Fragile X SyndromePhase 2NCT02126995
A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
A Phase 2 study testing MG01CI extended-release tablet in people with Fragile X Syndrome, run by Alcobra Ltd. at 12 sites in 2 countries.
Trial summary
This study is a multisite, randomized, double-blind, placebo-controlled, phase 2 study of MG01CI (low dose and high dose once daily) for 6 weeks compared with placebo in a 1:1 ratio of 60 adolescent and adult subjects with Fragile X Syndrome (FXS). Read more about this study
- Who is it for?
- Ages 15 to 55 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- MG01CI extended-release tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 12 sitesin 2 countries
- Study dates
- Jun 2014to Jun 2015
- 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 15 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subject is a man or a non-pregnant, non-lactating woman aged 15 to 55 years, inclusive, at the Randomization Visit.
- Subject has Fragile X Syndrome with a molecular genetic confirmation of the full Fragile X Mental Retardation (FMR1) mutation (≥200 CGG repetitions).
- Subject has a score of 12 or greater on the inattentive subscale of the ADHD RS IV (as rated by the investigator in a clinical interview of the parent/legal authorized guardian/consistent caregiver).
- Current treatment with no more than 3 prescribed psychotropic medications. Anti epileptic medications are permitted and are not counted as psychotropic medications if they are used for treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
- Permitted concomitant psychotropic medications (except anti-epileptic medications and stimulants; see 4b and 4d) must be at a stable dose and dosing regimen for at least 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
- Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
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- Subjects with a history of seizure disorder who are currently receiving treatment with anti-epileptics must have been seizure-free for 3 months preceding Screening, or must be seizure-free for 3 years if not currently receiving anti-epileptics.
- Stimulant medications must be at a stable dose and dosing regimen for 12 weeks prior to Screening and must remain stable during the period between Screening and the end of the treatment period (Week 6/early termination), unless the subject is washing out; see Exclusion Criterion 4.
- Behavioral treatments (excluding psychotherapy; see exclusion criteria) must be stable for 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
- Subject has a parent, legal authorized guardian or consistent caregiver who interacts with the subject for at least 10 hours per week and is able to provide weekly rating forms of the subject's behavior.
- Male and Female subjects of childbearing potential must agree to use an effective contraceptive throughout the study (eg, oral contraceptives or Norplant®; a reliable double barrier method of birth control [diaphragms with contraceptive jelly; cervical caps with contraceptive jelly; condoms with contraceptive foam]; intrauterine devices; vasectomy; or abstinence) and for at least a month after the study, and females must have a negative serum pregnancy test at the Screening Visit and a negative urine pregnancy test at the Baseline Visit. Females of childbearing potential are defined as women who are between menarche and 2 years post-menopause and who are not surgically sterilized. Male and female subjects who are not sexually active, and who agree to be abstinent throughout the study, will not be required to use birth control.
- Subject and caregiver are able to attend the clinic regularly and reliably.
- Subject is able to swallow tablets and capsules.
- For subjects who are not their own legal guardian, subject's parent/legal authorized guardian is able to understand, read, write, and speak English fluently to complete the study-related materials (or Hebrew for Israeli subjects).
- For subjects who are not their own legal guardian, subject's parent/legal authorized guardian is able to understand and sign an informed consent form to participate in the study.
- If subject is his/her own legal guardian, he/she can understand and sign informed consent to participate in the study.
- If subject is not their own legal guardian, the subject provides assent for participation in the study, if the subject has the cognitive ability to provide assent
Who cannot join
- Treatment within the 2 weeks prior to randomization (and throughout the clinical trial) with lithium, acamprosate, racemic baclofen, investigational metabotropic glutamate receptor subtype 5 (mGluR5) medications, d-cycloserine, oxytocin, carbetocin, modafinil, armodafinil, benzodiazepines (unless used for seizure control), memantine, amantadine, bupropion, or any medication in the statin class.
- Treatment within the 2 weeks prior to Screening with monoamine oxidase (MAO) inhibitors, tricyclic antidepressants, l-dopa, cisplatin, phenobarbital or phenytoin.
- Current treatment with an N-methyl-D-aspartate (NMDA) antagonist.
