Trial Outcomes & Findings for Double-Blinded, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Biochemical Activity of Intravenous Cpn10 Administration in Subjects With Mild to Moderate SLE. (NCT NCT01838694)

NCT ID: NCT01838694

Last Updated: 2017-01-30

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 30 participants
• Primary outcome timeframe: 4 weeks
• Results posted on: 2017-01-30

Participant Flow

Participant milestones

Measure	Placebo Multiple doses of matched vehicle (no active ingredients) administered intravenously over 60 minutes. Placebo	Ala-Cpn10 Recombinant minimally modified Chaperonin10 (Cpn10) Multiple doses in the range of 10mg twice weekly to 100mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10
Overall Study STARTED	8	22
Overall Study COMPLETED	8	21
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Placebo Multiple doses of matched vehicle (no active ingredients) administered intravenously over 60 minutes. Placebo	Ala-Cpn10 Recombinant minimally modified Chaperonin10 (Cpn10) Multiple doses in the range of 10mg twice weekly to 100mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10
Overall Study Adverse Event	0	1

Baseline Characteristics

Double-Blinded, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Biochemical Activity of Intravenous Cpn10 Administration in Subjects With Mild to Moderate SLE.

Baseline characteristics by cohort

Measure	Placebo n=8 Participants Multiple doses of matched vehicle (no active ingredients) administered intravenously over 60 minutes. Placebo	Ala-Cpn10 n=22 Participants Recombinant minimally modified Chaperonin10 (Cpn10) Multiple doses in the range of 10mg twice weekly to 100mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10	Total n=30 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants	18 Participants n=7 Participants	24 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Age, Continuous	50 years STANDARD_DEVIATION 18 • n=5 Participants	43 years STANDARD_DEVIATION 15 • n=7 Participants	45 years STANDARD_DEVIATION 15 • n=5 Participants
Gender Female	7 Participants n=5 Participants	21 Participants n=7 Participants	28 Participants n=5 Participants
Gender Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants	22 participants n=7 Participants	30 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks

Population: Analysis Population reflects participants for whom adequate analyzable samples were collected

Outcome measures

Measure	Placebo n=5 Participants Multiple doses of matched vehicle (no active ingredients) administered intravenously over 60 minutes. Placebo	Ala-Cpn10 - 10mgs in Mild SLE n=5 Participants Recombinant minimally modified Chaperonin10 (Cpn10) 10mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10	Ala-Cpn10 - 30mgs in Mild SLE n=4 Participants Recombinant minimally modified Chaperonin10 (Cpn10) 30mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10	Ala-Cpn10 - 100mgs in Mild SLE n=3 Participants Recombinant minimally modified Chaperonin10 (Cpn10) 100mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10	Ala-Cpn10 - 30mgs in Moderate SLE n=1 Participants Recombinant minimally modified Chaperonin10 (Cpn10) 30mg twice weekly administered intravenously by infusion over 60 minutes. Ala-Cpn10
Change From Baseline Serum Interleukin 6 (IL-6) Levels at the End of Active Dosing, Comparing Treatment to Placebo Cohort.	1.6 percentage change from baseline Standard Deviation 24.7	987.7 percentage change from baseline Standard Deviation 2202.1	-82.5 percentage change from baseline Standard Deviation 20.4	-29.7 percentage change from baseline Standard Deviation 59.1	5000 percentage change from baseline Standard Deviation 0

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Ala-Cpn10

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=8 participants at risk Multiple doses of matched vehicle (no active ingredients) administered intravenously over 60 minutes. Placebo	Ala-Cpn10 n=22 participants at risk Recombinant minimally modified Chaperonin10 (Cpn10) Multiple doses in the range (0.16mg/kg \[10mg twice weekly\] to 5mg/kg \[300mg twice weekly\]) administered intravenously by infusion over 60 minutes. Ala-Cpn10
Vascular disorders Hypotension	0.00% 0/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	4.5% 1/22 • Number of events 1 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks

Other adverse events

Measure	Placebo n=8 participants at risk Multiple doses of matched vehicle (no active ingredients) administered intravenously over 60 minutes. Placebo	Ala-Cpn10 n=22 participants at risk Recombinant minimally modified Chaperonin10 (Cpn10) Multiple doses in the range (0.16mg/kg \[10mg twice weekly\] to 5mg/kg \[300mg twice weekly\]) administered intravenously by infusion over 60 minutes. Ala-Cpn10
Eye disorders Eye pain	12.5% 1/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	0.00% 0/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks
Gastrointestinal disorders diarrhoea	12.5% 1/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	0.00% 0/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks
Gastrointestinal disorders dyspepsia	0.00% 0/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	9.1% 2/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks
Gastrointestinal disorders Mouth ulceration	12.5% 1/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	0.00% 0/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks
Gastrointestinal disorders Nausea	0.00% 0/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	13.6% 3/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks
General disorders Fatigue	12.5% 1/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	9.1% 2/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks
Immune system disorders cutaneous lupus erythematosus	12.5% 1/8 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks	0.00% 0/22 • Up to 30 days following each patient's discontinuation of the study, for up to 9 weeks

Additional Information

Mitchell Glass, M.D., EVP of R&D

Invion, Inc.

Phone: +61 7 3295 0500

Email: mitchell.glass@inviongroup.com

Results disclosure agreements

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

Restriction type: LTE60