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

Novartis Pharmaceuticals1 site in 1 country125 target enrollmentMay 8, 2024

ConditionsAtypical Hemolytic Uremic Syndrome

InterventionsIptcaopan 200 mg

DrugsIptcaopan 200 mg

Overview

Phase: Phase 3
Intervention: Iptcaopan 200 mg
Conditions: Atypical Hemolytic Uremic Syndrome
Sponsor: Novartis Pharmaceuticals
Enrollment: 125
Locations: 1
Primary Endpoint: Number of participants with adverse events and serious adverse events
Status: Recruiting
Last Updated: 9 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Detailed 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.

Registry

clinicaltrials.gov

Start Date

May 8, 2024

End Date

January 3, 2033

Last Updated

9 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Novartis Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Signed informed consent must be obtained prior to participation in the open label extension study
•Willing and able to comply with the study Schedule of Activities
•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
•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

•Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
•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
•Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
•History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
•Pregnant or nursing (lactating) women
•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.

Arms & Interventions

Iptacopan 200 mg

Open label , single arm

Intervention: Iptcaopan 200 mg

Outcomes

Primary Outcomes

Number of participants with adverse events and serious adverse events

Time Frame: Throughout the study duration, up to 4 years

Number of participants with adverse events and serious adverse events will be provided

Number of participants with abnormal safety laboratory parameters, vital signs and ECGs

Time Frame: Throughout the study duration, up to 4 years

Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided

Secondary Outcomes

Number of participants with absence of aHUS relapse without the use of anti-C5 antibody(Throughout the study duration, up to 4 years)
Number of participants with complete TMA response status without the use of anti-C5 antibody therapy(Throughout the study duration, up to 4 years)
Estimated glomerular filtration rate (eGFR)(Throughout study duration, up to 4 years)
Chronic kidney disease (CKD) stage(Throughout study duration, up to 4 years)
Number of participants by dialysis requirement status(Throughout the study duration, up to 4 years)
Number of participants with Thrombotic Microangiopathy (TMA) related adverse events(Throughout study duration, up to 4 years)