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

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What is this study about?

This clinical trial is focused on studying a condition known as Spinal Muscular Atrophy (SMA), a genetic disorder that affects the control of muscle movement. The study will evaluate a treatment called BIIB115, which is a solution for injection. The purpose of the study is to assess the safety and tolerability of this treatment when given to participants. The study will involve two groups: healthy adult male volunteers and children with SMA who have previously been treated with a medication called onasemnogene abeparvovec, also known as Zolgensma™.

The study will be conducted in two parts. In the first part, healthy adult male participants will receive a single dose of BIIB115 through an injection into the space around the spinal cord, a method known as intrathecal injection. In the second part, children with SMA will receive multiple doses of BIIB115 using the same method. The study aims to monitor any side effects and how the body processes the medication over time. Participants will be observed for any adverse events, which are any unwanted effects that occur during the study.

The trial will also measure the concentration of BIIB115 in the participants’ blood and spinal fluid to understand how the drug is absorbed and eliminated by the body. This information will help determine the appropriate dosage and frequency of administration for future treatments. The study is expected to continue for several years to gather comprehensive data on the safety and effectiveness of BIIB115 in treating Spinal Muscular Atrophy.

1 initial visit and screening

Upon joining the study, an initial visit is conducted to confirm eligibility. This involves a review of medical history and a physical examination to ensure good health for participation.

For pediatric participants, a genetic diagnosis of spinal muscular atrophy (SMA) is confirmed, and previous treatment with onasemnogene abeparvovec is verified.

2 first dose administration

The first dose of BIIB115 is administered through an injection into the spinal canal, known as intrathecal injection. This is done to deliver the medication directly to the central nervous system.

For healthy adult male participants, this is a single dose. For pediatric participants with SMA, multiple doses are administered over time.

3 monitoring and follow-up

After the dose administration, participants are monitored for any side effects or adverse reactions. This includes regular check-ups and assessments to ensure safety and tolerability.

For healthy adult male participants, monitoring continues up to 393 days. For pediatric participants, monitoring extends up to 720 days.

4 additional dose administration for pediatric participants

Pediatric participants receive additional doses of BIIB115 as part of the multiple ascending dose study. The frequency and number of doses are determined by the study protocol.

Each dose is followed by monitoring to assess the concentration of the medication in the body and to evaluate its effects.

5 final assessments and study completion

At the end of the study period, final assessments are conducted to evaluate the overall safety and effectiveness of the treatment.

Participants may undergo various tests and evaluations to measure the concentration of BIIB115 in the body and to determine the treatment’s impact on their condition.

Who Can Join the Study?

  • Part A – Must be a healthy male aged between 18 to 55 years.
  • Part A – Must have a body mass index (BMI) between 18 to 30. BMI is a measure of body fat based on height and weight.
  • Part A – Must be in good health as determined by the study doctor, based on medical history and health checks.
  • Part B – Must be aged between 0.5 to 12 years at the time of giving consent to participate.
  • Part B – Must weigh at least 7 kilograms at the time of giving consent.
  • Part B – Must have a genetic diagnosis of Spinal Muscular Atrophy (SMA). This means having specific changes in the genes related to SMA.
  • Part B – Must have at least one copy of the SMN2 gene. This gene is related to the production of a protein important for muscle function.
  • Part B – Must have received a treatment called onasemnogene abeparvovec, which is given through a vein, according to the approved guidelines.
  • Part B – Must have received the onasemnogene abeparvovec treatment at least 180 days before the first dose of the study medication.
  • Part B – Must have the potential for improvement due to less than optimal health status related to SMA, as determined by the study doctor.

Who Cannot Join the Study?

  • Participants who have not been previously treated with onasemnogene abeparvovec cannot join. This is a specific treatment for spinal muscular atrophy.
  • Participants who cannot receive an intrathecal (IT) bolus injection are excluded. This is a method of delivering medication directly into the space around the spinal cord.
  • Participants with any other medical condition that might interfere with the study results are not allowed to participate.
  • Participants who are unable to comply with the study procedures and requirements will be excluded.
  • Participants who have any allergies or reactions to the study medication or its components cannot take part.
  • Participants who are currently involved in another clinical trial are not eligible.
  • Participants who have a history of drug or alcohol abuse may be excluded.
  • Participants who are pregnant or breastfeeding cannot participate.
  • Participants who have any significant medical conditions that could affect their safety or the study outcomes are not allowed to join.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Centre for Human Drug Research Leiden The Netherlands
Universitair Ziekenhuis Gent Gent Belgium
Instytut Centrum Zdrowia Matki Polki Lodz Poland
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Instytut Pomnik Centrum Zdrowia Dziecka Warsaw Poland
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Hopital Beaujon Clichy France
Cmblif Cfzrllf Nrab Milan Italy
Uxvydaahfpotxutlrlkly Ebupg Aep Essen Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
04.10.2023
France France
Not recruiting
04.10.2023
Germany Germany
Not recruiting
04.10.2023
Italy Italy
Not recruiting
04.10.2023
Poland Poland
Not recruiting
04.10.2023
The Netherlands The Netherlands
Not recruiting
04.10.2023

Trial locations

Investigated drugs:

BIIB115 is a medication being studied to see how safe and tolerable it is for people. In this trial, it is given to healthy adult men and children with a condition called Spinal Muscular Atrophy (SMA). The medication is administered through an injection into the spinal fluid. The study aims to understand how the body handles the medication and to ensure it is safe for use in these groups of people.

Onasemnogene Abeparvovec (Zolgensma™) is a treatment that some children with Spinal Muscular Atrophy (SMA) have already received before participating in this study. It is a gene therapy designed to help improve muscle movement and strength in children with SMA. This therapy works by delivering a new, working copy of a gene that is missing or not working properly in these children, helping to improve their condition. In this trial, it is not being tested directly but is part of the participants’ previous treatment history.

Investigated diseases:

Spinal Muscular Atrophy – Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by the progressive degeneration of motor neurons in the spinal cord. This leads to muscle weakness and atrophy, primarily affecting the proximal muscles, which are closer to the center of the body. The disease progresses with a loss of motor function, impacting activities such as crawling, walking, sitting, and head control. SMA is classified into different types based on the age of onset and severity of symptoms. Over time, individuals with SMA may experience difficulties with breathing and swallowing due to muscle weakness. The progression rate and severity can vary significantly among individuals with the condition.

Trial ID:
2023-505643-39-00
Protocol code:
277HV101
NCT ID:
NCT05575011
Trial Phase:
Human Pharmacology (Phase I) – First administration to humans

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