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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06643481




Registration number
NCT06643481
Ethics application status
Date submitted
30/09/2024
Date registered
16/10/2024
Date last updated
24/06/2025

Titles & IDs
Public title
A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)
Scientific title
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)
Secondary ID [1] 0 0
2024-512536-29-00
Secondary ID [2] 0 0
CVHB937B12201
Universal Trial Number (UTN)
Trial acronym
ASTRALS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis (ALS) 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - VHB937
Other interventions - Placebo

Experimental: Arm 1 - I.V. infusions

Placebo comparator: Arm 2 - I.V. infusions


Treatment: Other: VHB937
VHB937 solution for infusion

Other interventions: Placebo
Solution for infusion
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS)
Timepoint [1] 0 0
Baseline to DB Week 40
Secondary outcome [1] 0 0
ALS Functional Rating Scale Revised (ALSFRS-R) total score
Timepoint [1] 0 0
Baseline to DB Week 40 or until death or PAV (whichever occurs first) and Baseline to OLE Week 100 or until death or PAV (whichever occurs first
Secondary outcome [2] 0 0
Slow Vital Capacity (SVC) (% of predicted normal value)
Timepoint [2] 0 0
Baseline to DB Week 40 or until death or PAV (whichever occurs first) and Baseline to OLE Week 100 or until death or PAV (whichever occurs first)
Secondary outcome [3] 0 0
Ratio to baseline in Neurofilament Light (NfL) concentration in serum
Timepoint [3] 0 0
DB up to Week 40; DB and OLE up to Week 100]
Secondary outcome [4] 0 0
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [4] 0 0
Baseline to end of study
Secondary outcome [5] 0 0
Time to death and Time to event (death or PAV, whichever comes first).
Timepoint [5] 0 0
Baseline to DB Week 40
Secondary outcome [6] 0 0
Time to death and Time to event (death or PAV, whichever comes first) - endpoints referring to treatment policy estimand
Timepoint [6] 0 0
Baseline to OLE Week 100, and Baseline to end of study
Secondary outcome [7] 0 0
Patient Global Impression of change in functional ability and ALS symptom severity (PGI-C)
Timepoint [7] 0 0
DB up to Week 40; DB and OLE up to Week 100
Secondary outcome [8] 0 0
Change in QoL from baseline as measured with Amyotrophic Lateral Sclerosis Assessment Questionnaire -5 (ALSAQ-5)
Timepoint [8] 0 0
DB up to Week 40; DB and OLE up to Week 100
Secondary outcome [9] 0 0
Change in Clinician Global Impression of change in functional ability and ALS symptom severity (CGI-C)
Timepoint [9] 0 0
DB up to Week 40; DB and OLE up to Week 100
Secondary outcome [10] 0 0
Change in QoL from baseline as measured with EuroQoL 5 Dimension 5 Level (EQ-5D-5L)
Timepoint [10] 0 0
DB up to Week 40; DB and OLE up to Week 100
Secondary outcome [11] 0 0
Change in QoL from baseline as measured with 12-item Short form health survey (SF-12)
Timepoint [11] 0 0
DB up to Week 40; DB and OLE up to Week 100
Secondary outcome [12] 0 0
Pharmacokinetics (PK) of VHB937-CMAX
Timepoint [12] 0 0
Day 1 to end of study
Secondary outcome [13] 0 0
Pharmacokinetics (PK) of VHB937-TMAX
Timepoint [13] 0 0
Day 1 to end of study
Secondary outcome [14] 0 0
Pharmacokinetics (PK) of VHB937-CTROUGH
Timepoint [14] 0 0
Day 1 to end of study
Secondary outcome [15] 0 0
To assess immunogenicity (IG) of VHB937
Timepoint [15] 0 0
Day 1 up to end of study
Secondary outcome [16] 0 0
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CMAX
Timepoint [16] 0 0
Screening to Week 12
Secondary outcome [17] 0 0
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-TMAX
Timepoint [17] 0 0
Screening to Week 12
Secondary outcome [18] 0 0
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CTROUGH
Timepoint [18] 0 0
Screening to Week 12

