Investigation of Sulindac in Male Fragile X Syndrome Patients Aged 13-40

• Status: Not yet recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: Male

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Healx Limited
• Treatments: Sulindac

Criteria

Inclusion Criteria:

• Subject and caregiver are both able to understand English clearly and caregiver can read and write English to complete study assessments

• Males aged 13-40 years (inclusive)

• Has FXS with molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats).

• Weight ≥45 kg

• 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

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

• Individual or family history of myocardial infarction, TIA, peripheral arterial disease, stroke or other significant cardiovascular disease

• Chronic use of NSAIDs or other anti-inflammatory agents

• Currently taking or have taken any cannabidiol (CBD) preparation within 30 days prior to randomization

• Currently taking or have taken sulindac within 30 days prior to randomization

• Changes in chronic psychotropic or anti-convulsant (where taken for reasons other than seizure control) drug treatment within 30 days prior to randomization

• 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 current or suspected symptoms of COVID-19 including, but not limited to fever, dry cough, new loss or change of smell or taste, myalgia, fatigue, or a positive COVID-19 test.

• Has abnormal baseline laboratory assessments including, but not limited to alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >1.5 × the upper limit of normal (ULN), total bilirubin or creatinine >1.0 × ULN, or other clinically relevant laboratory abnormality

• Has a clinically significant abnormal electrocardiogram (ECG), heart rate, or blood pressure 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