US-based Aevi Genomic Medicine has initiated its Phase I/II clinical trial with the enrolment and dosing of its first patient with AEVI-001 (NFC-1) to treat 22q11.2 deletion syndrome (22q DS) in children.

AEVI-001 is an investigational glutamate modulator designed to relieve psychiatric symptoms associated with 22q DS patients caused due to mutations in RANBP1 gene that disrupts glutamatergic signalling.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The Phase I/II trial will evaluate the changes in symptoms observed in three neuropsychiatric disorders, anxiety, attention deficit hyperactivity disorder (ADHD) and autism spectrum disorders (ASD), which are often found in patients affected by 22q DS.

Aevi Genomic Medicine chief scientific officer Garry Neil said: “We are pleased to have the first patient enrolled into this important study.

“We are pleased to have the first patient enrolled into this important study.”

“Patients with 22q Deletion Syndrome often suffer from a range of debilitating psychiatric conditions, including anxiety disorders, ASD, and ADHD, with many patients progressing to psychosis and schizophrenia.”

22qDS is a condition caused by the deletion of a small piece of chromosome 22 and is characterised by heart and facial abnormalities, as well as cleft palate.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The trial is to be conducted at the Children’s Hospital of Philadelphia (CHOP), US, and will enrol 22q DS patients aged between 12 and 17 years with a diagnosis of anxiety, ASD, or ADHD.

It will be ensured that the patients have a confirmed deletion of the RANBP1 gene and will receive five weeks of open-label treatment with AEVI-001.

The primary endpoints will be the changes from baseline on established metrics related to the three neuropsychiatric disorders.