Trial Outcomes & Findings for Trial of Antimycobacterial Therapy in Sarcoidosis (NCT NCT01074554)

NCT ID: NCT01074554

Last Updated: 2016-12-12

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 30 participants
• Primary outcome timeframe: Baseline to 8 weeks
• Results posted on: 2016-12-12

Participant Flow

One patient who was randomized to the CLEAR regimen did not have lesions compatible with sarcoidosis at baseline. This patient did not proceed with study participation and was removed from number of patients that started the study.

Participant milestones

Measure	Antibiotics This study will compare the effects of antibiotics or placebo on resolution of cutaneous sarcoidosis lesions.	Lactose Tablets Patients who are randomized to control arm will receive an equivalent number of lactose tablets.
Overall Study STARTED	14	15
Overall Study COMPLETED	11	11
Overall Study NOT COMPLETED	3	4

Reasons for withdrawal

Measure	Antibiotics This study will compare the effects of antibiotics or placebo on resolution of cutaneous sarcoidosis lesions.	Lactose Tablets Patients who are randomized to control arm will receive an equivalent number of lactose tablets.
Overall Study Adverse Event	3	3
Overall Study Lost to Follow-up	0	1

Baseline Characteristics

Trial of Antimycobacterial Therapy in Sarcoidosis

Baseline characteristics by cohort

Measure	Antibiotics n=14 Participants This study will compare the effects of antibiotics or placebo on resolution of cutaneous sarcoidosis lesions.	Lactose Tablets n=15 Participants Patients who are randomized to control arm will receive an equivalent number of lactose tablets.	Total n=29 Participants Total of all reporting groups
Age, Continuous	55 years n=5 Participants	53 years n=7 Participants	53 years n=5 Participants
Gender Female	9 Participants n=5 Participants	10 Participants n=7 Participants	19 Participants n=5 Participants
Gender Male	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 8 weeks

Outcome measures

Measure	Antibiotic Regimen n=14 Participants The Antibiotic Regimen consists of Levaquin 750 mg loading on day 1, then 500 mg po QD and Ethambutol 15-25 mg/kg for a maximum of 1200mg QD and Azithromycin 500mg on day 1, then 250 mg po QD and Rifampin 5-10 mg/kg for a maximum of 300mg po QD. All four drugs are given concomitantly.	Placebo Regimen n=15 Participants The placebo regimen consists of Lactose tablets, one for each antibiotic with equivalent pills
Change in Lesion Size at the Completion of Antibiotic Therapy, Measured on a Continuous Scale; Change Will be Determined by Change in Diameter of the Lesions	-8.4 mm Standard Deviation 14	0.07 mm Standard Deviation 32.

PRIMARY outcome

Timeframe: Baseline to 8 weeks

Number of patients with a decrease in Granuloma Burden (only in those patients having granulomas present at baseline biopsy)

Outcome measures

Measure	Antibiotic Regimen n=10 Participants The Antibiotic Regimen consists of Levaquin 750 mg loading on day 1, then 500 mg po QD and Ethambutol 15-25 mg/kg for a maximum of 1200mg QD and Azithromycin 500mg on day 1, then 250 mg po QD and Rifampin 5-10 mg/kg for a maximum of 300mg po QD. All four drugs are given concomitantly.	Placebo Regimen n=7 Participants The placebo regimen consists of Lactose tablets, one for each antibiotic with equivalent pills
Granuloma Burden	7 participants	0 participants

SECONDARY outcome

Timeframe: Baseline to 8 weeks

Characterization of lesion severity was conducted using Modified Sarcoidosis Activity and Severity Index (SASI), measuring erythema, induration and desquamation. The modification was that the same scale was applied to any part of the body, instead of the face alone. The scale range is 0 (no problem) to 72 (very severe).

Outcome measures

Measure	Antibiotic Regimen n=14 Participants The Antibiotic Regimen consists of Levaquin 750 mg loading on day 1, then 500 mg po QD and Ethambutol 15-25 mg/kg for a maximum of 1200mg QD and Azithromycin 500mg on day 1, then 250 mg po QD and Rifampin 5-10 mg/kg for a maximum of 300mg po QD. All four drugs are given concomitantly.	Placebo Regimen n=15 Participants The placebo regimen consists of Lactose tablets, one for each antibiotic with equivalent pills
Change in Modified Sarcoidosis Activity and Severity Index (SASI) at Completion of Therapy.	-2.9 units on a scale Standard Deviation 2.5	-0.6 units on a scale Standard Deviation 2.1

Adverse Events

Antibiotics

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Lactose Tablets

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Antibiotics n=15 participants at risk This study will compare the effects of antibiotics or placebo on resolution of cutaneous sarcoidosis lesions.	Lactose Tablets n=15 participants at risk Patients who are randomized to control arm will receive an equivalent number of lactose tablets.
Gastrointestinal disorders diarrhea	6.7% 1/15 • Number of events 1 • 8 weeks	6.7% 1/15 • Number of events 1 • 8 weeks
Musculoskeletal and connective tissue disorders joint pain	6.7% 1/15 • Number of events 1 • 8 weeks	6.7% 1/15 • Number of events 1 • 8 weeks
General disorders insomnia	6.7% 1/15 • Number of events 1 • 8 weeks	0.00% 0/15 • 8 weeks
General disorders pill burden	0.00% 0/15 • 8 weeks	6.7% 1/15 • Number of events 1 • 8 weeks

Additional Information

Dr. Wonder Drake

Vanderbilt University School of Medicine

Phone: 615-322-2035

Email: wonder.drake@vanderbilt.edu

Results disclosure agreements

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