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

Novartis

Status and phase: Enrolling; Phase 3

Conditions: Atypical Hemolytic Uremic Syndrome

Treatments: Drug: Iptcaopan 200 mg

Study type: Interventional

Funder types: Industry

Identifiers

NCT05795140

CLNP023F12001B

Details and patient eligibility

About

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.

Full description

The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.

Enrollment: 125 estimated patients

Sex: All

Ages: 18 to 100 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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125 participants in 1 patient group

Iptacopan 200 mg

Experimental group

Description:

Open label , single arm

Treatment:

Drug: Iptcaopan 200 mg