Fragile X SyndromePhase 2/3MetNCT03479476
A Trial of Metformin in Individuals With Fragile X Syndrome
A Phase 2/3 study testing Metformin in people with Fragile X Syndrome and Fragile X Mental Retardation Syndrome, run by University of California, Davis at 3 sites in 2 countries.
Trial summary
This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. Read more about this study
- Who is it for?
- Ages 6 to 25 years
- Conditions the study is for
- Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked
- What is being tested?
- Metformin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 30 Apr 2018to 22 May 2024
- Phase
- Phase 2/3
- 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 6 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Molecular genetic confirmation of the full FMR1 mutation or mosaicism.
- Males and non-pregnant, non-lactating females age 6 to 25 years, inclusive.
- Ability of subject and/or caregiver to understand, read, write, and speak English fluently to complete study-related materials.
- IQ ≤ 79 as measured by the Leiter-III at screening.
- Participant is able to speak at least occasional 3-word phrases.
- Participant and parent/caregiver are willing to participate in the protocol and able to attend the clinic regularly and reliably.
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- Stable concomitant medication dose and dosing regimen for at least 4 weeks prior to the screening/baseline visit, and the intention to maintain a stable regimen of allowed concomitant medications for the full duration of the study.
- Stable behavioral/educational treatments for at least 4 weeks prior to the screening/baseline visit.
- Sexually active women of childbearing potential must be using a medically acceptable method of birth control for the duration of the study and have a negative urine pregnancy test collected at the initial screening/baseline visit.
- For participants who are not their own legal guardian, the parent/legal authorized representative is able to understand and sign an informed consent to participate in the study.
Who cannot join
- Non-cooperation or inability to follow through with the study protocol.
- Life-threatening medical problem or other major systemic illness that compromises health or safety and/or would interfere with the study.
- History of intolerable adverse events with metformin.
- Current or recent metformin treatment (within the past year).
- Body mass index (BMI) less than 2 standard deviations for age.
- Serum creatinine > 1.4 mg/dl (female) or > 1.5 mg/dl (male) at screening.
Show all 9Show fewer
- History of metabolic acidosis or a condition with lactic acidosis.
- Severe B12 deficiency.
- Pregnancy at screening or unwillingness to use acceptable method of birth control, if applicable.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Canada2 sites
University of Alberta
Edmonton, Alberta, T6G2R3
St Justine Hospital
Montreal, Quebec, H3T 1C5
United States1 site
UC Davis MIND Institute
Sacramento, California, 95817
About this study
Official title: A Double-Blind, Placebo-Controlled Trial of Metformin in Individuals With Fragile X Syndrome
This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.
Read the detailed description
This is a multi-site study for fragile X syndrome (FXS) patients aged 6 to 25 years inclusive. It is a randomized, double-blind, placebo-controlled trial of metformin (also known as Glumetza, Glucophage, Fortamet), a type 2 diabetes medication that can also improve obesity and excessive appetite.
Metformin has emerged as a candidate drug for the targeted treatment of FXS based on animal studies showing rescue of multiple phenotypes in the FXS model. Metformin may contribute to normalizing signaling pathways in FXS in the central nervous system, which may include activities of mTOR and PI3K, both of which have shown to be pathogenically overactive in FXS. In addition, metformin inhibits phosphodiesterase, which would lead to correction of cAMP levels, and MMP9 production, which is also elevated in FXS. Looking at the potential signaling pathways, metformin appears to be a good candidate for targeting several of the intracellular functions in neurons disrupted in FXS and, therefore, has potential to rescue several types of symptoms in individuals with FXS. The researchers have utilized metformin in the clinical treatment of over 20 individuals with FXS between the ages of 4 and 58 years and have found the medication to be well tolerated and to provide benefits not only in lowering weight gain and normalizing appetite but also in language and behavior. In this controlled trial, the researchers hope to further assess metformin's safety and benefits in the areas of language and cognition, eating and weight loss, and overall behavior.
Each participant will be involved in this trial for a period of 4 months. This will include 3 visits to the UC Davis MIND Institute and 5 phone calls. At each visit, the researchers will assess behavioral, cognitive, and language development. The researchers will also assess the side effects of the study medication throughout the trial.
Study record updated 9/26/2018 to reflect the following IRB-approved protocol modifications: Erroneous references to "digital" books for primary outcome measure were removed, as this measure is administered using print (non-digital) materials. Eligibility criteria were updated to include subjects with IQ of up to and including 79 on the Leiter-III at screening. The Hyperphagia Questionnaire (HQ-CT) was replaced by the Anxiety Depression and Mood Screen (ADAMS) as a secondary outcome measure.
