Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
NCT ID: NCT04823052
Last Updated: 2022-12-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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WITHDRAWN
PHASE2
INTERVENTIONAL
2022-05-25
2022-10-19
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
One capsule, twice a day
Placebo
Placebo Capsule
Sulindac (HLX-0201), dose strength 1
One capsule, twice a day
Sulindac (HLX-0201), dose strength 1
Sulindac (HLX-0201) Capsule
Sulindac (HLX-0201), dose strength 2
One capsule, twice a day
Sulindac (HLX-0201), dose strength 2
Sulindac (HLX-0201) Capsule
Gaboxadol (HLX-0206)
One capsule, twice a day
Gaboxadol (HLX-0206)
Gaboxadol (HLX-0206) Capsule
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
13 Years
40 Years
MALE
No
Sponsors
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Healx Limited
INDUSTRY
Responsible Party
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Principal Investigators
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Elizabeth Berry-Kravis
Role: PRINCIPAL_INVESTIGATOR
Rush University Medical Center & Children's Hospital
Locations
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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
Countries
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Other Identifiers
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HLX002-0201
Identifier Type: -
Identifier Source: org_study_id
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