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Trial record 2 of 9 for:    "Storage Pool Platelet Disease"

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

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ClinicalTrials.gov Identifier: NCT00467831
Recruitment Status : Terminated (insufficient enrollment)
First Posted : May 1, 2007
Results First Posted : August 2, 2013
Last Update Posted : August 2, 2013
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Hermansky-Pudlak Syndrome (HPS)
Pulmonary Fibrosis
Oculocutaneous Albinism
Platelet Storage Pool Deficiency
Metabolic Disease
Interventions Drug: Losartan
Drug: Zileuton
Drug: N-Acetylcysteine
Drug: Pravastatin
Drug: Erythromycin
Enrollment 3
Recruitment Details Participants were recruited from April 2007 to November 2012 at the NIH Clinical Center.
Pre-assignment Details  
Arm/Group Title Multi-Drug Regimen
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 3
Completed 0
Not Completed 3
Reason Not Completed
Death             2
Withdrawal by Subject             1
Arm/Group Title Multi-Drug Regimen
Hide Arm/Group Description

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 Number of Baseline Participants 3
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
<=18 years
0
   0.0%
Between 18 and 65 years
3
 100.0%
>=65 years
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants
51  (9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
0
   0.0%
Male
3
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants
3
1.Primary Outcome
Title Survival at 2 Years
Hide Description The number of subjects surviving after 24 months on study.
Time Frame 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Multi-Drug Regimen
Hide Arm/Group Description:

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 Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
0
Time Frame Overall study: 24 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Multi-Drug Regimen
Hide Arm/Group Description

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.

All-Cause Mortality
Multi-Drug Regimen
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Multi-Drug Regimen
Affected / at Risk (%) # Events
Total   2/3 (66.67%)    
Respiratory, thoracic and mediastinal disorders   
Respiratory failure * 1 [1]  2/3 (66.67%)  2
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.1)
[1]
respiratory failure due to end stage lung disease resulting in death
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Multi-Drug Regimen
Affected / at Risk (%) # Events
Total   3/3 (100.00%)    
Blood and lymphatic system disorders   
anemia  1  2/3 (66.67%)  2
Hepatobiliary disorders   
elevated liver enzyme levels  1  2/3 (66.67%)  2
Respiratory, thoracic and mediastinal disorders   
upper respiratory infection * 1  2/3 (66.67%)  3
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.1)
The study was terminated because enrollment was too low. The results shown are not statistically significant.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Thomas Markello
Organization: NHGRI/NIH
Phone: 301-451-1305
EMail: markellot@mail.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )
ClinicalTrials.gov Identifier: NCT00467831     History of Changes
Other Study ID Numbers: 070132
07-HG-0132 ( Other Identifier: NHGRI IRB )
First Submitted: April 28, 2007
First Posted: May 1, 2007
Results First Submitted: June 3, 2013
Results First Posted: August 2, 2013
Last Update Posted: August 2, 2013