Key Insights

Highlights

Success Rate

83% trial completion

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 49/100

Termination Rate

11.1%

2 terminated out of 18 trials

Success Rate

83.3%

-3.2% vs benchmark

Late-Stage Pipeline

17%

3 trials in Phase 3/4

Results Transparency

50%

5 of 10 completed with results

Key Signals

5 with results83% success

Data Visualizations

Phase Distribution

11Total
P 1 (3)
P 2 (5)
P 3 (2)
P 4 (1)

Trial Status

Completed10
Recruiting2
Unknown2
Terminated2
Enrolling By Invitation1
Active Not Recruiting1

Trial Success Rate

83.3%

Benchmark: 86.5%

Based on 10 completed trials

Clinical Trials (18)

Showing 18 of 18 trials
NCT05619900Recruiting

Registry of Patients Diagnosed With Lysosomal Storage Diseases

NCT06036693Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

NCT03153319Phase 1Active Not Recruiting

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

NCT05368038Enrolling By Invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05687474Completed

Baby Detect : Genomic Newborn Screening

NCT03632213Phase 2Unknown

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

NCT01586455Phase 1Completed

Human Placental-Derived Stem Cell Transplantation

NCT03370653Phase 2CompletedPrimary

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil

NCT00748969Phase 2Terminated

Clinical Trial of Growth Hormone in MPS I, II, and VI

NCT00176917Phase 2Completed

Stem Cell Transplantation for Hurler

NCT01707433Completed

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01675674Terminated

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT00299000Phase 4CompletedPrimary

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

NCT00104234Phase 3CompletedPrimary

Study of rhASB in Patients With Mucopolysaccharidosis VI

NCT00067470Phase 3CompletedPrimary

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

NCT00048711Phase 2CompletedPrimary

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

NCT00048620Phase 1CompletedPrimary

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

NCT00005900Unknown

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Showing all 18 trials

Research Network

Activity Timeline