Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Authorised, recruiting
Participants planned
7
Countries
1
Sites
1
Neurodevelopmental disorders
Evaluate the effect of treatment on patients' motor skills by assessing their motor skills before and after treatment with the 88-item Gross Motor Function Measure (GMFM-88).
Key facts
- Sponsor
- Centre Hospitalier Universitaire De Montpellier
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 8 Apr 2026 → ongoing
- Decision date (initial)
- 2025-08-14
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- PTC Therapeutics · Montpellier University Hospital
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Therapy
Evaluate the effect of treatment on patients' motor skills by assessing their motor skills before and after treatment with the 88-item Gross Motor Function Measure (GMFM-88).
Secondary objectives 4
- Evaluate the effect of treatment on cognitive development, using developmental or cognitive scales depending on the patient's age (Bayley III, WPPSI, WISC, Vineland),
- Evaluate the overall benefit on patient quality of life using a patient quality of life scale (CP-CHILD) and a parent quality of life scale (PedsQL TM (Pediatric Quality of Life Inventory TM)).
- Assessing treatment benefit using the CGI (Clinical Global Impression) scale
- Assess treatment tolerance.
Conditions and MedDRA coding
Neurodevelopmental disorders
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 26.0 | PT | 10064062 | Neurodevelopmental disorder | 100000004873 |
Study design 2 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | treatment period start of treatment within 2 months of inclusion for a 12-month period
|
Not Applicable | None | ||
| 2 | assessment of physical and cognitive abilities évaluation de la qualité de vie : CGI – CP CHILD
Évaluations motrice (échelles GMFM 88 +/-) (durée 30 – 90 min heures)
Évaluations cognitive (Vineland & WPPSI/WISC Bayley III selon âge) (1h30) vineland par les parents
Evaluation à l'inclusion, 6 mois et 12 mois de traitement
|
Not Applicable | None |
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-514852-34-00 | Prospective pilot study of L-dopa treatment in patients with a neurodevelopmental disorder linked to a pathogenic variant of the CTNNB1 gene | Centre Hospitalier Universitaire De Montpellier |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Ages 1 to 15 inclusive
- Carrier of a pathogenic variant of CTNNB1.
- Patient presenting with dystonia.
- Patient willing to comply with the contraception requirements outlined in the protocol
Exclusion criteria 9
- Contraindication to treatment with L-dopa and carbidopa or one of its excipients
- Current treatment with L-dopa, dopamine agonist or dopamine blocker,
- Failure to obtain informed consent signed by both parents or legal guardians, and to obtain the child's assent if possible.
- Patient not enrolled in or benefiting from a social security scheme
- Person participating in another research project with an exclusion period still in progress
- Patient presenting with a gastro-duodenal ulcer.
- Patient presenting with open-angle glaucoma
- Patient presenting with orthostatic hypotension
- Person who is pregnant or who wishes to become pregnant within 12 months after inclusion.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- GMFM-88 motor score before treatment (Day 1) and at 6 months
Secondary endpoints 5
- GMFM-88 motor score before treatment (Day 1) and at 12 months
- Changes in language/development/cognition assessment scores (Bayley III, WPPSI, WISC, Vineland), before treatment and at 1 year after treatment
- Changes in quality-of-life scores (Caregiver Priorities & Child Health Index of Life with Disabilities (CP-CHILD), Clinical Global Impression of severity scale (CGI)) before treatment, at 6 months and at 1 year after initiation of treatment
- Tolerance assessment (number and severity of adverse events, clinical follow-up, clinical, neurological and osteoarticular examination, patient logbook).
