Amyotrophic Lateral Sclerosis (ALS)Phase 2UNITE-ALSNCT07023835
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
A Phase 2 study testing 50 mg Usnoflast and 75 mg Usnoflast in people with Amyotrophic Lateral Sclerosis (ALS), run by Zydus Therapeutics Inc. at 49 sites in 12 countries.
Trial summary
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- 50 mg Usnoflast and 75 mg Usnoflast
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- 49 sitesin 12 countries
- Study dates
- 17 Sep 2025to Oct 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of probable or definite Amyotrophic lateral sclerosis, according to the revised version of the El Escorial World Federation of Neurology criteria
- Time since onset of first symptom of Amyotrophic lateral sclerosis ≤24 months. Date of Amyotrophic lateral sclerosis symptom onset. For the purposes of this study, the date of symptom onset will be defined as the date the subject first had symptoms of their disease, i.e., limb weakness, dysarthria, dysphagia, shortness of breath, or fasciculations, from the screening visit.
- Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised score of ≥35 at screening.
- Slow vital capacity: ≥60% of predicted capacity at the screening visit.
- Be able to swallow capsules.
- Either not currently receiving riluzole/sodium phenylbutyrate and taurursodiol/tofersen or on a stable dose of riluzole/sodium phenylbutyrate and taurursodiol/tofersen for at least 4 weeks before the screening visit. Subjects receiving riluzole/sodium phenylbutyrate and taurursodiol/tofersen are expected to remain on the same dose throughout the duration of the study.
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- Either not currently receiving edaravone or on edaravone treatment. Subjects receiving edaravone must have completed at least 1 cycle of treatment before the screening visit and are expected to continue with a stable dose of edaravone treatment throughout the duration of the study.
- Capable of providing informed consent and complying with study procedures in the opinion of the investigator
- Completion in the randomized, double blind Usnoflast study (main study).
- Subjects who elect to continue treatment after completion of Usnoflast phase 2b study must enrol in the OLE within 28 days of the completion of Week 36 visit of the main study.
- Provide a new informed consent to enter the OLE phase.
Who cannot join
- Presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse that would impair the ability of the subject to provide informed consent, in the opinion of the investigator.
- Serious illness (e.g., pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with intravenous antibiotics, antivirals, or antifungals within 4 weeks of screening; chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the judgment of the investigator.
- Active herpes zoster infection within 2 months prior to the screening visit.
- Any medical condition that promotes suicidal attempt or behavior within 6 months prior to the screening visit and in the opinion of the investigator might interfere with subject's participation in the study or is a risk for a suicide attempt.
- History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or active cancer or another medically significant illness other than Amyotrophic lateral sclerosis, precluding safe participation of subject in this study in the opinion of the investigator.
- Known allergy, sensitivity, or intolerance to Investigational product or excipients.
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- Subjects who have taken concomitant medications that are substrates of drug metaboliz-ing enzymes (Cytochrome P450 1A2 and/or Cytochrome P450 2B6) within 7 days or 5 half-lives of the medication (whichever is longer) before the first dose of Investigational product and throughout the study.
- 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 the first dose of Investigational product administration.
- Use of any investigational drug concurrently or within 4 weeks or 5 half-lives (whichever is longer) prior to the first dose of Investigational product administration.
- Any clinically significant condition and/or laboratory significant value that would prevent the subject from participating in the study in the opinion of the investigator.
- Received a live vaccine within 14 days before the screening visit or planning to receive during the study duration.
- Subjects who have received stem cell or gene therapy for Amyotrophic lateral sclerosis at any time in the past.
- Following laboratory test values at screening:
- Alanine aminotransferase or Aspartate aminotransferase values >3.0 × Upper Limit of Normal
- Bilirubin >1.5 × Upper Limit of Normal unless the subject has documented Gilbert's syndrome (isolated bilirubin >1.5 × Upper Limit of Normal is acceptable if bilirubin is fractionated, and direct bilirubin is <35%)
- Estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2
- For those participating in the optional Cerebrospinal fluid collection, contraindications to lumbar puncture including but not limited to lumbar scoliosis, coagulopathy, infection at site of puncture, or use of anticoagulants.