- While stimulants will not be excluded from the trial, the subject and the parent/legal authorized guardian may decide to stop stimulant medication prior to the study upon discussion with the investigator at the Screening visit. If stimulant medication is stopped at screening, a two-week washout is required. A subject that decides to washout from stimulants will be excluded from the trial if a stimulant is administered after the Screening visit or during the course of the trial.
- Subject is planning to commence psychotherapy or cognitive behavior therapy (CBT) during the period of the study or had begun psychotherapy or CBT within 6 weeks prior to Screening.
- A subject who started psychotherapy or CBT for the first time within 6 weeks prior to Screening is excluded.
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- If a subject was previously receiving psychotherapy or CBT, and is resuming the therapy (such as return from summer vacation), then the subject is eligible for the study if the same therapy was resumed at least 2 weeks before screening.
- History of or current cardiovascular, renal, hepatic, respiratory, or gastrointestinal disease that may interfere with the absorption, distribution, metabolism, or excretion of the study medication, or that may interfere with the interpretation of the safety, tolerability, or efficacy of the study medication.
- History of or current cerebrovascular disease or clinically significant brain trauma.
- Current major depressive disorder (subject must be free of the most recent episode for 3 months prior to randomization).
- History of a Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5)-defined substance use disorder in the 3 months prior to Screening.
- Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or ECG, as measured at Screening.
- Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.
- Enrollment in another clinical trial within the 30 days preceding Screening.
- Any psychiatric condition (eg, schizophrenia or personality disorder as diagnosed by DSM-IV) or clinically significant or unstable medical or surgical condition that may preclude safe and complete study participation as determined by the investigator using medical history, physical examination, neurological examination, laboratory tests, and electrocardiograms. Common diseases such as mild hypertension, well-controlled type 2 diabetes mellitus (hemoglobin A1C [Hgb A1C] <6.5%), etc. are allowed per the investigator's judgment as long as they are stable and controlled by medical therapy that is constant for at least 8 weeks before randomization and subsequently throughout the study. If there are any concerns about the suitability of the subject's medical or surgical condition, the investigator should review the subject's history with the medical monitor. Subjects with autism spectrum disorder or anxiety disorder will be allowed.
- Subject has known or suspected human immune deficiency virus-positive status or has diseases such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.
- Subject has a history of an allergy or sensitivity to B-complex vitamins.
- Subject has used mega-dose vitamin B6/pyridoxine during the 28 days before the Randomization Visit. Subjects will be allowed to have a 28-day washout of mega-dose vitamin B6/pyridoxine after the Screening visit. Routine multivitamin supplements will be allowed.
- Subject has used high-dose supplements of omega-3 fatty acids ≥ 500 mg (such as softgels, capsules, or fish oils; regular daily dietary consumption of fish is allowed) or folic acid supplements (other than routine multivitamin supplements) at any time during the 2 weeks before the Randomization Visit.
- Subject is related to anyone employed by the sponsor, investigator, or study staff.
- Subject has any condition that, in the principal investigator's opinion, would place the subject at risk or influence the conduct of the study or interpretation of results.
- Subject is pregnant, lactating, or using an inadequate contraceptive method. -
Trial locations and contacts
12 sites in 2 countries.
12 sites in 2 countries
United States11 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
RUSH University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Duke University Medical Center
Durham, North Carolina, 27710
Baylor College of Medicine Research
Houston, Texas, 77030
Suburban Research Associates
Media, Pennsylvania, 19063
Southwest Autism Research & Resource Center
Phoenix, Arizona, 85006
University of California Davis Pediatrics
Sacramento, California, 95817
Univ. of Washington/Seattle Children's Hospital
Seattle, Washington, 98121
About this study
Official title: A 6-week, Randomized, Multicenter, Double-blind, Parallel, Flexed and Fixed-dose Study of MG01CI (Metadoxine Extended-release) Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
This study is a multisite, randomized, double-blind, placebo-controlled, phase 2 study of MG01CI (low dose and high dose once daily) for 6 weeks compared with placebo in a 1:1 ratio of 60 adolescent and adult subjects with Fragile X Syndrome (FXS). Following Screening, subjects will be randomized to MG01CI or matching placebo at Baseline (Day 0) and the 6 week Double-blind Treatment Period will begin on Day 1.
