RecruitingPHASE2INTERVENTIONAL

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

This research is investigating a medicine called risdiplam for very young babies diagnosed with spinal muscular atrophy (SMA). SMA is a genetic condition that affects muscles. The study wants to find out how risdiplam moves through the bodies of infants under 20 days old at their first dose, and whether it is safe for them. By carefully studying these things, researchers hope to learn more about how to best treat SMA in newborns. This could lead to better care options for families facing this condition, helping to improve the health and well-being of these babies.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Hoffmann-La Roche

Enrolment target

Start

26 Apr 2024

Estimated completion

30 Nov 2026

Muscular Atrophy, Spinal

What is this study about?

This research study is focused on a medicine called risdiplam, which is being investigated for spinal muscular atrophy (SMA). SMA is a rare genetic condition that affects the nerves that control muscle movement, leading to muscle weakness and wasting. This particular study is looking at how risdiplam works in the bodies of newborn babies with SMA.

The main goals are to understand how the medicine is absorbed, distributed, processed, and removed by a baby's body (this is called pharmacokinetics), and to check if it's safe for them. The study specifically includes babies who are under 20 days old when they receive their first dose of the medicine. By gathering this important information, doctors can learn more about how to use risdiplam effectively and safely for the very youngest patients.

Finding out how medicines work in newborns is really important because their bodies are different from older children and adults. This research aims to provide valuable insights that could help improve future treatments and care for infants born with SMA, giving them the best possible start in life.

Key takeaways

This study is for newborn babies (under 20 days old) with SMA.
It's looking at a medicine called risdiplam to understand its safety and how it works in infants.
Participation involves regular check-ups and monitoring by medical staff.
The goal is to improve care for babies with SMA.
You can withdraw your baby from the study at any time.

Who may be eligible?

This study is looking for newborn babies who are less than 20 days old at the time of their first dose of medication. They must have a confirmed diagnosis of spinal muscular atrophy (SMA) through genetic testing or newborn screening. Babies should have been born at 37 weeks gestation or later and be recovering well from any recent illnesses.

There are also some things that would mean a baby couldn't join the study. For example, if they show severe signs of SMA Type 0, or if they have certain heart problems. Also, if a baby is currently taking or has recently taken certain other medications, they might not be able to participate.

Parents or caregivers will need to be open to the possibility of using feeding tubes (like a nasogastric tube or gastrostomy tube) if the doctors recommend it to make sure the baby gets enough food, water, and medicine during the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my baby under 20 days old?
Has my baby been diagnosed with SMA through testing?
Was my baby born at 37 weeks gestation or later?
Is my baby currently well enough to potentially join a study?
Am I open to the possibility of a feeding tube for my baby if recommended by doctors?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve your baby receiving the study medicine, risdiplam. Doctors and nurses would regularly check your baby's health, including taking blood samples to understand how the medicine is affecting them and ensuring their safety. They would also monitor your baby's development and any changes related to their SMA.

You would have regular visits to the clinic for these assessments. The study team would explain the schedule of visits and what each one involves. Throughout the study, medical staff would be closely observing your baby and providing care. The total duration of participation in this study would be explained to you before you make any decisions.

Potential risks and benefits

Like all medical studies, there could be potential benefits and risks. Your baby might benefit from receiving risdiplam, which is being studied for SMA. However, because this is a research study, we don't know for sure if it will help your baby, or what all the effects might be. Potential risks could include side effects from the medicine, discomfort from blood tests or other procedures, or issues if a feeding tube becomes necessary. The study team will explain all known risks. Remember, taking part is completely voluntary, and you have the right to withdraw your baby from the study at any time without explaining why, and this will not affect their future medical care.

Locations (13)

Ann and Robert H. Lurie Children Hospital of Chicago
Verified postcode
Chicago, United States· Recruiting
University Of Michigan
Verified postcode
Ann Arbor, United States· Recruiting
Clinic for Special Children.
Verified postcode
Gordonville, United States· Recruiting
Hopital Universitaire des Enfants Reine Fabiola
Verified postcode
Brussels, Belgium· Recruiting
CHR Citadelle
Verified postcode
Liège, Belgium· Recruiting
Children'S Hospital of Eastern Ontario
Verified postcode
Ottawa, Canada· Recruiting
Universitatsklinikum Essen
Verified postcode
Essen, Germany· Recruiting
Fondazione Serena Onlus - CENTRO CLINICO NEMO
City only
Milano, Italy· Recruiting
Fondazione Policlinico Univeristario A. Gemelli
City only
ROMA, Italy· Recruiting
UMC Utrecht
Verified postcode
Utrecht, Netherlands· Recruiting
OUS (Oslo University Hospital), Rikshospitalet
Verified postcode
Oslo, Norway· Recruiting
Uniwersyteckie Centrum Kliniczne
Verified postcode
Gdansk, Poland· Recruiting

Common questions

What is spinal muscular atrophy (SMA)?

SMA is a genetic condition that causes muscle weakness. It affects the nerve cells that control movement, making muscles weaker over time.

What is risdiplam?

Risdiplam is a medicine being tested for SMA. It aims to help the body produce more of a protein that is needed for healthy nerves and muscles.

Why are you only studying very young babies?

Studying very young babies helps us understand how the medicine works in their developing bodies, which is different from older children or adults. This information is crucial for safe and effective treatment early on.

What does 'pharmacokinetics' mean?

'Pharmacokinetics' is a medical term that means how a medicine moves through the body – how it's absorbed, where it goes, how it's changed, and how it leaves the body.

Will my baby get special care if they join the study?

Yes, your baby will receive close medical attention and monitoring from the study team throughout their participation.

How to find out more

Reference Study ID Number: BN44619 https://forpatients.roche.com/

global-roche-genentech-trials@gene.com 888-662-6728 (U.S. Only) Official trial record

Always speak to your GP or specialist before deciding to take part in a study.