- Subjects with history of epilepsy within 6 months of screening visit.
- Surgery within last 3 months or planned major surgery within next 3 months from the date of screening (other than minor cosmetic surgery and minor dental surgery).
- Use or intended use of any medications/products known to alter drug absorption, metabolism, or elimination processes, including St. John's Wort, within 4 weeks of screening and up to end of study. Use of such medication will be considered on a case-by-case basis as per the opinion of the investigator and/or independent medical monitor.
- Receiving an elemental diet or parenteral nutrition.
- Received blood transfusion within 3 months prior to screening.
- Subjects with Human immunodeficiency virus, hepatitis B, hepatitis C, coronary artery disease, or active gastrointestinal condition that might interfere with drug absorption.
- Inability to be venipunctured or those not able to tolerate venous puncture.
- Employee of the investigator or study site, with direct involvement in the proposed study or other studies under the direction of that investigator or study site, as well as family members of employees of investigator or the investigator.
- Any condition not mentioned in any of above criteria that, as per the investigator, would hinder participation of the subject in the study. This may include, but not limited to, considerations of safety, compliance, or other factors that could impact the integrity of the study or the well-being of the subject.
- If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of Investigational product. If male of reproductive capacity, unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of Investigational product.
- For Open Label Extension
- Discontinued IP prematurely in the double-blind phase of the study for reasons other than tracheostomy or permanent-assisted ventilation.
- Treatment with or use of any restricted medications.
- Any ongoing AE that, in the opinion of the site investigator, is clear contraindication to the IP.
- Unstable cardiac or other life-threatening disease emergent during the randomized, double-blind study
- Any major medical history or other evidence of severe illness or any other conditions that would make the subject, in the opinion of the investigator, unsuitable for the study.
- If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of IP. If male of reproductive capacity, unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of IP.
Trial locations and contacts
49 sites in 12 countries. Contact the study team or a site near you to ask about taking part.
49 sites in 12 countries
United States15 sites, 15 recruiting
- Recruiting
Zydus US007
Atlanta, Georgia, 30322
Jane Bordeau, JrBord@emory.edu
- Recruiting
Zydus US010
Boston, Massachusetts, 02114
Kyle Molinari, usnoflasthealey@mgb.org
- Recruiting
Zydus US001
Dallas, Texas, 75206
Reham Azab, razab@texasneurology.com
- Recruiting
Zydus US006
Detroit, Michigan, 48202
Kelly Tundo, KCIACH1@hfhs.org
- Recruiting
Zydus US002
Houston, Texas, 77030
Kimberly Esparaza, houneukesparza@outlook.com
- Recruiting
Zydus US015
La Jolla, California, 92037
Rosemarie Previte, rprevite@health.ucsd.edu
- Recruiting
Zydus US014
Lincoln, Nebraska, 68510
Veronica Botti, veronica@somnos.com
Gary Pattee
- Recruiting
Zydus US005
New Britain, Connecticut, 06053
Honora C Dalamagas, hdalamagas@hfsc.org
- Not yet recruiting
Zydus US008
Orange, California, 92868
Jeein Kim, jeeink2@hs.uci.edu
- Recruiting
Zydus US009
Pittsburgh, Pennsylvania, 15212
Megan Hendricks, Megan.Hendricks@ahn.org
- Recruiting
Zydus US004
Richmond, Virginia, 23298
Adriana Clegg, adriana.clegg@vcuhealth.org
- Recruiting
Zydus US013
San Francisco, California, 94109
Valentina Mikhalenko, Valentina.Mikhalenko@sutterhealth.org
- Recruiting
Zydus US011
Seattle, Washington, 98122
Kelly Robertson, Kelly.Robertson@Swedish.org
- Recruiting
Zydus US012
Tampa, Florida, 80045
Jamie Reddish, jreddish@usf.edu
