A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS

This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS. The purpose of this open-label extension study is to evaluate the long-term safety, tolerability, and efficacy of iptacopan in subjects with aHUS who have completed the treatment period of the aHUS phase III trial and have benefited from iptacopan treatment

CT.gov Identifier
NCT05795140
EudraCT Identifier
N/A
CTIS:
2022-502965-34-00
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
May 2024 - Dec 2032
Sex
Female & Male
Age
18 - 100 Years
Inclusion and Exclusion Criteria
Inclusion Criteria:
  • 1. Signed informed consent must be obtained prior to participation in the open label
  • extension study
  • 2. Willing and able to comply with the study Schedule of Activities
  • 3. Participants who have completed the full study treatment period of any prior "Novartis
  • sponsored" iptacopan Phase 3 clinical trial in aHUS, are still on iptacopan study
  • treatment and derive benefit from it as per Investigator's judgement
  • 4. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and
  • Haemophilus influenzae infections should be up to date (i.e., any boosters required
  • should be administered according to local guidelines)
Exclusion Criteria:
  • 1. Concomitant treatment with any complement inhibitor as well as concomitant treatment
  • with any of the prohibited drugs
  • 2. Any comorbidity or medical condition (including but not limited to any active systemic
  • bacterial, viral or fungal infection or malignancy) that, in the opinion of the
  • Investigator could put the participant at risk
  • 3. Active infection or history of recurrent invasive infections caused by encapsulated
  • bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus
  • influenzae
  • 4. History of hypersensitivity to iptacopan or its excipients or to drugs of similar
  • chemical classes
  • 5. Pregnant or nursing (lactating) women
  • 6. Women of child-bearing potential, defined as all women physiologically capable of
  • becoming pregnant, unless they are using effective methods of contraception during
  • dosing of investigational drug and for 1 week after stopping of investigational drug.
  • Other protocol-defined inclusion/exclusion criteria may apply.

Protocol Summary

This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.

The purpose of this open-label extension study is to evaluate the long-term safety, tolerability, and efficacy of iptacopan in subjects with aHUS who have completed the treatment period of the aHUS phase III trial and have benefited from iptacopan treatment

Study Locations

To view all trial locations, click here

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