Overview
Sponsor-declared trial summary
Dominant Optic Atrophy
The main objective is to evaluate the tolerability of 3 grams per day of nicotinamide in DOA/DOA+ patients to ensure that this treatment does not induce ophthalmological or neurological toxic side effects.
Key facts
- Sponsor
- Centre Hospitalier Universitaire D Angers
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 27 Dec 2023 → 14 Apr 2026
- Decision date (initial)
- 2023-09-13
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
The main objective is to evaluate the tolerability of 3 grams per day of nicotinamide in DOA/DOA+ patients to ensure that this treatment does not induce ophthalmological or neurological toxic side effects.
Secondary objectives 4
- To evaluate the efficacy of treatment on visual function by electroretinogram, measuring visual acuity, visual field and thickness of visual fibers in the optic disc using OCT (optical coherence tomography).
- Biological efficacy will be assessed by measuring nicotinamide levels in patients' blood (plasma and whole blood) at baseline and follow-up.
- To evaluate the effectiveness of treatment on patients' quality of life using the NEI VFQ 25 self-questionnaire.
- To evaluate the neurological efficacy of treatment in DOA+ forms.
Conditions and MedDRA coding
Dominant Optic Atrophy
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- Major patients
- Patients with DOA or DOA+ due to a heterozygous pathogenic variant in the OPA1 gene
- Nicotinamide-naïve patients (> 3 months)
- Patients able to take oral medication, and comply with specific study procedures
- Patients affiliated with or benefiting from a social security scheme
- Signature of voluntary, free and informed consent to participate in the study
Exclusion criteria 10
- Asymptomatic patients (= healthy carriers of an OPA1 mutation who have not developed optic neuropathy)
- Patients with other associated severe ophthalmological pathologies (advanced glaucoma, retinal pathology).
- Patients treated with Idebenone
- Patients with transaminase levels (ASAT and/or ALAT) twice the high normal value
- Pregnant, breast-feeding or parturient women
- Patients with a contraindication to nicotinamide
- Person deprived of liberty by administrative or judicial decision
- Patients under legal protection
- Persons under compulsory psychiatric care
- Persons unable to give consent
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The primary endpoint will be assessed by the number of patients with reported ophthalmological and/or neurological adverse events.
Secondary endpoints 4
- Number of patients with improved visual function at follow-up visits compared with baseline at inclusion.
- Increase in plasma and whole blood nicotinamide levels in all patients, comparing baseline at inclusion with two follow-up periods.
- Differences on the NEI-VFQ-25 scale between M0 and M6
- Clinical neurological improvement at follow-up visits compared with baseline at inclusion.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
NICOBION 500 mg, comprimé pelliculé
PRD352725 · Product
- Active substance
- Nicotinamide
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 3 g gram(s)
- Max total dose
- 3 g gram(s)
- Max treatment duration
- 6 Month(s)
- Authorisation status
- Authorised
- ATC code
- A11HA01 — NICOTINAMIDE
- Marketing authorisation
- 223 822-0
- MA holder
- TEOFARMA S.R.L.
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Centre Hospitalier Universitaire D Angers
- Sponsor organisation
- Centre Hospitalier Universitaire D Angers
- Address
- 4 Rue Larrey
- City
- Angers Cedex 9
- Postcode
- 49933
- Country
- France
Scientific contact point
- Organisation
- Centre Hospitalier Universitaire D Angers
- Contact name
- coordinating investigator
Public contact point
- Organisation
- Centre Hospitalier Universitaire D Angers
- Contact name
- coordinating investigator
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 25 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| France | 2023-12-27 | 2026-04-14 | 2024-01-23 | 2025-10-14 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 10 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2023-506214-52-00 | 3 |
| Protocol (for publication) | D4_ Patient facing documents_Questionnaire DN4 | 1 |
| Protocol (for publication) | D4_ Patient facing documents_Questionnaire NEI-VFQ-25 | 1 |
| Protocol (for publication) | D4_Patient facing documents_Carnet patient | 2 |
| Protocol (for publication) | D4_Patient facing documents_Carnet patient_Tracked changes | 2 |
| Recruitment arrangements (for publication) | K-Recruitment and Informed consent procedure | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_ patient CHU Angers | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_ patient hors CHU Angers | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | SmPC_Nicobion | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2023-506214-52-00 | 3 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-06-09 | France | Acceptable 2023-09-12
|
2023-09-13 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-10-18 | France | Acceptable 2024-12-16
|
2024-12-19 |