Clinical trial NCT00467831 (phase 1/phase 2, status: terminated)
Conditions: hermansky-pudlak syndrome (hps), pulmonary fibrosis, oculocutaneous albinism, platelet storage pool deficiency, metabolic disease
ICD codes: J84.10, J84.112, E84.0, J84.178, Z86.39, O99.280, O99.281, O99.282, O99.283, O99.284, O99.285
Drugs: losartan, zileuton, n-acetylcysteine, pravastatin, erythromycin
SMILES: CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=C1)C1=CC=CC=C1C1=NNN=N1, CC(N(O)C(N)=O)C1=CC2=CC=CC=C2S1, CC(=O)N[C@@H](CS)C(O)=O, CC(C)C1=C(C(=O)NC2=CC=CC=C2)C(=C(N1CC[C@@H](O)C[C@@H](O)CC(O)=O)C1=CC=C(F)C=C1)C1=CC=CC=C1, [H][C@]12SC(C)(C)[C@@H](N1C(=O)[C@H]2NC(=O)[C@H](N)C1=CC=CC=C1)C(O)=O
Reason stopped: 
    insufficient enrollment
  
Eligibility criteria:

        -  INCLUSION CRITERIA:

        To be eligible for this protocol, participants must:

          -  Have a molecular diagnosis of HPS-1 or HPS-4

          -  Be 18-70 years of age

          -  Have the expectation to live more than 3 months, i.e., an FVC greater than or equal to
             30% of predicted

          -  Have evidence of severe pulmonary fibrosis, i.e.:

               1. A FVC less than or equal to 45% of predicted

               2. Reduced exercise tolerance lasting longer than 1 week on the Dyspnea Perception
                  Scale

               3. No evidence of improvement in pulmonary fibrosis within the past year, as defined
                  by an FVC increase of 10% or a DLco increase of 15%.

          -  Be available, willing, and able to come to the NIH Clinical Center for admission every
             3 months.

        EXCLUSION CRITERIA:

          -  An explanation for interstitial lung disease other than HPS, including but not limited
             to radiation, sarcoidosis, hypersensitivity pneumonitis, bronchiolitis obliterans
             organizing pneumonia, cancer

          -  Pregnancy or lactation

          -  History of ethanol abuse or recreational drug use in the past two years

          -  History of human immunodeficiency virus (HIV) or chronic viral hepatitis infection

          -  Chronic use of high-dose steroids (greater than 10 mg prednisone/day) intended for
             ongoing treatment of their interstitial lung disease

          -  Use of any of the following within 28 days of enrollment: investigational therapy,
             cytotoxic/immunosuppressive agents other than corticosteroids, including but not
             limited to azathioprine, cyclosphosphamide, methotrexate, cyclosporine, colchicine,
             interferon gamma-1b, bosentan;

          -  Any severe medical complication including but not be limited to uncontrolled seizures,
             repeated transient ischemic attacks, severe ataxia, uncontrolled migraine headaches,
             diplopia, repeated episodes of syncope, an untreated psychiatric disorder, recent
             myocardial infarction (past 6 months), unstable angina, clinically relevant and
             untreated arrhythmias, uncontrolled hypotension or hypertension (systolic blood
             pressure less than 80 or greater than 180 mm Hg), myocarditis, severe congestive left
             sided heart failure, hepatomegaly not due to right heart failure, renal glomerular
             impairment (creatinine clearance less than 35 ml/min/1.73 m(2)), pancreatitis, toxic
             thyroiditis, life-threatening malignancy;NOTE: right sided heart failure due to
             pulmonary hypertension as a result of pulmonary fibrosis will not be considered an
             exclusion criteria.

          -  Significant laboratory abnormalities, including but not limited to serum potassium
             less than 3.0 or greater than 5.4 mEq/L, SGPT greater than 100 U/L, CK greater than
             700 U/L, hemoglobin less than 9.0 g/dL, platelets less than 70 k/mm(3), leukocyte
             count less than 2.0 k/microL;

          -  For women of child-bearing age, failure to have an effective method of birth control.
             Oral contraceptives will be considered inadequate without a second method due to risk
             of reduced efficacy of BCP while taking Zileuton.

          -  Severe psychiatric disease untreated. Inability to give informed consent after reading
             or having the consent read to the participant in their native language. Any concern
             that there is a therapeutic misconception will be evaluated by genetic counselor
             and/or appropriate mental health professionals prior to acceptance into the study