Trial Outcomes & Findings for Safety and Efficacy Study of ELND005 as an Adjunctive Maintenance Treatment in Bipolar I Disorder (NCT NCT01674010)

NCT ID: NCT01674010

Last Updated: 2019-10-21

Results Overview

The study was terminated early so no efficacy analysis was done, safety data are reported.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 309 participants
• Primary outcome timeframe: up to 48 weeks
• Results posted on: 2019-10-21

Participant Flow

Participant milestones

Measure	Open Treatment Phase 1 ELND005 500 mg BID ELND005 500mg BID for 16 weeks	Phase 2 Placebo Double Blind Randomization (Phase 2)	Phase 2 ELND005 500 mg BID Randomization Phase 2 ELND005 500 mg BID
Open Treatment Phase 1 STARTED	309	0	0
Open Treatment Phase 1 COMPLETED	129	0	0
Open Treatment Phase 1 NOT COMPLETED	180	0	0
Randomization Phase 2 STARTED	0	65	64
Randomization Phase 2 COMPLETED	0	3	3
Randomization Phase 2 NOT COMPLETED	0	62	61

Reasons for withdrawal

Measure	Open Treatment Phase 1 ELND005 500 mg BID ELND005 500mg BID for 16 weeks	Phase 2 Placebo Double Blind Randomization (Phase 2)	Phase 2 ELND005 500 mg BID Randomization Phase 2 ELND005 500 mg BID
Open Treatment Phase 1 Adverse Event	10	0	0
Open Treatment Phase 1 Death	1	0	0
Open Treatment Phase 1 Lost to Follow-up	19	0	0
Open Treatment Phase 1 Physician Decision	9	0	0
Open Treatment Phase 1 Protocol Violation	7	0	0
Open Treatment Phase 1 Sponsor Decision	79	0	0
Open Treatment Phase 1 Did not remain in a stable remission	30	0	0
Open Treatment Phase 1 Withdrawal by Subject	25	0	0
Randomization Phase 2 Adverse Event	0	1	1
Randomization Phase 2 Lost to Follow-up	0	1	2
Randomization Phase 2 Mood episode recurrence	0	8	2
Randomization Phase 2 Physician Decision	0	0	2
Randomization Phase 2 Pregnancy	0	1	1
Randomization Phase 2 Protocol Violation	0	1	1
Randomization Phase 2 Sponsor Decision	0	42	45
Randomization Phase 2 Withdrawal by Subject	0	8	7

Baseline Characteristics

Safety and Efficacy Study of ELND005 as an Adjunctive Maintenance Treatment in Bipolar I Disorder

Baseline characteristics by cohort

Measure	All Study Participants n=309 Participants
Age, Continuous Phase 1(n=309)	44.3 years STANDARD_DEVIATION 11.09 • n=5 Participants
Age, Continuous Phase 2 Placebo (n=65)	44.5 years STANDARD_DEVIATION 10.65 • n=5 Participants
Age, Continuous Phase 2 ELND005 500mg BID(n=64)	44.6 years STANDARD_DEVIATION 10.61 • n=5 Participants
Sex/Gender, Customized Phase 1 Male	146 participants n=5 Participants
Sex/Gender, Customized Phase 1 Female	163 participants n=5 Participants
Sex/Gender, Customized Phase 2 Placebo Male	34 participants n=5 Participants
Sex/Gender, Customized Phase 2 Placebo Female	31 participants n=5 Participants
Sex/Gender, Customized Phase 2 ELND005 500 BID Male	32 participants n=5 Participants
Sex/Gender, Customized Phase 2 ELND005 500 BID Female	32 participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino Phase 1	23 participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino Phase 2 Placebo	4 participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino Phase 2 ELND005 500 BID	6 participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino Phase 1	284 participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino Phase 2 Placebo	60 participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino Phase 2 ELND005 500 BID	58 participants n=5 Participants
Race/Ethnicity, Customized Unknown Phase 1	2 participants n=5 Participants
Race/Ethnicity, Customized Unknown Phase 2 Placebo	1 participants n=5 Participants
Race/Ethnicity, Customized Unknown Phase 2 ELND005 500 BID	0 participants n=5 Participants
Region of Enrollment North America	210 participants n=5 Participants
Region of Enrollment Europe	99 participants n=5 Participants

PRIMARY outcome

Timeframe: up to 48 weeks

Population: TEAEs reported for Phase 2 were from the entire study period

The study was terminated early so no efficacy analysis was done, safety data are reported.

