Healx Is Recruiting Study Participants for Its IMPACT-FXS Study

Healx_Trial logos_Fragile x-colour

Investigating Multiple Pathways And Combined Treatments for FXS

Healx is recruiting adolescent and adult (13-40 years old) males with Fragile X syndrome (FXS) for its IMPACT-FXS study in the US. This Phase 2, placebo controlled study will evaluate the safety, tolerability and efficacy of two potential medications, HLX-0201 (sulindac, at 2 different doses) and HLX-0206 (gaboxadol). The aim is to confirm whether these drug candidates are safe and work as intended to reduce symptoms of FXS.

This study has an umbrella design, meaning that not one, but several drug candidates will be evaluated under the same protocol and against placebo. Investigating HLX-0201 and HLX-0206 comprises the first stage of the study, and then, in the next stage, Healx is aiming to add other candidates. This innovative approach allows Healx to identify suitable mono- and/or combination candidates to be evaluated in upcoming trials.

About the Trial

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 blinded condition. This gives a 75% chance of receiving an active treatment.

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

Why this study?

IMPACT-FXS is the world’s first umbrella trial for the investigation of therapies for Fragile X syndrome (FXS). The innovative design allows multiple compounds to be evaluated in parallel, and in sequence, in order to identify potential combination medications in subsequent stages of development. Healx has and continues to work closely with FXS patient organizations like FRAXA Research Foundation to redevelop these compounds for FXS, ensuring that patient insights are embedded into the study.

During the 3-month trial participation, individuals with FXS will be closely followed and symptoms will be carefully evaluated. The study participant will meet with their doctor six times in the clinic, as well as twice by phone. The evaluations cover the majority of the potential difficulties experienced by someone affected by FXS: learning disabilities, behavioural and emotional problems, as well as their general clinical condition. The comprehensive evaluation will consist of questionnaires, physical and further medical examinations. There is a 75% chance of receiving an active drug vs a 25% chance of receiving a placebo.

Enrollment Information

Rush University Medical Center
Recruiting

Loren Escot
(312) 942-4036
loren_escot@rush.edu

University of Massachusetts Chan Medical School
Recruiting

(774) 455-4100
ChildResearch@umassmed.edu

University of Colorado School of Medicine,
Children's Hospital Colorado
Not Yet Recruiting

Hannah Flemming
Hannah.Fleming@childrenscolorado.org

Mt Sinai Hospital
Not Yet Recruiting

Karli Hedstrom
karli.hedstrom@mssm.edu

Kennedy Krieger Institute
Not Yet Recruiting

Nadia Moore
(443) 923-7619
MooreNa@kennedykrieger.org

University of Miami,
Mailman Centre for Child Development
Not Yet Recruiting

Yaneysy Penate
(646) 527-1042
yxp303@med.miami.edu

Emory University
Not Yet Recruiting

Jean Luan McColl
jean.luan@emory.edu