The first 4 weeks of the treatment period will be a dose-optimization period,
Read more
All subjects will start with two daily tablets: low dose metadoxine or matching blinded placebo. At weekly visits/phone assessments, the investigator will evaluate the dose based upon the investigator's assessment of safety and tolerability. If the subject demonstrates safety or tolerability concerns with the low dose after 1 or 2 weeks of treatment, then the subject will be discontinued. If there are no concerns about safety and tolerability after 2 weeks of treatment, then the dose will be increased to high dose or placebo. If at the high dose there are concerns about safety and tolerability, then the dose will be either kept the same or reduced to low dose for the remainder of the treatment period.
There will be a 2-week Follow-up Period after the last dose of study treatment or early termination.
Read the detailed description
This study is a multisite, randomized, double-blind, placebo-controlled, phase 2 study of MG01CI (low and high doses of metadoxine once daily) for 6 weeks compared with placebo in a 1:1 ratio of 60 adolescent and adult subjects with FXS. Following Screening, subjects will be randomized to MG01CI or matching placebo at Baseline (Day 0) and the 6 week Double-blind Treatment Period will begin on Day 1.
The first 4 weeks of the treatment period will be a dose-optimization period, during which the subject's dose of MG01CI or placebo will be optimized. Investigators and subjects will be blinded with regard to whether the subject is taking active drug or placebo. Subjects will be blinded to anticipated dose (low vs high dose), while Investigators will not be blinded to anticipated dose, low dose vs high dose. Every two weeks subjects will receive 2-week supply treatment. A phone follow-up assessment of safety and tolerability will occur during titration after 1 and 3 weeks of treatment; if the investigator has any significant concerns regarding safety and tolerability, the subject will be assessed at the site at an unscheduled visit. All subjects will be assessed at the site after 2 weeks and 4 weeks of treatment.
All subjects will start with either low dose or matching blinded placebo (2 tablets daily). At weekly visits/phone assessments, the investigator will evaluate the dose based upon the investigator's assessment of safety and tolerability. If the subject demonstrates safety or tolerability concerns with the low dose after 1 or 2 weeks of treatment, then the subject will be discontinued. If there are no concerns about safety and tolerability after 2 weeks of treatment, then the dose will be increased to 2 tablets of either high dose of active treatment or placebo. If at high dose there are concerns about safety and tolerability, then the dose will be either kept the same or reduced to low dose for the remainder of the treatment period.
The last 2 weeks of the treatment period will be a dose-maintenance period. During the dose-maintenance period, the subject will maintain his or her optimal dose as determined at the end of the dose-optimization period. A phone follow-up assessment of safety and tolerability will occur after 5 weeks of treatment (after 1 week of dose maintenance). If the investigator has any significant concerns regarding safety and tolerability, the subject will be assessed at the site at an unscheduled visit. The subject will be assessed at the site after 6 weeks of treatment (after 2 weeks of dose maintenance).
There will be a 2-week Follow-up Period after the last dose of study treatment or early termination.
- Conditions
- Fragile X Syndrome
- Being tested
- MG01CI extended-release tablet
- Keywords
- Fragile X Syndrome, Fra(X) Syndrome, FRAXA Syndrome
- Medical subject headings
- Fragile X Syndrome, Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MG01CI extended-release tabletDrug
Also called Metadoxine (pyridoxol L-2-pyrrolidone-5-carboxylate)
Metadoxine, MG01CI extended-release tablet taken for 6 weeks treatment. Starting with a dose of 700 mg for 2 weeks followed by a 2 weeks of 1400 mg and then 2 weeks of 700 mg or 1400 mg.
| Study group | Role | What they receive |
|---|---|---|
| Metadoxine Immediate/Slow-release | Experimental | Flexed and fixed-dose Metadoxine Immediate/Slow-release 700 mg and 1400 mg administered orally once daily Receives: Drug: MG01CI extended-release tablet |
| Placebo | Placebo comparator | Placebo tablet identical in appearance to study investigational product. Administered orally once daily Receives: Drug: MG01CI extended-release tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of efficacy of MG01CI by Attention Deficit Hyperactivity Disorder (ADHD) Rating Scale (ADHD RS-IV)
To evaluate the efficacy of MG01CI (Metadoxine Extended-release) once daily compared with placebo in the treatment of symptoms of FXS in adults and adolescents as measured by the inattentive subscale of the Attention Deficit Hyperactivity Disorder (ADHD) Rating Scale (ADHD RS-IV) (as rated by the investigator in a clinical interview of the parent/legal authorized guardian/consistent caregiver).
