GNT0006 Gene Therapy Trial in Patients With LGMDR9

Launched by ATAMYO THERAPEUTICS · Feb 3, 2022

Keywords

ClinConnect Summary

The GNT0006 Gene Therapy Trial is a research study aimed at testing a new treatment for patients with a condition called LGMDR9. This condition affects muscle function, particularly the muscles that help with breathing. The trial is happening in two stages: the first stage will look at the safety of different doses of the treatment and whether it works, while the second stage will compare the treatment to a placebo (a substance with no active treatment) to confirm its effects. The study is currently recruiting participants aged 16 and older who are able to walk, have been diagnosed with LGMDR9, and show moderate muscle impairment related to breathing.

To be eligible for the trial, participants need to have a confirmed diagnosis based on their symptoms and genetic testing. However, people with certain health issues, like heart problems or specific antibodies that could interfere with the treatment, cannot take part. Those who join the trial can expect to receive either the gene therapy or a placebo, and they will be closely monitored throughout the study to assess their safety and health. This trial is an important step toward finding new therapies for LGMDR9, and participation could contribute to future treatments for this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Female and male ambulant patients
• • 2. Patients ≥ 16 years old
• • 3. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
• • 4. Moderate diaphragmatic muscle impairment
• Exclusion Criteria:
• • 1. Detectable serum neutralizing antibodies against AAV9
• • 2. Cardiomyopathy