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

NCT05795140 | Recruiting Phase 3 FDA Drug

• 1 other identifier: CLNP023F12001B
• Study Type: interventional
• Target: 125
• Locations: 4 countries
• Sites: 6

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.

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

Outcome Measures

Primary Outcomes (2)

• Number of participants with adverse events and serious adverse events

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

  Throughout the study duration, up to 4 years

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

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

  Throughout the study duration, up to 4 years

Secondary Outcomes (6)

• 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

Study Arms (1)

Iptacopan 200 mg

EXPERIMENTAL

Open label , single arm

Drug: Iptcaopan 200 mg

Interventions

Iptcaopan 200 mg DRUG

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

Also known as: LNP023

Iptacopan 200 mg

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (6)

Novartis Investigative Site

São Paulo, São Paulo, 05403 000, Brazil

RECRUITING

Novartis Investigative Site

Rio de Janeiro, 22270-60, Brazil

RECRUITING

Novartis Investigative Site

Beijing, 100034, China

RECRUITING

Novartis Investigative Site

Ostrava, Poruba, 708 52, Czechia

RECRUITING

Novartis Investigative Site

Nagpur, Maharashtra, 440015, India

RECRUITING

Novartis Investigative Site

Pune, Maharashtra, 411011, India

RECRUITING

MeSH Terms

Conditions

Atypical Hemolytic Uremic Syndrome; Thrombotic Microangiopathies; Glomerulonephritis, Membranoproliferative

Condition Hierarchy (Ancestors)

Hemolytic-Uremic Syndrome; Uremia; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Glomerulonephritis; Nephritis; Immune System Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 21, 2023
• First Posted: April 3, 2023
• Study Start: May 8, 2024
• Primary Completion (Estimated): December 23, 2032
• Study Completion (Estimated): January 3, 2033
• Last Updated: July 16, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

CZ (1); IN (2); BR (2); CN (1)