- Conditions
- Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked, Genetic Diseases, X-Linked, Trinucleotide Repeat Expansion, Fra(X) Syndrome
- Being tested
- Placebo Medication, Metformin
- Keywords
- Congenital Abnormalities, Metformin, Nervous System Diseases, Chromosome Disorders
- Medical subject headings
- Fragile X Syndrome, X-Linked Intellectual Disability, Genetic Diseases, X-Linked, Intellectual Disability, Neurobehavioral Manifestations, Sex Chromosome Disorders, Congenital Abnormalities, Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo MedicationDrug
Also called Placebo
Placebo liquid or capsules given in parallel to active medication.
MetforminDrug
Also called Glumetza, Glucophage, Fortamet, Riomet
Active medication.
| Study group | Role | What they receive |
|---|---|---|
| Placebo Medication | Placebo comparator | The placebo will be dosed in a weight-dependent manner. Liquid placebo will be provided to any participants unable to swallow the placebo capsules. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study. Receives: Drug: Placebo Medication |
| Active Metformin Medication | Active comparator | The active metformin medication will be dosed in a weight-dependent manner. Liquid metformin (100mg/cc) will be provided to any participants unable to swallow the metformin capsules. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study. Receives: Drug: Metformin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in the Expressive Language Sampling (ELS) mean Number of Different Words (NDW) score
The ELS is collected from shared interactions around a wordless picture book involving the participant and the examiner. The primary outcome measure will be Number of Different Words (NDW) derived from transcripts of audiorecorded samples of spoken language taken from two sampling contexts (conversation and narration), according to procedures described by Abbeduto and colleagues (Abbeduto et al., 1995; Kover et al., 2012). Samples are collected pre- and post- treatment using different books on each occasion. The mean of NDW in conversation and NDW in narration will be computed, and statistically adjusted through analysis of covariance for differences in talkativeness as outlined in Conners et al., 2018. The mean NDW score ranges from 1 to infinite/unspecified. An increase (positive change) in score from baseline to follow-up indicates improvement. The greater the increase, the greater the improvement.
Measured: Baseline, End of Treatment/Week 16
Secondary outcomes (8)
Change from baseline in the FXS-normed Aberrant Behavior Checklist - Community Edition (ABC-C)
The ABC-C is a 58-item caregiver-rated behavior scale used to examine treatment effects on challenging behaviors for individuals with FXS in the following domains: (1) irritability, agitation, crying; (2) lethargy, social withdrawal; (3) stereotypic behavior; (4) hyperactivity, noncompliance; and (5) inappropriate speech. Analysis will be performed utilizing the Sansone et al. (2012) FXS-normed ABC scoring measures.
Measured: Baseline, Week 8, End of Treatment/Week 16
Improvement of symptoms in FXS using the Clinical Impression - Improvement (CGI-I) scale
The CGI is a clinician-rated scale utilizing history from the caregiver and incorporating it into a clinical rating, first for severity, and then for clinical follow-up. The CGI-S will be used at the baseline assessment to judge symptom severity as 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; and 7 = Among the most extremely ill. The CGI-I will be used at the Week 8 and End of Treatment/Week 16 visits to judge the change in clinical impression as 1 = Very Much Improved; 2 = Much Improved; 3 = Minimally Improved; 4 = No Change; 5 = Minimally Worse; 6 = Much Worse; and 7 = Very Much Worse.
Measured: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Visual Analog Scale (VAS)
The VAS will be used to measure the severity of three specific behavioral symptoms targeted in this study: behavior problems, language abilities, and eating behavior. For each behavior the caregiver is instructed to mark their impression of the behavior at the baseline visit and again at the Week 8 and End of Treatment/Week 16 visits. The calculated distance in cm between the visit marks thereby demonstrates whether each behavior stayed the same, improved, or worsened during the study and by how much. The scale is from 0 cm (defined as "worst behavior") to 10 cm ("behavior not a problem").
Measured: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Vineland Adaptive Behavior Scales-Third Edition (VABS-III) Adaptive Behavior Composite Score
The VABS-III is a caregiver survey interview that measures the personal and social skills of individuals from birth through adulthood. It was designed to assess handicapped and non-handicapped persons in their personal and social functioning and is appropriate for individuals of all ages. The Adaptive Behavior Composite (ABC) score is calculated from the caregiver responses using age-adjusted scoring tables. ABC scores range from 20 to 160 and indicate low (20-70), moderately low (70-85), adequate (85-115), moderately high (115-130), or high (130-160) overall adaptive functioning.
