Clinical trials of three experimental new treatments for Fragile X are accepting participants
3/22/2012
There are active trials with two different mGluR5 antagonists - one from Novartis, the other from Roche - and arbaclofen from Seaside Therapeutics - in large scale trials.

Novartis AFQ056 – an mGluR5 antagonist

Ages 12-45
This is a placebo-controlled period of 4 months with 8 visits to study site.   Patients can be on up to 2 other psychoactive drugs when starting trial (any 2).  Travel reimbursement is available.  Participants can continue on the drug after the trial – this open label extension phase has 11 visits to study site over 2 years.

NEW: Ages 5-11: Child PK Trial
at Rush University Medical Center in Chicago; Dr. Berry-Kravis, Principal Investigator, and at Vanderbilt University in Nashville, Tennessee; Dr. Veenstra-VanderWeele, Principal investigator. This intensive study requires 7 visits with lots of blood draws over 2 months; 3 of the visits are overnight stays at the hotel by the hospital. Anyone can be in this trial – there are no medication, IQ, or behavioral restrictions. Travel costs may be covered including flights, hotel, and distance travel. People who complete it will be accepted into the open-label extension Phase III trial directly (will not have to do placebo-controlled phase of that trial), if and when that opens. 
PK = Pharmacokinetics which may be simply defined as what the body does to a drug.

 

Seaside STX209 – a GABA(b) agonist, chemically related to the existing drug baclofen

Ages 5-50
This is a Phase III double-blind placebo-controlled 2-month-long trial with 5-6 visits to the study site. Participants have to discontinue taking SSRIs and antidepressants to be in this trial. Some travel cost reimbursement is available, and participants can continue taking the drug after completing the placebo-controlled trial (open label extension phase).

  

Roche RO4917523 – an mGluR5 antagonist

NOW RECRUITING Ages 16-50
This is a phase II trial with a placebo-controlled period of 3 months. Certain prescribed medications are allowed during the trial. There are 7 visits to the study site over 4-5 months. Travel costs are covered.

Contacts, locations & details for all trials