Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04823052




Registration number
NCT04823052
Ethics application status
Date submitted
24/03/2021
Date registered
30/03/2021
Date last updated
8/12/2022

Titles & IDs
Public title
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
Scientific 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)
Secondary ID [1] 0 0
HLX002-0201
Universal Trial Number (UTN)
Trial acronym
IMPACT-FXS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fragile X Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sulindac (HLX-0201), dose strength 1
Treatment: Drugs - Sulindac (HLX-0201), dose strength 2
Treatment: Drugs - Placebo
Treatment: Drugs - Gaboxadol (HLX-0206)

Placebo Comparator: Placebo - One capsule, twice a day

Active Comparator: Sulindac (HLX-0201), dose strength 1 - One capsule, twice a day

Active Comparator: Sulindac (HLX-0201), dose strength 2 - One capsule, twice a day

Active Comparator: Gaboxadol (HLX-0206) - One capsule, twice a day


Treatment: Drugs: Sulindac (HLX-0201), dose strength 1
Sulindac (HLX-0201) Capsule

Treatment: Drugs: Sulindac (HLX-0201), dose strength 2
Sulindac (HLX-0201) Capsule

Treatment: Drugs: Placebo
Placebo Capsule

Treatment: Drugs: Gaboxadol (HLX-0206)
Gaboxadol (HLX-0206) Capsule
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
NIH Cognitive Toolbox
Timepoint [1] 0 0
Day 70
Primary outcome [2] 0 0
Clinical Global Impression - I
Timepoint [2] 0 0
Day 70
Primary outcome [3] 0 0
Aberrant Behavior Checklist
Timepoint [3] 0 0
Day 70
Primary outcome [4] 0 0
Anxiety, Depression, and Mood Scale
Timepoint [4] 0 0
Day 70
Primary outcome [5] 0 0
FXS Domain Specific Concerns
Timepoint [5] 0 0
Day 70
Secondary outcome [1] 0 0
To assess the safety and tolerability of each dose
Timepoint [1] 0 0
Day 70
Secondary outcome [2] 0 0
Kiddie Test of Attentional Performance (KiTAP)
Timepoint [2] 0 0
Day 70
Secondary outcome [3] 0 0
Emotional Faces Tobii Eye Tracking
Timepoint [3] 0 0
Day 70
Secondary outcome [4] 0 0
EEG
Timepoint [4] 0 0
Day 70
Secondary outcome [5] 0 0
CGI-S
Timepoint [5] 0 0
Day 70

Eligibility
Key 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
Minimum age
13 Years
Maximum age
40 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
25/05/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
19/10/2022
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Children's Health Queensland Hospital and Health Service - Brisbane
Recruitment hospital [2] 0 0
Fragile X Alliance Clinic - Caulfield
Recruitment hospital [3] 0 0
Murdoch Children's Research Institute - Melbourne
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment postcode(s) [2] 0 0
3161 - Caulfield
Recruitment postcode(s) [3] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
New York

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Healx Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04823052
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Elizabeth Berry-Kravis
Address 0 0
Rush University Medical Center & Children's Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04823052