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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02841267 | A Trial of PF-06252616 in Ambulatory Participants With LGMD2I | PHASE1 | COMPLETED | 19 | — | — | Jul 1, 2016 | Jan 1, 2019 | Oct 19, 2020 | 1 | United States |
Adverse events include subject-reported symptoms as well as clinically-significant changes in laboratory testing, vital signs, and suicide screening (based on the Columbia Suicide Severity Rating Scale).
| Arm | Type | Description |
|---|---|---|
| Low Dose, Cohort 1 | ACTIVE_COMPARATOR | 4 subjects will be enrolled in cohort 1 and will receive an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, if no stopping rules have been met, subjects will be receive an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. |
| Middle dose, Cohort 2 | ACTIVE_COMPARATOR | 8 subjects will be enrolled in cohort 2 and receive 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. |
| High dose, Cohort 3 | ACTIVE_COMPARATOR | 8 subjects will be enrolled in cohort 3 and receive 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Name | Type | Description |
|---|---|---|
| PF 06252616 | DRUG | - |
Inclusion Criteria: 1. Male and female patients age ≥ 18 2. Diagnosis of LGMD2I as defined by clinical presentation consistent with LGMD2I and FKRP gene testing showing biallelic alterations known or likely to be pathogenic. Diagnosis must be confirmed in subject's medical history and by genetic te...