Measured: 6 weeks
Secondary outcomes (1)
Evaluation of efficacy of MG01C as measured by total score on the ADHD RS-IV.
To evaluate the efficacy of MG01CI (Metadoxine Extended-release) once daily compared with placebo in the treatment of symptoms of FXS in adults and adolescents as measured by the total score on the ADHD RS-IV.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2014 (estimated)
- Study startJun 2014
- Primary completionJun 2015
- Study completionJun 2015
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
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alcobra Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elizabeth Berry-Kravis, MD, PhD, Principal investigator, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02126995
- Sponsor's protocol ID
- AL014
- Status last verified
- Dec 2014
- First submitted
- 29 Apr 2014
- Last updated
- 6 Jul 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 6-week, Randomized, Multicenter, Double-blind, Parallel, Flexed and Fixed-dose Study of MG01CI (Metadoxine Extended-release) Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT02126995
- Acronym / sponsor ID
- AL014
- Conditions
- Fragile X Syndrome
- Keywords
- Fragile X Syndrome, Fra(X) Syndrome, FRAXA Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Alcobra Ltd.Industry (pharma, biotech, devices)
- Study start
- Jun 2014
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 30 Apr 2014 (estimated)
- 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 a multisite, randomized, double-blind, placebo-controlled, phase 2 study of MG01CI (low dose and high dose once daily) for 6 weeks compared with placebo in a 1:1 ratio of 60 adolescent and adult subjects with Fragile X Syndrome (FXS). Following Screening, subjects will be randomized to MG01CI or matching placebo at Baseline (Day 0) and the 6 week Double-blind Treatment Period will begin on Day 1.
The first 4 weeks of the treatment period will be a dose-optimization period,
All subjects will start with two daily tablets: low dose metadoxine or matching blinded placebo. At weekly visits/phone assessments, the investigator will evaluate the dose based upon the investigator's assessment of safety and tolerability. If the subject demonstrates safety or tolerability concerns with the low dose after 1 or 2 weeks of treatment, then the subject will be discontinued. If there are no concerns about safety and tolerability after 2 weeks of treatment, then the dose will be increased to high dose or placebo. If at the high dose there are concerns about safety and tolerability, then the dose will be either kept the same or reduced to low dose for the remainder of the treatment period.
There will be a 2-week Follow-up Period after the last dose of study treatment or early termination.
Detailed description
This study is a multisite, randomized, double-blind, placebo-controlled, phase 2 study of MG01CI (low and high doses of metadoxine once daily) for 6 weeks compared with placebo in a 1:1 ratio of 60 adolescent and adult subjects with FXS. Following Screening, subjects will be randomized to MG01CI or matching placebo at Baseline (Day 0) and the 6 week Double-blind Treatment Period will begin on Day 1.
The first 4 weeks of the treatment period will be a dose-optimization period, during which the subject's dose of MG01CI or placebo will be optimized. Investigators and subjects will be blinded with regard to whether the subject is taking active drug or placebo. Subjects will be blinded to anticipated dose (low vs high dose), while Investigators will not be blinded to anticipated dose, low dose vs high dose. Every two weeks subjects will receive 2-week supply treatment. A phone follow-up assessment of safety and tolerability will occur during titration after 1 and 3 weeks of treatment; if the investigator has any significant concerns regarding safety and tolerability, the subject will be assessed at the site at an unscheduled visit. All subjects will be assessed at the site after 2 weeks and 4 weeks of treatment.
All subjects will start with either low dose or matching blinded placebo (2 tablets daily). At weekly visits/phone assessments, the investigator will evaluate the dose based upon the investigator's assessment of safety and tolerability. If the subject demonstrates safety or tolerability concerns with the low dose after 1 or 2 weeks of treatment, then the subject will be discontinued. If there are no concerns about safety and tolerability after 2 weeks of treatment, then the dose will be increased to 2 tablets of either high dose of active treatment or placebo. If at high dose there are concerns about safety and tolerability, then the dose will be either kept the same or reduced to low dose for the remainder of the treatment period.
The last 2 weeks of the treatment period will be a dose-maintenance period. During the dose-maintenance period, the subject will maintain his or her optimal dose as determined at the end of the dose-optimization period. A phone follow-up assessment of safety and tolerability will occur after 5 weeks of treatment (after 1 week of dose maintenance). If the investigator has any significant concerns regarding safety and tolerability, the subject will be assessed at the site at an unscheduled visit. The subject will be assessed at the site after 6 weeks of treatment (after 2 weeks of dose maintenance).
