Tuberous Sclerosis ComplexPhase 2NCT02849457

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

A Phase 2 study testing Early Vigabatrin in people with Tuberous Sclerosis Complex, run by Martina Bebin at 13 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure Read more about this study

Who is it for?: Ages 1 day to 6 months
Conditions the study is for: Tuberous Sclerosis Complex
What is being tested?: Early Vigabatrin
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 84 participants
Where: 13 sitesUnited States
Study dates: Dec 2016to 5 May 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 1 day to 6 months
All sexes
No healthy volunteers
Child

Who may be able to join

less than or equal to 6 months of age
No history of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
Meet genetic or clinical diagnostic criteria for TSC, the latter based on current recommendations for diagnostic evaluation, such as physical exam, neuroimaging, echocardiogram

Who cannot join

Is greater than 6 months of age
Has not been diagnosed with TSC
History of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
Has received any anticonvulsant medication including vigabatrin, other anti-seizure therapeutic agent including cannabidiol
Has received an oral mTOR inhibitor such as everolimus or sirolimus
Has taken an investigational drug, including but not limited to cannabidiol, as part of a research study 30 days prior to enrollment, or plans on taking an investigational drug at any time during the duration of the study
Is currently enrolled, or plans on enrolling at any time during the duration of the study, in an experimental behavioral early intervention study
Has a history of being born prematurely (born less than <30 weeks gestation at the time of delivery)

Trial locations and contacts

13 sites in 1 country.

City, postcode or hospital

13 sites in 1 country

United States13 sites

University of Alabama at Birmingham
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02215
Cincinnati's Children Hospital Medical Center
Cincinnati, Ohio, 45229
Duke University
Durham, North Carolina, 37710
University of Texas Health Science Center at Houston
Houston, Texas, 77054
University of California, Los Angeles
Los Angeles, California, 90095
Stanford University
Palo Alto, California, 94304
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Beaumont Children's Hospital
Royal Oak, Michigan, 48073
Minnesota Epilepsy Group, PA
Saint Paul, Minnesota, 55102
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University in St. Louis
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical Trial for Infants With TSC

Read the detailed description

The central hypothesis of this Phase IIb trial is that early identification of electroencephalography (EEG) biomarkers and early treatment versus delayed treatment with vigabatrin in infants with tuberous sclerosis complex (TSC) will have a positive impact on developmental outcomes at 24 months of age. It would also prevent or lower the risk of developing infantile spasms and refractory seizures. This preventative approach would be expected to result in more favorable long-term cognitive, behavioral, developmental and psychiatric outcomes and significantly improve overall quality of life. It is a randomized, double-blind, placebo-controlled clinical trial design. Successful completion of this trial will also advance the field by demonstrating the value of systematic surveillance with EEG in asymptomatic infants with TSC.

Conditions: Tuberous Sclerosis Complex
Being tested: Early Vigabatrin, Delayed Vigabatrin (Placebo)
Medical subject headings: Tuberous Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Early VigabatrinDrug
Also called Sabril
Subjects randomized to vigabatrin will be treated with vigabatrin 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study and continue to be followed until 36 months of age.
Delayed Vigabatrin (Placebo)Drug
Subjects randomized to placebo will be treated with matching placebo at 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study and continue to be followed until 36 months of age.

Study groups in NCT02849457
Study group	Role	What they receive
Delayed Vigabatrin (Placebo)	Placebo comparator	Randomization will only occur after detection of epileptiform activity on EEG. Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age. Receives: Drug: Early Vigabatrin; Drug: Delayed Vigabatrin (Placebo)
Early Vigabatrin	Experimental	Randomization will only occur after detection of epileptiform activity on EEG. Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age. Receives: Drug: Early Vigabatrin
Watchful Waiting (Control Group)	No intervention	Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.

What is being measured

The primary outcome is the main question the study is designed to answer.