- GMFM-88 motor score between 6 and 12 months of treatment
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 3
PRD12273754 · Product
- Active substance
- Levodopa
- Pharmaceutical form
- CAPSULE FOR ORAL USE
- Route of administration
- ORAL
- Max daily dose
- 3 mg/kg milligram(s)/kilogram
- Max total dose
- 3 mg/kg milligram(s)/kilogram
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Not Authorised
- ATC code
- N04BA02 — LEVODOPA AND DECARBOXYLASE INHIBITOR
- MA holder
- UNIVERSITY HOSPITAL OF MONTPELLIER
- Paediatric formulation
- No
- Orphan designation
- No
PRD12671437 · Product
- Active substance
- Carbidopa
- Pharmaceutical form
- CAPSULE FOR ORAL USE
- Route of administration
- ORAL
- Max daily dose
- 0.75 mg/kg milligram(s)/kilogram
- Max total dose
- 0.75 mg/kg milligram(s)/kilogram
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Not Authorised
- ATC code
- N04BA02 — LEVODOPA AND DECARBOXYLASE INHIBITOR
- MA holder
- UNIVERSITY HOSPITAL OF MONTPELLIER
- Paediatric formulation
- No
- Orphan designation
- No
PRD12273869 · Product
- Active substance
- Carbidopa
- Pharmaceutical form
- CAPSULE FOR ORAL USE
- Route of administration
- ORAL
- Max daily dose
- 0.3 mg/Kg milligram(s)/kilogram
- Max total dose
- 0.3 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Not Authorised
- ATC code
- N04BA02 — LEVODOPA AND DECARBOXYLASE INHIBITOR
- MA holder
- UNIVERSITY HOSPITAL OF MONTPELLIER
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Centre Hospitalier Universitaire De Montpellier
- Sponsor organisation
- Centre Hospitalier Universitaire De Montpellier
- Address
- 191 Avenue Du Doyen Gaston Giraud
- City
- Montpellier Cedex 5
- Postcode
- 34295
- Country
- France
Scientific contact point
- Organisation
- Centre Hospitalier Universitaire De Montpellier
- Contact name
- Agathe ROUBERTIE
Public contact point
- Organisation
- Centre Hospitalier Universitaire De Montpellier
- Contact name
- Agathe ROUBERTIE
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Authorised, recruiting | 7 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Centre Hospitalier Universitaire De Montpellier
Neuropédiatrie, 191 Avenue Du Doyen Gaston Giraud, 34295, Montpellier Cedex 5
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 | 2026-04-08 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 33 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_2024-514852-34-01_PROTOCOL_FP | 3 |
| Protocol (for publication) | D1_2024-514852-34-01_PROTOCOL_FP_CTNNB1 | 4 |
| Protocol (for publication) | D1_2024-514852-34-01_PROTOCOL_TRACK_FP | 3 |
| Protocol (for publication) | D1_2024-514852-34-01_PROTOCOL_TRACK_FP_CTNNB1 | 4 |
| Protocol (for publication) | D1_2024-514852-34-01_PROTOCOL_TRACK_NFP | 3 |
| Recruitment arrangements (for publication) | K1_2024-514852-34-01_recruitment_procedure | 1 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_logbook_Kid_FP | 1 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_Logbook_Kid_FP | 2 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_Logbook_Kid_TRACK_FP | 2 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_logbook_Parents_FP | 1 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_Logbook_Parents_FP | 2 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_Logbook_Parents_TRACK_FP | 2 |
| Subject information and informed consent form (for publication) | D4_2024-514852-34-01_Questionnaires and scales_Track_NFP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetKid 6-12_FP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetKid 6-12_Track_FP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetKid 6-12_Track_NFP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetTeen13-15_FP | 4 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetTeen13-15_Track_FP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetTeen13-15_Track_FP | 4 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformationSheetTeen13-15_Track_NFP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_Informed_Consent_FP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_Informed_Consent_TRACK_FP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformedSheetLegalRepresentative_FP | 4 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformedSheetLegalRepresentative_Track_FP | 2 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformedSheetLegalRepresentative_TRACK_FP | 4 |
| Subject information and informed consent form (for publication) | L1_2024-514852-34-01_InformedSheetLegalRepresentative_Track_NFP | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_2024-514852-34-01_SmPC_APOTEX | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_2024-514852-34-01_SmPC_SINEMET | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_2024-514852-34-01_SmPC_SINEMET | 1 |
| Synopsis of the protocol (for publication) | D1_2024-514852-34-01_SYNOPSIS_FP | 5 |
| Synopsis of the protocol (for publication) | D1_2024-514852-34-01_SYNOPSIS_FP_CLEAN | 2.0 |
| Synopsis of the protocol (for publication) | D1_2024-514852-34-01_SYNOPSIS_FP_TRACK | 4 |
| Synopsis of the protocol (for publication) | D1_2024-514852-34-01_SYNOPSIS_TRACK_FP | 5 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-04-30 | France | Acceptable 2025-08-11
|
2025-08-14 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-11-14 | France | Acceptable 2026-01-12
|
2026-01-29 |