The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05230537
Recruitment Status : Recruiting
First Posted : February 9, 2022
Last Update Posted : April 19, 2024
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The purpose of this study is to assess the effect of Iptacopan to prevent conversion of early or intermediate age-related macular degeneration (AMD) eyes to new incomplete retinal pigment epithelium and outer retinal atrophy (iRORA) or late AMD.

Condition or disease Intervention/treatment Phase
Age-Related Macular Degeneration Drug: Iptacopan (LNP023) Drug: Placebo Phase 2

Detailed Description:

This is a multicenter, randomized, participant and investigator masked, placebo controlled, proof-of-concept study to assess the safety and efficacy of Iptacopan (LNP023) in participants with early to intermediate age-related macular degeneration in one eye and neovascular age-related macular degeneration in the other eye. All enrolled participants must have early/intermediate AMD in one eye, with at least one high risk optical coherence tomography (OCT) feature (study eye) and neovascular AMD in the other eye (fellow eye).

Participants who meet all of the eligibility criteria will be randomized at the Baseline/Day 1 visit in a 1:1 ratio into one of two treatment arms:

  • Iptacopan (LNP023) oral capsules
  • Placebo oral capsules Approximately 146 participants (73 per arm) will be treated worldwide.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 146 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is a multicenter, randomized, participant and investigator masked, placebo controlled, proof-of-concept study to assess the safety and efficacy of LNP023 in participants with early to intermediate age-related macular degeneration (e/iAMD) in one eye and neovascular age-related macular degeneration (nAMD) in the other eye.
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description: Investigator and Participant
Primary Purpose: Treatment
Official Title: A Randomized, Participant and Investigator Masked, Placebo-controlled, Multicenter, Proof-of-concept Study to Assess the Safety and Efficacy of LNP023 (Iptacopan) in Patients With Early and Intermediate Age-related Macular Degeneration
Actual Study Start Date : February 17, 2022
Estimated Primary Completion Date : November 25, 2026
Estimated Study Completion Date : January 8, 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: Iptacopan (LNP023)
Iptacopan (LNP023) oral use capsules
 Drug: Iptacopan (LNP023)
oral capsules
Placebo Comparator: Placebo
Placebo matched to study drug, oral use capsules
 Drug: Placebo
oral capsules


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Development of new incomplete retinal pigment epithelium & outer retinal atrophy or late age-related macular degeneration (AMD) in the early/intermediate AMD eye as determined by optical coherence tomography (OCT) & supported by multimodal imaging [ Time Frame: Baseline/Day 1 through Month 24 ]
    OCT and other imaging will be performed using spectral domain OCT or swept source OCT machines


Secondary Outcome Measures :
  1. The incidence of ocular and non-ocular adverse events (AEs) [ Time Frame: Baseline/Day 1 through Month 24 ]
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.

  2. Change in Early Treatment Diabetic Retinopathy Study (ETDRS) (Standard Luminance) best corrected visual acuity (BCVA) scores in the early/intermediate AMD eye [ Time Frame: Baseline/Day 1 through Month 24 ]
    Best corrected visual acuity (BCVA) will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart. The number of letters read correctly, for each eye, will be recorded. Participants at sites in some countries may conduct BCVA testing using numerical charts rather than letter charts.

  3. Change in ETDRS low luminance visual acuity (LLVA) scores in the early/intermediate AMD eye [ Time Frame: Baseline/Day 1 through Month 24 ]
    ETDRS low luminance visual acuity (LLVA) scores will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart. The number of letters read correctly, for each eye, will be recorded. Participants at sites in some countries may conduct BCVA testing using numerical charts rather than letter charts.

  4. Change in contrast sensitivity (CS) scores in the early/intermediate AMD eye [ Time Frame: Baseline/Day 1 through Month 24 ]
    Pelli-Robson contrast sensitivity measurements will be performed using a Pelli-Robson contrast sensitivity wall chart and recording the number of correct letters read.

  5. Change in Standard luminance manifold contrast vision meter contrast sensitivity (MCVM-CS) scores in the early/intermediate AMD eye [ Time Frame: Baseline/Day 1 through Month 24 ]
    MCVM contrast sensitivity measurements will be performed using an automated device that uses an adaptive algorithm to rapidly measure a contrast sensitivity function in 5-10 minutes.

  6. Pharmacokinetics - concentrations of LNP023 related to trough samples [ Time Frame: Baseline/Day 1 through Month 24 ]
    Bioanalytical determination of plasma LNP023 concentrations related to trough samples using a LC-MS (mass spectrometry) method.

  7. Change in low luminance manifold contrast vision meter (MCVM) contrast sensitivity scores in the early/intermediate AMD eye [ Time Frame: Baseline/Day 1 through Month 24 ]
    MCVM contrast sensitivity measurements will be performed using an automated device that uses an adaptive algorithm to rapidly measure a contrast sensitivity function in 5-10 minutes.


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   50 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female participants ≥ 50 years of age
  • Diagnosis of early or intermediate age-related macular degeneration (AMD) in the study eye as determined by the investigator on fundus examination
  • Study eye (early/intermediate AMD eye) must have at least one high risk optical coherence tomography (OCT) feature (as defined by a central reading center).
  • Diagnosis of neovascular AMD (nAMD) in the fellow eye as determined by the investigator.
  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection are required prior to the start of the treatment with LNP023.
  • If not received previously, vaccination against Haemophilius influenzae infection should be given, if available and according to local regulations.

Exclusion Criteria:

  • History or current diagnosis of ECG abnormalities indicating significant safety risk, such as clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia and clinically significant second or third degree atrioventricular block (AV block) without a pacemaker.
  • History of familial long QT syndrome or known family history of Torsades de Pointes
  • History of stroke or myocardial infarction during the 6-month period prior to Baseline/Day 1, any current clinically significant arrhythmias, or any advanced cardiac or severe pulmonary hypertension
  • History of end stage kidney disease requiring dialysis or renal transplant
  • History of malignancy of any organ system
  • History of solid organ or bone marrow transplantation
  • History of recurrent meningitis or history of meningococcal infections despite vaccination
  • History of immunodeficiency diseases, including a positive Human Immunodeficiency Virus test result at Screening
  • Active Hepatitis B (HBV) or Hepatitis C (HCV) infection
  • History of hypersensitivity to any of the study treatments or excipients or to drugs of similar chemical classes or clinically relevant sensitivity to fluorescein dye as assessed by the Investigator.
  • Evidence of cRORA or exMNV in the study eye based on multimodal imaging as determined by the central reading center.
  • Participants who have current active TB as evidenced by clinical, radiographic and laboratory tests.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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): NCT05230537


Contacts
Layout table for location contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
Show Show 30 study locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05230537    
Other Study ID Numbers: CLNP023E12201
2021-001797-31 ( EudraCT Number )
First Posted: February 9, 2022    Key Record Dates
Last Update Posted: April 19, 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.
URL: https://clinicalstudydatarequest.com/

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Age-related macular degeneration
Macular degeneration
vision loss, macula damage
retina damage
dry macular degeneration
 wet macular degeneration
AMD
Best Corrected Visual Acuity
Neovascular AMD
OCT
Additional relevant MeSH terms:
Layout table for MeSH terms
Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases