Last Update: Jun 03, 2024
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
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLNP023F12001B
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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 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.

Atypical Hemolytic Uremic Syndrome
Phase3
Recruiting
150
May 08, 2024
Jan 03, 2033
All
18 Years - (Adult, Older Adult)

Interventions

Drug

Iptcaopan 200 mg

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

Eligibility 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.

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