BridgeMD
Tests treatment safety and results for Duchenne Muscular Dystrophy
Official title A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov ID: NCT06817382
What this study is testing
What is INS1201?
INS1201 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 4, men only
You may be able to join if
- Participant must be male at birth, 3 to \<5 years of age, inclusive (Part 1) and 2 to \<3 years of age (Part 2), at the time of legally authorized...
- Ambulatory -as defined as the ability to walk at least 10 meters unassisted (ie, without personal assistance or use of any assistive devices) Note...
- Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not...
- Able to cooperate with motor assessment testing.
- Has received vaccinations recommended for the participant's age and DMD disease according to Centers for Disease Control and Prevention (CDC) Child...
You likely can't join if
- Prior treatment with gene or cell-based therapy at any time.
- Oligonucleotide-based exon skipping or small molecule stop codon readthrough-promoting therapies for at least 6 months prior to enrolment.
- Has left ventricular ejection fraction \< 50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy.
- Has cardiac arrhythmia or significant electrocardiogram (ECG) interval abnormalities.
- Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study at any time during...
- The presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic/allergic...
See the full eligibility criteria
- Participant must be male at birth, 3 to \<5 years of age, inclusive (Part 1) and 2 to \<3 years of age (Part 2), at the time of legally authorized representative (LAR) signing and dating the informed consent form.
- Ambulatory -as defined as the ability to walk at least 10 meters unassisted (ie, without personal assistance or use of any assistive devices) Note: children who have not yet developed the ability to walk by the time of...
- Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already...
- Able to cooperate with motor assessment testing.
- Has received vaccinations recommended for the participant's age and DMD disease according to Centers for Disease Control and Prevention (CDC) Child and Adolescent Immunization Schedule by Age, World Health Organization...
- Prior treatment with gene or cell-based therapy at any time.
- Oligonucleotide-based exon skipping or small molecule stop codon readthrough-promoting therapies for at least 6 months prior to enrolment.
- Has left ventricular ejection fraction \< 50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy.
- Has cardiac arrhythmia or significant electrocardiogram (ECG) interval abnormalities.
- Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study at any time during this study.
- The presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic/allergic, behavioural disease, infection, unhealed injury, malignancy, concomitant...
- Creates unnecessary risks for undergoing gene transfer;
- Might compromise the participant's ability to comply with the protocol-required testing or procedures; or
- Might compromise the participant's well-being, safety, or clinical interpretability.
- Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
- Has signs of clinically significant symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
- Has contraindications for IT administration of the product or for lumbar puncture, such as anatomical abnormalities, bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or significant clotting...
- Demonstrates cognitive or developmental delay or impairment that could confound assessment of motor development in the opinion of the Investigator.
- Total serum anti-AAV9 antibody titers of \> 1:50 as determined by ELISA within 14 days of Day 1. Note: Other inclusion/exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Davis, California, United States
- Los Angeles, California, United States
- Palo Alto, California, United States
- San Diego, California, United States
- Atlanta, Georgia, United States
- Rochester, New York, United States
- Columbus, Ohio, United States
- Memphis, Tennessee, United States
- Norfolk, Virginia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years to 4 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Davis, California, United States; Los Angeles, California, United States; Palo Alto, California, United States; San Diego, California, United States; Atlanta, Georgia, United States and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.