Eligibility
Key inclusion criteria
* are 18 years of age or older
* male or female, if of childbearing potential, strict contraception required
* have ALS confirmed by the trial doctors using different tests.
* have mild symptoms of ALS as measured by the ALSFRS-R questionnaire (total score >=30).
* have had symptoms of ALS (weakness) within 24 months of taking part in this trial.
* have not received treatment for ALS or are currently on a stable dose of an approved treatment for ALS.
* have the ability to slowly exhale a volume of air at least 60% of what is expected for the participant's sex, height and age.
Minimum age
18 Years
Maximum age
100 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Use of other investigational drugs within 5 half-lives of screening, or within 30 days (e.g., small molecules) / or until the expected pharmacodynamic effect has returned to baseline (e.g., biologics), whichever is longer; or longer if required by local regulations.
* Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 24 weeks after stopping study medication.
* History or current diagnosis of cardiac conditions or ECG abnormalities indicating significant risk of safety for participants in the study.
* Clinical evidence of liver or renal disease/injury.
* Laboratory evidence of hematological abnormalities
* Presence of unstable psychiatric disease, cognitive impairment, neurological disease other than ALS, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the investigator's opinion.
* Participants that reported 'yes' on any suicidal ideation section except for the "Non-Suicidal Self-Injurious Behavior" in the past 2 years as per C-SSRS.
* Presence of cancer, HIV, Hep B, Hep C, tuberculosis, uncontrolled diabetes
* History of active severe respiratory disease, including Chronic Obstructive Pulmonary Disease, interstitial lung disease or pulmonary fibrosis.
* Taking any prohibited medications

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/10/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
20/06/2028
Actual
Sample size
Target
225
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - North Ryde
Recruitment hospital [2] 0 0
Novartis Investigative Site - Randwick
Recruitment hospital [3] 0 0
Novartis Investigative Site - Herston
Recruitment hospital [4] 0 0
Novartis Investigative Site - Cauldfield
Recruitment hospital [5] 0 0
Novartis Investigative Site - SouthPort
Recruitment postcode(s) [1] 0 0
2109 - North Ryde
Recruitment postcode(s) [2] 0 0
2031 - Randwick
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
3162 - Cauldfield
Recruitment postcode(s) [5] 0 0
4215 - SouthPort
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Nebraska
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Tennessee
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Belgium
State/province [11] 0 0
Leuven
Country [12] 0 0
Belgium
State/province [12] 0 0
Liege
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
Canada
State/province [14] 0 0
Quebec
Country [15] 0 0
China
State/province [15] 0 0
Beijing
Country [16] 0 0
Denmark
State/province [16] 0 0
Aalborg
Country [17] 0 0
Denmark
State/province [17] 0 0
Kobenhavn N V
Country [18] 0 0
France
State/province [18] 0 0
Nice
Country [19] 0 0
France
State/province [19] 0 0
Tours
Country [20] 0 0
Germany
State/province [20] 0 0
Luebeck
Country [21] 0 0
Germany
State/province [21] 0 0
Munchen
Country [22] 0 0
Ireland
State/province [22] 0 0
Dublin
Country [23] 0 0
Japan
State/province [23] 0 0
Miyagi
Country [24] 0 0
Korea, Republic of
State/province [24] 0 0
Gyeongsangnam Do
Country [25] 0 0
Korea, Republic of
State/province [25] 0 0
Seoul
Country [26] 0 0
Netherlands
State/province [26] 0 0
Utrecht
Country [27] 0 0
Poland
State/province [27] 0 0
Bydgoszcz
Country [28] 0 0
Poland
State/province [28] 0 0
Krakow
Country [29] 0 0
Poland
State/province [29] 0 0
Warszawa
Country [30] 0 0
Spain
State/province [30] 0 0
Catalunya
Country [31] 0 0
Sweden
State/province [31] 0 0
Malmo
Country [32] 0 0
Sweden
State/province [32] 0 0
Umea
Country [33] 0 0
Switzerland
State/province [33] 0 0
Basel
Country [34] 0 0
Switzerland
State/province [34] 0 0
St Gallen
Country [35] 0 0
United Kingdom
State/province [35] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06643481