Clinical trial • Phase III • Ophthalmology
ELAMIPRETIDE TRIHYDROCHLORIDE for Dry age-related macular degeneration (Dry AMD)
Phase III trial of ELAMIPRETIDE TRIHYDROCHLORIDE for Dry age-related macular degeneration (Dry AMD).
Overview
- Trial Therapeutic Area
- Ophthalmology
- Trial Disease
- Dry age-related macular degeneration (Dry AMD)
- Trial Stage
- Phase III
- Drug Modality
- Peptide/protein/enzyme
Key dates
- Initial CTIS Submission Date
- 05-06-2024
- First CTIS Authorization Date
- 20-09-2024
Trial design
Randomised, placebo: 'the placebo for this trial will be composed of excipients used to manufacture the investigational drug but without the active drug substance.' placebo arm matched to active; patients receive placebo for 96 weeks. dose not specified for placebo; schedule matched once daily.-controlled Phase III trial across 17 sites in Italy, Germany, Spain and others.
- Randomised
- Yes
- Comparator
- Placebo: 'The placebo for this trial will be composed of excipients used to manufacture the investigational drug but without the active drug substance.' Placebo arm matched to active; patients receive placebo for 96 weeks. Dose not specified for placebo; schedule matched once daily.
- Target Sample Size
- 265
- Trial Duration For Participant
- 728
Eligibility
Recruits 265 Vulnerable population not selected. Subjects must be able to provide informed consent (adults ≥55 years). Caregivers may assist with IMP administration and dosing diary entries; consent is provided by the participant (no assent procedures described)..
- Vulnerable Population
- Vulnerable population not selected. Subjects must be able to provide informed consent (adults ≥55 years). Caregivers may assist with IMP administration and dosing diary entries; consent is provided by the participant (no assent procedures described).
Inclusion criteria
- {"criterion_text":"- Adults ≥ 55 years of age with at least 1 eye with dry AMD with photoreceptor loss, as determined at the Screening Visit by the presence of extrafoveal GA, as determined by the Reading Center primarily by FAF."}
- {"criterion_text":"- Women of childbearing potential must agree to use 1 of the following methods of birth control"}
- {"criterion_text":"- Male subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception (e.g., abstinence, dual method of contraception)"}
- {"criterion_text":"- GA in the study eye at the Screening Visit may be multi-focal"}
- {"criterion_text":"- Able to administer IMP"}
- {"criterion_text":"- Able to provide informed consent and willing to comply with all site visits, examinations, daily IMP administrations and dosing diary entries, and other conditions of the trial protocol"}
Exclusion criteria
- {"criterion_text":"- Concurrent disease in the study eye during the trial"}
- {"criterion_text":"- Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial or might confound trial results"}
- {"criterion_text":"- Participation in other investigational drug or device clinical trials"}
- {"criterion_text":"- Inability to comply with trial or follow-up procedures"}
- {"criterion_text":"- Prior participation in any elamipretide trial"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Rate of change in the macular area of photoreceptor loss (defined as an Ellipsoid Zone – Retinal Pigment Epithelium (EZ-RPE) thickness of 0μm) assessed by spectral domain-optical coherence tomography (SD-OCT) and Ellipsoid Zone (EZ) mapping at Week 48","definition_or_measurement_approach":"Defined as EZ-RPE thickness of 0 μm; assessed by spectral domain-optical coherence tomography (SD-OCT) and Ellipsoid Zone (EZ) mapping at Week 48."}
Secondary endpoints
- {"endpoint_text":"- Efficacy: Rate of change in the macular area of photoreceptor loss (defined as an EZ-RPE thickness of 0μm) assessed by SD-OCT and EZ mapping at Week 72","definition_or_measurement_approach":"Defined as EZ-RPE thickness of 0 μm; assessed by SD-OCT and EZ mapping at Week 72."}
- {"endpoint_text":"- Efficacy: Rate of change in the macular area of photoreceptor loss (defined as an EZ-RPE thickness of 0μm) assessed by SD-OCT and EZ mapping at Week 96","definition_or_measurement_approach":"Defined as EZ-RPE thickness of 0 μm; assessed by SD-OCT and EZ mapping at Week 96."}
- {"endpoint_text":"- Efficacy: Proportion of subjects gaining ≥ 10 letters (2 lines) in Low Luminance Best-Corrected Visual Acuity (LL BCVA) from baseline at Week 48","definition_or_measurement_approach":"Change from baseline in LL BCVA; proportion of subjects gaining ≥10 letters at Week 48."}
- {"endpoint_text":"- Efficacy: Proportion of subjects gaining ≥ 15 letters (3 lines) in LL BCVA from baseline at Week 48","definition_or_measurement_approach":"Change from baseline in LL BCVA; proportion of subjects gaining ≥15 letters at Week 48."}
Recruitment
- Registry Or Advocacy Recruitment
- True, Fundacion Aiken De La Comunitat Valenciana (listed as a patient organisation site)
- Planned Sample Size
- 265
- Recruitment Window Months
- 39
- Consent Approach
- Informed consent obtained from each participant (participants must be able to provide informed consent). Subject information and ICF documents are provided (multiple L1/L2 ICF and other subject information documents exist); materials available in multiple languages (English and multiple local language versions present: CZ, DE, ES, HU, IT as indicated by protocol synopses and ICF documents). Caregivers may assist with administration; a 'Pregnant Partner' information/ICF document is available. No assent process described (adults only, ≥55).
