Outcome measures in SMA type II and III

Outcome measures in SMA type II and III - SMA-Europe

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00000437

Enrollment

160

Registered

2010-06-22

Start date

2010-12-07

Completion date

Unknown

Last updated

2026-04-27

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: Measurements obtained. Hammesmith Function Scale, Motor Function Measure (MFM 20 Items), Egen Scale,Peds QL (for neuromuscular disease), 6 Minute Walking Test, Step Activity Monitor (for 1 w

Eligibility

Sex/Gender

All

Age

30 Months to 24 Years

Inclusion criteria

Inclusion criteria: -confirmed diagnosis of SMA II or III non ambulant patient: age 30 month - 18 years ambulant patient: age 30 month - 24 years

Exclusion criteria

Exclusion criteria: Therapy with valproate

Design outcomes

Primary

Measure	Time frame
Validation of outcome measure in SMA II and III	—

Secondary

Measure	Time frame
More perception about progress in SMA II and III	—

Countries

Belgium, France, Germany, Italy, Netherlands, Spain, United Kingdom

Contacts

Public ContactRudolf Korinthenberg

UNIVERSITAETSKLINIKUM FREIBURG Zentrum für Kinderheilkunde und Jugendmedizin Klinik II: Neuropädiatrie und Muskelerkrankungen

rudolf.korinthenberg@uniklinik-freiburg.de49 761 270-4315

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: May 1, 2026