Overview
Sponsor-declared trial summary
Phase
Phase II and Phase III (Integrated)
Status
Ongoing, recruiting
Participants planned
14
Countries
1
Sites
1
Otoferlin gene-mediated hearing loss
To evaluate the long term safety of AAVAnc80-hOTOF, including late-occurring adverse events.
Key facts
- Sponsor
- Akouos Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 11 Nov 2024 → ongoing
- Decision date (initial)
- 2024-07-24
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Akouos Inc.
External identifiers
- EU CT number
- 2024-512584-31-00
- ClinicalTrials.gov
- NCT06696456
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To evaluate the long term safety of AAVAnc80-hOTOF, including late-occurring adverse events.
Secondary objectives 1
- To evaluate the long term efficacy of AAVAnc80-hOTOF
Conditions and MedDRA coding
Otoferlin gene-mediated hearing loss
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Long Term Follow-Up This is an observational, post-intervention long term follow-up study to evaluate the long term safety and
efficacy of AAVAnc80-hOTOF gene therapy in individuals with OTOF-mediated hearing loss who received a single
intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.
|
Not Applicable | None |
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2023-505128-76-00 | A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals with Sensorineural Hearing Loss due to Biallelic Otoferlin Gene (OTOF) Mutations | Akouos Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Individuals with OTOF-mediated hearing loss who received an intracochlear administration of AAVAnc80- hOTOF in the AK-OTOF-101 clinical trial (either Part A or Part B).
- Legally authorized representative and/or participant willingness to comply with all study requirements, as evidenced by successful completion of the informed consent process or permission and participant assent process (where appropriate).
Exclusion criteria 1
- Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study. Potential participants will not be excluded based on their sex, gender, race, or ethnicity.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 7
- Safety: Incidence and frequency of AEs
- Safety: Concomitant Medications
- Safety: Clinical labs: chemistries, hematology (CBC with differential), urinalysis
- Safety: Physical examination and vital signs
- Safety: Otoscopic examination
- Safety: Vestibular screening
- Safety: Tympanometry
Secondary endpoints 4
- Efficacy: Diagnostic ABR testing threshold
- Efficacy: Age-appropriate behavioral audiometry
- Efficacy: Standard word or speech recognition testing
- Efficacy: Standard auditory questionnaires
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10381505 · Product
- Active substance
- AAVANC80-5HOTOF
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRACOCHLEAR
- Max daily dose
- 810000000000 Other
- Max total dose
- 810000000000 Other
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- AKOUOS, INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2477
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Akouos Inc.
- Sponsor organisation
- Akouos Inc.
- Address
- 645 Summer Street Suite 200
- City
- Boston
- Postcode
- 02210-2135
- Country
- United States
Scientific contact point
- Organisation
- Akouos Inc.
- Contact name
- Akouos Clinical Trials
Public contact point
- Organisation
- Akouos Inc.
- Contact name
- Akouos Clinical Trials
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| Medpace Inc. ORG-100026760
|
Cincinnati, United States | On site monitoring, Code 10, Code 12, Code 13, Other, Code 2, Code 5, Data management, E-data capture, Code 8, Code 9 |
| Boyd Consultants Limited ORG-100013894
|
Dublin 11, Ireland | Code 12 |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruiting | 2 | 1 |
| Rest of world
United Kingdom, Taiwan, United States, Canada
|
— | 12 | — |
Investigational sites
Sant Joan De Deu Barcelona Hospital
Pediatric Otorhinolaryngology, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
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 |
|---|---|---|---|---|---|
| Spain | 2024-11-11 | 2024-11-11 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 12 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-512584-31-00_AK-OTOF-LTFU_redacted_for publication | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_ES_Akouos | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent 12-17 years old_Akouos_redacted_for publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent ICF_Akouos_redacted_AR | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parent_Akouos_redacted_for publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Travel vendor ICF_Akouos_redacted_AR | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_travel vendor_Akouos_redacted_for publication | 1.0 |
| Subject information and informed consent form (for publication) | L2_CaregiverRetentionBrochure-V1_29Aug2024_ES | 1 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_FAQ_Bank Transfer_Akouos_for publication | 10.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_PECard_Akouos_for publication | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ENG_2024-512584-31-00_AK-OTOF-LTFU_redacted_for publication | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ES_2024-512584-31-00_AK-OTOF-LTFU_redacted_for publication | 1 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-04-16 | Spain | Acceptable 2024-06-05
|
2024-07-24 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-10-04 | Spain | Acceptable 2024-06-05
|
2024-10-04 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-12-18 | Spain | Acceptable 2024-06-05
|
2024-12-18 |
| 4 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-02-07 | Spain | Acceptable | 2025-03-14 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-08-05 | Spain | Acceptable | 2025-08-05 |
| 6 | SUBSTANTIAL MODIFICATION | SM-2 | 2026-01-09 | Spain | Acceptable 2026-03-13
|
2026-03-17 |