To study if iptacopan is safe, tolerable and effective in patients with aHUS

Phase 3

• Conditions: Health Condition 1: D593- Hemolytic-uremic syndrome

• Registration Number: CTRI/2024/07/070874
• Lead Sponsor: ovartis Healthcare Pvt Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: ot Yet Recruiting
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

1. Approximately 100 male and female participants, =18 years of age, with a diagnosis of aHUS prior to screening.

2. Participants must have completed the full study treatment period of a Novartis sponsored iptacopan Phase 3 clinical trial in aHUS, are still on iptacopan study treatment and derive benefit from it as per Investigators judgement.

3. Already vaccinated against bacteria such as Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. If the patient has not been previously vaccinated, or if a booster is required, vaccine will be given if available and according to local regulations, at least 2 weeks prior to first dosing of iptacopan.

Exclusion Criteria

1. Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the drugs listed in Section 6.8.2

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified