Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruitment ended
Participants planned
360
Countries
5
Sites
17
Dry Age-Related Macular Degeneration (Dry AMD)
Evaluate the efficacy of once daily subcutaneous (SC) injections of elamipretide in subjects who have dry age-related macular degeneration (AMD)
Key facts
- Sponsor
- Stealth Biotherapeutics Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Eye Diseases [C11]
- Trial duration
- 20 Nov 2024 → ongoing
- Decision date (initial)
- 2024-09-23
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- Stealth BioTherapeutics Inc
External identifiers
- EU CT number
- 2024-511482-11-00
- ClinicalTrials.gov
- NCT06373731
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Pharmacokinetic, Efficacy
Evaluate the efficacy of once daily subcutaneous (SC) injections of elamipretide in subjects who have dry age-related macular degeneration (AMD)
Secondary objectives 2
- Evaluate the safety and tolerability of once daily SC injections of elamipretide
- Evaluate the Pharmacokinetic (PK) profile of elamipretide and its metabolites
Conditions and MedDRA coding
Dry Age-Related Macular Degeneration (Dry AMD)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10075567 | Dry age-related macular degeneration | 100000004853 |
Study design 3 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Screening Period After patients consent, patients will undergo Screening procedures as described in the protocol. Patients who complete Screening and continue to meet all trial requirements, including all Inclusion Criteria and none of the Exclusion Criteria, may be randomized at the Baseline Visit (Day 1) and enter the Treatment Period. Screening Period lasting for a maximum of 28 days.
|
Not Applicable | None | ||
| 2 | Masked Treatment Period The Treatment Period begins on the day of the Baseline Visit (Day 1) and lasts for approximately 96 weeks. At the Baseline Visit (Day 1), following completion of all Baseline procedures as described in the protocol, including reconfirming of eligibility, patients will be randomized 2:1 to treatment with either elamipretide or placebo.
Patients (and caregivers if needed) will be provided with a dosing diary and trained on the procedure for administration of the IMP and recording of the location and time of the IMP administration daily in the dosing diary. Subject (or trained caregiver) will administer IMP at the clinical trial site, unless otherwise specified.
|
Randomised Controlled | Double | [{"id":159537,"code":1,"name":"Subject"},{"id":159536,"code":2,"name":"Investigator"}] | Elamipretide: Patients on this arm will receive treatment with elamipretide for 96 weeks after randomisation Placebo: Patients on this arm will receive treatment with placebo for 96 weeks after randomisation |
| 3 | Follow-up Period At the Week 96 Visit, patients will have a 4 week safety follow-up until week 100.
|
Not Applicable | None | Safety Follow-up: The 4-week Safety Follow-Up Period will begin after completion of the Week 96 Visit. Patients will return to the clinical trial site at Week 100 for final safety assessments, as described in the protocol and return used and unused IMP not previously returned. |
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2016-000126-19 | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment with Elamipretide in Patients Hospitalized with Congestion due to Heart Failure, Estudio de fase II, aleatorizado, doble ciego, controlado con placebo, para evaluar los efectos cardiacos y renales del tratamiento a corto plazo con elamipretida en pacientes hospitalizados con congestión debida a insuficiencia cardiaca | |
| 2017-002447-15 | A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension, 3. fázisú, randomizált, kettős vak, párhuzamos csoportos, placebo-kontrollos vizsgálat a napi rendszerességgel, szubkután alkalmazott elamipretid injekciók hatásosságának és biztonságosságának felmérésére elsődleges mitokondriális miopátiában szenvedő betegek körében, amelyet egy nyílt címkés kezelési időszak követ, Sperimentazione di fase 3, randomizzata, in doppio cieco, a gruppi paralleli, controllata con placebo per valutare l’efficacia e la sicurezza di iniezioni sottocutanee giornaliere di elamipretide in soggetti con miopatia mitocondriale primaria seguita da un’estensione del trattamento in aperto | |
| 2021-003907-16 | A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD), III. fázisú, randomizált, kettős vak, párhuzamos csoportos, placebokontrollált vizsgálat a napi subcutan elamipretid injekciók hatásosságának és biztonságosságának értékelésére patogén nukleáris DNS-mutációkból (nPMD) eredő primer mitokondriális betegségben szenvedő betegeknél , Estudio de fase III aleatorizado, doble ciego, de grupos paralelos y controlado con placebo para evaluar la eficacia y la seguridad de las inyecciones subcutáneas diarias de elamipretida en sujetos con enfermedad mitocondrial primaria causada por mutaciones patogénicas del ADN nuclear (EMPn), Studio di fase III, randomizzato, in doppio cieco, a gruppi paralleli, controllato con placebo mirato a valutare l’efficacia e la sicurezza di iniezioni sottocutanee giornaliere di elamipretide in soggetti con patologia mitocondriale primaria dovuta a mutazioni patogenetiche del DNA nucleare (nMPD), Studio di fase III, randomizzato, in doppio cieco, a gruppi paralleli, controllato con placebo mirato a valutare l’efficacia e la sicurezza di iniezioni sottocutanee giornaliere di elamipretide in soggetti con patologia mitocondriale primaria dovuta a mutazioni patogenetiche del DNA nucleare (nMPD) | |
| 2011-002824-42 | A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Safety, Tolerability and Efficacy of Intravenous Bendavia™ (MTP-131) on Reperfusion Injury in Patients Treated with Standard Therapy Including Primary PCI and Stenting for ST-segment Elevation Myocardial Infarction | |
| 2015-005615-32 | A Phase 2 Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Effects of 4 Weeks Treatment with Subcutaneous Elamipretide on Left Ventricular Function in Subjects with Stable Heart Failure with Preserved Ejection Fraction, Eine randomisierte, doppelblinde, placebokontrollierte Phase-II-Studie zur Beurteilung der Wirkungen einer 4-wöchigen Behandlung mit subkutanem Elamipretid auf die linksventrikuläre Funktion bei Patienten mit stabiler Herzinsuffizienz und erhaltener Ejektionsfraktion, Eine randomisierte, doppelblinde, placebokontrollierte Phase-II-Studie zur Beurteilung der Wirkungen einer 4-wöchigen Behandlung mit subkutanem Elamipretid auf die linksventrikuläre Funktion bei Patienten mit stabiler Herzinsuffizienz und erhaltener Ejektionsfraktion, Eine randomisierte, doppelblinde, placebokontrollierte Phase-II-Studie zur Beurteilung der Wirkungen einer 4-wöchigen Behandlung mit subkutanem Elamipretid auf die linksventrikuläre Funktion bei Patienten mit stabiler Herzinsuffizienz und erhaltener Ejektionsfraktion, Eine randomisierte, doppelblinde, placebokontrollierte Phase-II-Studie zur Beurteilung der Wirkungen einer 4-wöchigen Behandlung mit subkutanem Elamipretid auf die linksventrikuläre Funktion bei Patienten mit stabiler Herzinsuffizienz und erhaltener Ejektionsfraktion, Eine randomisierte, doppelblinde, placebokontrollierte Phase-II-Studie zur Beurteilung der Wirkungen einer 4-wöchigen Behandlung mit subkutanem Elamipretid auf die linksventrikuläre Funktion bei Patienten mit stabiler Herzinsuffizienz und erhaltener Ejektionsfraktion | |
| 2014-005724-10 | A Phase 2 Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Effects of Multiple Subcutaneous Injections of Elamipretide on Left Ventricular Function in Subjects with Stable Heart Failure with Reduced Ejection Fraction, Studio di Fase II randomizzato, in doppio cieco, controllato con placebo per valutare gli effetti di iniezioni sottocutanee multiple di elamipretide sulla funzione ventricolare sinistra in soggetti con insufficienza cardiaca stabile e frazione di eiezione ridotta |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- 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.
- Women of childbearing potential must agree to use 1 of the following methods of birth control
- 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)
- GA in the study eye at the Screening Visit may be multi-focal
- Able to administer IMP
- 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 5
- Concurrent disease in the study eye during the trial
- 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
- Participation in other investigational drug or device clinical trials
- Inability to comply with trial or follow-up procedures
- Prior participation in any elamipretide trial
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- 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
Secondary endpoints 4
- 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
- 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
- Efficacy: Proportion of subjects gaining ≥ 10 letters (2 lines) in Low Luminance Best-Corrected Visual Acuity (LL BCVA) from baseline at Week 48
- Efficacy: Proportion of subjects gaining ≥ 15 letters (3 lines) in LL BCVA from baseline at Week 48
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD5932196 · Product
- Active substance
- Elamipretide Trihydrochloride
- Other product name
- Elamipretide liquid cartridge formulation
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 40 mg/ml milligram(s)/millilitre
- Max total dose
- 27160 mg/ml milligram(s)/millilitre
- Max treatment duration
- 96 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- STEALTH BIOTHERAPEUTICS INC.
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Stealth Biotherapeutics Inc.
- Sponsor organisation
- Stealth Biotherapeutics Inc.
- Address
- 123 Highland Avenue Suite 201
- City
- Needham
- Postcode
- 02494-3005
- Country
- United States
Scientific contact point
- Organisation
- Stealth Biotherapeutics Inc.
- Contact name
- Jim Carr
Public contact point
- Organisation
- Stealth Biotherapeutics Inc.
- Contact name
- Rekha Sathyanarayana
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Ora Europe Limited ORG-100044931
|
London, United Kingdom | On site monitoring, Code 12, Other, Code 2, Code 5 |
Locations
5 EU/EEA countries · 17 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Ongoing, recruitment ended | 25 | 1 |
| Germany | Ongoing, recruitment ended | 15 | 4 |
| Hungary | Ongoing, recruitment ended | 14 | 2 |
| Italy | Ongoing, recruitment ended | 20 | 5 |
| Spain | Ongoing, recruitment ended | 21 | 5 |
| Rest of world
United Kingdom, United States, New Zealand
|
— | 265 | — |
Investigational sites
Universitaetsklinikum Tuebingen AöR
Ophthalmology, Elfriede-Aulhorn-Strasse 7, Nordstadt, Tuebingen
Klinikum rechts der Isar der TU Muenchen AöR
Ophthalmology, Ismaninger Strasse 22, Au-Haidhausen, Munich
Augenzentrum Am St Franziskus-Hospital Muenster
Ophthalmology, Hohenzollernring 74, Herz-Jesu, Munster
Universitaetsklinikum Regensburg AöR
Ophthalmology, Franz-Josef-Strauss-Allee 11, Grass-Oberisling, Regensburg
Nozologen Kft.
Ophthalmology, Varady Antal Utca 10 Fszt. 5, 7621, Pecs
University Of Szeged
Ophthalmology, Koranyi Fasor 10-11, 6720, Szeged
Ospedale San Raffaele S.r.l.
Ophthalmology, Via Olgettina 60, 20132, Milan
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Ophthalmology, Largo Francesco Vito 1, 00168, Rome
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Ophthalmology, Via Francesco Sforza 28, 20122, Milan
Azienda Sanitaria Universitaria Friuli Centrale
Ophthalmology, Via Pozzuolo 330, 33100, Udine
ASST Fatebenefratelli Sacco
Ophthalmology, Via Giovanni Battista Grassi 74, 20157, Milan
Fundacion Aiken De La Comunitat Valenciana
Ophthalmology, Calle De Pizarro 15 Bajo, 46004, Valencia
Valles Ophthalmology Research S.L.
Ophthalmology, Carrer Pedro I Pons 1, 08195, Sant Cugat Del Valles
Oftalmologia Vistahermosa S.L.
Ophthalmology, Avenida De La Ilustracion 1, Poligono Industrial De Burjassot, Burjassot
Centro De Oftalmologia Barraquer S.A.
Ophthalmology, Calle Muntaner 314, 08021, Barcelona
Hospital Universitario Puerta De Hierro De Majadahonda
Ophthalmology, Calle De Joaquin Rodrigo 2, 28222, Majadahonda
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Czechia | 2025-01-30 | 2025-03-18 | 2025-06-24 | ||
| Germany | 2024-12-17 | 2025-02-26 | 2025-06-30 | ||
| Hungary | 2024-11-20 | 2024-12-02 | 2025-06-24 | ||
| Italy | 2025-02-03 | 2025-02-13 | 2025-07-14 | ||
| Spain | 2024-12-20 | 2024-12-26 | 2025-06-30 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 54 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-511482-11-00_Redacted | 3.0 |
| Protocol (for publication) | D4_Patient facing documents_EQ-5D-5L Questionnaire_UK ENG_Redacted | 1.2 |
| Protocol (for publication) | D4_Patient facing documents_VILL-33 Questionnaire_UK ENG_Redacted | 2.0 |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements | n/a |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements | n/a |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements and material_Redacted | n/a |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Dosing Video for patients_Redacted | Rev 2 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_IMP Training Checklist_Redacted | n/a |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Instruction for Use sheet_Redacted | 1.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Patient Diary_Redacted | 2.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Patient ID Card_Redacted | 1.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Thank you email for trial participation | 1.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Thank you email for trial participation | 1.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Thank you email for trial participation | 1.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Thank you email for trial participation | 1.0 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_Thank you email for trial participation | 1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Data Protection_Redacted | 1.1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Main_Redacted | 2.0.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Main_Redacted | 2.0.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Main_Redacted | 2.0.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Main_Redacted | 2.0.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Main_Redacted | 2.0.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Pregnant Partner_Redacted | V1.2.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Pregnant Partner_Redacted | 1.4.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Pregnant Partner_Redacted | 1.2.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF_Pregnant Partner_Redacted | 1.1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Redacted | 1.6.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Dosing Video for patients | Rev 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Dosing Video for patients | Rev 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Dosing Video for patients | n/a |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Dosing Video for patients_Redacted | Rev 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_IMP Training Checklist_Redacted | 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_IMP Training Checklist_Redacted | n/a |
| Subject information and informed consent form (for publication) | L2_Other subject information material_IMP Training Checklist_Redacted | n/a |
| Subject information and informed consent form (for publication) | L2_Other subject information material_IMP Training Checklist_Redacted | n/a |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Instruction for Use sheet_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Instruction for Use sheet_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Instruction for Use sheet_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Instructions for Use sheet_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient Diary_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient Diary_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient Diary_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient Diary_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient ID Card_Redacted | 1.1 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient ID Card_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient ID Card_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Patient ID Card_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Summary_ EN_2024-511482-11-00 | 2.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_CZ 2024-511482-11-00_Redacted | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_DE 2024-511482-11-00_Redacted | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ENG 2024-511482-11-00_Redacted | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ES 2024-511482-11-00_Redacted | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_HU 2024-511482-11-00_Redacted | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_IT 2024-511482-11-00_Redacted | 3.0 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-06-05 | Germany | Acceptable 2024-09-19
|
2024-09-20 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-10-04 | Acceptable | 2024-10-23 | |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-10-11 | Germany | Acceptable | 2024-10-24 |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-10-14 | Acceptable | 2024-11-11 | |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-05-02 | Germany | Acceptable 2025-06-26
|
2025-06-27 |
| 6 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-12-16 | Germany | Acceptable 2026-02-11
|
2026-02-11 |