Geography
- Total Number Of Sites
- 17
- Total Number Of Participants
- 95
Italy
- Earliest CTIS Part Ii Submission Date
- 16-08-2024
- Latest Decision Or Authorization Date
- 12-02-2026
- Processing Time Days
- 545
- Number Of Sites
- 5
- Number Of Participants
- 20
Sites
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- Ophthalmology
- Principal Investigator Name
- Francesco Maria Bandello
- Principal Investigator Email
- bandello.francesco@hsr.it
- Contact Person Name
- Francesco Maria Bandello
- Contact Person Email
- bandello.francesco@hsr.it
- Site Name
- ASST Fatebenefratelli Sacco
- Department Name
- Ophthalmology
- Principal Investigator Name
- Giovanni Staurenghi
- Principal Investigator Email
- giovanni.staurenghi@unimi.it
- Contact Person Name
- Giovanni Staurenghi
- Contact Person Email
- giovanni.staurenghi@unimi.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Ophthalmology
- Principal Investigator Name
- Antonio Baldascino
- Principal Investigator Email
- antonio.baldascino@policlinogemelli.it
- Contact Person Name
- Antonio Baldascino
- Contact Person Email
- antonio.baldascino@policlinogemelli.it
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- Ophthalmology
- Principal Investigator Name
- Francesco Viola
- Principal Investigator Email
- francesco.viola@unimi.it
- Contact Person Name
- Francesco Viola
- Contact Person Email
- francesco.viola@unimi.it
- Site Name
- Azienda Sanitaria Universitaria Friuli Centrale
- Department Name
- Ophthalmology
- Principal Investigator Name
- Paolo Lanzetta
- Principal Investigator Email
- paolo.lanzetta@uniud.it
- Contact Person Name
- Paolo Lanzetta
- Contact Person Email
- paolo.lanzetta@uniud.it
Germany
- Earliest CTIS Part Ii Submission Date
- 12-09-2024
- Latest Decision Or Authorization Date
- 11-02-2026
- Processing Time Days
- 517
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Klinikum rechts der Isar der TU Muenchen AöR
- Department Name
- Ophthalmology
- Principal Investigator Name
- Mathias Maier
- Principal Investigator Email
- annette.schuster@mri.tum.de
- Contact Person Name
- Mathias Maier
- Contact Person Email
- annette.schuster@mri.tum.de
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Ophthalmology
- Principal Investigator Name
- Alexandra Schweig
- Principal Investigator Email
- alexandra.schweig@med.uni-tuebingen.de
- Contact Person Name
- Alexandra Schweig
- Contact Person Email
- alexandra.schweig@med.uni-tuebingen.de
- Site Name
- Augenzentrum Am St Franziskus-Hospital Muenster
- Department Name
- Ophthalmology
- Principal Investigator Name
- Clemens Lange
- Principal Investigator Email
- clemens.lange@augen-franzikus.de
- Contact Person Name
- Clemens Lange
- Contact Person Email
- clemens.lange@augen-franzikus.de
- Site Name
- Universitaetsklinikum Regensburg AöR
- Department Name
- Ophthalmology
- Principal Investigator Name
- Andreea Gamulescu
- Principal Investigator Email
- andreea.gamulescu@klinik.uni-regensburg.de
- Contact Person Name
- Andreea Gamulescu
- Contact Person Email
- andreea.gamulescu@klinik.uni-regensburg.de
Spain
- Earliest CTIS Part Ii Submission Date
- 04-09-2024
- Latest Decision Or Authorization Date
- 16-02-2026
- Processing Time Days
- 530
- Number Of Sites
- 5
- Number Of Participants
- 21
Sites
- Site Name
- Hospital Universitario Puerta De Hierro De Majadahonda
- Department Name
- Ophthalmology
- Principal Investigator Name
- Jose Maria Ruiz Moreno
- Principal Investigator Email
- josemaria.ruiz@uclm.es
- Contact Person Name
- Jose Maria Ruiz Moreno
- Contact Person Email
- josemaria.ruiz@uclm.es
- Site Name
- Centro De Oftalmologia Barraquer S.A.
