MARVEL-1, Freeline’s Phase I/II clinical study exploring the potential of FLT190 investigational gene therapy to treat Fabry disease is recruiting participants now
Fabry disease is caused by changes in a gene called GLA that contains the instructions for cells in the human body to make an enzyme called alpha-Gal A. Having too little alpha-Gal A causes build-up of a fatty substance in cells and tissues throughout the body. This results in the symptoms of Fabry disease.
FLT190 is an investigational gene therapy that is designed to deliver a working version of the GLA gene to people with Fabry disease. To get the GLA gene into the cells, it is packaged into the outer ‘coat’ of a virus, which does not cause infections. FLT190 uses a modified virus called adeno-associated virus, or AAV, to deliver the GLA gene into the liver cells. Using the new GLA gene, the alpha-Gal A enzyme is then made by the liver cells and released into the bloodstream where it can quickly reach many other parts of the body. The alpha-Gal A can then be taken up by organs and tissues to help prevent the build-up of fatty substances that cause symptoms in Fabry disease.
FLT190 is delivered to the participant by a one-time slow intravenous infusion. Participants then also receive additional medications to prevent and manage their body’s immune response to FLT190.
MARVEL-1 is a Phase I/II clinical study designed to investigate safety of FLT190 and to determine if it can potentially improve symptoms in people with Fabry disease.
Eligible Participants
In addition to other criteria, to take part, the participant must:
– Be male, aged 18 or older
– Have a confirmed diagnosis of classic Fabry disease
Learn More
The MARVEL-1 study is taking place in multiple countries in Europe and North America.
For more information, go to www.clinicaltrials.gov and search for NCT04040049 or email clinicaltrials@freeline.life.
MRC-2023-009