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

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ClinicalTrials.gov Identifier: NCT05935215
Recruitment Status : Recruiting
First Posted : July 7, 2023
Last Update Posted : April 15, 2024
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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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.

Condition or disease Intervention/treatment Phase
Atypical Hemolytic Uremic Syndrome Drug: Iptacopan Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Detailed Description:

The study is designed as a multicenter, single-arm, open label study to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in participants with aHUS. It consists of a screening period of up to 8 weeks followed by a 12-Month Core Treatment period and 12-Month Extension Treatment period.

The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS
Actual Study Start Date : February 28, 2024
Estimated Primary Completion Date : January 25, 2029
Estimated Study Completion Date : July 21, 2029

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: iptacopan 200 mg b.i.d.
open label arm of iptacopan 200 mg b.i.d.
 Drug: Iptacopan
Open Label
Other Name: LNP023


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of participants free of TMA manifestation [ Time Frame: 12 months ]
    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.


Secondary Outcome Measures :
  1. Percentage of participants free of TMA manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies [ Time Frame: 12 months, 24 months ]
    Absence of thrombotic microangiopathy (TMA) manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies, without the use of anti-C5 antibody during iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.

  2. Percentage of participants free of TMA manifestation [ Time Frame: 24 months ]
    Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 24 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.

  3. Time to TMA manifestation [ Time Frame: 12 months, 24 months ]
    Time to thrombotic microangiopathy (TMA) manifestation

  4. Change from baseline in platelets [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in platelets at month 12 and month 24.

  5. Change from baseline in LDH [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in lactate dehydrogenase (LDH) at month 12 and month 24.

  6. Change from baseline in hemoglobin [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in hemoglobin at month 12 and month 24.

  7. Change from baseline in serum creatinine [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in serum creatinine at month 12 and month 24.

  8. Change from baseline in UPCR [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in urine protein to creatinine ratio (UPCR) at month 12 and month 24.

  9. Change from baseline in eGFR [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in estimated glomerular filtration rate (eGFR) at month 12 and month 24.

  10. Change from baseline in CKD stage [ Time Frame: Baseline, month 12, month 24 ]
    Change from baseline in chronic kidney disease (CKD) stage at month 12 and month 24.

  11. Number of participants who require dialysis [ Time Frame: month 12 and month 24 ]
    Dialysis requirement status (Yes/ No)

  12. Percentage of participants with TMA related events. [ Time Frame: month 12 and month 24 ]
    Percentage of participants with thrombotic microangiopathy (TMA) related events.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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 weight-based dosage regimen of anti-C5 antibody treatment for at least 3 months prior to the screening visit.

  • Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) for at least 3 months prior to entering the screening period as defined by:

    1. Hematological normalization in platelet count ≥150 x 109/L and LDH below upper limit of normal [ULN], and
    2. Stable or improving kidney function as defined by ≤15% increase in serum creatinine.
  • 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.

Exclusion Criteria:

  • 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05935215


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
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Japan
Novartis Investigative Site Recruiting
Iruma-gun, Saitama, Japan, 350-0495
Turkey
Novartis Investigative Site Recruiting
Ankara, Turkey, 06500
Novartis Investigative Site Recruiting
Mersin, Turkey, 33079
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05935215    
Other Study ID Numbers: CLNP023F12302
First Posted: July 7, 2023    Key Record Dates
Last Update Posted: April 15, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Atypical Hemolytic Uremic Syndrome
aHUS, thrombotic microangiopathy
TMA
Additional relevant MeSH terms:
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Azotemia
Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Syndrome
Hemolysis
Disease
Pathologic Processes
Uremia
Kidney Diseases
Urologic Diseases
Female Urogenital Diseases
 Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Cytopenia