Active not recruitingPHASE3INTERVENTIONAL

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

This research study is investigating a new medication, BBP-418 (ribitol), for a specific type of muscle weakness called LGMD2I. Many people with this condition experience progressive muscle weakening, especially in their shoulders, hips, and thighs. The study's main goal is to check how safe this new medication is over a long period and if it can effectively help manage the condition. Participants between 12 and 60 years old who have a confirmed diagnosis of LGMD2I are invited. We want to understand if BBP-418 can make a positive difference in the lives of those affected. This is a crucial step in developing new treatments for LGMD2I.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

ML Bio Solutions, Inc.

Enrolment target

Start

31 May 2023

Estimated completion

01 Jul 2027

Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

What is this study about?

This study is focusing on a specific type of muscular dystrophy called Limb-Girdle Muscular Dystrophy type 2I, often shortened to LGMD2I. This condition causes muscles, particularly around the hips and shoulders, to gradually weaken. There isn't a cure for LGMD2I yet, so researchers are constantly looking for new ways to help people manage their symptoms and improve their quality of life.

Here, scientists are testing a new medication called BBP-418, also known as ribitol. They want to find out two main things: firstly, if BBP-418 is safe for people with LGMD2I to take over a longer period, and secondly, if it can effectively help with their muscle weakness. To do this, some participants will receive BBP-418, while others will receive a 'placebo', which looks like the medicine but contains no active ingredients. This is a standard way to ensure any improvements seen are genuinely due to the study medication.

This is a 'Phase 3' study, which means it's one of the larger, final stages of testing a new medication before it can potentially be made widely available. The results from this study will be very important in understanding if BBP-418 could become a future treatment option for LGMD2I. The study includes a wide age range of participants, from 12 to 60 years, because the researchers want to gather information from many people who live with this condition.

Key takeaways

This study is for people with a specific muscle condition called LGMD2I.
It's testing a new medication, BBP-418, to see if it's safe and helps improve symptoms.
Participants will either receive the new medication or a 'dummy' pill (placebo).
It's a long-term study with regular clinic visits and health checks.
You can be aged 12 to 60 to take part (18 to 60 in Europe).
Your decision to join or leave the study won't affect your regular medical care.

Who may be eligible?

To join this study, people need to have a confirmed diagnosis of LGMD2I through genetic testing and show some signs of muscle weakness, either in their shoulders and hips or in their arms and legs. Participants must be between 12 and 60 years old (though in Europe, it's 18-60 years old). They also need to weigh more than 30 kg and be able to understand the study and agree to take part, with a parent or guardian's help if they are under 18.

There are also some reasons why someone might not be able to join. For example, if you have other serious health problems like heart, kidney, lung, liver, or hormone issues that aren't related to LGMD2I, or if your kidney function is below a certain level. If you've had major surgery like for scoliosis that might make it hard to do the study tests, or if the doctors think joining the study might not be safe for you, you also might not be eligible. Women who could have children and men who could father children must agree to use effective contraception during the study and for some time after.

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.

Do I have a confirmed genetic diagnosis of LGMD2I with some current muscle weakness?
Am I between 12 and 60 years old (18-60 if I live in Europe)?
Do I weigh more than 30 kilograms?
Am I generally healthy, without other serious health conditions not related to LGMD2I, or severe kidney problems?
Am I willing and able to attend all study appointments and complete all required tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given either the study medication (BBP-418) or a placebo (a dummy medicine) on a long-term basis. You'll have regular visits to the clinic where doctors and nurses will check your health, measure your muscle strength, and perform other tests to see how you're responding to the treatment. These tests might include blood and urine samples, and possibly some small muscle biopsies (where a tiny piece of muscle is taken for examination). You'll also have your vital signs (like blood pressure and heart rate) checked, and potentially heart recordings (ECG). These visits will be scheduled over a significant period. The study team will explain the full schedule of visits and procedures in detail, so you know exactly what to expect and how much time it might involve. You will also have follow-up checks after you finish taking the medication. The total duration of your participation will be explained by the study team.

Potential risks and benefits

Taking part in a study like this might offer some potential benefits, such as receiving a new medication that could help manage your LGMD2I, or getting very close medical attention from specialists. However, there are also potential risks, as BBP-418 is still under investigation. These risks could include side effects from the medication, discomfort from tests like blood draws or biopsies, or simply the time commitment. It's important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (21)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Arkansas Children's Hospital
Verified postcode
Little Rock, United States
University of California Irvine
Verified postcode
Irvine, United States
University of Colorado Anschutz Medical Campus
Verified postcode
Aurora, United States
University of Florida
Unverified
Gainsville, United States
University of Iowa
Verified postcode
Iowa City, United States
University of Kansas Medical Center
Verified postcode
Kansas City, United States
Kennedy Krieger Institute
Verified postcode
Baltimore, United States
University of Minnesota, Twin Cities
Verified postcode
Minneapolis, United States
Washington University School of Medicine
Verified postcode
St Louis, United States
Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront
Verified postcode
Portland, United States
The Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
University of Pennsylvania
Verified postcode
Philadelphia, United States

Common questions

What is LGMD2I?

LGMD2I is a type of muscular dystrophy that causes weakness in muscles, especially around the hips and shoulders, and can affect other muscles too.

What is BBP-418?

BBP-418, also known as ribitol, is a new medication being tested to see if it can help people with LGMD2I.

Will I definitely get the active medication?

No, this is a 'blinded' study, meaning some participants will receive BBP-418 and others will receive a placebo, and neither you nor the doctors will know which one you are getting until the study ends.

Who can join the study?

Generally, people aged 12 to 60 with a confirmed diagnosis of LGMD2I and certain health requirements may be able to join, though there are specific criteria.

What does a 'Phase 3' study mean?

Phase 3 means this is a later stage of research, where the medication is tested on more people to confirm its safety and effectiveness before it can potentially be approved for general use.