Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT05935215 | Recruiting Phase 3 FDA Drug

• 1 other identifier: CLNP023F12302
• Study Type: interventional
• Target: 50
• Locations: 8 countries
• Sites: 31

Brief Summary

The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.

Conditions

Atypical Hemolytic Uremic Syndrome

Keywords

Atypical Hemolytic Uremic Syndrome; aHUS,; thrombotic microangiopathy; TMA

Outcome Measures

Primary Outcomes (1)

• Percentage of participants free of TMA manifestation

  Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.

  12 months

Secondary Outcomes (12)

• Percentage of participants free of TMA manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies

  12 months, 24 months

• Percentage of participants free of TMA manifestation

  24 months

• Time to TMA manifestation

  12 months, 24 months

• Change from baseline in platelets

  Baseline, month 12, month 24

• Change from baseline in LDH

  Baseline, month 12, month 24

Study Arms (1)

iptacopan 200 mg b.i.d.

EXPERIMENTAL

open label arm of iptacopan 200 mg b.i.d.

Drug: Iptacopan

Interventions

Iptacopan DRUG

Open Label

Also known as: LNP023

iptacopan 200 mg b.i.d.

Eligibility Criteria

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

You may qualify if:

• Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded.
• . Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period.
• In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period.
• Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:
• Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and
• Stable kidney function as defined by serum creatinine values within ±15% during the Screening period
• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.
• If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.

You may not qualify if:

• History of aHUS disease relapse while on anti-C5 antibody treatment.
• eGFR \< 30 ml/min/1.73m\^2
• Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.
• Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.
• Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation
• Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
• Any medical condition deemed likely to interfere with the patient's participation in the study

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (31)

Novartis Investigative Site

Nanjing, Jiangsu, 210009, China

WITHDRAWN

Novartis Investigative Site

Beijing, 100034, China

ACTIVE NOT RECRUITING

Novartis Investigative Site

Beijing, 100730, China

ACTIVE NOT RECRUITING

Novartis Investigative Site

Shanghai, 200025, China

WITHDRAWN

Novartis Investigative Site

Bordeaux, 33076, France

RECRUITING

Novartis Investigative Site

Paris, 75015, France

RECRUITING

Novartis Investigative Site

Paris, 75970, France

RECRUITING

Novartis Investigative Site

Rouen, 76031, France

RECRUITING

Novartis Investigative Site

Toulouse, 31054, France

RECRUITING

Novartis Investigative Site

Tours, 37044, France

RECRUITING

Novartis Investigative Site

Essen, 45147, Germany

RECRUITING

Novartis Investigative Site

Kiel, 24105, Germany

RECRUITING

Novartis Investigative Site

Ranica, BG, 24020, Italy

RECRUITING

Novartis Investigative Site

Milan, MI, 20122, Italy

RECRUITING

Novartis Investigative Site

Roma, RM, 00168, Italy

RECRUITING

Novartis Investigative Site

Matsumoto-shi, Nagano, 3908510, Japan

RECRUITING

Novartis Investigative Site

Iruma-gun, Saitama, 3500495, Japan

RECRUITING

Novartis Investigative Site

Bunkyo Ku, Tokyo, 113-8655, Japan

RECRUITING

Novartis Investigative Site

Santiago Compostela, A Coruna, 15706, Spain

RECRUITING

Novartis Investigative Site

Barcelona, 08036, Spain

RECRUITING

Novartis Investigative Site

Córdoba, 14004, Spain

RECRUITING

Novartis Investigative Site

Málaga, 29010, Spain

RECRUITING

Novartis Investigative Site

Seville, 41013, Spain

RECRUITING

Novartis Investigative Site

Valencia, 46026, Spain

RECRUITING

Novartis Investigative Site

Izmir, Balcova, 35340, Turkey (Türkiye)

WITHDRAWN

Novartis Investigative Site

Köseköy, Kocaeli, 41380, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Ankara, Yenimahalle, 06500, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Mersin, Yenisehir, 33110, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Glasgow, Scotland, G51 4TF, United Kingdom

RECRUITING

Novartis Investigative Site

Newcastle upon Tyne, Tyne and Wear, NE7 7DN, United Kingdom

RECRUITING

Novartis Investigative Site

London, NW1 2BU, United Kingdom

RECRUITING

MeSH Terms

Conditions

Atypical Hemolytic Uremic Syndrome; Thrombotic Microangiopathies

Interventions

iptacopan

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

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

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

Study Record Dates

• First Submitted: June 29, 2023
• First Posted: July 7, 2023
• Study Start: February 28, 2024
• Primary Completion (Estimated): July 21, 2028
• Study Completion (Estimated): July 19, 2029
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

JP (3); GB (3); IT (3); TU (4); CN (4); ES (6); FR (6); DE (2)