Health Condition 1: G121- Other inherited spinal muscular atrophy
Conditions
Interventions
Intervention1: ZYIL1 Capsules: Route :- Oral
Strength : 25 and 50 mg
Duration : 56 days
Control Intervention1: Placebo of ZYIL1 Capsules: Route :- Oral
Strength : 25 and 50 mg Duration : 56 days
Sponsors
Zydus Lifesciences Limited
Eligibility
Inclusion criteria
Inclusion criteria: 1.Male and/or female patients aged between 18 and 80 years (inclusive at screening). 2. Diagnosis of probable or definite ALS, according to the revised version of the El Escorial World Federation of Neurology criteria. Refer Appendix III - El Escorial Criteria. 3. Time since onset# of first symptom of ALS =9 months 4. Slow Vital Capacity (SVC) = 50% of the predicted value 5. Be able to swallow the study capsules during study 6. Either not currently receiving riluzole or on a stable dose of riluzole for at least 4 weeks before the screening visit. Participants receiving riluzole are expected to remain on the same dose throughout the duration of the study 7. Either not currently receiving edaravone or on edaravone treatment. Participants receiving edaravone must have completed at least 1 cycle of treatment before the screening visit and are expected to continue with stable dose edaravone treatment throughout the duration of the study
Exclusion criteria
Exclusion criteria: 1)With significant cognitive impairment, psychiatric disease, other neurodegenerative disorder (e.g., Parkinson disease or AD), substance abuse other causes of neuromuscular weakness, or any other condition that would make the participants unsuitable for participating in the study or could interfere with assessment or completing the study in the opinion of the Investigator 2. History of recent serious infection (e.g., pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with IV antibiotics, antivirals, or antifungals within 4 weeks of screening; or chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the Investigator’s judgment 3. With active herpes zoster infection within 2 months prior to the screening visit 4. A documented history of attempted suicide within 6 months prior to the screening visit, or in the Investigator’s judgment are at risk for a suicide attempt 5. History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or another medically significant illness other than ALS precluding their safe participation in this study 6. Participants who are pregnant or are currently breastfeeding 7. A known history of allergy to any ingredients of ZYIL1 8. Patients taking concomitant medicines within 7 days or 5 half-lives of the medication (whichever is longer) prior to first dose of study drug administration till end of the study, which are substrate of CYP1A2 enzymes (e.g., alosetron, caffeine, duloxetine, melatonin, ramelteon, tasimelteon, tizanidine etc.) and CYP2B6 enzymes (e.g., bupropion, efavirenz etc.). 9. Use of any steroids, colchicine or anti-IL-1 inhibitors within 7 days or 5 half-lives of the medication (whichever is longer) prior to first dose of study drug administration. 10. Use of any investigational drugs concurrently or within 4 weeks or 5 half-lives (whichever is longer), prior to first dose of study drug administration. 11. Any clinically significant and/or laboratory significant value or other instability that would prevent the patient from participating in the study as determined by the Investigator. 12. Received a live vaccine within 14 days before the screening visit or planning to receive during the study 13. Participants who have received stem cell or gene therapy for ALS at any time in the past
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To measure the efficacy of the treatment using the slope of progression with the revised Amyotrophic Lateral Sclerosis Functional Rating ScaleTimepoint: Baseline to Day 84 | — |
Secondary
| Measure | Time frame |
|---|---|
| safety & tolerability of ZYIL1 in patients with ALSTimepoint: Baseline to Day 84;evaluate pharmacokinetics of ZYIL1Timepoint: Baseline to Day 84;evaluate effect of ZYIL1 on neurofilament biomarkerTimepoint: Baseline to Day 84 | — |
Countries
India
Contacts
Public ContactDr Bhargav Darji
Zydus lifesciences Ltd (Formerly Cadila Healthcare Ltd.)
Outcome results
None listed