Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05935215 | Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS) | PHASE3 | RECRUITING | 50 | — | — | Feb 28, 2024 | Jul 19, 2029 | Apr 29, 2026 | 31 | China, France +6 |
| NCT04889430 | Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy | PHASE3 | COMPLETED | 34 | — | — | Jan 17, 2022 | Apr 11, 2026 | Jun 1, 2026 | 36 | United States, Brazil +6 |
Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.
The number/percentage of participants treated with iptacopan achieving complete thrombotic microangiopathy (TMA) response during 26 weeks of study treatment. Complete TMA Response is defined as (1) hematological normalization in platelet count (platelet count ≥150 x 10\^9/L) and LDH (below ULN), and (2) improvement in kidney function (≥ 25% serum creatinine reduction from baseline), maintained for two measurements obtained at least four weeks apart, and any measurement in between
Long term (one year) safety, tolerability and efficacy of iptacopan via 1) safety evaluations including adverse events/serious adverse events, safety laboratory parameters, vital signs etc. after 52 weeks of study treatment, and 2) efficacy evaluations including complete TMA response, hematological parameters (platelets, LDH, hemoglobin), eGFR, PROs after 52 weeks of study treatment
| Arm | Type | Description |
|---|---|---|
| iptacopan 200 mg b.i.d. | EXPERIMENTAL | open label arm of iptacopan 200 mg b.i.d. |
| Iptacopan 200 mg b.i.d | EXPERIMENTAL | Single arm open-label with 50 adult patients receiving 200mg oral twice daily doses of iptacopan |
| Name | Type | Description |
|---|---|---|
| Iptacopan | DRUG | Open Label |
Inclusion Criteria: * Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded. •. Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 ...