Evaluation of clinical and laboratory parameters of adult patients with SMA under treatment with nusinersen

Evaluation of clinical and laboratory parameters of adult patients with SMA under treatment with nusinersen - ADULT-SMA

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00015702

Enrollment

350

Registered

2018-10-23

Start date

2018-10-19

Completion date

Unknown

Last updated

2025-04-07

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

Conditions

G12.1

Interventions

Group 1: The treatment of SMA with nusinersen is within its approval. Motor function scores (HFMSE, RULM, 6MWT) were performed during the treatment.

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Inclusion criteria

Inclusion criteria: genetically confirmed 5q-SMA Planned treatment with Nusinersen Patient is able to give informed consent

Exclusion criteria

Exclusion criteria: Pregnancy < 18 y.

Design outcomes

Primary

Measure	Time frame
Change of Hammersmith functional Evaluation Score (HFMSE) at 6 and 12 months after treatment with nusinersen	—

Secondary

Measure	Time frame
Change in Revised Upper Limb Module (RULM) score after 6 and 12 months treatment with nusinersen	—

Countries

Germany

Contacts

Public ContactTim Hagenacker

Klinik für Neurologie Universitätsklinikum Essen

tim.hagenacker@uk-essen.de+492017236513

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 28, 2026