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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03833128 | A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders | PHASE1 | COMPLETED | 24 | — | — | Apr 4, 2019 | Mar 21, 2022 | Dec 9, 2022 | 7 | United States, France +1 |
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
| Arm | Type | Description |
|---|---|---|
| Group 1 - Part A | EXPERIMENTAL | REN001 Low Dose oral once daily x 12 weeks |
| Group 2 - Part A | EXPERIMENTAL | REN001 High Dose oral once daily x 12 weeks |
| Group 3 - Part B | EXPERIMENTAL | REN001 High Dose oral once daily x 12 weeks |
| Name | Type | Description |
|---|---|---|
| Low Dose REN001 | DRUG | Oral |
| High Dose REN001 | DRUG | Oral |
Inclusion Criteria: Subjects must give written, signed and dated informed consent Confirmed diagnosis of FAOD A diagnostic acylcarnitine profile, in blood or cultured fibroblasts A stable treatment regimen for at least 30 days prior to enrollment Exclusion Criteria: Unstable or poorly controll...