Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Natural history and biomarker study in adult SMA types 2–4 with nusinersen treatment
2019-005007-40Phase 4Completed
- Sponsor
- UZ Leuven
- Start
- Jan 2020
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 4, open-label trial of nusinersen in spinal muscular atrophy (SMA) (2 arms), sponsored by UZ Leuven. Primary endpoint: Change in clinical and functional characteristics. Started Jan 2020; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
Beyond the preview
Go deeper on 2019-005007-40.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2019-005007-40.