E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10021263 |
E.1.2 | Term | IgA nephropathy |
E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective is to evaluate the long-term safety and tolerability of iptacopan in eligible participants |
|
E.2.2 | Secondary objectives of the trial |
The secondary objective is to characterize the clinical benefit (efficacy) of iptacopan in eligible participants receiving open-label iptacopan. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Signed informed consent must be obtained prior to participation in the REP; participants should be able to communicate well with the Investigator, understand and comply with the requirements of the study. - For LNP023X2203, patients must have completed part 1 or part 2 of the trial. For other parent trials, patients must have completed the entire parent trial duration defined by the respective protocol - eGFR* ≥ 20 ml/min/1.73m2 *eGFR calculated using the CKD-EPI formula (or modified MDRD formula according to specific ethnic groups and local practice guidelines) -Per investigator’s clinical judgement, the patient may benefit from receiving the open-label treatment of iptacopan 200 mg b.i.d. -Prior Vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e. any boosters required administered according to local regulations. -All patients must be on supportive care regimen of ACEi or ARB* as per KDIGO guidelines (KDIGO 2021). * Patients with allergies or intolerance to ACEi and ARB are eligible for the study but the Investigator should clearly document the reasons for not being on maximal ACEi/ARB dose in the source documents. |
|
E.4 | Principal exclusion criteria |
- Participants who are screen or baseline failed in any of the iptacopan parent studies in IgAN or who prematurely withdrew from iptacopan parent studies for any reason. - Evidence of severe urinary obstruction or difficulty in voiding; any urinary tract disorder other than IgAN at screening and before dosing with iptacopan. - Current (within 4 weeks prior to study treatment administration in the REP) acute kidney injury (AKI) defined by AKIN criteria. - Presence of Rapidly Progressive Glomerulonephritis (RPGN) as defined by 50% decline in eGFR within the last 3 months. - Patients treated with immunosuppressive or other immunmodulatory agents such as but not limited to cyclophosphamide, rituximab, infliximab, eculizumab, canakinumab, mycophenolate mofetil (MMF) or mycophenolate sodium (MPS), cyclosporine, tacrolimus, sirolimus, everolimus and/or systemic corticosteroids exposure (>7.5 mg/d prednisone/prednisolone equivalent) 90 days prior to first study drug administration. Rituximab requires 180 days wash out. Participants treated with endothelin (receptor) antagonists within 90 days prior to first study drug administration. - Use of other investigational drugs at the time of enrolment, or within 5 half-lives of enrolment or within 30 days whichever is longer. - History of recurrent invasive infections caused by encapsulated organisms, such as meningococcus, pneumococcus and H. influenzae. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability endpoints (including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs).
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Date of first administration of (Day 1) to 7 days after the date of the last actual administration of study treatment |
|
E.5.2 | Secondary end point(s) |
- Annualized total eGFR slope - Change from baseline in eGFR - Log transformed ratio to baseline in UPCR, UACR |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Screening visit, Months 1, 3, 6, 9, 12 and every 6 months |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 50 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Colombia |
Malaysia |
Singapore |
Hong Kong |
Taiwan |
Australia |
Brazil |
Canada |
China |
India |
Israel |
Japan |
Korea, Republic of |
Mexico |
Russian Federation |
Thailand |
United Kingdom |
United States |
Viet Nam |
Belgium |
Czechia |
Denmark |
Finland |
France |
Germany |
Hungary |
Italy |
Netherlands |
Norway |
Spain |
Sweden |
Türkiye |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 7 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 14 |
E.8.9.2 | In all countries concerned by the trial years | 8 |