Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Alogabat · 1 trial · 1 indication
Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with autism spectrum disorder (ASD) (AUC)
Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with ASD (CL/F)
| Arm | Type | Description |
|---|---|---|
| Part 1 Adult Alogabat Dose (Age 15-17) | EXPERIMENTAL | In Part 1 of the study participants will receive alogabat once a day (QD). |
| Part 1 Age-adjusted Dose (Age 10-14) | EXPERIMENTAL | In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat. |
| Part 1 Age-adjusted Dose (Age 5-9) | EXPERIMENTAL | In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat. |
| Part 2 Cohort 1 | EXPERIMENTAL | In Part 2 of the study, the dosing will depend upon the results of Part 1 with two different dose levels per cohort. Doses can be age-adjusted. |
| Part 2 Cohort 2 | EXPERIMENTAL | In Part 2 of the study, the dosing will depend upon the interim results with two different dose levels per cohort. Doses can be age-adjusted. |
| Part 1 Optional Cohort | EXPERIMENTAL | If dose adjustments (e.g., increase or decrease in dose) are required, particularly due to uncertainty of the clearance estimates (e.g., due to high variability) or over-/underprediction of the pediatric clearance versus adult clearance, additional participants may be recruited in any of the of the 3 age-groups in order to confirm the exposure equivalence. A total of two optional cohorts may be utilized in this study, allocated to Part 1 and/or Part 2. |
| Part 2 Optional Cohort | EXPERIMENTAL | In Part 2 of the study, the dosing will depend upon the interim results with two different dose levels per cohort. Doses can be age-adjusted. |
| Name | Type | Description |
|---|---|---|
| Alogabat | DRUG | Alogabat will be administered QD with dose depending on cohort and age of the participant. |
Inclusion Criteria: * Clinical diagnosis of AS and a genetic subtype of deletion on chromosome 15q11q13 confirmed by a historical molecular diagnosis * The participant's general health status, in the context of the disease under study, allows them to participate in a clinical trial in the opinion o...
Alogabat is an investigational small molecule being studied for the treatment of Angelman Syndrome, a rare genetic disorder. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities. The drug is being evaluated in children and adolescents aged 5 to 17 years with Angelman Syndrome who have the deletion genotype.
Alogabat is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded over-the-counter under the ticker symbol RHHBY. The company is conducting clinical trials to evaluate the safety and efficacy of Alogabat in patients with Angelman Syndrome.
Alogabat is in Phase 2 clinical development. It is an investigational drug and has not received approval from the U.S. Food and Drug Administration or any other regulatory agency. The drug is being studied in a completed Phase 2 trial that enrolled 48 participants with Angelman Syndrome.
Alogabat has one completed Phase 2 clinical trial registered under NCT05630066. The study investigated the pharmacokinetics, safety, and proof of mechanism of Alogabat in children and adolescents aged 5-17 years with Angelman Syndrome and the deletion genotype. The trial was conducted in the United States, Australia, France, Germany, Italy, and Spain.
Alogabat is a small molecule therapeutic being developed for Angelman Syndrome. The specific molecular target of Alogabat has not been disclosed in available information, so its precise mechanism of action is not publicly detailed. The drug is being studied to understand its pharmacokinetics and provide proof of mechanism in the target patient population.