Measured: Baseline, End of Treatment/Week 16
Change from baseline in the Anxiety Depression and Mood Screen (ADAMS) overall score
The ADAMS consists of a series of 28 questions relating to the participant's symptoms of anxiety, depression, and mood. The sum of responses on a scale of 0 to 3 for each of the 28 questions generates the overall score, which ranges from 0 to 84. Lower scores indicate lower severity of symptoms, while higher scores indicate higher severity of symptoms. It will be completed by the caregiver at baseline, Week 8, and End of Treatment/Week 16 visits.
Measured: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Child Sleep Habits Questionnaire (CSHQ) overall score
The CSHQ consists of a series of questions relating to the sleep habits of children. It will be completed by caregivers of all participants, regardless of participant age, at the baseline, Week 8, and End of Treatment/Week 16 visits.
Measured: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Swanson, Nolan and Pelham Questionnaire (SNAP-IV) overall score
The SNAP-IV, based on DSM-V criteria for ADHD, is a caregiver-rated questionnaire that effectively identifies those with and without ADHD and accurately predicts presentation specifier (inattention, hyperactivity/impulsivity, and combined). Its psychometric properties and clinical utility have been demonstrated in multiple studies since its introduction in 2001, and it has been found to be reliable and well validated with normative data from both parents and teachers. It will be completed by caregivers of all participants at baseline, Week 8, and End of Treatment/Week 16.
Measured: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Pediatric Quality of Life Questionnaire (PedsQL) Parent Proxy overall score
The PedsQL Parent Proxy consists of a series of questions relating to a child's quality of life and is administered to the caregiver of the participant. The parent proxy module designed for children 8-12 years of age will be administered to the caregivers of all participants, regardless of participant age, because the questions therein are most appropriate for the overall study population's cognitive age and ability. It will be completed at the baseline, Week 8, and End of Treatment/Week 16 visits. For any participants not in school, questions pertaining to "school" will be replaced with references to "work" or other activities in their life.
Measured: Baseline, Week 8, End of Treatment/Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2018
- Study start30 Apr 2018
- Primary completion22 May 2024
- Study completion22 May 2024
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.
- More trials run by University of California, DavisThe sponsor's other registered studies.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Collaborators
- University of Alberta, St. Justine's Hospital
- Responsible party
- Sponsor
- Investigators
- Randi J Hagerman, MD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03479476
- Sponsor's protocol ID
- 1068417
- Acronym
- Met
- Status last verified
- Jan 2025
- First submitted
- 20 Mar 2018
- Last updated
- 17 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled Trial of Metformin in Individuals With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT03479476
- Acronym / sponsor ID
- Met / 1068417
- Conditions
- Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked, Genetic Diseases, X-Linked, Trinucleotide Repeat Expansion, Fra(X) Syndrome
- Keywords
- Congenital Abnormalities, Metformin, Nervous System Diseases, Chromosome Disorders
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Collaborators
- University of Alberta, St. Justine's Hospital
- Study start
- 30 Apr 2018 (actual)
- Primary completion
- 22 May 2024 (actual)
- Study completion
- 22 May 2024 (actual)
- First posted
- 27 Mar 2018 (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 a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.
Detailed description
This is a multi-site study for fragile X syndrome (FXS) patients aged 6 to 25 years inclusive. It is a randomized, double-blind, placebo-controlled trial of metformin (also known as Glumetza, Glucophage, Fortamet), a type 2 diabetes medication that can also improve obesity and excessive appetite.
Metformin has emerged as a candidate drug for the targeted treatment of FXS based on animal studies showing rescue of multiple phenotypes in the FXS model. Metformin may contribute to normalizing signaling pathways in FXS in the central nervous system, which may include activities of mTOR and PI3K, both of which have shown to be pathogenically overactive in FXS. In addition, metformin inhibits phosphodiesterase, which would lead to correction of cAMP levels, and MMP9 production, which is also elevated in FXS. Looking at the potential signaling pathways, metformin appears to be a good candidate for targeting several of the intracellular functions in neurons disrupted in FXS and, therefore, has potential to rescue several types of symptoms in individuals with FXS. The researchers have utilized metformin in the clinical treatment of over 20 individuals with FXS between the ages of 4 and 58 years and have found the medication to be well tolerated and to provide benefits not only in lowering weight gain and normalizing appetite but also in language and behavior. In this controlled trial, the researchers hope to further assess metformin's safety and benefits in the areas of language and cognition, eating and weight loss, and overall behavior.
