Males aged 13-40 years old with fragile X syndrome needed for a research study

Healx is conducting a Phase 2, placebo-controlled study in adolescent and adult males with fragile X syndrome (FXS).  The purpose of this study is to evaluate the safety, tolerability and efficacy of HLX-0201 and HLX-0206 in males with FXS, meaning that this trial aims to confirm whether these drug candidates are safe and work as intended to reduce symptoms of FXS.

This study has a so-called umbrella design, meaning that not one, but several drug candidates will be evaluated in parallel and against placebo. Investigating HLX-0201 and HLX-0206 comprises the first stage of the study, and then, in the next stage, we are aiming to add other candidates. These candidates will be added to the current study in an adaptive manner. This innovative approach allows Healx to identify the best mono- or combination and evaluate them in upcoming trials.

About the Trial

Who can participate?

Males aged 13–40 years old with the full mutation of fragile X syndrome may be eligible to participate.

What will happen in the study?

The first stage of the study will investigate HLX-0201 (at 2 different doses) and HLX-0206, compared to placebo. Study participants will be randomly assigned to one of these four treatment groups in a blinded condition. This gives a 75% chance of receiving an active treatment.

During the study, some of the procedures that participants will take part in include cognitive and behavioral assessments, medical history, medication diaries, blood work and EEG (only at some sites).

What are the good things that can happen from this research?

This research will examine candidate medications that could be -if found efficacious and safe through the research- new treatments for males with FXS (HLX-0201 and HLX-0206).

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 patients 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. Doses of HLX-0201 are confirmed as well tolerated with few adverse events reported. HLX-0206 has been tested in approximately 4500 patients previously, where it was generally safe and well tolerated.

Will you/your child be paid to complete this study?

The study participant may be reimbursed as follows:

  • A daily limit of $30.00 per person applies to meal reimbursements for the study participant and their caregiver during site visits.
  • Travel costs will be reimbursed as incurred upon proof of receipt. Mileage will be reimbursed at the current Internal Revenue Service (IRS) rate for mileage.

If the study participant needs to travel to the study site, Healx will reimburse the reasonable costs caused by travelling to see the study doctor for the study participant and their caregiver.  Due to the length of the visits, the study participant and their caregiver will also be reimbursed (or provided a voucher) for meals depending on the timing of the study participant’s visit.

Reimbursement will be issued in the form of a cheque, a pre-loaded debit card, direct deposit or cash.  The form of reimbursement will be determined by study staff.

Who can I contact for more information about this study?

Please contact the study site closest to you for more details.

Name Location Study Coordinator Email Status
Rush University Medical Center Chicago, Illinois Loren Escot Email Recruiting
University of Massachusetts Chan Medical School Worcester, Massachusetts Taylor Merk Email Recruiting
University of Colorado School of Medicine, Children’s Hospital Colorado Aurora, Colorado Hannah Flemming Email Not Yet Recruiting
Emory University Atlanta, Georgia Jean Luan McColl Email Not Yet Recruiting
University of Miami, Mailman Centre for Child Development Miami, Florida Yaneysy Penate Email Not Yet Recruiting
Mt Sinai Hospital New York City, New York Karli Hedstrom Email Not Yet Recruiting
Kennedy Krieger Institute Baltimore, Maryland Nadia A. Moore Email Not Yet Recruiting

You can also visit the IMPACT-FXS ClinicalTrials.gov post or the IMPACT-FXS website for more details.

Interested in Participating?

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