restrictions are in place

Restriction type: OTHER