Each participant will be involved in this trial for a period of 4 months. This will include 3 visits to the UC Davis MIND Institute and 5 phone calls. At each visit, the researchers will assess behavioral, cognitive, and language development. The researchers will also assess the side effects of the study medication throughout the trial.
Study record updated 9/26/2018 to reflect the following IRB-approved protocol modifications: Erroneous references to "digital" books for primary outcome measure were removed, as this measure is administered using print (non-digital) materials. Eligibility criteria were updated to include subjects with IQ of up to and including 79 on the Leiter-III at screening. The Hyperphagia Questionnaire (HQ-CT) was replaced by the Anxiety Depression and Mood Screen (ADAMS) as a secondary outcome measure.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo Medication | Placebo comparator | Drug: Placebo MedicationThe placebo will be dosed in a weight-dependent manner. Liquid placebo will be provided to any participants unable to swallow the placebo capsules. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study. |
| Active Metformin Medication | Active comparator | Drug: MetforminThe active metformin medication will be dosed in a weight-dependent manner. Liquid metformin (100mg/cc) will be provided to any participants unable to swallow the metformin capsules. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study. |
Who may qualify
- Ages
- 6 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Molecular genetic confirmation of the full FMR1 mutation or mosaicism.
- Males and non-pregnant, non-lactating females age 6 to 25 years, inclusive.
- Ability of subject and/or caregiver to understand, read, write, and speak English fluently to complete study-related materials.
- IQ ≤ 79 as measured by the Leiter-III at screening.
- Participant is able to speak at least occasional 3-word phrases.
Key exclusion criteria (9 listed)
- Non-cooperation or inability to follow through with the study protocol.
- Life-threatening medical problem or other major systemic illness that compromises health or safety and/or would interfere with the study.
- History of intolerable adverse events with metformin.
- Current or recent metformin treatment (within the past year).
- Body mass index (BMI) less than 2 standard deviations for age.
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 |
|---|---|---|
| Placebo MedicationAlso: PlaceboPlacebo liquid or capsules given in parallel to active medication. | Drug | Placebo Medication |
| MetforminAlso: Glumetza, Glucophage, Fortamet, RiometActive medication. | Drug | Active Metformin Medication |
Primary outcome
Change from baseline in the Expressive Language Sampling (ELS) mean Number of Different Words (NDW) score
Time frame: Baseline, End of Treatment/Week 16
The ELS is collected from shared interactions around a wordless picture book involving the participant and the examiner. The primary outcome measure will be Number of Different Words (NDW) derived from transcripts of audiorecorded samples of spoken language taken from two sampling contexts (conversation and narration), according to procedures described by Abbeduto and colleagues (Abbeduto et al., 1995; Kover et al., 2012). Samples are collected pre- and post- treatment using different books on each occasion. The mean of NDW in conversation and NDW in narration will be computed, and statistically adjusted through analysis of covariance for differences in talkativeness as outlined in Conners et al., 2018. The mean NDW score ranges from 1 to infinite/unspecified. An increase (positive change) in score from baseline to follow-up indicates improvement. The greater the increase, the greater the improvement.
8 secondary outcomes
Change from baseline in the FXS-normed Aberrant Behavior Checklist - Community Edition (ABC-C)
Time frame: Baseline, Week 8, End of Treatment/Week 16
Improvement of symptoms in FXS using the Clinical Impression - Improvement (CGI-I) scale
Time frame: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Visual Analog Scale (VAS)
Time frame: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Vineland Adaptive Behavior Scales-Third Edition (VABS-III) Adaptive Behavior Composite Score
Time frame: Baseline, End of Treatment/Week 16
Change from baseline in the Anxiety Depression and Mood Screen (ADAMS) overall score
Time frame: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Child Sleep Habits Questionnaire (CSHQ) overall score
Time frame: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Swanson, Nolan and Pelham Questionnaire (SNAP-IV) overall score
Time frame: Baseline, Week 8, End of Treatment/Week 16
Change from baseline in the Pediatric Quality of Life Questionnaire (PedsQL) Parent Proxy overall score
Time frame: Baseline, Week 8, End of Treatment/Week 16
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 17 Jan 2025.
- Learn more or sign up for the study here!Link provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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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.
Randi J Hagerman, MD principal investigator
University of California, Davis
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Metformin in people with Fragile X Syndrome and Fragile X Mental Retardation Syndrome, run by University of California, Davis at 3 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 9 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 17 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Canada, United States. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Change from baseline in the Expressive Language Sampling (ELS) mean Number of Different Words (NDW) score (time frame: Baseline, End of Treatment/Week 16). 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 17 Jan 2025.