Evaluate long-term safety, tolerability and efficacy of iptacopan in study participants with aHUS

A multicenter, 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 - CLNP023F12001B

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2031250834

Enrollment

Registered

2026-03-24

Start date

2026-05-18

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Atypical Hemolytic Uremic Syndrome

Interventions

Iptacopan 200 mg

Eligibility

Sex/Gender

All

Inclusion 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

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.

Design outcomes

Primary

Measure	Time frame
Safety evaluations including AE/SAEs, safety laboratory parameters, vital signs and ECGs through study duration	—

Countries

Brazil, China, Czech Republic, India, Japan

Contacts

Public ContactHideki Maruyama

Novartis Pharma. K.K.

rinshoshiken.toroku@novartis.com+81-120-003-293

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026