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BIIB115

Researchers are currently studying a medication called BIIB115 for potential use in treating spinal muscular atrophy (SMA), particularly in children who have previously received gene therapy. This clinical trial is designed to evaluate the safety, tolerability, and how the drug moves through the body in both healthy adult males and children with SMA. The study is divided into two parts: Part A involves healthy volunteers receiving a single dose of either BIIB115 or placebo, while Part B focuses on children with SMA who previously received onasemnogene abeparvovec (Zolgensma™) gene therapy. This research aims to provide important insights into how BIIB115 might benefit patients with SMA and establish appropriate dosing guidelines for potential future treatments.

Table of Contents

What is BIIB115?

BIIB115 is an investigational drug currently being studied for the treatment of Spinal Muscular Atrophy (SMA). This medication is particularly being evaluated in children with SMA who have previously been treated with another therapy called onasemnogene abeparvovec (also known by its brand name Zolgensma™)[1]. BIIB115 is administered as an injection directly into the spinal canal, which is called an intrathecal (IT) bolus injection. This delivery method allows the medication to reach the cerebrospinal fluid (CSF), which surrounds the brain and spinal cord.

What is Spinal Muscular Atrophy (SMA)?

Spinal Muscular Atrophy is a rare genetic disease that affects the motor neurons in the spinal cord and brainstem. These neurons are responsible for controlling muscle movement throughout the body. When these neurons don’t function properly, muscles become weak and may waste away (atrophy). SMA can affect a person’s ability to walk, eat, and even breathe. It is a serious condition that often appears in infancy or early childhood, though there are varying types with different levels of severity[1].

Current Clinical Study of BIIB115

BIIB115 is currently being investigated in a Phase 1 clinical trial. This trial aims to evaluate the safety, tolerability, and how the body processes the drug (pharmacokinetics) in both healthy adult males and children with SMA who have previously received onasemnogene abeparvovec treatment[1].

The main objectives of this study are:

  • To assess the safety of BIIB115 by monitoring for adverse events (unwanted health problems that may or may not be caused by the study drug)
  • To understand how the body processes BIIB115 by measuring drug levels in both the blood (serum) and cerebrospinal fluid (CSF)
  • To determine appropriate dosing for future studies

Study Design and Structure

The clinical trial for BIIB115 is divided into two parts[1]:

Part A: Single Dose in Healthy Adult Males

This part involves healthy adult male volunteers who will receive a single dose of either BIIB115 or a placebo. Important features of Part A include:

  • Participants will be randomly assigned to one of four different dose groups (Dose 1, 2, 3, or 4) or a placebo group
  • Neither the researchers nor the participants will know whether they’re receiving BIIB115 or placebo (this is called “blinding” or “masking”)
  • Each participant will receive a single injection into the spinal canal (intrathecal injection)
  • The treatment and follow-up period will last for 13 months
  • Participants will have up to 6 clinic visits and 4 telephone calls during this period

Part B: Multiple Doses in Children with SMA

This part focuses specifically on children with SMA who have previously been treated with onasemnogene abeparvovec. Key aspects include:

  • Children will be assigned to receive either Dose 3 or Dose 4 of BIIB115
  • Each participant will receive two doses of BIIB115 at different time points
  • Both researchers and participants will know they are receiving BIIB115 (this is called “open-label”)
  • The treatment and follow-up period will last for 25 months
  • Participants will have up to 14 clinic visits and 6 telephone calls

In both parts of the study, participants will remain under medical supervision for 24 hours after each dose to monitor for any immediate health issues or side effects[1].

Who Can Participate in the Study?

The study includes two distinct groups of participants[1]:

  1. Healthy Adult Male Volunteers: These participants do not have SMA and will participate in Part A of the study.
  2. Pediatric SMA Patients: Children who have been diagnosed with Spinal Muscular Atrophy and have previously received treatment with onasemnogene abeparvovec (Zolgensma™). These participants will take part in Part B of the study.

How is BIIB115 Administered?

BIIB115 is given as an intrathecal (IT) bolus injection. This means the medication is injected directly into the spinal canal, where it can enter the cerebrospinal fluid surrounding the brain and spinal cord[1].

For healthy volunteers in Part A, this is a one-time injection. For children with SMA in Part B, they will receive two separate injections at different time points during the study.

What Outcomes Are Being Measured?

The study is collecting several types of data to evaluate BIIB115[1]:

Primary Outcome:

  • Safety assessment: The number of participants who experience adverse events (AEs) or serious adverse events (SAEs). An adverse event is any unwanted medical occurrence during the study, while a serious adverse event is one that results in death, is life-threatening, requires hospitalization, causes disability, or is otherwise medically significant.

Secondary Outcomes:

  • How BIIB115 moves through the body, including:
    • Concentration levels of BIIB115 in the cerebrospinal fluid and blood
    • How long BIIB115 stays in the body (terminal elimination half-life)
    • The total amount of drug exposure over time (area under the curve or AUC)
    • The highest concentration reached (maximum observed concentration or Cmax)
    • How long it takes to reach the highest concentration (time to maximum concentration or Tmax)

Safety Monitoring During the Study

Throughout the study, participants will be closely monitored for any side effects or adverse events. Safety is the primary concern, which is why[1]:

  • Participants stay in the clinic for 24 hours after receiving each dose
  • Multiple follow-up visits and phone calls are scheduled throughout the study period
  • Any adverse events are thoroughly documented and evaluated
  • Blood and cerebrospinal fluid samples are collected to monitor drug levels and body responses

The extensive follow-up periods (13 months for Part A and 25 months for Part B) allow researchers to identify both short-term and long-term effects of BIIB115 treatment[1].

