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A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

ConditionALS (Amyotrophic Lateral Sclerosis)
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18-65
SponsorShanghai Zhimeng Biopharma, Inc.

About this trial

The goal of this clinical trial is to learn if drug CB03-154 works to treat ALS in adults. It will also learn about the safety of drug CB03-154.

The main questions it aims to answer are:

* Does drug CB03-154 have an effect on delaying disease progression, improving function, and prolonging survival in adult ALS patients? * What medical problems do patients have when taking drug CB03-154? Researchers will compare drug CB03-154 to a placebo (a look-alike substance that contains no drug) to see if drug CB03-154 works to treat ALS.

Participants (adult ALS patients) will:

* Take drug CB03-154 or a placebo every day for 39 weeks (an additional 39 weeks would be required if entering the open-label extension phase). * Visit the clinic approximately every 2-3 months for checkups and tests, and there is also telephone follow-up in between. * Keep a diary of daily medication (CB03-154 or other concomitant medications), and if there are any unplanned medications, the reason (disease or symptoms) also need be recorded.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Agree to follow the treatment plan and trial procedures of this study, and sign the written informed consent form.

Male or female, aged 18 to 65 years, inclusive.

The weight of subjects during the screening period must not be less than 45 kg, and the BMI must not be less than 18 kg/m2.

Diagnosed according to the Revised EI Escorial diagnostic criteria set by the World Federation of Neurology: definite ALS, probable ALS, lab supported probable ALS, or possible ALS.

Disqualifiers

Significant cognitive impairment, mental disorders (such as schizophrenia, bipolar disorder), other neurodegenerative diseases (such as Parkinson's disease, Alzheimer's disease, frontotemporal dementia, etc.), substance abuse or other causes leading to neuromuscular weakness (such as myasthenia gravis), or other conditions that may interfere with the participants' participation in clinical study or, in the investigator's judgment, may interfere with outcome assessment or affect the completion of the trial.

Serum aspartate aminotransferase (AST), alanine aminotransferase (ALT), or bilirubin>2.0× upper limit of normal at screening.

Estimated glomerular filtration rate <59 mL/min/1.73m2 at Screening (using the Cockcroft-Gault formula to calculate eGFR: eGFR (mL/min/1.73m2) = Ccr × 0.84 × 1.73 / BSA; Ccr (mL/min) = [(140 - age) × weight (kg)] / [72 × Scr (mg/dL)], females multiply the result by 0.85, and Scr is the serum creatinine; BSA (m2) = 0.007184 × weight (kg)^0.425 × height (cm)^0.725).

D-dimer>2.0× upper limit of normal or venous ultrasound of the lower limbs shows deep vein thrombosis at screening or a history of venous thrombosis.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Test drug CB03-154 5mg group

    Drug

    One CB03-154 tablet (5mg/tablet) once daily.

  • Test drug CB03-154 10mg group

    Drug

    Two CB03-154 tablets (5mg/tablet) once daily.

  • Test drug CB03-154 15mg group

    Drug

    Three CB03-154 tablets (5mg/tablet) once daily.

  • Placebo Group

    Drug

    Three Placebo tablets once daily.

Treatment groups

240 Participants
are divided into 4 treatment groups
Group A: CB03-154 5mgExperimental treatment 1 intervention
Group B: CB03-154 10mgExperimental treatment 1 intervention
Group C: CB03-154 15mgExperimental treatment 1 intervention
Group D: Placebo 15mgPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Changes from Baseline to Week 39 in ALS Functional Rating Scale-Revised (ALSFRS-R).(Part A) Changes from Baseline to Week 78 in ALS Functional Rating Scale-Revised (ALSFRS-R).(Part B)

Time frame
From baseline to the entire period of double-blind period, assessed up to 39 weeks. (Part A) From baseline to the entire period of open-label extension, assessed up to 78 weeks. (Part B)

Secondary outcomes

1

A joint rank analysis of function (Functional Rating Scale-Revised , ALSFRS-R) and survival(ALS/SURV)score at Week 39.(Part A) A joint rank analysis of function (Functional Rating Scale-Revised , ALSFRS-R) and survival(ALS/SURV)score at Week 78.(Part B)

Time frame
From baseline to the entire period of double-blind period, assessed up to 39 weeks. (Part A) From baseline to the entire period of open-label extension, assessed up to 78 weeks. (Part B)
2

Change from Baseline and Slope of decline from Baseline in the Functional Rating Scale-Revised (ALSFRS-R) score at Weeks 8, 16, 28, 39, 51, 63 and 78.

Time frame
From baseline to the entire period of double-blind period, assessed up to 39 weeks. (Part A) From baseline to the entire period of open-label extension, assessed up to 78 weeks. (Part B)
3

Change from Baseline and slope of decline in the Rasch Overall ALS Disability Scale (ROADS) score at Weeks 8, 16, 28, 39, 51, 63, and 78.

Time frame
From baseline to the entire period of double-blind period, assessed up to 39 weeks. (Part A) From baseline to the entire period of open-label extension, assessed up to 78 weeks. (Part B)
4

Time from baseline to the earliest occurrence of 1 of the following events: first use of Permanent Assisted Ventilation (PAV) for >22 hours per day for >7 consecutive days OR death.

Time frame
From baseline to the entire period of double-blind period, assessed up to 39 weeks. (Part A) From baseline to the entire period of open-label extension, assessed up to 78 weeks. (Part B)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shanghai Zhimeng Biopharma, Inc.

Lead sponsor

Source ClinicalTrials.gov
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