Success Metrics

Clinical Success Rate
75.0%

Based on 9 completed trials

Completion Rate
75%(9/12)
Active Trials
3(19%)
Results Posted
33%(3 trials)
Terminated
3(19%)

Phase Distribution

Ph phase_2
5
31%
Ph not_applicable
1
6%
Ph phase_4
2
13%
Ph phase_3
6
38%

Phase Distribution

0

Early Stage

5

Mid Stage

8

Late Stage

Phase Distribution14 total trials
Phase 2Efficacy & side effects
5(35.7%)
Phase 3Large-scale testing
6(42.9%)
Phase 4Post-market surveillance
2(14.3%)
N/ANon-phased studies
1(7.1%)

Highest Phase Reached

Phase 4

Trial Status & Enrollment

Completion Rate

75.0%

9 of 12 finished

Non-Completion Rate

25.0%

3 ended early

Currently Active

3

trials recruiting

Total Trials

16

all time

Status Distribution
Active(3)
Completed(9)
Terminated(3)
Other(1)

Detailed Status

Completed9
Terminated3
Recruiting2
unknown1
Active, not recruiting1

Development Timeline

Analytics

Development Status

Total Trials
16
Active
3
Success Rate
75.0%
Most Advanced
Phase 4

Trials by Phase

Phase 25 (35.7%)
Phase 36 (42.9%)
Phase 42 (14.3%)
N/A1 (7.1%)

Trials by Status

unknown16%
completed956%
terminated319%
active_not_recruiting16%
recruiting213%

Recent Activity

3 active trials
Showing 5 of 16
recruiting

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
recruitingphase_3

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

NCT04808505
active_not_recruitingphase_3

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505
terminatedphase_3

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

NCT03822013
completedphase_3

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362
View all 16 trials

Clinical Trials (16)

Showing 16 of 16 trials
NCT06121011

A Global Prospective Observational Registry of Patients With Pompe Disease

Recruiting
NCT04808505Phase 3

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Recruiting
NCT03911505Phase 3

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Active Not Recruiting
NCT03822013Phase 3

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Terminated
NCT03729362Phase 3

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Completed
NCT03910621Phase 4

Safety and Efficacy of Miglustat in Chinese NPC Patients

Completed
NCT00517153Phase 2

Miglustat in Niemann-Pick Type C Disease

Completed
NCT00319046Phase 3

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

Completed
NCT02030015Phase 4

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Terminated
NCT02520934Not Applicable

Miglustat on Gaucher Disease Type IIIB

Unknown
NCT01451879

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Completed
NCT00418847Phase 2

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

Completed
NCT00742092Phase 2

Miglustat in Cystic Fibrosis

Completed
NCT01760564Phase 3

Application of Miglustat in Patients With Niemann-Pick Type C

Completed
NCT00945347Phase 2

Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?

Completed
NCT00537602Phase 2

Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Terminated

All 16 trials loaded

Drug Details

Intervention Type
DRUG
Total Trials
16