RecruitingPhase 3

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

Brazil, China, Czechia125 participantsStarted 2024-05-08

Plain-language 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.

Who can participate

Age range18 Years – 100 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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 criteria

✕. 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.

What they're measuring

Number of participants with adverse events and serious adverse events

Timeframe: Throughout the study duration, up to 4 years

Number of participants with abnormal safety laboratory parameters, vital signs and ECGs

Timeframe: Throughout the study duration, up to 4 years

Trial details

NCT IDNCT05795140

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2032-12-23

Contact for this trial

Novartis Pharmaceuticals

+41613241111 novartis.email@novartis.com

View on ClinicalTrials.gov More Atypical Hemolytic Uremic Syndrome trials