Cognitive Assessment Scores and Developmental Impact
The primary outcome measure will be the standardized Cognitive scale scores on the Bayley Scales of Infant and Toddler Development- Third Edition at 24 months. The Cognitive scale Composite score is a standard score derived from the observed and elicited performance of the child on cognitive assessment tasks, with a mean of 100 and standard deviation of 10. The range for the Cognitive scale Composite score is 55 to 145. The score is calculated using standard procedures available in the manual for this measure. A higher score is considered better performance. The Bayley Scales of Infant and Toddler Development at 24 months will be used for the data analysis and to compare the developmental impact of early versus delayed treatment with vigabatrin.
Measured: 24 months

Secondary outcomes (7)

Number of Subjects That Develop Seizures When Treated With Study Drug During the Randomized Phase of the Study.
Evaluate the number of subjects that develop seizures when treated with vigabatrin or placebo as a seizure prevention.
Measured: 24 months
Time to the Subject's First Clinical Seizure From Randomization
Time to the subject's first clinical seizure will be measured for both subjects on placebo and vigabatrin.
Measured: 24 months
Count of Participants With Drug Resistant Epilepsy at 24 Months of Age.
The count of participants with drug resistant epilepsy. Drug resistant epilepsy classified according to International League Against Epilepsy (ILAE) definition, specifically defined as any participant on 2 or more anti-seizure medications experiencing persistent seizures (seizures occurring within 3 months of the 24 month participant visit).
Measured: 24 months
Evaluate Vineland II ABC Scores and Impact of Early Versus Late Treatment
The range for the Vineland-II Adaptive Behavior Composite is 20 to 160
The Vineland-II ABC standard score has a mean of 100 and standard deviation of 15, with higher scores indicating better overall adaptive functioning.
The ABC standard score is a composite derived from obtained scores on the Communication, Daily Living Skills, Socialization, and Motor Skills domains on the Vineland-II and is calculated according to standardized procedures described in the Vineland-II manual.
Measured: 12 months, 24 months and 36 months
Evaluate Autism Diagnostic Observation Schedule 2nd Edition (ADOS2) Scores and Impact of Early Versus Late Treatment
Evaluate ADOS2 scores and the impact of early versus late treatment at 24 and 36 months.
Measured: 24 months and 36 months
Number of Subjects With Vigabatrin Related Adverse Events and Severe Adverse Events
Number of subjects with vigabatrin related adverse events, severe adverse events as assessed by CTCAE v4.0 and risk evaluation and mitigation strategy (REMS) measures as required by the FDA.
Measured: 24 months
EEG Biomarker for Developing Epilepsy
Feasibility of the routine 1 hour video EEG in determining the EEG biomarker for developing epilepsy. Outcome was determined as the number of participants developing seizures amongst those developing the biomarker (epileptiform activity).
Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 20165 May 2023

First posted29 Jul 2016 (estimated)
Study startDec 2016
Primary completion26 Apr 2023
Study completion5 May 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Tuberous Sclerosis ComplexStudies 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: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 84 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02849457
Sponsor's protocol ID: PREVeNT
Other IDs: 1U01NS092595-01A1
Status last verified: Jul 2024
First submitted: 13 Jul 2016
Last updated: 19 Aug 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Yes: De-identified data will be shared with National Database for Autism Research (NDAR)

Publications

Bebin EM, Peters JM, Porter BE, McPherson TO, O'Kelley S, Sahin M, Taub KS, Rajaraman R, Randle SC, McClintock WM, Koenig MK, Frost MD, Northrup HA, Werner K, Nolan DA, Wong M, Krefting JL, Biasini F, Peri K, Cutter G, Krueger DA; PREVeNT Study Group. Early Treatment with Vigabatrin Does Not Decrease Focal Seizures or Improve Cognition in Tuberous Sclerosis Complex: The PREVeNT Trial. Ann Neurol. 2023 Aug 28:10.1002/ana.26778. doi: 10.1002/ana.26778. Online ahead of print.
PubMed 37638552
van der Poest Clement E, Jansen FE, Braun KPJ, Peters JM. Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex. Paediatr Drugs. 2020 Feb;22(1):73-84. doi: 10.1007/s40272-019-00376-0.
PubMed 31912454