- Recruiting
Zydus US003
Winston-Salem, North Carolina, 27157
Mozdeh Mirandi, mozhdeh.marandi@advocatehealth.org
France6 sites, 6 recruiting
- Not yet recruiting
Zydus 124
Clermont-Ferrand, France
Sophia Sickout Arondo, ssickoutarondo@chu-clermontferrand.fr
- Not yet recruiting
Zydus 122
Limoges, France
Clemence Labetoulle, clemence.labetoulle@chu-limoges.fr
- Recruiting
Zydus 123
Montpellier, France
Sebastien Alphandery, s-alphandery@chu-montpellier.fr
- Recruiting
Zydus 125
Nice, France
Carole Barré, barre.c2@chu-nice.fr
- Recruiting
Zydus 120
Paris, France
Nadia Osman, nadia.osman@icm-institute.org
- Recruiting
Zydus 121
Tours, France
Amélie Legrand, a.legrand@chu-tours.fr
Germany6 sites, 6 recruiting
- Not yet recruiting
Zydus 130
Dresden, Germany
Marian Kollaske, Marian.Kollaske@ukdd.de
- Not yet recruiting
Zydus 133
Hanover, Germany
Michaela Renke, Michaela.renke@diakovere.de
- Not yet recruiting
Zydus 134
Jena, Germany
Anna-Maria Rähse, Anna-Maria.Raehse@med.uni-jena.de
- Not yet recruiting
Zydus 131
Lübeck, Germany
Janina von der Gablentz, janina.gablentz@uni-luebeck.de
- Not yet recruiting
Zydus 132
Rostock, Germany
Sophie Fischer, Sophie.Fischer@med.uni-rostock.de
- Not yet recruiting
Zydus 135
Ulm, Germany
Svetlana Götz, svetlana.goetz@uniklinik-ulm.de
Italy5 sites, 5 recruiting
- Recruiting
Zydus 150
Brescia
Chiara Colombi, chiara.colombi@centrocliniconemo.it
- Not yet recruiting
Zydus 154
Genova, Italy
Carlotta Gennaro, carlotta.gennaro@hsanmartino.it
- Not yet recruiting
Zydus 153
Milan, Italy
Claudia Panciroli, panciroli.claudia@hsr.it
- Not yet recruiting
Zydus 151
Modena, Italy
Giulia Gianferrari, gianferrari.giulia@aou.mo.it
- Recruiting
Zydus 152
Naples, Italy
Lucia Aruta, lucia.aruta@unina.it
Spain4 sites, 4 recruiting
- Not yet recruiting
Zydus 180
Alicante, Spain
Alexandra Muñoz Ambit, florence.sm54@gmail.com
- Not yet recruiting
Zydus 181
Barcelona, Spain
Cristina Terrafeta Pastor, cterrafeta@idibell.cat
- Not yet recruiting
Zydus 182
Málaga, Spain
Adrian Roldán, adrianroldan.neurologia@gmail.com
- Recruiting
Zydus 183
Valencia, Spain
Silvia Perez y Albertos, silvia_perez@iislafe.es
Australia3 sites, 3 recruiting
- Recruiting
Zydus 200
Caulfield South, Australia
Nicole Panerio, nicole.panerio@calvarycare.org.au
- Not yet recruiting
Zydus 202
Southport
Vincent Sapaen, Vincent.sapaen@health.qld.gov.au
- Recruiting
Zydus 201
Sydney, New South Wales
Richard Gan, richard.gan@mq.edu.au
Sweden3 sites, 3 recruiting
- Recruiting
Zydus 191
Malmö, Sweden
Jonas Näslund, jonas.naslund@skane.se
- Not yet recruiting
Zydus 190
Stockholm, Sweden
Charlotta Molin Edlund, charlotta.molin-edlund@regionstockholm.se
- Not yet recruiting
Zydus 192
Umeå, Sweden
Lena Bylund, lena.bylund@regionvasterbotten.se
Canada2 sites, 1 recruiting
- Active, not recruiting
Zydus 100
Québec, Quebec, QC H4A 3T2
- Not yet recruiting
Zydus 101
Toronto, Ontario, ON M4N 3M5
Anita Seghatoleslam, masoumeh.seghatoleslam@sri.utoronto.ca
Poland2 sites, 2 recruiting
- Not yet recruiting
Zydus 170
Krakow, Poland
Anna Soltysiak, anna.soltysiak@cmlinden.com
- Not yet recruiting
Zydus 171
Warsaw, Poland
Wioleta Drózd, wioleta.drozd@neuroprotect.pl
Belgium1 site, 1 recruiting
- Not yet recruiting
Zydus 110
Leuven, Belgium
Ann D'hondt, ann.dhondt@uzleuven.be
Ireland1 site, 1 recruiting
- Not yet recruiting
Zydus 140
Dublin, Ireland
Toyosi Atoyebi, toyosiatoyebi@rcsi.com
Netherlands1 site, 1 recruiting
- Recruiting
Zydus 160
Utrecht, Netherlands
Tommy Bunte, T.Bunte-3@umcutrecht.nl
About this study
Official title: A Phase 2b, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Usnoflast Administered to Adult Subjects With ALS
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
Read the detailed description
A phase 2b, randomized, double-blind, placebo-controlled, parallel-group, multicenter 36 weeks study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis followed by 16 weeks open label extension study.
