A phase 2, randomized, double-blind, placebo-controlled parallel group study of VHB937 in Amyotrophic Lateral Sclerosis (ALS) over 40 weeks followed by an Open-label Extension (ASTRALS)

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Brief summary

The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS) [Time Frame: Baseline to DB Week 40]

Detailed description

ALSFRS-R total score [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)], Slow Vital Capacity (SVC) (% of predicted normal value) [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)], Ratio to baseline in Neurofilament Light (NfL) concentration in serum [Timeframe: DB up to Week 40; DB and OLE up to Week 100], Safety and tolerability parameters including adverse events, laboratory data, vital signs, electrocardiogram (ECG), Columbia Suicide Severity Rating Scale (C-SSRS) [Time Frame: Baseline to DB Week 40, Baseline to OLE Week 100, and Baseline to last scheduled visit or end of study], Time to death and Time to event (death or PAV, whichever comes first). [Timeframe: Baseline to DB Week 40], Time to death and Time to event (death or PAV, whichever comes first) – endpoints referring to treatment policy estimand [Time Frame: Baseline to OLE Week 100, and Baseline to end of study], Clinician and Patient Global Impression of change in functional ability and ALS symptom severity (CGI-C and PGI-C) [Time Frame: DB up to Week 40; DB and OLE up to Week 100], Change in QoL from baseline as measured with Amyotrophic Lateral Sclerosis Assessment Questionnaire -5 (ALSAQ-5), EuroQoL 5 Dimension 5 Level (EQ-5D-5L), 12-item Short form health survey (SF-12) [Time Frame: DB up to Week 40; DB and OLE up to Week 100], PK in serum and CSF and Anti-VHB937 antibodies (ADAs) in serum [Time Frame for serum: DB up to Week 40, DB and OLE up to Week 100; DB and OLE up to last scheduled visit; For CSF: DB Week 12]

Related papers

No related papers found yet.

Interventions

DRUGVHB937

DRUGPlacebo 00 mg/ 00mL

DRUGconcentrate for solution for infusion (Placebo to VHB937)

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS) [Time Frame: Baseline to DB Week 40]	—

Secondary

Measure	Time frame
ALSFRS-R total score [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)], Slow Vital Capacity (SVC) (% of predicted normal value) [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)], Ratio to baseline in Neurofilament Light (NfL) concentration in serum [Timeframe: DB up to Week 40; DB and OLE up to Week 100], Safety and tolerability parameters including adverse events, laboratory data, vital signs, electrocardiogram (ECG), Columbia Suicide Severity Rating Scale (C-SSRS) [Time Frame: Baseline to DB Week 40, Baseline to OLE Week 100, and Baseline to last scheduled visit or end of study], Time to death and Time to event (death or PAV, whichever comes first). [Timeframe: Baseline to DB Week 40], Time to death and Time to event (death or PAV, whichever comes first) – endpoints ref	—

Measure

Time frame

ALSFRS-R total score [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)], Slow Vital Capacity (SVC) (% of predicted normal value) [Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first); Baseline to OLE Week 100 or until death or PAV (whichever occurs first)], Ratio to baseline in Neurofilament Light (NfL) concentration in serum [Timeframe: DB up to Week 40; DB and OLE up to Week 100], Safety and tolerability parameters including adverse events, laboratory data, vital signs, electrocardiogram (ECG), Columbia Suicide Severity Rating Scale (C-SSRS) [Time Frame: Baseline to DB Week 40, Baseline to OLE Week 100, and Baseline to last scheduled visit or end of study], Time to death and Time to event (death or PAV, whichever comes first). [Timeframe: Baseline to DB Week 40], Time to death and Time to event (death or PAV, whichever comes first) – endpoints ref

—

Countries

Belgium, Denmark, Finland, France, Germany, Ireland, Italy, Netherlands, Poland, Spain, Sweden

Outcome results

None listed