RecruitingPhase 3NCT05795140

Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS

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


Sponsor

Novartis Pharmaceuticals

Enrollment

125 participants

Start Date

May 8, 2024

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 18 YearsMax Age: 100 Years

Inclusion Criteria4

  • Signed informed consent must be obtained prior to participation in the open label extension study
  • Willing and able to comply with the study Schedule of Activities
  • 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
  • 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 Criteria6

  • Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
  • 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
  • Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
  • History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
  • Pregnant or nursing (lactating) women
  • 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.

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Interventions

DRUGIptcaopan 200 mg

Open label, participant specific kits, hard gelatin capsules to be taken twice a day


Locations(6)

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Novartis Investigative Site

Rio de Janeiro, Brazil

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Ostrava, Poruba, Czechia

Novartis Investigative Site

Nagpur, Maharashtra, India

Novartis Investigative Site

Pune, Maharashtra, India

View Full Details on ClinicalTrials.gov

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NCT05795140


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