Trial Outcomes & Findings for A Study of IDN-6556 in Subjects With NAFLD and Raised Transaminases (NCT NCT02077374)

NCT ID: NCT02077374

Last Updated: 2016-08-30

Results Overview

Back transformation from log-transformed analysis results to original scale in alanine aminotransferase (ALT) from Baseline to Day 28/ET between IDN-6556 vs Placebo

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

38 participants

Primary outcome timeframe

Baseline to Day 28/ET

Results posted on

2016-08-30

Participant Flow

In total, 71 subjects were screened at 7 sites, and 38 subjects were randomized and treated to a BID treatment with either placebo or IDN-6556 25 mg with 19 subjects in each arm.

38 subjects were randomized and treated (19 each arm). 2 subjects on placebo discontinued before Day 28 and 1 discontinued after Day 28, but returned for their Day 56 visit, so 17 subjects completed the study. However, all 38 were included in the analyses using the last results provided for the 2 subjects at early termination.

Participant milestones

Participant milestones
Measure	IDN-6556 IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo Placebo Matching Placebo BID for 28 days
Overall Study STARTED	19	19
Overall Study COMPLETED	19	17
Overall Study NOT COMPLETED	0	2

Reasons for withdrawal

Reasons for withdrawal
Measure	IDN-6556 IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo Placebo Matching Placebo BID for 28 days
Overall Study Withdrawal by Subject	0	1
Overall Study Protocol Violation	0	1

Baseline Characteristics

A Study of IDN-6556 in Subjects With NAFLD and Raised Transaminases

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days	Total n=38 Participants Total of all reporting groups
Age, Categorical Between 18 and 65 years	17 Participants n=5 Participants	16 Participants n=7 Participants	33 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Age, Continuous	46 years n=5 Participants	55 years n=7 Participants	52 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	7 Participants n=7 Participants	14 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Region of Enrollment United States	19 participants n=5 Participants	19 participants n=7 Participants	38 participants n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 28/ET

Population: The full analysis set (FAS) included all subjects who were randomized and received at least one dose of study drug. For the FAS, subjects were assigned to the treatment group based on the randomization schedule, regardless of the treatment they actually received. All efficacy analyses were conducted on the FAS.

Mean change in alanine aminotransferase (ALT) from Baseline to Day 28/ET between IDN-6556 vs Placebo

Outcome measures

Outcome measures
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days
Change in Alanine Aminotransferase (ALT)	-37.8 U/L Standard Deviation 42.7	-14.9 U/L Standard Deviation 22.5

PRIMARY outcome

Timeframe: Baseline to Day 28/ET

Back transformation from log-transformed analysis results to original scale in alanine aminotransferase (ALT) from Baseline to Day 28/ET between IDN-6556 vs Placebo

Outcome measures

Outcome measures
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days
Relative Percent Change in Alanine Aminotransferase (ALT)	-39.2 Relative percent change Standard Deviation 26.2	-13.9 Relative percent change Standard Deviation 21.8

SECONDARY outcome

Timeframe: Day 28/ET

Difference in the change in aspartate aminotransferase (AST) from Baseline to Day 28/ET in units per liter (U/L) between IDN-6556 and placebo.

Outcome measures

Outcome measures
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days
Change in Aspartate Aminotransferase (AST)	-6.7 U/L Interval -63.1 to 20.0	-5.2 U/L Interval -41.9 to 27.9

SECONDARY outcome

Timeframe: Day 28/ET

Difference in the change in Caspase-cleaved cytokeratin serum levels (cCK18/M30) in units per liter (U/L) from baseline to Day 28/ET between IDN-6556 and placebo

Outcome measures

Outcome measures
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days
Levels of cCK18/M30	-183.0 U/L Interval -640.0 to 190.0	14.0 U/L Interval -2056.0 to 571.0

SECONDARY outcome

Timeframe: Day 28/ET

Difference in the change of caspase 3/7 (Relative Light Units) from baseline to Day 28/ET between IDN-6556 and Placebo

Outcome measures

Outcome measures
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days
Levels of Caspase 3/7 RLU	-287.0 RLU Interval -2194.0 to 417.0	68.0 RLU Interval -1779.0 to 1229.0

SECONDARY outcome

Timeframe: Day 28/ET

Difference in the change in full-length cytokeratin 18 (flCK18/M65) in units per liter (U/L) from Baseline to Day 28/ET between IDN-6556 and placebo

Outcome measures

Outcome measures
Measure	IDN-6556 n=19 Participants IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 Participants Placebo Matching Placebo BID for 28 days
Levels of flCK18/M65	-296.0 U/L Interval -1216.0 to 225.0	4.0 U/L Interval -2364.0 to 724.0

Adverse Events

IDN-6556

Serious events: 1 serious events

Other events: 10 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	IDN-6556 n=19 participants at risk IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 participants at risk Placebo Matching Placebo BID for 28 days
Gastrointestinal disorders Gastrointestinal haemorrhage	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Skin and subcutaneous tissue disorders Cellulitis	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.

Other adverse events

Additional Information

Jean L. Chan, MD

Conatus Pharmaceuticals Inc.

Phone: (858) 376-2632

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	IDN-6556 n=19 participants at risk IDN-6556 capsules, 25 mg IDN-6556: 25 mg BID for 28 days	Placebo n=19 participants at risk Placebo Matching Placebo BID for 28 days
Blood and lymphatic system disorders Haemorrhagic anaemia	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Endocrine disorders Hyperthyroidism	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Abdominal distension	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Frequent bowel movements	10.5% 2/19 • Number of events 2 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
General disorders Chest pain	10.5% 2/19 • Number of events 2 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
General disorders Injection site erythema	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Immune system disorders Hypersensitivity	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Infections and infestations Bronchitis	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Infections and infestations Cellulitis	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Infections and infestations Pharyngitis streptococcal	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Infections and infestations Urinary tract infection	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	10.5% 2/19 • Number of events 2 • Adverse events data were collected up to Day 56.
Infections and infestations Viral upper respiratory tract infection	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Musculoskeletal and connective tissue disorders Groin pain	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Nervous system disorders Hypoaesthesia	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Nervous system disorders Headache	10.5% 2/19 • Number of events 2 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Renal and urinary disorders Renal failure acute	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Skin and subcutaneous tissue disorders Psoriasis	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Blood and lymphatic system disorders Monoclonal B-cell lymphocytosis	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Abdominal pain upper	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Diarrhoea	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Dry mouth	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Dyspepsia	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Flatulence	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Haematochezia	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Nausea	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Gastrointestinal disorders Vomiting	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
General disorders Injection site pain	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
General disorders Injection site swelling	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
General disorders Local Swelling	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
General disorders Oedema peripheral	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
General disorders Pyrexia	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.	0.00% 0/19 • Adverse events data were collected up to Day 56.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.
Respiratory, thoracic and mediastinal disorders Sinus congestion	0.00% 0/19 • Adverse events data were collected up to Day 56.	5.3% 1/19 • Number of events 1 • Adverse events data were collected up to Day 56.