IMPACT-FXS clinical trial

We are sponsoring a Phase 2 clinical trial (clinicaltrials.gov identifier NCT04823052) to explore the safety and efficacy of several potential medications for fragile X syndrome. The IMPACT-FXS study (which stands for Investigating Multiple Pathways And Combined Treatments for FXS) is the world’s first umbrella trial for the investigation of therapies for FXS, and will evaluate several compounds first as monotherapies and then as combination therapies in order to identify the best medication. Check your eligibility with our quick questionnaire.

What is the aim of the study?
This first phase of this research study will test two investigational medications (HLX-0201 and HLX-0206) in 13- to 40- year-old boys and men with fragile X syndrome (FXS). The purpose of this study is to test the safety and tolerability of the medications, and to see if they reduce symptoms of FXS.

What are the study procedures & duration?
Potential participants with FXS will be screened carefully for participation in the study. If selected, the participant will be randomly assigned to one of four groups: three treatment groups and one control group, which acts as a baseline for comparison.

This is a blinded study, which means neither the participant nor the study doctor will know which treatment the participant is receiving (although, in the event of a circumstance where a doctor needs to know which group a participant is assigned to, the study doctor will be able to find out what treatment they are receiving).

The study will take 14 weeks in total. During the study, procedures that participants will take part in will include cognitive and behavioural assessments, medical history, medication diaries, blood work and EEG (at some sites).

What are the good things that can happen from this research?
This research will examine candidate medications that could be new treatments for males with FXS to see if the medications are safe and have the intended effects.

HLX-0201 is a medication that has been approved in the US for indications other than FXS by the US Food and Drug Administration (FDA), an authority that regulates medicines. HLX-0206 is an investigational medicine that has been examined in a number of clinical trials involving children and adults for neurological conditions.

It is Healx’s hope that this study will lead to a better understanding of these study medications as potential treatments for FXS. The information gained in this study may be useful in treating other people diagnosed with FXS in the future.

What are the bad things that can happen from this research?
In a research study, not all of the risks or side effects may be known before you start the study. Everyone in the research study will be monitored continuously and carefully for side effects. Regular, biweekly visits, as well as an independent safety monitoring board will assure the close follow up of the participants.

Of note, HLX-0201 and HLX-0206 have known safety profiles: HLX-0201 has already been approved in the US and the EU for several decades, however in different indications than fragile X syndrome. Doses of HLX-0201 are confirmed as well tolerated with few adverse events reported. HLX-0206 has been tested in approximately 4500 people diagnosed with fragile X syndrome previously, where it was generally safe and well tolerated.

What are the eligibility criteria to take part in the study?

You may be eligible to take part if the following criteria apply to you:

• Male
• Fragile X syndrome suspected or already diagnosed
• Between 13 and 40 years of age
• Weight of 45kg or more
• Willing and able to take oral tablets
• Willing and able to travel to study sites
• Not diagnosed with diabetes mellitus type 1 or type 2

Other criteria might prevent you from being allowed to participate in the study (e.g. diagnosis of other conditions or current use of certain medication). Take our survey to see if you're eligible.