5 completed, 2 active not recruiting, 1 recruiting, 1 terminated. 4 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 1, Terminated 1, Unknown 1, Withdrawn 1 |
| Phase 1 | 1 | Terminated 1 |
Follow Hermanski-Pudlak Syndrome: new trials by email Literature Review of Hermanski-Pudlak Syndrome trials Research Report on Hermanski-Pudlak Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| 23andMe IPF Research Study No intervention - Idiopathic Pulmonary Fibrosis | Completed | 23andMe, Inc. | 690 | 2020-06-05 | 2024-12-09 | |
| Efficacy and Safety of Pirfenidone Treatment in HPS-ILD Pirfenidone - Hermansky Pudlak Syndrome, Interstitial Lung Disease | Phase 2 | Unknown | Jesse Roman | 50 | 2019-12-01 | 2019-12-10 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis Pulmonary function test, Chest CT, Sample collection - Hermansky Pudlak Syndrome | Completed | Vanderbilt University | 55 | 2015-03-01 | 2020-06-24 | |
| Clinical and Pathophysiological Investigations Into Erdheim Chester Disease Myelofibrosis, Gaucher Disease | Completed | National Human Genome Research Institute (NHGRI) | 88 | 2011-08-01 | 2019-07-26 | |
| Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome Mesalamine, Infliximab, Corticosteroids - Hermanski-Pudlak Syndrome, Colitis | Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2007-08-07 | 2017-07-02 | |
| Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome Losartan, Zileuton, N-Acetylcysteine - Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis | Phase 1 / Phase 2 | Terminated | National Human Genome Research Institute (NHGRI) | 3 | 2007-04-01 | 2013-08-02 |
| Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome Pirfenidone, Placebo - Albinism, Inborn Errors of Metabolism | Phase 2 | Completed | William Gahl, M.D. | 35 | 2005-09-01 | 2017-10-16 |
| Study of Megakaryocytes From Patients With Abnormal Platelet Vesicles Blood Coagulation Disorders | Completed | National Human Genome Research Institute (NHGRI) | 3 | 2004-06-29 | 2017-07-02 | |
| Analysis of Specimens From Individuals With Pulmonary Fibrosis Pulmonary Fibrosis, Healthy Volunteers | Active not recruiting | National Human Genome Research Institute (NHGRI) | 315 | 2004-06-09 | 2026-10-21 | |
| Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome Hermansky-Pudlak Syndrome (HPS) | Recruiting | National Human Genome Research Institute (NHGRI) | 600 | 1995-11-06 | 2026-10-13 |
Pulmonary Fibrosis (6) · Platelet Storage Pool Deficiency (4) · Albinism (3) · Albinism, Oculocutaneous (2) · Lung Diseases (2) · Metabolic Diseases (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1)
Amino Acid Metabolism, Inborn Errors (11) · Blood Coagulation Disorders, Inherited (11) · Blood Platelet Disorders (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Eye Diseases (11) · Eye Diseases, Hereditary (11) · Genetic Diseases, Inborn (11) · Hematologic Diseases (11) · Hemic and Lymphatic Diseases (11) · Hemorrhagic Disorders (11) · Hypopigmentation (11) · Nutritional and Metabolic Diseases (11) · Pigmentation Disorders (11) · Skin Diseases (11) · Skin Diseases, Genetic (11)
pirfenidone (2) · Acetylcysteine (1) · Adalimumab (1) · Adrenal Cortex Hormones (1) · Erythromycin (1) · Infliximab (1) · Losartan (1) · Mercaptopurine (1) · Mesalamine (1) · Pravastatin (1) · Respiratory Physiological Phenomena (1) · Specimen Handling (1) · Tacrolimus (1) · zileuton (1)
National Human Genome Research Institute (NHGRI) (5) · 23andMe, Inc. (1) · Jesse Roman (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · RTI International (1) · Vanderbilt University (1) · William Gahl, M.D. (1)
United States (11) · Puerto Rico (1)
Wendy J Introne, M.D. (2) · Curt Scharfe, MD, PhD (1) · Jesse Roman, MD (1) · Kevin J O'Brien, C.R.N.P. (1) · Lisa R. Young, MD (1) · Suyash Shringarpure (1) · Thomas Markello, M.D. (1)
PMID 29445374, PMID 30369044, PMID 31315600, PMID 35142618
U54HL127672 (1)