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

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. The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS. To provide supporting evidence on the benefit/risk relationship of iptacopan in subjects with aHUS derived from the pivotal Phase III study CLNP023F12301 (switching from anti-C5 antibody therapy to iptacopan therapy).

CT.gov Identifier
NCT05935215
EudraCT Identifier
N/A
CTIS:
2023-504550-35-00
Sponsor
Novartis
Collaborator
N/A
Study Contact Information
+41 61 324 0000
Recruiting
Contact Us

Study Details

Diseases
Glomerulonephritis and atypical hemolytic uremic syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome; C3 Glomerulopathy; Complement Factor H Deficiency
Study Drug
Iptacopan
Genes
CFI
CFH
Study Dates
Feb 2024 - Jul 2028
Sex
Female & Male
Age
18 - 100 Years
Inclusion and Exclusion Criteria
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:
  • Hematological normalization in platelet count ≥150 x 10^9/L and LDH below upper limit of normal [ULN], andStable kidney function as defined by serum creatinine values within ±15% during the Screening periodVaccination 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^2Active 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 transplantationFemale patients who are pregnant or breastfeeding, or intending to conceive during the course of the studyAny medical condition deemed likely to interfere with the patient's participation in the study

Protocol 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.

The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.

To provide supporting evidence on the benefit/risk relationship of iptacopan in subjects with aHUS derived from the pivotal Phase III study CLNP023F12301 (switching from anti-C5 antibody therapy to iptacopan therapy).

Study Locations

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