A Phase I/II clinical trial with SENS-501 in children suffering from severe to profound hearing loss due to Otoferlin (OTOF) mutations

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sensorion

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Otorhinolaryngologic Diseases [C09]

Trial duration

4 Sep 2024 → ongoing

Decision date (initial)

2024-01-17

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Sensorion

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy, Others, Dose response

Part 1: To assess safety, tolerability of SENS-501 following unilateral injection into the cochlea.
Part 2: To assess the efficacy following unilateral injection SENS-501 into the cochlea.

Secondary objectives 3

1. Part 1: To assess the efficacy following unilateral injection SENS-501 into the cochlea.
2. Part 2: To assess the safety and tolerability following unilateral injection SENS-501 into the cochlea
3. Part 1 and 2: To evaluate the clinical performance, safety and usability of SPHYNX Pump and Catheter.

Conditions and MedDRA coding

Bilateral severe to profound hearing loss due to Otoferlin deficiency.

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, National Agency For The Safety Of Medicine And Health Products

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Children (male or female) ≥ 6 to ≤ 31 months at the time of inclusion.
2. Severe to profound hearing loss assessed by ABR
3. Biallelic mutation in the Otoferlin gene
4. Presence of OAEs
5. Documented normal cochlea and internal auditory canals
6. Patients with intact vestibular function

Exclusion criteria 7

1. History of chronic, acute, or major disease, or unspecified reasons, that in the opinion of the Investigator, makes the participant unsuitable for participation in the study or constitutes an unacceptable risk.
2. Have been dosed in a previous gene therapy clinical trial.
3. Patients with a prior or current cochlear implant.
4. Any contraindication to the surgery determined by the surgeon or anesthesia determined by the anesthesiologist, or designee, or history of therapy known as ototoxic (e.g., cisplatin, high dose and long treatment with aminoglycosides, etc.) for an extended period (more than 2 weeks).
5. Participation in any other interventional clinical trial (in accordance with the definition in Art. 2.2(1) of REGULATION (EU) No 536/2014).
6. Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or might interfere with the evaluation of study treatment.
7. Anticipated noncompliance with the protocol requirements

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Part 1: Number of study-related adverse events and serious adverse events (through trial completion)
2. Part 2: Number of patients who demonstrate an improvement of hearing threshold measured by ABR (through trial completion)

Secondary endpoints 4

1. Part 1: Number of patients who demonstrate an improvement of hearing threshold measured by ABR (through trial completion)
2. Part 2 : Number of study-related adverse events and serious adverse events (through trial completion)
3. Part 1 and 2: Number of patients who demonstrate an improvement of hearing threshold measured by PTA (through trial completion)
4. Part 1 and 2: Clinical performance and usability of SPHYNX Pump and Catheter (SPHYNX pump injection log, user feedback questionnaire, usability questionnaire - through trial completion)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sensorion

Sponsor organisation

Sensorion

Address

375 Rue Du Professeur Blayac

City

Montpellier

Postcode

34080

Country

France

Scientific contact point

Organisation

Sensorion

Contact name

Lionel HOVSEPIAN

Public contact point

Organisation

Sensorion

Contact name

Lionel HOVSEPIAN

Third parties 2

Locations

3 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 74 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

10 submissions — initial application plus substantial / non-substantial modifications