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex
RecruitingPhase 3
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
Tuberous Sclerosis Complex
Not yet recruitingPhase 2
Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)
Tuberous Sclerosis Complex, Epilepsy
Active, not recruitingPhase 4
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
Lennox Gastaut Syndrome, Dravet Syndrome

All Tuberous Sclerosis Complex trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical Trial for Infants With TSC
ClinicalTrials.gov ID: NCT02849457
Acronym / sponsor ID: PREVeNT
Conditions: Tuberous Sclerosis Complex
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 84 participants (actual)
Lead sponsor: Martina BebinUniversity, hospital or other
Collaborators: National Institute of Neurological Disorders and Stroke (NINDS)
Study start: Dec 2016
Primary completion: 26 Apr 2023 (actual)
Study completion: 5 May 2023 (actual)
First posted: 29 Jul 2016 (estimated)
Results: Posted 19 Aug 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 3

Arm	Type	Receives
Delayed Vigabatrin (Placebo)	Placebo comparator	Drug: Early Vigabatrin, Drug: Delayed Vigabatrin (Placebo)Randomization will only occur after detection of epileptiform activity on EEG. Participants randomized to this arm will be treated with matching placebo until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on placebo, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
Early Vigabatrin	Experimental	Drug: Early VigabatrinRandomization will only occur after detection of epileptiform activity on EEG. Participants randomized to this arm will be treated with vigabatrin until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on vigabatrin, they will be eligible for Open label vigabatrin. Participants will be followed until 36 months of age.
Watchful Waiting (Control Group)	No intervention	–Enrolled participants in this arm are those who never develop EEG abnormalities or clinical seizures during the length of the study. While all participants who enrolled in the study started in this group, participants were randomized upon development of EEG epileptiform activity. All participants who completed the study without developing EEG epileptiform activity or clinical seizures are reported in this group.

Who may qualify

Ages: 1 day to 6 months
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (3 listed)

less than or equal to 6 months of age
No history of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
Meet genetic or clinical diagnostic criteria for TSC, the latter based on current recommendations for diagnostic evaluation, such as physical exam, neuroimaging, echocardiogram

Key exclusion criteria (8 listed)

Is greater than 6 months of age
Has not been diagnosed with TSC
History of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
Has received any anticonvulsant medication including vigabatrin, other anti-seizure therapeutic agent including cannabidiol
Has received an oral mTOR inhibitor such as everolimus or sirolimus

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
Early VigabatrinAlso: SabrilSubjects randomized to vigabatrin will be treated with vigabatrin 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study and continue to be followed until 36 months of age.	Drug	Delayed Vigabatrin (Placebo), Early Vigabatrin
Delayed Vigabatrin (Placebo)Subjects randomized to placebo will be treated with matching placebo at 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study and continue to be followed until 36 months of age.	Drug	Delayed Vigabatrin (Placebo)

Primary outcome

Cognitive Assessment Scores and Developmental Impact
Time frame: 24 months
The primary outcome measure will be the standardized Cognitive scale scores on the Bayley Scales of Infant and Toddler Development- Third Edition at 24 months. The Cognitive scale Composite score is a standard score derived from the observed and elicited performance of the child on cognitive assessment tasks, with a mean of 100 and standard deviation of 10. The range for the Cognitive scale Composite score is 55 to 145. The score is calculated using standard procedures available in the manual for this measure. A higher score is considered better performance. The Bayley Scales of Infant and Toddler Development at 24 months will be used for the data analysis and to compare the developmental impact of early versus delayed treatment with vigabatrin.

