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PF-06252616 · 2 trials · 2 indications
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. |
| 1 | PLACEBO_COMPARATOR | - |
| 2 | PLACEBO_COMPARATOR | - |
| 3 | PLACEBO_COMPARATOR | - |
| 4 | PLACEBO_COMPARATOR | - |
| 5 | PLACEBO_COMPARATOR | Repeat dose of PF-06252616, IV infusion, single dose - 10.0 miligram per kilogram |
| 6 | PLACEBO_COMPARATOR | - |
| 7 | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| PF 06252616 | DRUG | - |
| PF-06252616 | BIOLOGICAL | 1.0 milligram per kilogram of PF-06252616, IV infusion, single dose |
| Placebo | DRUG | Placebo for PF-06252616, IV infusion, single dose |
| PF-06252161 | BIOLOGICAL | 10.0 milligram per kilogram of PF-06252616, IV infusion, single dose |
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...
PF-06252616 is an investigational small molecule developed by Pfizer, Inc. (ticker PFE). It has been studied in limb girdle muscular dystrophy type 2I, also known as LGMD2I, and in healthy adult volunteers. It remains in Phase 1 clinical development and is not an approved therapy.
PF-06252616 was studied as an investigational treatment for ambulatory adults with limb girdle muscular dystrophy type 2I, or LGMD2I. One Phase 1 trial, NCT02841267, enrolled 19 ambulatory participants with LGMD2I in the United States. The program is in Phase 1 and no approved use exists.
Pfizer, Inc., which trades under the ticker PFE, is the developer of PF-06252616. Pfizer sponsored the clinical studies of the compound, including the Phase 1 trial in ambulatory participants with LGMD2I and the Phase 1 study in healthy subjects.
PF-06252616 is in Phase 1 clinical development. Both of its recorded Phase 1 studies have completed: NCT02841267 in ambulatory participants with LGMD2I and NCT01616277 in healthy subjects. The compound is investigational and has not been approved by the FDA.
PF-06252616 has been evaluated in two completed Phase 1 trials. NCT02841267 studied ambulatory participants with LGMD2I and enrolled 19 people in the United States. NCT01616277 evaluated safety and tolerability in 86 healthy subjects, also in the United States. No trials are currently active.
Yes, PF-06252616 and PF 06252616 refer to the same investigational compound from Pfizer. The only difference is the hyphen in the code name, so search results and trial records using either spelling point to the same Phase 1 small molecule program.