There will be a 2-week Follow-up Period after the last dose of study treatment or early termination.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Metadoxine Immediate/Slow-release | Experimental | Drug: MG01CI extended-release tabletFlexed and fixed-dose Metadoxine Immediate/Slow-release 700 mg and 1400 mg administered orally once daily |
| Placebo | Placebo comparator | Drug: MG01CI extended-release tabletPlacebo tablet identical in appearance to study investigational product. Administered orally once daily |
Who may qualify
- Ages
- 15 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- Subject is a man or a non-pregnant, non-lactating woman aged 15 to 55 years, inclusive, at the Randomization Visit.
- Subject has Fragile X Syndrome with a molecular genetic confirmation of the full Fragile X Mental Retardation (FMR1) mutation (≥200 CGG repetitions).
- Subject has a score of 12 or greater on the inattentive subscale of the ADHD RS IV (as rated by the investigator in a clinical interview of the parent/legal authorized guardian/consistent caregiver).
- Current treatment with no more than 3 prescribed psychotropic medications. Anti epileptic medications are permitted and are not counted as psychotropic medications if they are used for treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
- Behavioral treatments (excluding psychotherapy; see exclusion criteria) must be stable for 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
Key exclusion criteria (22 listed)
- Treatment within the 2 weeks prior to randomization (and throughout the clinical trial) with lithium, acamprosate, racemic baclofen, investigational metabotropic glutamate receptor subtype 5 (mGluR5) medications, d-cycloserine, oxytocin, carbetocin, modafinil, armodafinil, benzodiazepines (unless used for seizure control), memantine, amantadine, bupropion, or any medication in the statin class.
- Treatment within the 2 weeks prior to Screening with monoamine oxidase (MAO) inhibitors, tricyclic antidepressants, l-dopa, cisplatin, phenobarbital or phenytoin.
- Current treatment with an N-methyl-D-aspartate (NMDA) antagonist.
- While stimulants will not be excluded from the trial, the subject and the parent/legal authorized guardian may decide to stop stimulant medication prior to the study upon discussion with the investigator at the Screening visit. If stimulant medication is stopped at screening, a two-week washout is required. A subject that decides to washout from stimulants will be excluded from the trial if a stimulant is administered after the Screening visit or during the course of the trial.
- Subject is planning to commence psychotherapy or cognitive behavior therapy (CBT) during the period of the study or had begun psychotherapy or CBT within 6 weeks prior to Screening.
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 |
|---|---|---|
| MG01CI extended-release tabletAlso: Metadoxine (pyridoxol L-2-pyrrolidone-5-carboxylate)Metadoxine, MG01CI extended-release tablet taken for 6 weeks treatment. Starting with a dose of 700 mg for 2 weeks followed by a 2 weeks of 1400 mg and then 2 weeks of 700 mg or 1400 mg. | Drug | Metadoxine Immediate/Slow-release, Placebo |
Primary outcome
Evaluation of efficacy of MG01CI by Attention Deficit Hyperactivity Disorder (ADHD) Rating Scale (ADHD RS-IV)
Time frame: 6 weeks
To evaluate the efficacy of MG01CI (Metadoxine Extended-release) once daily compared with placebo in the treatment of symptoms of FXS in adults and adolescents as measured by the inattentive subscale of the Attention Deficit Hyperactivity Disorder (ADHD) Rating Scale (ADHD RS-IV) (as rated by the investigator in a clinical interview of the parent/legal authorized guardian/consistent caregiver).
1 secondary outcome
Evaluation of efficacy of MG01C as measured by total score on the ADHD RS-IV.
Time frame: 6 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2016.
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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, MD, PhD principal investigator
Rush University Medical Center
12 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 MG01CI extended-release tablet in people with Fragile X Syndrome, run by Alcobra Ltd. at 12 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 17 inclusion and 22 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: finished, as of the sponsor's last update on 6 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Israel. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Evaluation of efficacy of MG01CI by Attention Deficit Hyperactivity Disorder (ADHD) Rating Scale (ADHD RS-IV) (time frame: 6 weeks). 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 6 Jul 2016; AzTrials last refreshed its copy on 6 Nov 2026.