This Open Label Extension will be a multicenter, 16-week, single arm study to confirm the long-term safety and efficacy of Usnoflast in subjects with ALS. Eligible subjects of all three arms of the main study will be recruited in the OLE phase and will receive Usnoflast (75 mg) for a total of 16 weeks BID (oral capsule administration).
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- 50 mg Usnoflast, 75 mg Usnoflast, Placebo
- Keywords
- Amyotrophic Lateral Sclerosis,, ALS, Usnoflast
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
50 mg UsnoflastDrug
Also called Not any
50 mg Usnoflast (50 mg Usnoflast capsules and matching placebo of 25 mg capsule)
75 mg UsnoflastDrug
Also called Not any
75 mg Usnoflast (25 mg + 50 mg Usnoflast capsules)
PlaceboDrug
Also called Not any
Matching placebo of 25 mg and 50 mg
| Study group | Role | What they receive |
|---|---|---|
| 50 mg Usnoflast | Active comparator | 50 mg Usnoflast capsules and matching placebo of 25 mg capsule under fasting conditions twice a day orally for 36 weeks Receives: Drug: 50 mg Usnoflast |
| 75 mg Usnoflast | Active comparator | 25 mg + 50 mg Usnoflast capsules under fasting conditions twice a day orally for 36 weeks Receives: Drug: 75 mg Usnoflast |
| Placebo | Placebo comparator | Matching placebo of 25 mg and 50 mg under fasting conditions twice a day orally for 36 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of Usnoflast versus placebo assessed using the revised ALSFRS-R total score
Change in disease progression from baseline through Week 36 as measured by ALSFRS-R total score
Measured: From baseline through Week 36
Efficacy of Usnoflast versus placebo assessed using the survival
Change in disease progression from baseline through Week 36 as measured by survival
Survival is defined based on the time of death or permanent-assisted ventilation (PAV). PAV is defined as the use of invasive or noninvasive mechanical ventilation for >22 hours daily for >7 consecutive days
Measured: From baseline through Week 36
Effect of Usnoflast versus placebo on survival in Open label extension phase
Time from baseline to the occurrence of death or Permanent-assisted ventilation (>22 hours daily for >7 days)
Measured: From baseline through Week 16
Number of participants with treatment emergent adverse events in open label extension
Time from baseline
Measured: From baseline through Week 16
Number of participants with Serious adverse events in open label extension
Time from baseline
Measured: From baseline through Week 16
Secondary outcomes (18)
Effect of Usnoflast versus placebo on survival
Time from baseline to the occurrence of death or Permanent-assisted ventilation (>22 hours daily for >7 days)
Measured: From baseline through Week 36
Evaluate the effect of Usnoflast versus placebo on Slow vital capacity
Change in Slow vital capacity from baseline to Week 36
Measured: from baseline to Week 36
Evaluate the effect of Usnoflast versus placebo on serum levels of Neurofilament light chain protein
Change in serum levels of Neurofilament light chain protein from baseline to Week 36
Measured: From baseline to Week 36
Evaluate the effect of Usnoflast versus placebo on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score and various functional items/domains of the ALSFRS-R total score
To determine the rate of disease progression using the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score and scores of various functional items/domains of the ALSFRS-R total score from baseline to Week 36
Measured: From baseline to Week 36
Evaluate and compare the effect of Usnoflast versus placebo on overall health-related quality of life
Change in Amyotrophic Lateral Sclerosis assessment questionnaire-40 (ALSAQ-40) score from baseline to Week 36
Measured: From baseline to Week 36
Number of participants with treatment emergent adverse events
Time from baseline
Measured: From baseline to Week 36
Number of participants with Serious adverse events
Time from baseline
Measured: From baseline to Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Peak steady state plasma concentration (Cmax,ss)
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time to reach peak plasma concentration at steady state (Tmax,ss)
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Accumulation index calculated as a ratio of AUCtau (last dose)/AUCtau (first dose)
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Area under Plasma concentration vs. time curve till the last time point: AUC0-t
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Area under Plasma concentration vs. time curve extrapolated to the infinity: AUC0-∞
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Area under Plasma concentration vs. time curve based on extrapolation: AUCextap
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Elimination rate constant (Kel)
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Elimination half-life (t1/2)
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in Cerebrospinal fluid
The concentration of Usnoflast in CSF.
