Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as ADX-038 Dose Level 2, siRNA
ADX-038 · 4 trials · 9 indications
Evaluate the proportion of patients with treatment emergent adverse events (TEAEs) with ADX-038 and telitacicept versus those on ADX-038 alone.
Rate of change in the area of total EZ loss in the study eye from baseline to Month 12
Incidence and severity of TEAEs
To evaluate the safety and tolerability of ADX-038 in HVs by incidence, relationship, and severity of adverse events and serious adverse events
Evaluate the safety and tolerability of ADX-038 by incidence, relationship, and treatment-emergent adverse events and serious adverse events.
| Arm | Type | Description |
|---|---|---|
| Part A - ADX-038 Monotherapy | EXPERIMENTAL | - |
| Part B - ADX-038 Combination Therapy | EXPERIMENTAL | - |
| Active - ADX-038 | EXPERIMENTAL | - |
| Placebo - Saline | PLACEBO_COMPARATOR | - |
| ADX-038 Dose Level 1 - IgAN | EXPERIMENTAL | - |
| ADX-038 Dose Level 1 - C3G | EXPERIMENTAL | - |
| ADX-038 Dose Level 2 - IgAN | EXPERIMENTAL | - |
| Phase 1 - Active ADX-038 administered to HP | EXPERIMENTAL | For each cohort in Phase 1 (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-038): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule. |
| Phase 1- Placebo administered to HP | PLACEBO_COMPARATOR | For each cohort in Phase 1 (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-038): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule. |
| Phase 2a - ADX-038 administered to PNH participants | EXPERIMENTAL | This will be initiated at the dose level determined by the Safety Review Committee from SAD in HPs. The treatment of PNH participants is an open-label study. |
| Name | Type | Description |
|---|---|---|
| ADX-038 | DRUG | siRNA duplex oligonucleotide |
| Telitacicept | DRUG | fusion protein |
| Placebo | DRUG | Saline |
| ADX-038 Dose Level 2 | DRUG | siRNA duplex oligonucleotide |
| ADX-038 Dose Level 1 | DRUG | siRNA duplex oligonucleotide |
Inclusion Criteria: * Mean eGFR greater than or equal to 30 mL/min/1.73m2 * Clinical evidence of active kidney disease * Treated with supportive care including an ACE inhibitor or ARB if applicable * Willing to receive required vaccinations * Primary diagnosis of IgAN, C3G or IC-MPGN confirmed by a...