Clinical trial NCT05795140: Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Atypical Hemolytic Uremic Syndrome
Keywords: aHUS
iptacopan
atypical hemolytic uremic syndrome
thrombotic microangiopathy
TMA
dialysis
CKD
eGFR
PE/PI
Complement 3 Glomerulopathy (C3G)
IgAN
PNH
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2023-04-03
Enrollment: 125
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of participants with adverse events and serious adverse events
timeFrame: Throughout the study duration, up to 4 years
measure: Number of participants with abnormal safety laboratory parameters, vital signs and ECGs
timeFrame: Throughout the study duration, up to 4 years
Secondary outcomes:
measure: Number of participants with absence of aHUS relapse without the use of anti-C5 antibody
timeFrame: Throughout the study duration, up to 4 years
measure: Number of participants with complete TMA response status without the use of anti-C5 antibody therapy
timeFrame: Throughout the study duration, up to 4 years
measure: Estimated glomerular filtration rate (eGFR)
timeFrame: Throughout study duration, up to 4 years
measure: Chronic kidney disease (CKD) stage
timeFrame: Throughout study duration, up to 4 years
measure: Number of participants by dialysis requirement status
timeFrame: Throughout the study duration, up to 4 years
measure: Number of participants with Thrombotic Microangiopathy (TMA) related adverse events
timeFrame: Throughout study duration, up to 4 years
Other outcomes:
Locations:
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Beijing
country: China
facility: Novartis Investigative Site
city: Ostrava
country: Czechia
facility: Novartis Investigative Site
state: Poruba
city: Nagpur
country: India
facility: Novartis Investigative Site
state: Maharashtra
city: Pune
country: India
facility: Novartis Investigative Site
state: Maharashtra
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: