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Recruiting NCT05795140

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

Phase III Interventional Atypical Hemolytic Uremic Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Iptcaopan 200 mg.
Who it may be relevant to
Registry conditions: Atypical Hemolytic Uremic Syndrome. Basic parameters: 18 years — 100 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Brazil, China, Czechia, India, Japan +1
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug Iptcaopan 200 mg
    Open label, participant specific kits, hard gelatin capsules to be taken twice a day

Primary outcome measures

  • Number of participants with adverse events and serious adverse events [Time frame: Throughout the study duration, up to 4 years]
Secondary outcome measures (6)
  • Number of participants with absence of TMA manifestation without the use of anti-C5 antibody [Time frame: Throughout the study duration, up to 4 years]
  • Number of participants with complete TMA response status without the use of anti-C5 antibody therapy [Time frame: Throughout the study duration, up to 4 years]
  • Estimated glomerular filtration rate (eGFR) [Time frame: Throughout study duration, up to 4 years]
  • Chronic kidney disease (CKD) stage [Time frame: Throughout study duration, up to 4 years]
  • Number of participants by dialysis requirement status [Time frame: Throughout the study duration, up to 4 years]
  • Number of participants with Thrombotic Microangiopathy (TMA) related adverse events [Time frame: Throughout study duration, up to 4 years]

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 (e.g. CLNP023F12301 , CLNP023F12302), 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Brazil · 2 centers
  • Novartis Investigative Site — São Paulo
  • Novartis Investigative Site — Rio de Janeiro
India · 2 centers
  • Novartis Investigative Site — Nagpur
  • Novartis Investigative Site — Pune
Turkey (Türkiye) · 2 centers
  • Novartis Investigative Site — Ankara
  • Novartis Investigative Site — Mersin
China · 1 center
  • Novartis Investigative Site — Beijing
Czechia · 1 center
  • Novartis Investigative Site — Ostrava
Japan · 1 center
  • Novartis Investigative Site — Iruma-gun

Identifiers

NCT: NCT05795140 · CLNP023F12001B · 2022-502965-34-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