Trial Outcomes & Findings for LCP-Tacro vs. Azathioprine for the Treatment of Autoimmune Hepatitis (NCT NCT00608894)

NCT ID: NCT00608894

Last Updated: 2020-03-17

Results Overview

Percent of patients that achieve biochemical remission of (AIH) at Month 6 during treatment with LCP-Tacro + prednisone or azathioprine + prednisone. Biochemical remission is defined as ALT, total bilirubin and gamma globulin within normal limits.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 13 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2020-03-17

Participant Flow

Participant milestones

Measure	LCP-Tacro LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.
Overall Study STARTED	7	6
Overall Study COMPLETED	7	4
Overall Study NOT COMPLETED	0	2

Reasons for withdrawal

Measure	LCP-Tacro LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.
Overall Study Withdrawal by Subject	0	2

Baseline Characteristics

LCP-Tacro vs. Azathioprine for the Treatment of Autoimmune Hepatitis

Baseline characteristics by cohort

Measure	LCP-Tacro n=7 Participants LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine n=6 Participants Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Total n=13 Participants Total of all reporting groups
Age, Continuous	50 years n=5 Participants	56 years n=7 Participants	53 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	6 Participants n=7 Participants	9 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Percent of patients that achieve biochemical remission of (AIH) at Month 6 during treatment with LCP-Tacro + prednisone or azathioprine + prednisone. Biochemical remission is defined as ALT, total bilirubin and gamma globulin within normal limits.

Outcome measures

Measure	LCP-Tacro n=7 Participants LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine n=6 Participants Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.
Biochemical Remission of (AIH) at Month 6.	3 Participants	4 Participants

SECONDARY outcome

Timeframe: 3 months

Percent of patients who achieve biochemical remission by Month 3 during treatment with LCP-Tacro + prednisone or azathioprine + prednisone. Biochemical remission is defined as ALT, total bilirubin and gamma globulin within normal limits.

Outcome measures

Measure	LCP-Tacro n=7 Participants LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine n=4 Participants Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.
Biochemical Remission by Month 3.	2 Participants	4 Participants

SECONDARY outcome

Timeframe: 6 months

Percents of patients in each treatment group classified as having an incomplete response (defined as some or no improvement during therapy), a treatment failure (defined as permanent discontinuation of the regimen originally randomized to), or a case of relapse (recurrence following achievement of remission)

Outcome measures

Measure	LCP-Tacro n=7 Participants LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine n=6 Participants Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.
Incomplete Response, Treatment Failure, or a Case of Relapse at 6 Months Relapse	0 Participants	0 Participants
Incomplete Response, Treatment Failure, or a Case of Relapse at 6 Months Incomplete response	4 Participants	1 Participants
Incomplete Response, Treatment Failure, or a Case of Relapse at 6 Months Treatment failure	0 Participants	1 Participants

Adverse Events

LCP-Tacro

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Azathioprine

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	LCP-Tacro n=7 participants at risk LCP-Tacro tablets(1,2,and 5mg tacrolimus)+ prednisone tablets(5mg) LCP-Tacro (tacrolimus): LCP-Tacro(tacrolimus)tablets starting at 2 mg once daily, then adjusted to achieve and maintain target whole blood tacrolimus levels of 3 - 6 ng/mL, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.	Azathioprine n=6 participants at risk Azathioprine tablets(50mg)+ prednisone tablets(5mg) Azathioprine: Azathioprine tablets 50 - 100 mg (approximately 1 mg/kg) once daily, plus prednisone 30 mg/day for one week, then 20 mg/day for one week, then 15 mg/day for two weeks, then 10 mg/day through Month 6.
Gastrointestinal disorders Abdominal discomfort	14.3% 1/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	0.00% 0/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Gastrointestinal disorders Diarrhea	28.6% 2/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	16.7% 1/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Gastrointestinal disorders Nausea	14.3% 1/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	33.3% 2/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Gastrointestinal disorders Vomiting	14.3% 1/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	33.3% 2/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Nervous system disorders Headache	28.6% 2/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	0.00% 0/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Nervous system disorders Tremor	28.6% 2/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	0.00% 0/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Skin and subcutaneous tissue disorders Acne	14.3% 1/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	0.00% 0/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)
Vascular disorders Hypertension	14.3% 1/7 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)	16.7% 1/6 • 6 months One patient (AZA) crossed over to treatment with LCP - Tacro after 8 weeks of treatment and is included in the original treatment group to which he was randomized (AZA)

Additional Information

Director, Regulatory Affairs

Veloxis Pharmaceuticals, Inc.

Phone: 919-591-3090

Email: bbu@veloxis.com

Results disclosure agreements

• Principal investigator is a sponsor employee The study is a multicenter collaborative investigation and the clinical trial results are to be published as a collaborative manuscript. Authorship will reflect varying levels of individual contribution to the study by the individual PI's.
• Publication restrictions are in place

Restriction type: OTHER