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Part A: Cohort 4: BIIB115 Dose 4 for Spinal Muscular Atrophy

Phase 1
Recruiting
Research Sponsored by Biogen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up part a: day 1 to day 180, part b: day 1 to 720
Awards & highlights

Study Summary

This trial aims to test the safety and tolerability of a new drug called BIIB115 given as a single dose to healthy male participants and as multiple doses to pediatric patients with Spinal Muscular At

Who is the study for?
This trial is for healthy males aged 18-55 and children aged 0.5 to 12 with Spinal Muscular Atrophy (SMA) who've been treated with onasemnogene abeparvovec. Adults must have a BMI of 18-30 kg/m^2 and be in good health, while children need to weigh at least 7 kg and may benefit from treatment due to SMA.Check my eligibility
What is being tested?
The study tests the safety and tolerability of BIIB115, given through spinal injection, comparing it with a placebo. It's conducted in two parts: one-time dosing for healthy adults (Part A), and multiple doses for pediatric SMA patients previously treated with gene therapy (Part B).See study design
What are the potential side effects?
While specific side effects are not listed here, the trial aims to assess how safe BIIB115 is when administered via spinal injection which could include typical drug administration reactions like pain or discomfort at the injection site.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~part a: day 1 to day 180, part b: day 1 to 720
This trial's timeline: 3 weeks for screening, Varies for treatment, and part a: day 1 to day 180, part b: day 1 to 720 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Parts A and B: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
Part A: Terminal Elimination Half-Life (t½) of BIIB115 in CSF
Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum
Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum
+5 more

Trial Design

7Treatment groups
Experimental Treatment
Placebo Group
Group I: Part B: Cohort 6: BIIB115 Dose 4Experimental Treatment1 Intervention
Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 4, via IT bolus injectionat two separate time points.
Group II: Part B: Cohort 5: BIIB115 Dose 3Experimental Treatment1 Intervention
Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 3, via IT bolus injection at two separate time points.
Group III: Part A: Cohort 4: BIIB115 Dose 4Experimental Treatment1 Intervention
Participants will receive a single dose of BIIB115, Dose 4, via IT bolus injection, on Day 1.
Group IV: Part A: Cohort 3: BIIB115 Dose 3Experimental Treatment1 Intervention
Participants will receive a single dose of BIIB115, Dose 3, via IT bolus injection, on Day 1.
Group V: Part A: Cohort 2: BIIB115 Dose 2Experimental Treatment1 Intervention
Participants will receive a single dose of BIIB115, Dose 2, via IT bolus injection, on Day 1.
Group VI: Part A: Cohort 1: BIIB115 Dose 1Experimental Treatment1 Intervention
Participants will receive a single dose of BIIB115, Dose 1, via IT bolus injection, on Day 1.
Group VII: Part A: Cohorts 1-4: BIIB115-Matching PlaceboPlacebo Group1 Intervention
Participants will receive a single dose of BIIB115-matching placebo, via IT bolus injection, on Day 1.

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

BiogenLead Sponsor
639 Previous Clinical Trials
467,260 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,782 Previous Clinical Trials
8,066,254 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there a substantial number of medical facilities in the United States that are currently conducting this clinical trial?

"Twelve active sites, including Universitair Ziekenhuis Gent in Gent, Universitaetsklinikum Heidelberg in Heidelberg, and Centre For Human Drug Research in Leiden are currently enrolling participants. Additionally, there are a dozen other locations participating in this trial."

Answered by AI

Are there any vacancies available for patients to participate in this trial?

"Indeed, data available on clinicaltrials.gov indicates the ongoing recruitment of participants for this trial. The study was first posted on October 10th, 2022 and most recently updated on March 12th, 2024. There is a need to enlist 62 individuals from among the twelve participating sites."

Answered by AI

Is this research trial enrolling individuals older than 40 years?

"To be considered for this study, participants must range from 6 months to 55 years of age."

Answered by AI

What is the current number of individuals actively participating in this research endeavor?

"Indeed, data available on clinicaltrials.gov highlights the ongoing recruitment of participants for this investigation. The trial was posted online on October 10th, 2022 and most recently updated on March 12th, 2024. A total of 62 individuals are sought from a network spanning across 12 different sites."

Answered by AI

What are the criteria for individuals who may qualify to participate in this research?

"This study aims to enroll 62 individuals diagnosed with spinal muscular atrophy, aged between 6 months and 55 years. Eligible participants must meet specific criteria including a minimum weight of ≥7 kg at the time of consent, having a Survival Motor Neuron 2 (SMN2) copy number of ≥1, receiving treatment with onasemnogene abeparvovec for at least 180 days before their first dose of BIIB115, showing potential for improvement due to suboptimal clinical status linked to SMA as determined by the Investigator Key assessment, possessing a genetic diagnosis involving SMN1 gene deletion or mutation"

Answered by AI

Has Part A: Cohort 4 involving BIIB115 Dose 4 been granted approval by the Food and Drug Administration?

"Based on the phase of this trial, our assessment at Power indicates a safety rating of 1 for Part A: Cohort 4 involving BIIB115 Dose 4. This score reflects the preliminary nature of available data regarding its safety and effectiveness."

Answered by AI
~41 spots leftby May 2027