RecruitingRare disease

Study of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)

Fast replyInvestigational

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on children under 2 years of age who have Spinal Muscular Atrophy (SMA), a genetic condition that affects the nerves controlling muscle movement, causing muscle weakness and movement difficulties. The study will test a medicine called apitegromab, given through intravenous infusion (delivered directly into a vein).

The purpose of this research is to understand how apitegromab works in young children with SMA and to evaluate its effects on their movement abilities. All children in the study must already be receiving or have received other approved treatments for SMA, such as onasemnogene abeparvovec-xioi, nusinersen, or risdiplam.

The study will last for 48 weeks, during which children will receive regular infusions of the study medication. Throughout the study, doctors will monitor how the medicine moves through the body and its effects on muscle function. This is a double-blind study, which means neither the participants nor the doctors directly involved in their care will know which treatment each child receives.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Your eligibility for the study will be confirmed based on specific criteria, including being less than 2 years old and having a confirmed diagnosis of Spinal Muscular Atrophy (SMA).
A medical assessment will verify that you are currently receiving or have received approved therapy for SMA.
Your weight will be checked to ensure it meets the required minimum according to standard growth charts.
Step 2
Treatment initiation
You will receive apitegromab through an intravenous infusion (medicine given through a vein).
The study will be conducted in a double-blind format, which means neither you nor the doctor will know the exact treatment assignment.
Step 3
Regular monitoring
Your blood samples will be collected to measure the levels of apitegromab and a protein called myostatin in your body.
Your motor function (movement ability) will be regularly assessed using specific tests designed for infants.
The medical team will monitor your health and any possible side effects throughout the study.
Step 4
48-week assessment
At week 48, a detailed assessment of your motor skills will be performed using the BSID-4 GMS (a standardized test that measures movement abilities).
The results will be compared to your initial assessment to measure any changes in motor function.
Step 5
Study completion
The study is expected to continue until March 2029.
All observed effects and safety information will be documented throughout the study period.

Who can join the trial?

8 criteria

Must be less than 2 years old when parents/guardians provide consent for participation
Must have been born after 35 weeks of pregnancy and weighed at least 2.0 kilograms at birth
Must have a confirmed diagnosis of 5q autosomal recessive SMA (a genetic form of spinal muscular atrophy)
Must have confirmed presence of SMN2 gene copies (genes related to muscle function)
check
Must be currently receiving or have previously received approved treatment for SMA, either:
- Gene therapy (onasemnogene abeparvovec-xioi), or
- Ongoing treatment with medications that target SMN2 (nusinersen or risdiplam)
- Body weight must be at or above the 1st percentile for age according to World Health Organization growth charts when screened
- Must show delayed motor development for their age due to SMA, as determined by the doctor, or have a CHOP-INTEND score below 55 (CHOP-INTEND is a test that measures movement abilities in infants)

Who cannot join the trial?

12 criteria

History of severe adverse reactions to any monoclonal antibody (a type of medical treatment that targets specific proteins in the body)
Prior participation in any clinical trial involving investigational drugs within 5 half-lives or 30 days before screening
Major surgery within 6 months before screening or planned during the study period
Active or chronic infection, including hepatitis B, hepatitis C, or HIV
Any medical condition that could interfere with the study medication or put the patient at risk, as determined by the study doctor
History of cancer within the past 3 years (except successfully treated non-melanoma skin cancer)
Severe heart, liver, or kidney problems
Current use of medications that could interact with the study drug
Inability to comply with study procedures or visits
Pregnancy, breastfeeding, or planning to become pregnant during the study period
Known allergies to any components of the study medication
Any contraindication to MRI scanning (a type of medical imaging test)

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Katholieke Universiteit te Leuven
Leuven, Belgium
Responsive
Universitair Medisch Centrum Utrecht
Utrecht, The Netherlands
Responsive
Universitair Ziekenhuis Gent
Gent, Belgium

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting

Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Apitegromab is a medication being studied for treating Spinal Muscular Atrophy (SMA) in young children. It's designed to help improve muscle strength and motor function in patients with SMA. This medication works by targeting specific proteins involved in muscle growth and development. The treatment aims to help very young children (under 2 years old) who are affected by this rare genetic condition that affects muscle strength and movement.

Apitegromab

What is already known about the treatment

Apitegromab - A novel investigational medication administered through intravenous infusion, specifically designed for the treatment of Spinal Muscular Atrophy (SMA) in patients under 2 years of age. This monoclonal antibody works by targeting and inhibiting myostatin, a protein that naturally limits muscle growth and differentiation, thereby potentially improving muscle strength and motor function in SMA patients. Currently undergoing Phase 2 clinical trials, apitegromab represents a promising therapeutic approach that aims to enhance motor function outcomes in young children affected by SMA, with its development focused on establishing optimal dosing, safety profile, and efficacy through careful evaluation of pharmacokinetics and pharmacodynamics.

Investigated diseases

Spinal Muscular Atrophy - A genetic condition that affects the nerve cells responsible for controlling muscle movement. The disease causes progressive muscle weakness and atrophy, primarily affecting muscles used for crawling, walking, sitting up, and controlling head movement. The condition occurs when there is a problem with the survival motor neuron 1 (SMN1) gene, which is essential for maintaining motor neurons. Symptoms typically include muscle weakness, decreased muscle tone, and difficulties with movement and motor development. The severity and age of onset can vary among different individuals.