RecruitingPhase 3NCT05935215
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS
Sponsor
Novartis Pharmaceuticals
Enrollment
50 participants
Start Date
Feb 28, 2024
Study Type
INTERVENTIONAL
Conditions
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
Min Age: 18 YearsMax Age: 100 Years
Inclusion Criteria8
- 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:
- Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and
- 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 Criteria7
- 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
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Interventions
DRUGIptacopan
Open Label
Locations(31)
View Full Details on ClinicalTrials.gov
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NCT05935215
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