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

Primary Purpose

Fragile X Syndrome

Status
Withdrawn
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Sulindac (HLX-0201), dose strength 1
Sulindac (HLX-0201), dose strength 2
Placebo
Gaboxadol (HLX-0206)
Sponsored by
Healx Limited
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fragile X Syndrome focused on measuring Fragile X Syndrome, FXS

Eligibility Criteria

13 Years - 40 Years (Child, Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital Colorado
  • University of Miami
  • Emory University School of Medicine
  • Rush University Medical Center & Children's Hospital
  • Kennedy Krieger Insitute
  • University of Massachusetts Chan Medical School
  • Icahn School of Medicine (Mount Sinai)
  • Children's Health Queensland Hospital and Health Service
  • Fragile X Alliance Clinic
  • Murdoch Children's Research Institute

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Active Comparator

Active Comparator

Active Comparator

Arm Label

Placebo

Sulindac (HLX-0201), dose strength 1

Sulindac (HLX-0201), dose strength 2

Gaboxadol (HLX-0206)

Arm Description

One capsule, twice a day

One capsule, twice a day

One capsule, twice a day

One capsule, twice a day

Outcomes

Primary Outcome Measures

NIH Cognitive Toolbox
Clinical Global Impression - I
Aberrant Behavior Checklist
Anxiety, Depression, and Mood Scale
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.

Secondary Outcome Measures

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.
Kiddie Test of Attentional Performance (KiTAP)
Emotional Faces Tobii Eye Tracking
EEG
Resting state whole brain EEG spectra activity in 3 EEG assessments (EEG spectra, auditory evoked response potential (ERP) and Chirp modulated sweep.
CGI-S

Full Information

First Posted
March 24, 2021
Last Updated
December 7, 2022
Sponsor
Healx Limited
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1. Study Identification

Unique Protocol Identification Number
NCT04823052
Brief Title
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
Acronym
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)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Withdrawn
Why Stopped
Healx has experienced delays to the site activation of the study and this has had an adverse impact on the recruitment timeline which is delaying the progress of other projects in our FXS programme.
Study Start Date
May 25, 2022 (Actual)
Primary Completion Date
October 19, 2022 (Actual)
Study Completion Date
October 19, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Healx Limited

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome
Keywords
Fragile X Syndrome, FXS

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Subjects will be randomly assigned to 1 of 4 treatment arms. All treatment arms will be conducted in parallel. Fifteen subjects will be included in each arm. The study treatment will be blinded to patients, carers and physicians.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
One capsule, twice a day
Arm Title
Sulindac (HLX-0201), dose strength 1
Arm Type
Active Comparator
Arm Description
One capsule, twice a day
Arm Title
Sulindac (HLX-0201), dose strength 2
Arm Type
Active Comparator
Arm Description
One capsule, twice a day
Arm Title
Gaboxadol (HLX-0206)
Arm Type
Active Comparator
Arm Description
One capsule, twice a day
Intervention Type
Drug
Intervention Name(s)
Sulindac (HLX-0201), dose strength 1
Intervention Description
Sulindac (HLX-0201) Capsule
Intervention Type
Drug
Intervention Name(s)
Sulindac (HLX-0201), dose strength 2
Intervention Description
Sulindac (HLX-0201) Capsule
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo Capsule
Intervention Type
Drug
Intervention Name(s)
Gaboxadol (HLX-0206)
Intervention Description
Gaboxadol (HLX-0206) Capsule
Primary Outcome Measure Information:
Title
NIH Cognitive Toolbox
Time Frame
Day 70
Title
Clinical Global Impression - I
Time Frame
Day 70
Title
Aberrant Behavior Checklist
Time Frame
Day 70
Title
Anxiety, Depression, and Mood Scale
Time Frame
Day 70
Title
FXS Domain Specific Concerns
Description
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.
Time Frame
Day 70
Secondary Outcome Measure Information:
Title
To assess the safety and tolerability of each dose
Description
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.
Time Frame
Day 70
Title
Kiddie Test of Attentional Performance (KiTAP)
Time Frame
Day 70
Title
Emotional Faces Tobii Eye Tracking
Time Frame
Day 70
Title
EEG
Description
Resting state whole brain EEG spectra activity in 3 EEG assessments (EEG spectra, auditory evoked response potential (ERP) and Chirp modulated sweep.
Time Frame
Day 70
Title
CGI-S
Time Frame
Day 70

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elizabeth Berry-Kravis
Organizational Affiliation
Rush University Medical Center & Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Rush University Medical Center & Children's Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Kennedy Krieger Insitute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
University of Massachusetts Chan Medical School
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
10655
Country
United States
Facility Name
Icahn School of Medicine (Mount Sinai)
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Children's Health Queensland Hospital and Health Service
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Facility Name
Fragile X Alliance Clinic
City
Caulfield
State/Province
Victoria
ZIP/Postal Code
3161
Country
Australia
Facility Name
Murdoch Children's Research Institute
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia

12. IPD Sharing Statement

Learn more about this trial

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

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