Trial Outcomes & Findings for Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome (NCT NCT00467831)

Results Overview

The number of subjects surviving after 24 months on study.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

3 participants

Primary outcome timeframe

24 months

Results posted on

2013-08-02

Participants were recruited from April 2007 to November 2012 at the NIH Clinical Center.

Participant milestones
Measure	Multi-Drug Regimen Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily. Erythromycin : Erythromycin tablet, 333 mg by mouth three times daily. Losartan : Losartan potassium tablet, 25 mg by mouth every night at bedtime. Zileuton : Zileuton tablet, 1200 mg by mouth twice daily. N-Acetylcysteine : N-acetylcysteine solution, 600 mg by mouth three times daily. Pravastatin : Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal
Measure	Multi-Drug Regimen Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily. Erythromycin : Erythromycin tablet, 333 mg by mouth three times daily. Losartan : Losartan potassium tablet, 25 mg by mouth every night at bedtime. Zileuton : Zileuton tablet, 1200 mg by mouth twice daily. N-Acetylcysteine : N-acetylcysteine solution, 600 mg by mouth three times daily. Pravastatin : Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Overall Study Death	2
Overall Study Withdrawal by Subject	1

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

Baseline characteristics by cohort
Measure	Multi-Drug Regimen n=3 Participants Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily. Erythromycin : Erythromycin tablet, 333 mg by mouth three times daily. Losartan : Losartan potassium tablet, 25 mg by mouth every night at bedtime. Zileuton : Zileuton tablet, 1200 mg by mouth twice daily. N-Acetylcysteine : N-acetylcysteine solution, 600 mg by mouth three times daily. Pravastatin : Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	51 years STANDARD_DEVIATION 9 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

Timeframe: 24 months

The number of subjects surviving after 24 months on study.

Outcome measures
Measure	Multi-Drug Regimen n=2 Participants Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily. Erythromycin : Erythromycin tablet, 333 mg by mouth three times daily. Losartan : Losartan potassium tablet, 25 mg by mouth every night at bedtime. Zileuton : Zileuton tablet, 1200 mg by mouth twice daily. N-Acetylcysteine : N-acetylcysteine solution, 600 mg by mouth three times daily. Pravastatin : Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Survival at 2 Years	0 participants

Serious events: 2 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events
Measure	Multi-Drug Regimen n=3 participants at risk Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily. Erythromycin : Erythromycin tablet, 333 mg by mouth three times daily. Losartan : Losartan potassium tablet, 25 mg by mouth every night at bedtime. Zileuton : Zileuton tablet, 1200 mg by mouth twice daily. N-Acetylcysteine : N-acetylcysteine solution, 600 mg by mouth three times daily. Pravastatin : Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Respiratory, thoracic and mediastinal disorders Respiratory failure	66.7% 2/3 • Number of events 2 • Overall study: 24 months

Other adverse events
Measure	Multi-Drug Regimen n=3 participants at risk Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily. Erythromycin : Erythromycin tablet, 333 mg by mouth three times daily. Losartan : Losartan potassium tablet, 25 mg by mouth every night at bedtime. Zileuton : Zileuton tablet, 1200 mg by mouth twice daily. N-Acetylcysteine : N-acetylcysteine solution, 600 mg by mouth three times daily. Pravastatin : Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Hepatobiliary disorders elevated liver enzyme levels	66.7% 2/3 • Number of events 2 • Overall study: 24 months
Respiratory, thoracic and mediastinal disorders upper respiratory infection	66.7% 2/3 • Number of events 3 • Overall study: 24 months
Blood and lymphatic system disorders anemia	66.7% 2/3 • Number of events 2 • Overall study: 24 months

NHGRI/NIH

Phone: 301-451-1305