Amyotrophic Lateral Sclerosis (ALS)Phase 2ASTRALSNCT06643481
A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing VHB937 in people with Amyotrophic Lateral Sclerosis (ALS), run by Novartis Pharmaceuticals at 74 sites in 17 countries.
Trial summary
This is a multicenter, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early-stage ALS (within 2 years of ALS symptoms onset). Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- VHB937
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 251 participants
- Where
- 74 sitesin 17 countries
- Study dates
- 17 Oct 2024to 10 Jul 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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
Trial locations and contacts
74 sites in 17 countries.
74 sites in 17 countries
United States21 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Austin Neuromuscular Center
Austin, Texas, 78759
Massachusetts General Hospital
Boston, Massachusetts, 02114
Atrium Health
Charlotte, North Carolina, 28207
Univ of Cincinnati Medical Center
Cincinnati, Ohio, 45219
The Ohio State University
Columbus, Ohio, 43210
Duke University Health System
Durham, North Carolina, 27710
Nerve and Muscle Center of Texas
Houston, Texas, 77030
University Of Kansas Medical Center
Kansas City, Kansas, 66160
AMR Knoxville
Knoxville, Tennessee, 37920
University of California San Diego
La Jolla, California, 92037
Loma Linda University Health
Loma Linda, California, 92354
Keck Medical Center USC
Los Angeles, California, 90033
University of Miami
Miami, Florida, 33136
Lange Neurology PC
New York, New York, 10065
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Orlando Health Clinical Trials
Orlando, Florida, 32806
Temple University
Philadelphia, Pennsylvania, 19140
University of Rochester Medical Center
Rochester, New York, 14642
UC San Francisco Medical Center
San Francisco, California, 94143-0348
University of Washington Medical Center
Seattle, Washington, 98195
Germany9 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Hanover, 30559
Novartis Investigative Site
Lübeck, 23538
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, 68167
Novartis Investigative Site
Munich, Bavaria, 81675
Novartis Investigative Site
Münster, 48149
Novartis Investigative Site
Rostock, Mecklenburg-Vorpommern, 18057
Novartis Investigative Site
Ulm, 89081
Novartis Investigative Site
Würzburg, Bavaria, 97080
France5 sites
Novartis Investigative Site
Bron, 69677
Novartis Investigative Site
Lille, 59037
Novartis Investigative Site
Nice, 06001
Novartis Investigative Site
Paris, 75013
Novartis Investigative Site
Tours, 37044
Poland5 sites
Novartis Investigative Site
Bydgoszcz, 85-163
Novartis Investigative Site
Krakow, Poland, 31 531
Novartis Investigative Site
Krakow, 30-721
Novartis Investigative Site
Warsaw, 01-684
Novartis Investigative Site
Warsaw, 02-473
United Kingdom5 sites
Novartis Investigative Site
Farnborough, BR6 8ND
Novartis Investigative Site
London, SW17 0QT
Novartis Investigative Site
London, WC1N 3BG
Novartis Investigative Site
Sheffield, South Yorkshire, S10 2JF
Novartis Investigative Site
Stoke-on-Trent, ST4 6QG
Australia4 sites
Novartis Investigative Site
Caulfield South, Victoria, 3162
Novartis Investigative Site
Herston, Queensland, 4029
Novartis Investigative Site
North Ryde, New South Wales, 2109
Novartis Investigative Site
Southport, 4215
Italy4 sites
Novartis Investigative Site
Milan, MI, 20138
Novartis Investigative Site
Modena, MO, 41126
Novartis Investigative Site
Pisa, PI, 56126
Novartis Investigative Site
Torino, TO, 10126
Spain4 sites
Novartis Investigative Site
Barcelona, 08035
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, 08907
Novartis Investigative Site
Santiago Compostela, A Coruna, 15706
Novartis Investigative Site
Valencia, 46026
South Korea3 sites
Novartis Investigative Site
Seoul, 04763
Novartis Investigative Site
Seoul, 05505
Novartis Investigative Site
Yangsan, Gyeongsangnam-do, 50612
Sweden3 sites
Novartis Investigative Site
Malmö, 214 28
Novartis Investigative Site
Stockholm, 113 61
Novartis Investigative Site
Umeå, SE-90185
Belgium2 sites
Novartis Investigative Site
Leuven, Vlaams Brabant, 3000
Novartis Investigative Site
Liège, 4000
Canada2 sites
Novartis Investigative Site
Calgary, Alberta, T2N 4N1
Novartis Investigative Site
Montreal, Quebec, H4A 3T2
Denmark2 sites
Novartis Investigative Site
Aalborg, 9000
Novartis Investigative Site
Kobenhavn N V, 2400
Switzerland2 sites
Novartis Investigative Site
Basel, 4031
Novartis Investigative Site
Sankt Gallen, 9007
About this study
Official 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)
This is a multicenter, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early-stage ALS (within 2 years of ALS symptoms onset). The study comprises a core double-blind (DB) 40-week treatment period followed by an open label extension (OLE).
