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Sugar Alcohol
Ribitol for Limb-Girdle Muscular Dystrophy (Fortify Trial)
Phase 3
Recruiting
Research Sponsored by ML Bio Solutions, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 36 months
Awards & highlights
Fortify Trial Summary
This trial studies the safety & effectiveness of a long-term treatment for LGMD2I/R9 in patients aged 12-60. It covers the majority of existing patients.
Who is the study for?
This trial is for individuals aged 12-60 with genetically confirmed LGMD2I/R9, weighing over 30 kg. They must understand and consent to study procedures, use effective contraception if of childbearing or reproductive potential, and be able to complete all study tasks like biopsies. Exclusions include significant other diseases, severe kidney issues, recent surgeries affecting assessments, pregnancy/breastfeeding intentions within the study period through 12 weeks after last dose.Check my eligibility
What is being tested?
The trial tests BBP-418 (Ribitol) for safety and effectiveness in treating Limb Girdle Muscular Dystrophy type 2I (LGMD2I). Participants will receive either the investigational drug or a placebo over a long-term period to see how well it works compared to not receiving the active treatment.See study design
What are the potential side effects?
While specific side effects are not listed here, participants may experience adverse reactions related to BBP-418 (Ribitol), which could range from mild symptoms like nausea or headaches to more serious ones depending on individual responses and pre-existing health conditions.
Fortify Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 36 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~36 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from baseline in North Star Assessment for Limb Girdle Muscular Dystrophy following 36 months of treatment to assess efficacy of BBP-418 or placebo
Frequency and severity of treatment-emergent adverse events following 36 months of treatment to assess safety of BBP-418 or placebo
Secondary outcome measures
Change from baseline in 10 meter walk test velocity to assess the clinical efficacy of BBP-418 in patients with LGMD2I/R9
Change from baseline in pulmonary function as measured by FVC (percent predicted, performed in a sitting position) to assess the clinical efficacy of BBP-418 in patients with LGMD2I/R9
Change from baseline in the Performance of Upper Limb scale 2.0 to assess the clinical efficacy of BBP-418 in patients with LGMD2I/R9
Other outcome measures
Change from baseline in glycosylated Alpha dystroglycan / total glycosylated Alpha dystroglycan ratio to assess biomarkers for clinical efficacy of BBP-418 in patients with LGMD2I/R9
Change from baseline in total glycosylated Alpha dystroglycan to assess biomarkers for clinical efficacy of BBP-418 in patients with LGMD2I/R9
Fortify Trial Design
2Treatment groups
Active Control
Placebo Group
Group I: BBP-418Active Control2 Interventions
BBP-418 Granules for Oral Solution will be supplied as granules in tri-ply PET/Aluminum/PE sachets for unit dose. The number of sachets to reconstitute will depend on the applicable dose to be delivered, 9 g BID or 12 g BID, as determined by the weight of the participant. The granules will be reconstituted in water for oral administration.
Group II: Placebo to Match BBP-418Placebo Group2 Interventions
The placebo will be identical to the BBP-418 Granules for Oral Solution in appearance, packaging, labeling, and storage conditions.
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Who is running the clinical trial?
ML Bio Solutions, Inc.Lead Sponsor
2 Previous Clinical Trials
115 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am willing and able to follow all study requirements, including getting biopsies.I agree to use effective birth control during and for 12 weeks after the last dose.I haven't used ribose or sugar alcohol supplements in the last 90 days.I haven't been on any experimental treatments or devices in the last 90 days.I have never had gene therapy for LGMD2I/R9.I have a genetic diagnosis of LGMD2I/R9 and show symptoms.I haven't taken steroids for muscular dystrophy in the last 90 days.I haven't had surgery for scoliosis or similar conditions in the past year.My body weight is over 30 kg.I do not have any major health issues besides LGMD2I/R9.I am willing to use effective birth control during the study.I am willing and able to follow all study procedures.My kidneys work well.I do not have any blood or platelet disorders that prevent muscle biopsies.I don't have any health issues that could make this study unsafe for me.I am between 12 and 60 years old and weigh more than 30 kg.I have a confirmed genetic diagnosis of LGMD2I/R9 and it affects my health.I am between 12 and 60 years old.I understand the study and agree to participate.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo to Match BBP-418
- Group 2: BBP-418
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Do individuals over the age of twenty qualify for participation in this research initiative?
"This clinical trial is open to patients aged 12 and over, up until the age of 60."
Answered by AI
Has BBP-418 attained regulatory approval from the FDA?
"Our assessment of BBP-418's safety is a 3 since the trial has reached Phase 3, implying that there are sufficient data to support its efficacy and multiple rounds confirming its security."
Answered by AI
Could I potentially qualify for participation in this experiment?
"To meet the criteria of this study, participants must have limb girdle muscular dystrophy and reside between 12-60 years old. The medical trial is accepting up to 81 individuals."
Answered by AI
Is sign-up for this research study ongoing?
"As reported on clinicaltrials.gov, the recruitment for this medical trial has been halted since March 16th 2023. Initially posted in April 1st 2023, there are no longer any available spots but 89 other trials actively recruiting patients remain accessible."
Answered by AI
Who else is applying?
What site did they apply to?
Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront
What portion of applicants met pre-screening criteria?
Met criteria
Why did patients apply to this trial?
I am willing to do anything to support a cure for this disease.
PatientReceived 1 prior treatment
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