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New treatment option for Amyotrophic Lateral Sclerosis (ALS)
Official title A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
ClinicalTrials.gov ID: NCT07369076
What this study is testing
What is NB-4746 High dose?
NB-4746 High dose is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for amyotrophic lateral sclerosis (als).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this trial is to learn about the effects of NB-4746 compared with placebo in people with amyotrophic lateral sclerosis. The questions this trial aims to answer in comparing NB-4746 to placebo are: What adverse events associated with taking NB-4746 are reported during this trial?
- Phase 2: a mid-size study of how well it works
- Time commitment: about 12 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join if
- \- ALS Criteria for Phase 1b:
- Participants in Phase 1b must have:
- Diagnosis of ALS per Gold Coast Criteria; and
- Symptom onset ≤48 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in...
- ALS Criteria for Phase 2:
You likely can't join if
- Presence of tracheostomy or permanent assisted ventilation; defined as \> 22 hours daily of mechanical ventilation for more than 1 week (7 days).
- History or presence of clinically significant uncontrolled medical conditions (other than ALS) that include cardiovascular, respiratory, hepatic...
- Lifetime history of cancer except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic...
- Female participants who have a positive serum pregnancy test at Screening, positive urine pregnancy test on Day 1, or who are breastfeeding on Day 1.
- Has a spinal deformity or other condition that may prevent the performance of a lumbar puncture.
- International Normalized Ratio (INR) \>1.4, platelet count \<50,000/μL, or use of warfarin, heparin, or direct oral anticoagulants.
See the full eligibility criteria
- \- ALS Criteria for Phase 1b:
- Participants in Phase 1b must have:
- Diagnosis of ALS per Gold Coast Criteria; and
- Symptom onset ≤48 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region...
- ALS Criteria for Phase 2:
- Participants in Phase 2 must have:
- Diagnosis of ALS per Gold Coast Criteria; and
- Symptom onset ≤24 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region...
- Additional (All Participants):
- Male or female participants aged ≥18 years and ≤80 years at the time of signing informed consent.
- Participants must demonstrate functional oral intake and be independent of enteral nutritional support (e.g., PEG, G-tube) at the time of screening per Principal Investigator's judgement.
- Slow vital capacity (SVC) ≥60% of predicted at Screening.
- If taking riluzole, participant must be on a stable dose for ≥60 days prior to Screening lab blood draw.
- If taking edaravone, participant must have completed at least 1 cycle of edaravone prior to screening labs.
- Willing to adhere to contraceptive requirements during the study period as described in Appendix 1.
- Capable of giving signed informed consent, as described in Section 13.2.2, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
- Presence of tracheostomy or permanent assisted ventilation; defined as \> 22 hours daily of mechanical ventilation for more than 1 week (7 days).
- History or presence of clinically significant uncontrolled medical conditions (other than ALS) that include cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological...
- Lifetime history of cancer except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. Fully resected basal cell carcinoma and squamous...
- Female participants who have a positive serum pregnancy test at Screening, positive urine pregnancy test on Day 1, or who are breastfeeding on Day 1.
- Has a spinal deformity or other condition that may prevent the performance of a lumbar puncture.
- International Normalized Ratio (INR) \>1.4, platelet count \<50,000/μL, or use of warfarin, heparin, or direct oral anticoagulants.
- History of Class III/IV heart failure (per New York Heart Association [NYHA]).
- Inability to swallow or tolerate oral medications at Screening.
- Current participation in any other investigational drug study or receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) before Screening.
- Known sensitivity to the NB-4746 study drug or any of the formulation components.
- Has received NB-4746 at any time prior to initial Screening.
- Any other reason that, in the opinion of the Investigator, would confound the conduct of this study or the interpretation of the results or that could compromise the participant's safety.
- Currently taking or planning to receive tofersen for the treatment of ALS.
- If a participant does not transition to the OLE in ≤30 days following completion of the main study, the participant will need to be rescreened for laboratory criteria and contraception/pregnancy criteria.
The study team makes the final eligibility decision.
Where it's taking place
- Concord, New South Wales, Australia
- Randwick, New South Wales, Australia
- Brisbane, Queensland, Australia
- Bedford Park, South Australia, Australia
- Caulfield South, Victoria, Australia
- Nedlands, Western Australia, Australia
- Fredericton, New Brunswick, Canada
- Montreal, Quebec, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 12 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Concord, New South Wales, Australia; Randwick, New South Wales, Australia; Brisbane, Queensland, Australia; Bedford Park, South Australia, Australia; Caulfield South, Victoria, Australia; Nedlands, Western Australia, Australia and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.