Read the detailed description
The main questions this trial aims to answer in comparing VHB937 to placebo are:
- How long will participants live without needing permanent help from a machine to breathe after starting the trial treatment?
- What is the change in the participant's ability to perform daily activities? This will be measured using a questionnaire called the amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R).
- What adverse events are reported during this trial? An adverse event is any sign or symptom that participants have during a trial. Adverse events may or may not be caused by treatments in the trial. The trial doctors will check participants' ALS and general health throughout the trial.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- VHB937, Placebo
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, monoclonal antibody, motor neuron disease, MND
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VHB937Biological
VHB937 solution for infusion
PlaceboOther
Solution for infusion
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | I.V. infusions Receives: Biological: VHB937 |
| Arm 2 | Placebo comparator | I.V. infusions Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS)
To compare the efficacy of VHB937 vs. placebo on a composite of permanent assisted ventilation (PAV) free survival and function in DB epoch
Measured: Baseline to DB Week 40
Secondary outcomes (18)
ALS Functional Rating Scale Revised (ALSFRS-R) total score
To assess the efficacy of VHB937 on functional decline in DB and OLE epochs.
Measured: 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
Slow Vital Capacity (SVC) (% of predicted normal value)
To assess the efficacy of VHB937 in delaying decline in respiratory function in DB and OLE epochs.
Measured: 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)
Ratio to baseline in Neurofilament Light (NfL) concentration in serum
To assess the effect of VHB937 on a biomarker of neurodegeneration in DB and OLE epochs.
Measured: DB up to Week 40; DB and OLE up to Week 100]
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
To assess the safety and tolerability of VHB937 in DB and OLE epochs.
Measured: Baseline to end of study
Time to death and Time to event (death or PAV, whichever comes first).
To assess the efficacy of VHB937 vs. placebo on survival endpoints in DB epoch.
Measured: Baseline to DB Week 40
Time to death and Time to event (death or PAV, whichever comes first) - endpoints referring to treatment policy estimand
To assess the efficacy of early vs. delayed VHB937 administration on survival endpoints (DB VHB937 followed by OLE VHB937 vs. DB placebo followed by OLE VHB937)
Measured: Baseline to OLE Week 100, and Baseline to end of study
Patient Global Impression of change in functional ability and ALS symptom severity (PGI-C)
To assess change in ALS condition in DB and OLE epochs.
Measured: 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)
To assess Quality of Life (QoL) with VHB937 in DB and OLE epochs.
Measured: DB up to Week 40; DB and OLE up to Week 100
Change in Clinician Global Impression of change in functional ability and ALS symptom severity (CGI-C)
To assess change in ALS condition in DB and OLE epochs.
Measured: DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with EuroQoL 5 Dimension 5 Level (EQ-5D-5L)
To assess Quality of Life (QoL) with VHB937 in DB and OLE epochs.
Measured: DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with 12-item Short form health survey (SF-12)
To assess Quality of Life (QoL) with VHB937 in DB and OLE epochs.
