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

This study has been terminated.
(insufficient enrollment)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )
ClinicalTrials.gov Identifier:
NCT00467831
First received: April 28, 2007
Last updated: June 3, 2013
Last verified: June 2013
Results First Received: June 3, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: 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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were recruited from April 2007 to November 2012 at the NIH Clinical Center.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
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.


Participant Flow:   Overall Study
    Multi-Drug Regimen  
STARTED     3  
COMPLETED     0  
NOT COMPLETED     3  
Death                 2  
Withdrawal by Subject                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
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.


Baseline Measures
    Multi-Drug Regimen  
Number of Participants  
[units: participants]
  3  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     3  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  51  ± 9  
Gender  
[units: participants]
 
Female     0  
Male     3  
Region of Enrollment  
[units: participants]
 
United States     3  



  Outcome Measures

1.  Primary:   Survival at 2 Years   [ Time Frame: 24 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The study was terminated because enrollment was too low. The results shown are not statistically significant.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Thomas Markello
Organization: NHGRI/NIH
phone: 301-451-1305
e-mail: markellot@mail.nih.gov


Publications:

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
Study First Received: April 28, 2007
Results First Received: June 3, 2013
Last Updated: June 3, 2013
Health Authority: United States: Federal Government