Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06379789 | A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B | PHASE1 | RECRUITING | 130 | — | — | Sep 11, 2024 | Aug 14, 2047 | May 19, 2026 | 42 | United States, Australia +6 |
Part 1, 2B, and 2C
Part 1, 2B, and 2C
Part 1
Part 2A, 2B, and 2C
Part 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort 1 Dose Escalation for RDE | EXPERIMENTAL | Starting dose to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 1: Cohort 2 Dose Escalation for RDE | EXPERIMENTAL | Dose 2 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 1: Cohort 3 Dose Escalation for RDE | EXPERIMENTAL | Dose 3 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 1: Cohort 4 Dose Escalation for RDE | EXPERIMENTAL | Dose 4 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 2: Dose Expansion A | EXPERIMENTAL | Participants ≥18 Years of Age will receive the RDE of REGV131-LNP1265 determined by Part 1 |
| Part 2: Dose Expansion B | EXPERIMENTAL | Participants ≥12 to \<18 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1 |
| Part 2: Dose Expansion C | EXPERIMENTAL | Participants ≥2 to \<12 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1 |
| Name | Type | Description |
|---|---|---|
| REGV131 | DRUG | Administered per the protocol before LNP1265 |
| LNP1265 | DRUG | Administered per the protocol following REGV131 |
Key Inclusion Criteria: 1. Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \<0.02 IU/mL) or documented genotype known to produce severe hemophilia B 2. Currently taking FIX prophylaxis and previous experience with FIX therapy, ...