Success Metrics

Active Trials
2(67%)

Phase Distribution

Ph phase_1
1
33%
Ph phase_3
2
67%

Phase Distribution

1

Early Stage

0

Mid Stage

2

Late Stage

Phase Distribution3 total trials
Phase 1Safety & dosage
1(33.3%)
Phase 3Large-scale testing
2(66.7%)

Highest Phase Reached

Phase 3

Trial Status & Enrollment

Completion Rate

0.0%

0 of 0 finished

Non-Completion Rate

0.0%

0 ended early

Currently Active

2

trials recruiting

Total Trials

3

all time

Status Distribution
Active(3)

Detailed Status

Active, not recruiting2
Enrolling by invitation1

Development Timeline

Analytics

Development Status

Total Trials
3
Active
2
Success Rate
N/A
Most Advanced
Phase 3

Trials by Phase

Phase 11 (33.3%)
Phase 32 (66.7%)

Trials by Status

active_not_recruiting267%
enrolling_by_invitation133%

Recent Activity

2 active trials
Showing 3 of 3
enrolling_by_invitationphase_3

A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

NCT06431893
active_not_recruitingphase_3

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

NCT06247085
active_not_recruitingphase_1

Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

NCT03406611

Clinical Trials (3)

Showing 3 of 3 trials
NCT06431893Phase 3

A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

Enrolling By Invitation
NCT06247085Phase 3

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

Active Not Recruiting
NCT03406611Phase 1

Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

Active Not Recruiting

All 3 trials loaded

Drug Details

Intervention Type
DRUG
Total Trials
3