A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)
Type d'essai
Interventional
(ICTRP)
Intervention étudiée
Biological: VHB937Other: Placebo
(ICTRP)
Maladie en cours d'investigation
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)
Critères d'exclusion
non disponible
Lieu de l’étude
États-Unis, Australie, Belgique, Canada, Chine, Danemark, France, Allemagne, Irlande, Italie, Pays-Bas, Pologne, , Espagne, Suède, Suisse, Royaume-Uni, Finlande, Japon
(ICTRP)
Informations générales
Novartis Pharmaceuticals
(ICTRP)
Titre académique
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)
Plan de l'étude
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor). (ICTRP)
Critères d'évaluation principaux et secondaires
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)
Date d'enregistrement
non disponible
Inclusion du premier participant
non disponible
Sponsors secondaires
non disponible
Contacts supplémentaires
Novartis PharmaceuticalsNovartis Pharmaceuticals, novartis.email@novartis.com, 1-888-669-6682, Novartis Pharmaceuticals (ICTRP)
ID secondaires
2024-512536-29-00, CVHB937B12201 (ICTRP)
Résultats-Données individuelles des participants
non disponible
Informations complémentaires sur l'essai
https://clinicaltrials.gov/study/NCT06643481 (ICTRP)
Résultats de l'essai
Résumé des résultats
non disponible
Lien vers les résultats dans le registre primaire
non disponible