A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)
Tipo di studio
Interventional
(ICTRP)
Intervento studiato
Biological: VHB937Other: Placebo
(ICTRP)
Malattie studiate
Amyotrophic Lateral Sclerosis (ALS)
(ICTRP)
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.
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 (ICTRP)
Criteri di esclusione
non disponibile
Luogo dello studio
Stati Uniti, Australia, Belgio, Canada, China, Dänemark, Francia, Germania, Irlanda, Italia, Paesi Bassi, Polonia, , Spagna, Svezia, Svizzera, Regno Unito, Finnland, Giappone
(ICTRP)
Informazioni generali
Novartis Pharmaceuticals
(ICTRP)
Titolo accademico
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) (ICTRP)
Disegno dello studio
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor). (ICTRP)
Endpoint primari e secondari
The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS) (ICTRP)
ALS Functional Rating Scale Revised (ALSFRS-R) total score;Slow Vital Capacity (SVC) (% of predicted normal value);Ratio to baseline in Neurofilament Light (NfL) concentration in serum;Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs);Time to death and Time to event (death or PAV, whichever comes first).;Time to death and Time to event (death or PAV, whichever comes first) - endpoints referring to treatment policy estimand;Patient Global Impression of change in functional ability and ALS symptom severity (PGI-C);Change in QoL from baseline as measured with Amyotrophic Lateral Sclerosis Assessment Questionnaire -5 (ALSAQ-5);Change in Clinician Global Impression of change in functional ability and ALS symptom severity (CGI-C);Change in QoL from baseline as measured with EuroQoL 5 Dimension 5 Level (EQ-5D-5L);Change in QoL from baseline as measured with 12-item Short form health survey (SF-12);Pharmacokinetics (PK) of VHB937-CMAX;Pharmacokinetics (PK) of VHB937-TMAX;Pharmacokinetics (PK) of VHB937-CTROUGH;To assess immunogenicity (IG) of VHB937;Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CMAX;Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-TMAX;Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CTROUGH (ICTRP)
Data di registrazione
non disponibile
Inclusione del primo partecipante
non disponibile
Sponsor secondari
non disponibile
Contatti aggiuntivi
Novartis PharmaceuticalsNovartis Pharmaceuticals, novartis.email@novartis.com, 1-888-669-6682, Novartis Pharmaceuticals (ICTRP)
ID secondari
2024-512536-29-00, CVHB937B12201 (ICTRP)
Risultati-Dati individuali dei partecipanti
non disponibile
Ulteriori informazioni sullo studio
https://clinicaltrials.gov/study/NCT06643481 (ICTRP)
Risultati dello studio
Riepilogo dei risultati
non disponibile
Link ai risultati nel registro primario
non disponibile