Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40

Withdrawn before enrolling · Phase 2 · Has a placebo group

Conditions studied: Fragile X Syndrome

In brief

This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an outpatient setting.

Key facts

Study ID

NCT04823052

Run by

Healx Limited

People needed

0

Starts

2022-05-25

Expected to finish

2022-10-19

Last updated by the study team

2022-12-08

Who can join

Age: 13 and older, up to 40. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Subject consents to participate, or if the subject are not the subjects own legal guardian, offers assent supported by legally authorized representative consent. Caregiver also commits to the study requirements prior to any study-related procedures
• Willing and able to comply with the study procedures as specified in the protocol and to comply with the study drug administration
• Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments
• Males aged 13 to 40 years (inclusive)
• Has FXS with molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats). May have been confirmed historically or at Screening
• Weight ≥45 kg
• CGI-S score ≥4
• Is in general good health as deemed by the Investigator, determined by physical examination, medical history and laboratory tests
• If receiving sertraline, is on a stable, well-tolerated dose for the previous 3 months with no further changes anticipated
• Agrees not to discuss treatment outcomes on social media until subject has completed their End of Therapy visit

You may not qualify if…

• Active or history of peptic or gastric ulcer or hemorrhage
• Any chronic major medical comorbid condition deemed by the Investigator as presenting added risk to the subject, including but not limited to refractory hypertension, kidney disease, or liver disease
• Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication
• Unstable seizure disorder defined by any seizure within 6 months prior to baseline visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study entry
• Patients with cardiovascular disease risk factors: Uncontrolled high blood pressure (systolic blood pressure >150 mmHg), Unstable angina, History of documented myocardial infarction or cerebrovascular accident, NYHA Class III and IV heart failure, Known uncontrolled hyperlipidemia as LDL-C ≥190 mg/dL or triglycerides ≥ 500 mg/dL
• Chronic use of NSAIDs or other anti-inflammatory agents
• Currently taking or have taken any cannabidiol (CBD) preparation within 30 days prior to screening
• Currently taking or have taken sulindac or gaboxadol within 30 days prior to screening
• Currently taking GABAergic agents (i.e., acamprosate, baclofen, vigabatrin, tiagabine, riluzole, benzodiazepines, and gabapentin)
• Changes in psychotropic or anti-convulsant (where taken for reasons other than seizure control) drug treatment within 30 days prior to Screening
• Significant changes in any educational, behavioral and/or dietary interventions the month prior to Screening
• Planned initiation of new, or modification of ongoing, interventions during the study
• History of adverse effects of sulindac or other NSAIDs that would prevent safe study completion
• Unable or unwilling to take oral medication (whole capsule) or history of dysphagia or malabsorption
• Has abnormal baseline laboratory assessments including, but not limited to, ALT or AST or total bilirubin >1.5 × ULN, or other clinically relevant laboratory abnormality
• Has a clinically significant abnormal ECG, heart rate or BP at Screening as judged by the Investigator
• Has received an investigational drug (either approved or not approved) in any prior clinical study within 30 days or 5 half-lives (whichever is longer) prior to Screening

Where it is running

• Children's Hospital Colorado — Aurora, Colorado, United States
• University of Miami — Miami, Florida, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• Rush University Medical Center & Children's Hospital — Chicago, Illinois, United States
• Kennedy Krieger Insitute — Baltimore, Maryland, United States
• University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States
• Icahn School of Medicine (Mount Sinai) — New York, New York, United States
• Children's Health Queensland Hospital and Health Service — Brisbane, Queensland, Australia
• Fragile X Alliance Clinic — Caulfield, Victoria, Australia
• Murdoch Children's Research Institute — Melbourne, Victoria, Australia

Full record on ClinicalTrials.gov

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