Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Trofinetide · 2 trials · 1 indication
Percentage of subjects with treatment-emergent adverse events (TEAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects with withdrawals due to AEs
Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval \>500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of \>60 ms
Potentially clinically important changes in vital signs were defined in the study protocol as: systolic blood pressure (SBP) ≥180 mmHg and increased ≥20 mmHg from baseline; SBP ≤90 mmHg and decreased ≥20 mmHg from baseline; diastolic blood pressure (DBP) ≥ 105 mmHg and increased ≥15 mmHg from baseline; DBP ≤50 mmHg and decreased ≥15 mmHg from baseline; Pulse ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline
Potentially clinically important changes in body weight were defined in the study protocol as: Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline
Potentially clinically important changes in laboratory parameters were defined in the study protocol as: Sodium ≤125 mmol/L; Sodium ≥155 mmol/L; Potassium ≤3.0 mmol/L; Potassium ≥5.5 mmol/L; Chloride ≤85 mmol/L; Chloride ≥120 mmol/L; Calcium \<2.0 mmol/L; Calcium \>2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine \>1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x ULN; Glucose ≤2.48 mmol/L; Glucose ≥11 mmol/L; Albumin ≤26 g/L; Albumin ≥60 g/L; Protein ≤50 g/L; Protein ≥100 g/L; Alanine aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN
The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behaviour.
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.
| Arm | Type | Description |
|---|---|---|
| Trofinetide | EXPERIMENTAL | - |
| Drug - Trofinetide | EXPERIMENTAL | Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Placebo | PLACEBO_COMPARATOR | Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Name | Type | Description |
|---|---|---|
| Trofinetide | DRUG | Trofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Placebo | OTHER | Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks |
Inclusion Criteria: 1. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003 2. Met all entry criteria for the antecedent study 3. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator 4. Can still swallow the stu...
Trofinetide is an investigational small molecule being developed for the treatment of Rett Syndrome, a rare neurological disorder that primarily affects girls and women. It is currently in Phase 3 clinical development and has not been approved by the FDA.
Trofinetide is being developed by ACADIA Pharmaceuticals Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ACAD. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Rett Syndrome.
Trofinetide is in Phase 3 clinical development. Two Phase 3 trials have been completed, including a placebo-controlled study and an open-label extension study. The drug remains investigational and is not yet approved for commercial use.
Trofinetide has been studied in two completed Phase 3 trials. The first, NCT04181723, is a placebo-controlled study in 187 girls and women with Rett Syndrome. The second, NCT04279314, is an open-label extension study with 154 participants. Both trials were conducted in the United States.
Trofinetide is not FDA approved. It is an investigational drug currently in Phase 3 clinical development for Rett Syndrome. The completed trials include a placebo-controlled study and an open-label extension, but regulatory approval has not been granted.