Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Authorised, recruitment pending
Participants planned
20
Countries
1
Sites
6
Laminopathies
Evaluation of the effect of therapy with Deflazacort drops 0.9 mg/kg/day on clinical outcome and secretome in adult and pediatric patients with L-CMD.
Key facts
- Sponsor
- Azienda Ospedaliero Universitaria Pisana
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Decision date (initial)
- 2025-01-21
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-519672-12-00
- EudraCT number
- 2019-004426-24
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
Evaluation of the effect of therapy with Deflazacort drops 0.9 mg/kg/day on clinical outcome and secretome in adult and pediatric patients with L-CMD.
Conditions and MedDRA coding
Laminopathies
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-518695-32-00 | Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers | Azienda Ospedaliero Universitaria Pisana |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- Corticosteroid-naïve patients, aged 3 to 40 years of either sex and of Caucasian origin, with clinical and genetic diagnosis of L-CMD or EDMD2 with age onset before 5 years. Patients will be eligible if they can provide reproducible measurements of forced vital capacity (FVC) (variability <15% between two repeated measures of FVC) at the time of recruitment. Signature of informed consent by the patient, in the case of an adult individual, or parents/legal guardian, in the case of a minor individual.
Exclusion criteria 1
- Hypersensitivity to the active ingredient or any of the excipients Chronic therapy with corticosteroids or immunosuppressive drugs Diabetes mellitus or other comorbidities that strongly contraindicate the use of corticosteroids, such as Active tuberculosis, peptic ulcer, ocular herpes simplex, systemic fungal infections, psychosis; administration of live attenuated vaccine Hereditary fructose intolerance Pregnancy and lactation. Pregnancy and contraception guidelines for clinical trials are attached.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Evaluation of the effects of treatment with Deflazacort drops (0.9mg/kg/day in relation to the change of clinical parameters at each treatment time and follow up (T0-T6-T12-T15-T18).
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
DEFLAN 22,75 mg/ml gocce orali, sospensione
PRD716624 · Product
- Active substance
- Deflazacort
- Pharmaceutical form
- ORAL DROPS, SUSPENSION
- Route of administration
- BUCCAL USE
- Max daily dose
- 0.9 mg/Kg milligram(s)/kilogram
- Max total dose
- 3942 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- H02AB13 — DEFLAZACORT
- Marketing authorisation
- 026046033
- MA holder
- LABORATORI GUIDOTTI S.P.A.
- MA country
- Italy
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Labelled
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Azienda Ospedaliero Universitaria Pisana
- Sponsor organisation
- Azienda Ospedaliero Universitaria Pisana
- Address
- Via Roma 67
- City
- Pisa
- Postcode
- 56126
- Country
- Italy
Scientific contact point
- Organisation
- Azienda Ospedaliero Universitaria Pisana
- Contact name
- Clinical Trial Center
Public contact point
- Organisation
- Azienda Ospedaliero Universitaria Pisana
- Contact name
- Clinical Trial Center
Locations
1 EU/EEA country · 6 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Authorised, recruitment pending | 20 | 6 |
| Rest of world | — | 0 | — |
Investigational sites
Azienda Ospedaliera di Padova
Neuroscienze, Via Nicolo' Giustiniani 2, 35128, Padova
IRCCS Istituto Giannina Gaslini
DINOGMI, Via Gerolamo Gaslini 5, 16147, Genoa
IRCCS Foundation Istituto Neurologico Carlo Besta
Neuroimmunologia e Malattie Neuromuscolari, Via Giovanni Celoria 11, 20133, Milan
Azienda Ospedaliero Universitaria Pisana
UO Neurologia, Via Roma 67, 56126, Pisa
Azienda Unita Sanitaria Locale Di Bologna
UOC di Neuropsichiatria dell’età Pediatrica, Via Altura 3, 40139, Bologna
Ospedale Pediatrico Bambino Gesu
Unità di Malattie Neuromuscolari e Neurodegenerative, Piazza Di Sant'onofrio 4, 00165, Rome
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 7 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | Protocollo V5_2024-519672-12-00 | 5 |
| Recruitment arrangements (for publication) | Document not required under the CT Directive 2001 | 00 |
| Subject information and informed consent form (for publication) | Informativa e Assenso pazienti 7_13 anni | 4 |
| Subject information and informed consent form (for publication) | Informativa e Assenso pazienti 14_18 anni | 4 |
| Subject information and informed consent form (for publication) | Informativa e Consenso adulti | 5 |
| Subject information and informed consent form (for publication) | Informativa e Consenso genitori_tutore legale | 4 |
| Summary of Product Characteristics (SmPC) (for publication) | Document not submitted under the CT Directive 2001_20_EC | 00 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-11-15 | Italy | Acceptable 2024-12-09
|
2025-01-21 |