Amyotrophic Lateral Sclerosis study: BIIB147
Official title A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS)
ClinicalTrials.gov ID: NCT07855211
What this study is testing
What is BIIB147?
BIIB147 is an investigational medicine, being studied as a potential treatment for amyotrophic lateral sclerosis.
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
- In this study, researchers will learn more about the effects of BIIB147, in people with amyotrophic lateral sclerosis, also known as ALS.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 8 years
- 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 and older
You may be able to join if
- Key MAD Period
- Participants must have a diagnosis of ALS consistent with the Gold Coast criteria.
- No pathogenic variants, likely pathogenic variants, or variants of uncertain significance in the superoxide dismutase 1 (SOD1) or fused in sarcoma...
- Greater than or equal to (≥) 50% of predicted value for slow vital capacity (SVC) as adjusted for sex, age, and height (from the sitting position).
- If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit...
You likely can't join if
- MAD Period
- History or positive test result at screening for human immunodeficiency virus (HIV).
- History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study...
- Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
- Requires permanent ventilation (using \> 22 hours per day [h/day] invasive or non-invasive ventilation)
- History of a deep venous thrombosis or pulmonary embolism ≤2 years of screening, or since the date of ALS diagnosis, whichever is longer.
See the full eligibility criteria
- Key MAD Period
- Participants must have a diagnosis of ALS consistent with the Gold Coast criteria.
- No pathogenic variants, likely pathogenic variants, or variants of uncertain significance in the superoxide dismutase 1 (SOD1) or fused in sarcoma (FUS) genes (confirmed by central genetic testing).
- Greater than or equal to (≥) 50% of predicted value for slow vital capacity (SVC) as adjusted for sex, age, and height (from the sitting position).
- If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit end of study (EOS)/early termination (ET), unless the Investigator...
- Participants must have completed Study 303AS101 MAD period; all pre-dose assessments for the end of MAD treatment period visit (Day 113 for Cohorts A, B, C, and Day 169 for Cohort D) must be completed prior to dosing in...
- MAD Period
- History or positive test result at screening for human immunodeficiency virus (HIV). The requirement for testing at screening may be omitted if it is not permitted by local regulations.
- History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and/or is expected to be associated with elevations in NfL, in the opinion...
- Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
- Requires permanent ventilation (using \> 22 hours per day [h/day] invasive or non-invasive ventilation)
- History of a deep venous thrombosis or pulmonary embolism ≤2 years of screening, or since the date of ALS diagnosis, whichever is longer.
- Treatment with an approved ALS disease-modifying therapy other than riluzole or edaravone, or another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent...
- Current enrollment or a plan to enroll in any treatment clinical study. Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after...
- Any new condition or worsening of an existing condition that, according to the Investigator, would make the participant unsuitable for inclusion, could interfere with the assessment of safety, or would compromise the...
- Clinically significant abnormalities (based on most recent assessments in MAD period) in hematology, blood chemistry parameters, or 12-lead electrocardiogram (ECG) prior to first OLE visit that would make the...
The study team makes the final eligibility decision.
Where it is enrolling
- Scottsdale, Arizona, United States
- St Louis, Missouri, United States
- Austin, Texas, United States
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, but confirm specifics with the study team.
How long does this study last?
The study runs about 8 years 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 and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Scottsdale, Arizona, United States; St Louis, Missouri, United States; Austin, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.