Measured: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Effect of Usnoflast versus placebo on Cerebrospinal fluid levels of Neurofilament light chain protein
Change in Cerebrospinal fluid levels of Neurofilament light chain protein
Measured: From baseline to Week 36
To determine the rate of disease progression in open label extension
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score and scores of various functional items/domains of the ALSFRS-R total score from baseline to Week 16
Measured: From baseline to Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2025
- Study start17 Sep 2025
- Primary completionMar 2028 (estimated)
- Study completionOct 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.
- 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
- Randomized, double-blind, placebo-controlled, multicenter
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zydus Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Deven V Parmar, Study director, Zydus Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07023835
- Sponsor's protocol ID
- USNO.24.002
- Acronym
- UNITE-ALS
- Status last verified
- Jul 2026
- First submitted
- 2 Jun 2025
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Usnoflast Administered to Adult Subjects With ALS
- ClinicalTrials.gov ID
- NCT07023835
- Acronym / sponsor ID
- UNITE-ALS / USNO.24.002
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis,, ALS, Usnoflast
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 240 participants (estimated)
- Lead sponsor
- Zydus Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Sep 2025 (actual)
- Primary completion
- Mar 2028 (estimated)
- Study completion
- Oct 2028 (estimated)
- First posted
- 17 Jun 2025 (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.
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
Detailed description
A phase 2b, randomized, double-blind, placebo-controlled, parallel-group, multicenter 36 weeks study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis followed by 16 weeks open label extension study.
This Open Label Extension will be a multicenter, 16-week, single arm study to confirm the long-term safety and efficacy of Usnoflast in subjects with ALS. Eligible subjects of all three arms of the main study will be recruited in the OLE phase and will receive Usnoflast (75 mg) for a total of 16 weeks BID (oral capsule administration).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Randomized, double-blind, placebo-controlled, multicenter
| Arm | Type | Receives |
|---|---|---|
| 50 mg Usnoflast | Active comparator | Drug: 50 mg Usnoflast50 mg Usnoflast capsules and matching placebo of 25 mg capsule under fasting conditions twice a day orally for 36 weeks |
| 75 mg Usnoflast | Active comparator | Drug: 75 mg Usnoflast25 mg + 50 mg Usnoflast capsules under fasting conditions twice a day orally for 36 weeks |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo of 25 mg and 50 mg under fasting conditions twice a day orally for 36 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Diagnosis of probable or definite Amyotrophic lateral sclerosis, according to the revised version of the El Escorial World Federation of Neurology criteria
- Time since onset of first symptom of Amyotrophic lateral sclerosis ≤24 months. Date of Amyotrophic lateral sclerosis symptom onset. For the purposes of this study, the date of symptom onset will be defined as the date the subject first had symptoms of their disease, i.e., limb weakness, dysarthria, dysphagia, shortness of breath, or fasciculations, from the screening visit.
- Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised score of ≥35 at screening.
- Slow vital capacity: ≥60% of predicted capacity at the screening visit.
