Deflazacort TREATment in LMNArelated congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-519672-12-00

Acronym

TREAT LMNA

Enrollment

Registered

2025-01-21

Start date

Unknown

Completion date

Unknown

Last updated

2025-01-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Laminopathies

Brief summary

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).

Interventions

DRUGDEFLAN 22

DRUG75 mg/ml gocce orali

DRUGsospensione

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
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).	—

Countries

Italy

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026