Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VP 20629 · 1 trial · 1 indication
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs), defined as all AEs that start during study drug treatment (and up to 7 days after the last dose of the study drug) and were not seen at baseline, or were seen at baseline but increased in frequency and/or severity during study drug treatment (and up to 7 days after the last dose of study drug).
An abnormal laboratory finding which required an action or intervention by the investigator, or a finding judged by the investigator to represent a change beyond the range of normal physiologic fluctuation were reported as an adverse event. Treatment-emergent were events between first dose of study drug and 7 days after the last dose that were absent before treatment or that worsened relative to pretreatment state. Number of participants with Grade 3 or higher treatment-emergent adverse events for laboratory abnormalities were reported as clinically relevant laboratory changes.
Vital sign assessments included systolic blood pressure, diastolic blood pressure, heart rate, and temperature. Vital signs abnormalities reported as TEAEs were reported.
ECG included PR interval, QRS interval, QTcB interval, QTcF interval were considered as clinically significant ECG abnormalities.
| Arm | Type | Description |
|---|---|---|
| Single dose of VP 20629 or placebo | EXPERIMENTAL | Four groups of 8 subjects each will receive a single dose of VP 20629 (150 mg, 450 mg, 900 mg, or 1200 mg) or placebo. |
| Multiple doses of VP 20629 or placebo | EXPERIMENTAL | Three groups of 8 subjects each will receive multiple doses of VP 20629 (300 mg, 600 mg, or 900 mg total daily dose) or placebo. VP 20629 or placebo will be administered every 8 hours for 7 days with a single morning dose on Day 8. |
| Name | Type | Description |
|---|---|---|
| VP 20629 | DRUG | - |
| Placebo | DRUG | - |
Inclusion Criteria: 1. Be 18 to 45 years of age (inclusive). 2. Have a body mass index between 18 and 27 kg/m\^2 (inclusive). 3. Have a clinical presentation consistent with FA. 4. Have a confirmed diagnosis of FA with a defined expanded guanosine, adenine, adenine (GAA) triplet repeat number. 5. H...
VP 20629 is an investigational small molecule being developed for the treatment of Friedreich's Ataxia, a rare inherited disease that causes progressive damage to the nervous system. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
VP 20629 is being developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company. Takeda's stock is listed on the New York Stock Exchange under the ticker symbol TAK.
VP 20629 is in Phase 1 clinical development. A Phase 1 trial has been completed, and the drug remains investigational. It has not been approved by the FDA or any other regulatory agency.
VP 20629 has one completed clinical trial, NCT01898884, titled 'Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia.' This Phase 1 study enrolled 46 participants in the United States and was randomized, double-blind, and controlled.
VP 20629 is the primary name used for this investigational drug in clinical trials. No alternative names have been reported in the available clinical trial information.