- Department Name
- Ophthalmology
- Principal Investigator Name
- Santiago Abengoechea Hernandez
- Principal Investigator Email
- sah@barraquer.com
- Contact Person Name
- Santiago Abengoechea Hernandez
- Contact Person Email
- sah@barraquer.com
- Site Name
- Valles Ophthalmology Research S.L.
- Department Name
- Ophthalmology
- Principal Investigator Name
- Laura Sararols Ramsey
- Principal Investigator Email
- sruiz@omiq.es
- Contact Person Name
- Laura Sararols Ramsey
- Contact Person Email
- sruiz@omiq.es
- Site Name
- Fundacion Aiken De La Comunitat Valenciana
- Department Name
- Ophthalmology
- Principal Investigator Name
- Patricia Udaondo Mirete
- Principal Investigator Email
- draudaondo@gmail.com
- Contact Person Name
- Patricia Udaondo Mirete
- Contact Person Email
- draudaondo@gmail.com
- Site Name
- Oftalmologia Vistahermosa S.L.
- Department Name
- Ophthalmology
- Principal Investigator Name
- Roberto Gallego-Pinazo
- Principal Investigator Email
- robertogallegopinazo@gmail.com
- Contact Person Name
- Roberto Gallego-Pinazo
- Contact Person Email
- robertogallegopinazo@gmail.com
Hungary
- Earliest CTIS Part Ii Submission Date
- 12-08-2024
- Latest Decision Or Authorization Date
- 16-02-2026
- Processing Time Days
- 553
- Number Of Sites
- 2
- Number Of Participants
- 14
Sites
- Site Name
- University Of Szeged
- Department Name
- Ophthalmology
- Principal Investigator Name
- Edit Toth-Molnar
- Principal Investigator Email
- toth-molnar.edit@med.u-szeged.hu
- Contact Person Name
- Edit Toth-Molnar
- Contact Person Email
- toth-molnar.edit@med.u-szeged.hu
- Site Name
- Nozologen Kft.
- Department Name
- Ophthalmology
- Principal Investigator Name
- Balazs Varsanyi
- Principal Investigator Email
- varsanyi.balazs@ganglion.hu
- Contact Person Name
- Balazs Varsanyi
- Contact Person Email
- varsanyi.balazs@ganglion.hu
Czechia
- Earliest CTIS Part Ii Submission Date
- 28-08-2024
- Latest Decision Or Authorization Date
- 13-02-2026
- Processing Time Days
- 534
- Number Of Sites
- 1
- Number Of Participants
- 25
Sites
- Site Name
- Axon Clinical s.r.o.
- Department Name
- Ophthalmology
- Principal Investigator Name
- Jan Ernest
- Principal Investigator Email
- jan.ernest@axon-clinical.com
- Contact Person Name
- Jan Ernest
- Contact Person Email
- jan.ernest@axon-clinical.com
Sponsor
Primary sponsor
- Full Name
- Stealth Biotherapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Ora Europe Limited
- Responsibilities
- Sponsor duties codes: 1, 12, 15 (Site Contracts/payment management), 2, 5
Third parties
- {"country":"United Kingdom","full_name":"Ora Europe Limited","duties_or_roles":"Sponsor duties codes: 1, 12, 15 (Site Contracts/payment management), 2, 5","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Elamipretide
- Active Substance
- ELAMIPRETIDE TRIHYDROCHLORIDE
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS INJECTION
- Route
- Subcutaneous injection
- Authorisation Status
- MIA number: IMP11566/00001
- First In Class
- Yes
- Frequency
- Once daily
- Maximum Dose
- 40 mg per day
- Investigational Product Name
- The placebo for this trial will be composed of excipients used to manufacture the investigational drug but without the active drug substance.
- Modality
- Other
- Frequency
- Once daily (matched to active)
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