Key Insights

Highlights

Treatment Research

2 treatments under study, led by Ald101

Related Conditions

Often studied with 13 related conditions

Clinical Risk Assessment

Based on trial outcomes

High Risk

Score: 75/100

Termination Rate

20.0%

1 terminated out of 5 trials

Success Rate

0.0%

-86.6% vs benchmark

Late-Stage Pipeline

20%

1 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed with results

Key Signals

0

Data Visualizations

Phase Distribution

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

Trial Status

Unknown2
Enrolling By Invitation1
Terminated1
Not Yet Recruiting1

Clinical Trials (5)

Showing 5 of 5 trials
NCT00001215Enrolling By InvitationPrimary

Genetic Studies of Lysosomal Storage Disorders

NCT07740512Phase 2Not Yet RecruitingPrimary

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

NCT02000310UnknownPrimary

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

NCT02120235UnknownPrimary

Investigating Lysosomal Storage Diseases in Minority Groups

NCT00654433Phase 3Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Showing all 5 trials

Research Network

Common Treatments

  • Ald1011
  • Gemfibrozil1

Active Sponsors

  • O & O Alpan LLC2
  • National Human Genome Research Institute (NHGRI)1
  • Aldagen1
  • Polaryx Therapeutics, Inc.1

Top Investigators

Related Conditions

  • Gaucher Disease2
  • Peroxisomal Storage Diseases1
  • Pompe Disease1
  • Sandhoff Disease1
  • Inborn Errors Of Metabolism1

Activity Timeline