Ann: Positive FDA meetings enable INDs for three Phase 2 trials, page-11

It’s likely that competing trials are behind Neuren’s decision to hold its Angelman syndrome Phase 2 trial here in Australia.

Angelman syndrome

Ultragenyx Pharmaceutical + GeneTx Biotherapeutics commenced a Phase 1/2 Safety and Tolerability Study of GTX-102 in children with Angelman Syndrome (KIK-AS) in February last year. The estimated completion date is January next year. It’s a 6 month, 20 participant, open-label trial. GTX-102 is an antisense therapy, delivered by lumbar injection in 3-4 monthly doses and followed by a quarterly maintenance regimen. There are 5 trial sites, 4 in the US and 1 in Canada.

https://clinicaltrials.gov/ct2/show/NCT04259281?cond=Angelman Syndrome&draw=3

https://angelmansyndromenews.com/ne...tragenyx-may-support-restart-phase-1-2-trial/


Roche/Genentech is investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 in patients aged 1 to 12 years with Angelman Syndrome. The Tangelo Phase 1 study aims to enrol 66 participants. The study commenced last August and is due to complete in December next year. Like GTX-102, RO7248824 is an antisense therapy delivered by lumbar injection. Dosing is over a period of 8 weeks, with a minimum of approximately 4 weeks between each dose. There is a 12 month assessment period. The trial is happening at 5 US sites and 5 European sites.

https://clinicaltrials.gov/ct2/show/NCT04428281?term=BP41674&draw=2&rank=1


Ionis Pharmaceuticals intends to launch a clinical trial later this year to test ION582, another experimental antisense therapy. This will be a Phase 1/2a global trial, expected to enrol up to 50 patients.

https://angelmansyndromenews.com/ne...cal-trial-this-year-ion582-angelman-syndrome/

https://www.angelmanclinicaltrials.com/industrysponsors



Pitt-Hopkins syndrome

Pitt-Hopkins syndrome appears to have only one current study, being conducted by the Pitt Hopkins Research Foundation in conjunction with Arizona State University. The study is assessing an oral combination product of vancomycin, magnesium citrate and microbiota. The 10 participant Phase 2 trial commenced in September 2019 and is expected to complete in February next year. The 14 week study is being held at one centre in Arizona.

https://clinicaltrials.gov/ct2/show/NCT04132427



Phelan McDermid syndrome

Phelan McDermid syndrome appears to have two current studies.

One is a Phase 2 study of IGF-1, administered subcutaneously for 3 months. The trial is sponsored by the Icahn School of Medicine at Mount Sinai and National Institute of Mental Health (NIMH). The 35 participant trial commenced in 2012 and is expected to complete in April 2023. It is being held at just one US location.

https://clinicaltrials.gov/ct2/show/NCT01525901?term=phelan mcdermid syndrome&draw=2&rank=8


The other is a Phase 3 trial, investigating the effect of Lithium in patients with Autism Spectrum Disorder and Phelan-McDermid Syndrome. The trial is sponsored by Assistance Publique - Hôpitaux de Paris. The trial was supposed to start in January this year and complete in May 2023.  The lithium will be administered as an oral treatment in 22 patients in a 12 week trial being held at one location in France

https://clinicaltrials.gov/ct2/show/NCT04623398?term=phelan mcdermid syndrome&draw=2&rank=9