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

Fragile X Syndrome Clinical Trial

— IMPACT-FXS  

Official title:

A Phase 2, Placebo-controlled, Adaptive Design Study to Explore the Safety and Efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) and Possible Other Treatments in Adolescent and Adult Males With Fragile X Syndrome (FXS)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04823052
• Other study ID #: HLX002-0201
• Status: Withdrawn
• Phase: Phase 2
• Start date: May 25, 2022
• Est. completion date: October 19, 2022

Study information

• Verified date: December 2022
• Source: Healx Limited
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 19, 2022
• Est. primary completion date: October 19, 2022
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 13 Years to 40 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• Sulindac (HLX-0201), dose strength 1
Sulindac (HLX-0201) Capsule
• Sulindac (HLX-0201), dose strength 2
Sulindac (HLX-0201) Capsule
• Placebo
Placebo Capsule
• Gaboxadol (HLX-0206)
Gaboxadol (HLX-0206) Capsule

Locations

Country	Name	City	State
Australia	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
United States	Emory University School of Medicine	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Kennedy Krieger Insitute	Baltimore	Maryland
United States	Rush University Medical Center & Children's Hospital	Chicago	Illinois
United States	University of Miami	Miami	Florida
United States	Icahn School of Medicine (Mount Sinai)	New York	New York
United States	University of Massachusetts Chan Medical School	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Healx Limited

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	NIH Cognitive Toolbox		Day 70
Primary	Clinical Global Impression - I		Day 70
Primary	Aberrant Behavior Checklist		Day 70
Primary	Anxiety, Depression, and Mood Scale		Day 70
Primary	FXS Domain Specific Concerns	The Clinician/Caregiver FXS Domain Specific Concerns allows for the subject specific symptoms of concern to be assessed on an ongoing basis throughout the study. The specific concerns that correlate to the 6 domains (Clinician) or 3 Domains (Caregiver) will be assessed using a 7-point Likert scale.	Day 70
Secondary	To assess the safety and tolerability of each dose	Incidence of adverse events (AEs) and serious adverse events (SAEs) reported during the study. Change from baseline to Day 70 in physical examinations and assessment of vital signs.	Day 70
Secondary	Kiddie Test of Attentional Performance (KiTAP)		Day 70
Secondary	Emotional Faces Tobii Eye Tracking		Day 70
Secondary	EEG	Resting state whole brain EEG spectra activity in 3 EEG assessments (EEG spectra, auditory evoked response potential (ERP) and Chirp modulated sweep.	Day 70
Secondary	CGI-S		Day 70