Measured: DB up to Week 40; DB and OLE up to Week 100
Pharmacokinetics (PK) of VHB937-CMAX
CMAX - The maximum concentration of VHB937 in serum
Measured: Day 1 to end of study
Pharmacokinetics (PK) of VHB937-TMAX
TMAX - The time to reach the maximum concentration of VHB937 in serum
Measured: Day 1 to end of study
Pharmacokinetics (PK) of VHB937-CTROUGH
CTROUGH - Minimum observed concentration of VHB937 in serum
Measured: Day 1 to end of study
To assess immunogenicity (IG) of VHB937
To assess immunogenicity of Anti-VHB937 antibodies in serum
Measured: Day 1 up to end of study
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CMAX
CMAX - The maximum concentration of VHB937 in CSF
Measured: Screening to Week 12
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-TMAX
TMAX - The time to reach the maximum concentration of VHB937 in CSF
Measured: Screening to Week 12
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CTROUGH
CTROUGH - Minimum observed concentration of VHB937 in CSF
Measured: Screening to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2024
- Study start17 Oct 2024
- Primary completion23 Jun 2026
- Study completion10 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-blind, Randomized 2:1 ratio
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 251 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06643481
- Sponsor's protocol ID
- CVHB937B12201
- Other IDs
- 2024-512536-29-00
- Acronym
- ASTRALS
- Status last verified
- Jul 2026
- First submitted
- 30 Sep 2024
- Last updated
- 7 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT06643481
- Acronym / sponsor ID
- ASTRALS / CVHB937B12201
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, monoclonal antibody, motor neuron disease, MND
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 251 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 17 Oct 2024 (actual)
- Primary completion
- 23 Jun 2026 (actual)
- Study completion
- 10 Jul 2028 (estimated)
- First posted
- 16 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early-stage ALS (within 2 years of ALS symptoms onset). The study comprises a core double-blind (DB) 40-week treatment period followed by an open label extension (OLE).
Detailed description
The main questions this trial aims to answer in comparing VHB937 to placebo are:
- How long will participants live without needing permanent help from a machine to breathe after starting the trial treatment?
- What is the change in the participant's ability to perform daily activities? This will be measured using a questionnaire called the amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R).
- What adverse events are reported during this trial? An adverse event is any sign or symptom that participants have during a trial. Adverse events may or may not be caused by treatments in the trial. The trial doctors will check participants' ALS and general health throughout the trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Double-blind, Randomized 2:1 ratio
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: VHB937I.V. infusions |
| Arm 2 | Placebo comparator | Other: PlaceboI.V. infusions |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VHB937VHB937 solution for infusion | Biological | Arm 1 |
| PlaceboSolution for infusion | Other | Arm 2 |
Primary outcome
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
To compare the efficacy of VHB937 vs. placebo on a composite of permanent assisted ventilation (PAV) free survival and function in DB epoch
18 secondary outcomes
ALS Functional Rating Scale Revised (ALSFRS-R) total score
Time frame: 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
Slow Vital Capacity (SVC) (% of predicted normal value)
Time frame: 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)
Ratio to baseline in Neurofilament Light (NfL) concentration in serum
Time frame: DB up to Week 40; DB and OLE up to Week 100]
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline to end of study
Time to death and Time to event (death or PAV, whichever comes first).
Time frame: 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
Patient Global Impression of change in functional ability and ALS symptom severity (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)
Time frame: DB up to Week 40; DB and OLE up to Week 100
Change in Clinician Global Impression of change in functional ability and ALS symptom severity (CGI-C)
Time frame: DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with EuroQoL 5 Dimension 5 Level (EQ-5D-5L)
Time frame: DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with 12-item Short form health survey (SF-12)
Time frame: DB up to Week 40; DB and OLE up to Week 100
Pharmacokinetics (PK) of VHB937-CMAX
Time frame: Day 1 to end of study
Pharmacokinetics (PK) of VHB937-TMAX
Time frame: Day 1 to end of study
Pharmacokinetics (PK) of VHB937-CTROUGH
Time frame: Day 1 to end of study
To assess immunogenicity (IG) of VHB937
Time frame: Day 1 up to end of study
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CMAX
Time frame: Screening to Week 12
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-TMAX
Time frame: Screening to Week 12
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CTROUGH
Time frame: Screening to Week 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
74 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VHB937 in people with Amyotrophic Lateral Sclerosis (ALS), run by Novartis Pharmaceuticals at 74 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 74 sites in United States, Germany, France, Poland, United Kingdom and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026.