- Be able to swallow capsules.
Key exclusion criteria (34 listed)
- Presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse that would impair the ability of the subject to provide informed consent, in the opinion of the investigator.
- Serious illness (e.g., pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with intravenous antibiotics, antivirals, or antifungals within 4 weeks of screening; chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the judgment of the investigator.
- Active herpes zoster infection within 2 months prior to the screening visit.
- Any medical condition that promotes suicidal attempt or behavior within 6 months prior to the screening visit and in the opinion of the investigator might interfere with subject's participation in the study or is a risk for a suicide attempt.
- History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or active cancer or another medically significant illness other than Amyotrophic lateral sclerosis, precluding safe participation of subject in this study in the opinion of the investigator.
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 |
|---|---|---|
| 50 mg UsnoflastAlso: Not any50 mg Usnoflast (50 mg Usnoflast capsules and matching placebo of 25 mg capsule) | Drug | 50 mg Usnoflast |
| 75 mg UsnoflastAlso: Not any75 mg Usnoflast (25 mg + 50 mg Usnoflast capsules) | Drug | 75 mg Usnoflast |
| PlaceboAlso: Not anyMatching placebo of 25 mg and 50 mg | Drug | Placebo |
Primary outcome
Efficacy of Usnoflast versus placebo assessed using the revised ALSFRS-R total score
Time frame: From baseline through Week 36
Change in disease progression from baseline through Week 36 as measured by ALSFRS-R total score
Efficacy of Usnoflast versus placebo assessed using the survival
Time frame: From baseline through Week 36
Change in disease progression from baseline through Week 36 as measured by survival Survival is defined based on the time of death or permanent-assisted ventilation (PAV). PAV is defined as the use of invasive or noninvasive mechanical ventilation for >22 hours daily for >7 consecutive days
Effect of Usnoflast versus placebo on survival in Open label extension phase
Time frame: From baseline through Week 16
Time from baseline to the occurrence of death or Permanent-assisted ventilation (>22 hours daily for >7 days)
Number of participants with treatment emergent adverse events in open label extension
Time frame: From baseline through Week 16
Time from baseline
Number of participants with Serious adverse events in open label extension
Time frame: From baseline through Week 16
Time from baseline
18 secondary outcomes
Effect of Usnoflast versus placebo on survival
Time frame: From baseline through Week 36
Evaluate the effect of Usnoflast versus placebo on Slow vital capacity
Time frame: from baseline to Week 36
Evaluate the effect of Usnoflast versus placebo on serum levels of Neurofilament light chain protein
Time frame: From baseline to Week 36
Evaluate the effect of Usnoflast versus placebo on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score and various functional items/domains of the ALSFRS-R total score
Time frame: From baseline to Week 36
Evaluate and compare the effect of Usnoflast versus placebo on overall health-related quality of life
Time frame: From baseline to Week 36
Number of participants with treatment emergent adverse events
Time frame: From baseline to Week 36
Number of participants with Serious adverse events
Time frame: From baseline to Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in plasma
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Evaluate Pharmacokinetic of Usnoflast in Cerebrospinal fluid
Time frame: PK time pts: pre-dose, 2, 4, 6, 8 hours ±30 mins post morning dose at Day 1, Week 16, and Week 36
Effect of Usnoflast versus placebo on Cerebrospinal fluid levels of Neurofilament light chain protein
Time frame: From baseline to Week 36
To determine the rate of disease progression in open label extension
Time frame: From baseline to Week 16
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 1 Sep 2026.
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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.
Deven V Parmar study director
Zydus Therapeutics Inc.
49 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 50 mg Usnoflast and 75 mg Usnoflast in people with Amyotrophic Lateral Sclerosis (ALS), run by Zydus Therapeutics Inc. at 49 sites in 12 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 11 inclusion and 34 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: looking for participants now, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 49 sites in United States, France, Germany, Italy, Spain and 7 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?
Contact Farheen Shaikh (6094534751, fshaikh@zydustherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of Usnoflast versus placebo assessed using the revised ALSFRS-R total score (time frame: From baseline through Week 36). The study lists 5 primary outcomes in all. 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 1 Sep 2026.