Study Aspect Details
Study Drug BIIB115
Study Population Healthy adult male volunteers (Part A) and children with SMA previously treated with onasemnogene abeparvovec (Part B)
Administration Method Intrathecal (IT) bolus injection directly into the spinal canal
Study Design: Part A Randomized, blinded, placebo-controlled with single ascending doses; 13-month duration with 6 clinic visits and 4 phone calls
Study Design: Part B Open-label with multiple ascending doses; 25-month duration with 14 clinic visits and 6 phone calls
Primary Outcome Number of participants with adverse events and serious adverse events
Secondary Outcomes Concentration of BIIB115 in CSF and serum, half-life, area under the concentration-time curve, maximum concentration, and time to reach maximum concentration
Post-dose Monitoring 24-hour observation in clinic after each dose
Study Identifier NCT05575011

FAQ

  • What is BIIB115 and what is it being studied for? BIIB115 is an investigational drug being studied as a potential treatment for spinal muscular atrophy (SMA). In this clinical trial, researchers are specifically looking at how it might benefit children with SMA who have previously received gene therapy treatment with onasemnogene abeparvovec (Zolgensma™). The study aims to understand its safety profile and how the body processes different doses of the medication.
  • How is the clinical trial for BIIB115 structured? The trial is divided into two parts. Part A involves healthy adult male volunteers who receive a single dose of either BIIB115 or a placebo (an injection that looks like the study drug but contains no active medicine). Part B involves children with SMA who have previously received gene therapy. These children will receive two doses of BIIB115 at different time points. Part A is blinded, meaning neither participants nor researchers know who receives the actual drug, while Part B is open-label, so everyone knows the participants are receiving BIIB115.
  • How is BIIB115 administered to participants? BIIB115 is administered via intrathecal (IT) bolus injection. This means the medication is injected directly into the spinal canal, where cerebrospinal fluid flows around the brain and spinal cord. This method of delivery is chosen to help the medication reach the nervous system, which is important for treating conditions like SMA that affect the nerves and muscles.
  • What are researchers measuring in this BIIB115 trial? The primary focus is measuring the safety of BIIB115 by tracking adverse events and serious adverse events. Researchers are also measuring how the body processes the drug by looking at BIIB115 concentrations in both cerebrospinal fluid and blood serum. They're evaluating factors like how long the drug stays in the body (half-life), how high the concentration gets (maximum concentration), and the overall exposure to the drug over time.
  • How long will participants be involved in the BIIB115 trial? The time commitment varies between the two parts of the study. For Part A (healthy volunteers), the treatment and follow-up period lasts about 13 months and includes up to 6 clinic visits and 4 telephone calls. For Part B (children with SMA), the commitment is longer at approximately 25 months, with up to 14 clinic visits and 6 telephone calls. After receiving the drug, participants in both parts stay in the clinic for 24 hours for monitoring.

Ongoing Clinical Trials on BIIB115

  • Study on the Safety and Tolerability of BIIB115 for Children with Spinal Muscular Atrophy Previously Treated with Gene Therapy

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Poland

Glossary

  • Spinal Muscular Atrophy (SMA): A genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement. It causes muscle weakness and atrophy (wasting) due to loss of motor neurons in the spinal cord and brainstem.
  • BIIB115: An investigational drug being studied for the treatment of spinal muscular atrophy (SMA), particularly in children who have previously received gene therapy.
  • Onasemnogene abeparvovec (Zolgensma™): A gene therapy treatment for spinal muscular atrophy that provides a functional copy of the SMN1 gene to motor neurons.
  • Intrathecal (IT) bolus injection: A method of drug administration where medication is injected directly into the spinal canal, where cerebrospinal fluid circulates around the brain and spinal cord.
  • Cerebrospinal Fluid (CSF): The clear fluid that surrounds the brain and spinal cord, providing protection, delivering nutrients, and removing waste products.
  • Placebo: A substance that looks like the study drug but contains no active medicine. It's used in clinical trials to help determine if observed effects are due to the active drug or other factors.
  • Adverse Event (AE): Any undesirable medical occurrence in a study participant, whether or not it's considered related to the treatment being studied. This can include any unfavorable sign, symptom, or disease.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization, causes persistent disability, results in birth defects, or is otherwise medically significant.
  • Pharmacokinetics: The study of how drugs move through the body, including absorption, distribution, metabolism, and excretion.
  • Half-life (t½): The time it takes for the concentration of a drug in the body to be reduced by half.
  • Maximum Observed Concentration (Cmax): The highest concentration of a drug observed in the blood or other fluids after administration.
  • Area Under the Curve (AUC): A measure of the total exposure to a drug over time. AUC0-last refers to the area from time zero to the last measurable concentration, while AUCinf refers to the area from time zero to infinity.
  • Blinded Study: A study design where participants and/or researchers don't know who is receiving the study drug versus placebo, to prevent bias in assessing outcomes.
  • Open-label Study: A study where both participants and researchers know which treatment is being administered.
  • Cohort: A group of participants in a study who share a common characteristic or experience, such as receiving the same dose of medication.

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