Clinical Trials in Japan / NCT05935215
Recruiting
Phase 3
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT05935215 · tracked via the Priya Life Science Japan tracker
Condition(s) studied
Atypical Hemolytic Uremic Syndrome
Study summary
The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.
Eligibility
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 months prior to entering the screening period.
* In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period.
Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:
1. Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and
2. Stable kidney function as defined by serum creatinine values within ±15% during the Screening period
* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.
* If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.
Exclusion Criteria:
* History of aHUS disease relapse while on anti-C5 antibody treatment.
* eGFR \< 30 ml/min/1.73m\^2
* Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.
* Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.
* Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation
* Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
* Any medical condition deemed likely to interfere with the patient's participation in the study
Primary outcome measure(s)
- Percentage of participants free of TMA manifestation — 12 months
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.
Trial sites (31)
| Facility | City | Region | Status |
|---|---|---|---|
| Novartis Investigative Site | Nanjing | Jiangsu | Withdrawn |
| Novartis Investigative Site | Beijing | China | Active Not Recruiting |
| Novartis Investigative Site | Beijing | China | Active Not Recruiting |
| Novartis Investigative Site | Shanghai | China | Withdrawn |
| Novartis Investigative Site | Bordeaux | France | Recruiting |
| Novartis Investigative Site | Paris | France | Recruiting |
| Novartis Investigative Site | Paris | France | Recruiting |
| Novartis Investigative Site | Rouen | France | Recruiting |
| Novartis Investigative Site | Toulouse | France | Recruiting |
| Novartis Investigative Site | Tours | France | Recruiting |
| Novartis Investigative Site | Essen | Germany | Recruiting |
| Novartis Investigative Site | Kiel | Germany | Recruiting |
| Novartis Investigative Site | Ranica | BG | Recruiting |
| Novartis Investigative Site | Milan | MI | Recruiting |
| Novartis Investigative Site | Roma | RM | Recruiting |
| Novartis Investigative Site | Matsumoto-shi | Nagano | Recruiting |
| Novartis Investigative Site | Iruma-gun | Saitama | Recruiting |
| Novartis Investigative Site | Bunkyo Ku | Tokyo | Recruiting |
| Novartis Investigative Site | Santiago Compostela | A Coruna | Recruiting |
| Novartis Investigative Site | Barcelona | Spain | Recruiting |
| Novartis Investigative Site | Córdoba | Spain | Recruiting |
| Novartis Investigative Site | Málaga | Spain | Recruiting |
| Novartis Investigative Site | Seville | Spain | Recruiting |
| Novartis Investigative Site | Valencia | Spain | Recruiting |
| Novartis Investigative Site | Izmir | Balcova | Withdrawn |
| Novartis Investigative Site | Köseköy | Kocaeli | Recruiting |
| Novartis Investigative Site | Ankara | Yenimahalle | Recruiting |
| Novartis Investigative Site | Mersin | Yenisehir | Recruiting |
| Novartis Investigative Site | Glasgow | Scotland | Recruiting |
| Novartis Investigative Site | Newcastle upon Tyne | Tyne and Wear | Recruiting |
| Novartis Investigative Site | London | United Kingdom | Recruiting |
More Novartis Pharmaceuticals trials in Japan
Other trials for the same condition
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
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A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05935215 on ClinicalTrials.gov ↗ ← All trials in Japan