Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RO7248824 · 2 trials · 1 indication
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal. OOE part: From baseline OOE visit to final OOE visit (Day 420) or early termination.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal. OOE part: From baseline OOE visit to final OOE visit (Day 420) or early termination.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal. OOE part: From baseline OOE visit to final OOE visit (Day 420) or early termination.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal. OOE part: From baseline OOE visit to final OOE visit (Day 420) or early termination.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal.
MAD part: From the baseline MAD visit to the final MAD visit (D365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (D1092) or early withdrawal.
MAD part: From the baseline MAD visit to the final MAD visit (Day 365) or early withdrawal. LTE part: From the baseline LTE visit to final LTE visit (Day 1092) or early withdrawal.
| Arm | Type | Description |
|---|---|---|
| RO7248824 | EXPERIMENTAL | In Part 1 of the study RO7248824 and \[89Zr\]-labeled RO7248824 will be administered as a single bolus IT injection following a standard IT Administration procedure. In Part 2 of the study, it is planned to test up to 3 additional IT procedures. This part is tentative with regard to its conduct and the number of procedures that may be tested. |
| Cohort A1 RO7248824 | EXPERIMENTAL | Participants 5-12 years |
| Cohort A2 RO7248824 | EXPERIMENTAL | Participants 5-12 years |
| Cohort A3 RO7248824 | EXPERIMENTAL | Participants 5-12 years |
| Cohort A4 RO7248824 | EXPERIMENTAL | Participants 5-12 years |
| Cohort A5 RO7248824 | EXPERIMENTAL | Participants 5-12 years |
| Cohort B1 RO7248824 | EXPERIMENTAL | Participants 1-4 years |
| Cohort B2 RO7248824 | EXPERIMENTAL | Participants 1-4 years |
| Cohort B3 RO7248824 | EXPERIMENTAL | Participants 1-4 years |
| Cohort B4 RO7248824 | EXPERIMENTAL | Participants 1-4 years |
| Cohort B5 RO7248824 | EXPERIMENTAL | Participants 1-4 years |
| LTE: Cohort EA1 RO7248824 | EXPERIMENTAL | New participants (age 5-12) enrolling directly in the LTE part |
| LTE: Cohort EA2 RO7248824 | EXPERIMENTAL | Participants continuing from multiple ascending dose (MAD) cohorts A1 and A2 |
| LTE: Cohort EA3 RO7248824 | EXPERIMENTAL | Participants continuing from MAD cohorts A3 and A4 |
| LTE: Cohort EA4 RO7248824 | EXPERIMENTAL | Participants continuing from MAD Cohort A5 |
| LTE: Cohort EB1 RO7248824 | EXPERIMENTAL | New participants (age 1-4) enrolling directly into the LTE |
| LTE: Cohort EB2 RO7248824 | EXPERIMENTAL | Participants continuing from MAD cohorts B1 and B2 |
| LTE: Cohort EB3 RO7248824 | EXPERIMENTAL | Participants continuing from MAD cohorts B3 and B4 |
| LTE: Cohort EB4 RO7248824 | EXPERIMENTAL | Participants continuing from MAD Cohort B5 |
| OOE: RO7248824 | EXPERIMENTAL | Participants in the LTE part of the study will be given the opportunity to participate in the OOE part. |
| Name | Type | Description |
|---|---|---|
| RO7248824 | DRUG | A single dose of 10 mg RO7248824 will be used for this PET study in healthy participants. |
Inclusion Criteria: Informed Consent 1. Able and willing to provide written informed consent and to comply with the study protocol according to ICH and local regulations Age 2. Aged from 25 to to 55 years at the time of dosing Type of Participants and Disease Characteristics 3. Overtly hea...
RO7248824 is an investigational small molecule being developed for Angelman Syndrome, a rare genetic disorder. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is being studied to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in participants with Angelman Syndrome.
RO7248824 is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded under the ticker symbol RHHBY on the OTC market. The company is conducting clinical trials to investigate the drug's potential as a treatment for Angelman Syndrome.
RO7248824 is in Phase 1 clinical development. Two Phase 1 trials have been completed, with a total enrollment of 95 participants. The drug is investigational and has not received FDA approval. It is being studied for the treatment of Angelman Syndrome, a rare neurodevelopmental disorder.
RO7248824 has been studied in two completed Phase 1 clinical trials. The first trial, NCT04428281, evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in 74 participants with Angelman Syndrome across the United States, Italy, Netherlands, and Spain. The second trial, NCT04863794, assessed the distribution of the drug in the central nervous system following single intrathecal doses in 21 healthy male participants in the Netherlands.
RO7248824 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Angelman Syndrome. The drug has completed two Phase 1 trials, but it has not yet progressed to later-stage trials or received regulatory approval. Its safety and efficacy have not been established.