Fragile X SyndromePhase 2IMPACT-FXSNCT04823052
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
A Phase 2 study testing Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2 and Gaboxadol (HLX-0206) in people with Fragile X Syndrome, run by Healx Limited at 10 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 13 to 40 years, men only
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2 and Gaboxadol (HLX-0206)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 25 May 2022to 19 Oct 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 40 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 10Show fewer
- 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
Who cannot join
- 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
Show all 17Show fewer
- 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
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States7 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Insitute
Baltimore, Maryland, 21205
Rush University Medical Center & Children's Hospital
Chicago, Illinois, 60612
University of Miami
Miami, Florida, 33136
Icahn School of Medicine (Mount Sinai)
New York, New York, 10029
University of Massachusetts Chan Medical School
Worcester, Massachusetts, 10655
Australia3 sites
Children's Health Queensland Hospital and Health Service
Brisbane, Queensland, 4101
Fragile X Alliance Clinic
Caulfield, Victoria, 3161
Murdoch Children's Research Institute
Melbourne, Victoria, 3052
About this study
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)
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.
- Conditions
- Fragile X Syndrome
- Being tested
- Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2, Placebo, Gaboxadol (HLX-0206)
- Keywords
- Fragile X Syndrome, FXS
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sulindac (HLX-0201), dose strength 1Drug
Sulindac (HLX-0201) Capsule
Sulindac (HLX-0201), dose strength 2Drug
Sulindac (HLX-0201) Capsule
PlaceboDrug
Placebo Capsule
Gaboxadol (HLX-0206)Drug
Gaboxadol (HLX-0206) Capsule
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | One capsule, twice a day Receives: Drug: Placebo |
| Sulindac (HLX-0201), dose strength 1 | Active comparator | One capsule, twice a day Receives: Drug: Sulindac (HLX-0201), dose strength 1 |
| Sulindac (HLX-0201), dose strength 2 | Active comparator | One capsule, twice a day Receives: Drug: Sulindac (HLX-0201), dose strength 2 |
| Gaboxadol (HLX-0206) | Active comparator | One capsule, twice a day Receives: Drug: Gaboxadol (HLX-0206) |
What is being measured
The primary outcome is the main question the study is designed to answer.
NIH Cognitive Toolbox
Measured: Day 70
Clinical Global Impression - I
Measured: Day 70
Aberrant Behavior Checklist
Measured: Day 70
Anxiety, Depression, and Mood Scale
Measured: Day 70
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.
Measured: Day 70
Secondary outcomes (5)
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.
Measured: Day 70
Kiddie Test of Attentional Performance (KiTAP)
Measured: Day 70
Emotional Faces Tobii Eye Tracking
Measured: Day 70
EEG
Resting state whole brain EEG spectra activity in 3 EEG assessments (EEG spectra, auditory evoked response potential (ERP) and Chirp modulated sweep.
Measured: Day 70
CGI-S
Measured: Day 70
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2021
- Study start25 May 2022
- Primary completion19 Oct 2022
- Study completion19 Oct 2022
Why it 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Healx Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elizabeth Berry-Kravis, Principal investigator, Rush University Medical Center & Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04823052
- Sponsor's protocol ID
- HLX002-0201
- Acronym
- IMPACT-FXS
- Status last verified
- Dec 2022
- First submitted
- 24 Mar 2021
- Last updated
- 8 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04823052
- Acronym / sponsor ID
- IMPACT-FXS / HLX002-0201
- Conditions
- Fragile X Syndrome
- Keywords
- Fragile X Syndrome, FXS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason 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.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Healx LimitedIndustry (pharma, biotech, devices)
- Study start
- 25 May 2022 (actual)
- Primary completion
- 19 Oct 2022 (actual)
- Study completion
- 19 Oct 2022 (actual)
- First posted
- 30 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- 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.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboOne capsule, twice a day |
| Sulindac (HLX-0201), dose strength 1 | Active comparator | Drug: Sulindac (HLX-0201), dose strength 1One capsule, twice a day |
| Sulindac (HLX-0201), dose strength 2 | Active comparator | Drug: Sulindac (HLX-0201), dose strength 2One capsule, twice a day |
| Gaboxadol (HLX-0206) | Active comparator | Drug: Gaboxadol (HLX-0206)One capsule, twice a day |
Who may qualify
- Ages
- 13 to 40 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (17 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sulindac (HLX-0201), dose strength 1Sulindac (HLX-0201) Capsule | Drug | Sulindac (HLX-0201), dose strength 1 |
| Sulindac (HLX-0201), dose strength 2Sulindac (HLX-0201) Capsule | Drug | Sulindac (HLX-0201), dose strength 2 |
| PlaceboPlacebo Capsule | Drug | Placebo |
| Gaboxadol (HLX-0206)Gaboxadol (HLX-0206) Capsule | Drug | Gaboxadol (HLX-0206) |
Primary outcome
NIH Cognitive Toolbox
Time frame: Day 70
Clinical Global Impression - I
Time frame: Day 70
Aberrant Behavior Checklist
Time frame: Day 70
Anxiety, Depression, and Mood Scale
Time frame: Day 70
FXS Domain Specific Concerns
Time frame: Day 70
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.
5 secondary outcomes
To assess the safety and tolerability of each dose
Time frame: Day 70
Kiddie Test of Attentional Performance (KiTAP)
Time frame: Day 70
Emotional Faces Tobii Eye Tracking
Time frame: Day 70
EEG
Time frame: Day 70
CGI-S
Time frame: Day 70
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2022.
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A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elizabeth Berry-Kravis principal investigator
Rush University Medical Center & Children's Hospital
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2 and Gaboxadol (HLX-0206) in people with Fragile X Syndrome, run by Healx Limited at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 8 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
NIH Cognitive Toolbox (time frame: Day 70). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2022.