Key Insights

Highlights

Success Rate

60% trial completion

Treatment Research

10 treatments under study, led by Blood Parameters

Research Leaders

6 sponsors active, led by National Human Genome Research Institute (NHGRI)

Related Conditions

Often studied with 7 related conditions

Clinical Risk Assessment

Based on trial outcomes

High Risk

Score: 75/100

Termination Rate

33.3%

2 terminated out of 6 trials

Success Rate

60.0%

-26.6% vs benchmark

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

100%

3 of 3 completed with results

Key Signals

3 with results60% success

Data Visualizations

Phase Distribution

3Total
Phase 1/2 (1)
Phase 2 (2)

Trial Status

Completed3
Terminated2
Recruiting1

Trial Success Rate

60.0%

Benchmark: 86.6%

Based on 3 completed trials

Clinical Trials (6)

Showing 6 of 6 trials
NCT04068961CompletedPrimary

New Strategies of Genetic Study of Patients With Oculocutaneous Albinism

NCT06138509RecruitingPrimary

Peripheral Serotonin and Albinism

NCT00808106CompletedPrimary

Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism

NCT01663935Phase 2Terminated

Vision Response to Dopamine Replacement

NCT00001596Phase 2Completed

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

NCT00467831Phase 1/2Terminated

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

Showing all 6 trials

Research Network

Common Treatments

  • Blood Parameters1
  • Acetylcysteine1
  • Pirfenidone1
  • Pravastatin1
  • Zileuton1

Active Sponsors

  • National Human Genome Research Institute (NHGRI)3
  • University Hospital, Bordeaux1
  • URC-CIC Paris Descartes Necker Cochin1
  • Assistance Publique - Hôpitaux de Paris1
  • William Gahl, M.D.1

Top Investigators

Related Conditions

  • Albinism2
  • Platelet Storage Pool Deficiency2
  • Pulmonary Fibrosis2
  • Inborn Errors Of Metabolism1
  • Hermanskypudlak Syndrome Hps1

Activity Timeline