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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

125 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-05-08

Study Completion Date

2033-01-03

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Atypical Hemolytic Uremic Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Iptacopan 200 mg

Open label , single arm

Group Type EXPERIMENTAL

Iptcaopan 200 mg

Intervention Type DRUG

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

Interventions

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Iptcaopan 200 mg

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

Intervention Type DRUG

Other Intervention Names

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LNP023

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No