Outcome measures

Measure	Open Treatment Phase 1 ELND005 500 mg BID n=309 Participants ELND005 500mg BID for 16 weeks	Phase 2 Placebo n=65 Participants Double Blind Randomization (Phase 2)	Phase 2 ELND005 500 mg BID n=64 Participants Randomization Phase 2 ELND005 500 mg BID
Treatment Emergent Adverse Events	153 participants	45 participants	45 participants

SECONDARY outcome

Timeframe: up to 48 weeks

Population: Study was prematurely terminated, no data were collected for this Outcome Measure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 48 weeks

Population: Study was prematurely terminated, no data were collected for this Outcome Measure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 48 weeks

Population: Study was prematurely terminated, no data were collected for this Outcome Measure

Outcome measures

Outcome data not reported

Adverse Events

Open Treatment Phase 1 ELND005 500 mg BID

Serious events: 12 serious events

Other events: 153 other events

Deaths: 0 deaths

Phase 2 Placebo

Serious events: 3 serious events

Other events: 45 other events

Deaths: 0 deaths

Phase 2 ELND005 500 mg BID

Serious events: 3 serious events

Other events: 45 other events

Deaths: 0 deaths

Serious adverse events

Measure	Open Treatment Phase 1 ELND005 500 mg BID n=309 participants at risk ELND005 500mg BID for 16 weeks	Phase 2 Placebo n=65 participants at risk Double Blind Randomization (Phase 2)	Phase 2 ELND005 500 mg BID n=64 participants at risk Randomization Phase 2 ELND005 500 mg BID
Psychiatric disorders Depression	0.97% 3/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Psychiatric disorders Bipolar I Disorder	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Cardiac disorders Cardiac disorder	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Cardiac disorders Embolic stroke	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Injury, poisoning and procedural complications Food Poisoning	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.6% 1/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Gastrointestinal disorders Gastroenteritis	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Blood and lymphatic system disorders Hemolytic anemia	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.5% 1/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Psychiatric disorders Impulsive Behaviour	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Psychiatric disorders Mania	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.6% 1/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Infections and infestations Pneumonia	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.5% 1/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Psychiatric disorders Suicide attempt	0.32% 1/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Reproductive system and breast disorders Abortion spontaneous	0.00% 0/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.5% 1/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Eye disorders Diplopia	0.00% 0/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.6% 1/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Psychiatric disorders Intentional self-injury	0.00% 0/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.5% 1/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Musculoskeletal and connective tissue disorders Intervertebral disc degeneration	0.00% 0/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	1.5% 1/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	0.00% 0/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.

Other adverse events

Measure	Open Treatment Phase 1 ELND005 500 mg BID n=309 participants at risk ELND005 500mg BID for 16 weeks	Phase 2 Placebo n=65 participants at risk Double Blind Randomization (Phase 2)	Phase 2 ELND005 500 mg BID n=64 participants at risk Randomization Phase 2 ELND005 500 mg BID
Gastrointestinal disorders Gastrointestinal disorders	9.4% 29/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	18.5% 12/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	17.2% 11/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
General disorders General disorders and administration site conditions	3.2% 10/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	3.1% 2/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	7.8% 5/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Infections and infestations Infections and infestations	15.2% 47/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	30.8% 20/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	31.2% 20/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Injury, poisoning and procedural complications Injury poisoning and procedural complications	6.1% 19/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	23.1% 15/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	17.2% 11/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Investigations Investigations	4.2% 13/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	12.3% 8/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	12.5% 8/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Metabolism and nutrition disorders Metabolism and nutrition disorders	2.9% 9/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	3.1% 2/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	7.8% 5/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorders	5.8% 18/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	16.9% 11/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	15.6% 10/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Nervous system disorders Nervous system disorders	11.3% 35/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	20.0% 13/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	17.2% 11/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Psychiatric disorders Psychiatric disorders	20.4% 63/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	23.1% 15/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	20.3% 13/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders	1.3% 4/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	6.2% 4/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	6.2% 4/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	3.9% 12/309 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	4.6% 3/65 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.	6.2% 4/64 • Adverse events were recorded for each patient starting from the time the consent form was signed until the completion of the study.

Additional Information

Aleksandra Pastrak,MD, PhD, VP of Clinical Development and Medical Officer

Transition Therapeutics Ireland Limited

Phone: +1 416 263 1227

Email: apastrak@transitiontherapeutics.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: GT60