Registry-listed formulations

golodirsen · mesh:C000710673

Each study is counted once in the molecule profile. Registry products remain separate. Naming an ingredient does not establish that products are interchangeable or what participants received.

4 source entries across 3 studies. This list may not include all unresolved or historical entries.

GOLODIRSEN (SRP-4053)Registry product
Named ingredient; product remains distinct
Formulation reported by source: CONCENTRATE FOR SOLUTION FOR INFUSION
Named ingredient; exact form unconfirmed. This mention does not add to the molecule trial count.
View study and source
SRP-4053Registry intervention
Linked study entry; product equivalence not established
View study and source
Vyondys 53Registry intervention
Linked study entry; product equivalence not established
View study and source
Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]Registry intervention
Linked study entry; product equivalence not established
View study and source

Success Metrics

Completion Rate

50%(2/4)

Active Trials

1(20%)

Results Posted

100%(2 trials)

Terminated

2(40%)

Phase Distribution

Phase 2

20%

Phase 3

40%

Phase 4

20%

Phase Distribution

Early Stage

Mid Stage

Late Stage

Phase Distribution4 total trials

Phase 2Efficacy & side effects

1(25.0%)

Phase 3Large-scale testing

2(50.0%)

Phase 4Post-market surveillance

1(25.0%)

Highest Phase Reached

Phase 4

Trial Status & Enrollment

Completion Rate

50.0%

2 of 4 finished

Non-Completion Rate

50.0%

2 ended early

Currently Active

trials recruiting

Total Trials

all time

Status Distribution

Active(1)

Completed(2)

Terminated(2)

Detailed Status

Terminated2

Completed2

Enrolling by invitation1

Development Timeline

Analytics

Development Status

Total Trials

Active

Success Rate

0.0%

Most Advanced

Phase 3

Trials by Phase

Phase 21 (25.0%)

Phase 32 (50.0%)

Phase 41 (25.0%)

Trials by Status

enrolling_by_invitation120%

terminated240%

completed240%

Recent Activity

1 active trials

Showing 5 of 5

terminatedphase_4

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

NCT04708314

completedphase_3

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT02500381

enrolling_by_invitation

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340

terminatedphase_3

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

NCT03532542

completedphase_2

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

NCT04179409

Clinical Trials (5)

Showing 5 of 5 trials

NCT04708314Phase 4

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

Terminated

NCT02500381Phase 3

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

Completed

NCT06606340

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

Enrolling By Invitation

NCT03532542Phase 3

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Terminated

NCT04179409Phase 2

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

Completed

All 5 trials loaded

Drug Details

Intervention Type: Drug
Molecule Type: Oligonucleotide
Total Trials: 5

External References

ChEMBL

CHEMBL4297762

Names & Codes

Trade Names

vyondys 53

ATC Classification

M09AX08

Research Codes

NG-12-0163SRP-4053