7 secondary outcomes

Number of Subjects That Develop Seizures When Treated With Study Drug During the Randomized Phase of the Study.
Time frame: 24 months
Time to the Subject's First Clinical Seizure From Randomization
Time frame: 24 months
Count of Participants With Drug Resistant Epilepsy at 24 Months of Age.
Time frame: 24 months
Evaluate Vineland II ABC Scores and Impact of Early Versus Late Treatment
Time frame: 12 months, 24 months and 36 months
Evaluate Autism Diagnostic Observation Schedule 2nd Edition (ADOS2) Scores and Impact of Early Versus Late Treatment
Time frame: 24 months and 36 months
Number of Subjects With Vigabatrin Related Adverse Events and Severe Adverse Events
Time frame: 24 months
EEG Biomarker for Developing Epilepsy
Time frame: 24 months

Results and safety

Results posted to the registry on 19 Aug 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Delayed Vigabatrin (Placebo)	0	0
Early Vigabatrin	0	0
Watchful Waiting (Control Group)	84	72
Watchful Waiting (Open Label Vigabatrin)	0	0

Primary outcome: Cognitive Assessment Scores and Developmental Impact

Time frame: 24 months. Mean (Standard Deviation). Unit: score on a scale

Measure	Delayed Vigabatrin (Placebo) n = 23	Early Vigabatrin n = 29	Watchful Waiting (Control Group) n = 11	Watchful Waiting (Open Label Vigabatrin) n = 4
Value	83.9 (17.3)	80.9 (15.6)	97.3 (16.9)	85.0 (21.6)

Delayed Vigabatrin (Placebo) vs Early Vigabatrin: Mean Difference (Final Values): -0.6565 (95% CI -8.5567 to 7.2436), p = 0.8681, Mixed Models Analysis

Population analysed: The analysis population was the intent to treat population. Three participants withdrew prior to 24 months of age, and one participant did not complete the Bayley assessment at 24 months.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events occurring from enrollment through a patient's 36 month age visit (an average study duration of approximately 34 months). Note, patients could enroll at different ages, but had a common final study visit corresponding to age 36 months. Adverse events were assessed/collected at every study visit, and serious adverse events were collected in real time and reported to the medical safety monitor for the study.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Delayed Vigabatrin (Placebo), Pre-randomization	3 of 27 (11.1%)	4 of 27 (14.8%)	0 of 27
Early Vigabatrin, Pre-randomization	2 of 29 (6.9%)	4 of 29 (13.8%)	0 of 29
Delayed Vigabatrin (Placebo), Post-randomization	14 of 27 (51.9%)	15 of 27 (55.6%)	0 of 27
Early Post-randomization	15 of 29 (51.7%)	17 of 29 (58.6%)	0 of 29
Watchful Waiting (Control Group)	1 of 12 (8.3%)	7 of 12 (58.3%)	0 of 12
Watchful Waiting (Open Label Vigabatrin), Before Use of Vigabatrin	2 of 4 (50%)	3 of 4 (75%)	0 of 4
Watchful Waiting (Open Label Vigabatrin), After Starting Vigabatrin	2 of 4 (50%)	3 of 4 (75%)	0 of 4

Limitations and caveats (from the sponsor): COVID-19 impacted one of the secondary endpoints (ADOS2) because of the requirement for facemasks during the assessment which would invalidate the ADOS2. Therefore this secondary outcome measure abandoned.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 37638552Bebin EM, Peters JM, Porter BE, McPherson TO, O'Kelley S, Sahin M, Taub KS, Rajaraman R, Randle SC, McClintock WM, Koenig MK, Frost MD, Northrup HA, Werner K, Nolan DA, Wong M, Krefting JL, Biasini F, Peri K, Cutter G, Krueger DA; PREVeNT Study Group. Early Treatment with Vigabatrin Does Not Decrease Focal Seizures or Improve Cognition in Tuberous Sclerosis Complex: The PREVeNT Trial. Ann Neurol. 2023 Aug 28:10.1002/ana.26778. doi: 10.1002/ana.26778. Online ahead of print.
Derived from this study: PubMed 31912454van der Poest Clement E, Jansen FE, Braun KPJ, Peters JM. Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex. Paediatr Drugs. 2020 Feb;22(1):73-84. doi: 10.1007/s40272-019-00376-0.
Individual participant dataSharing planned: Yes. De-identified data will be shared with National Database for Autism Research (NDAR)

Related trials for Tuberous Sclerosis Complex

Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex
RecruitingPhase 3
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
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Not yet recruitingPhase 2
Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)
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Active, not recruitingPhase 4
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
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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.

Martina Bebin, MD, MPA principal investigator

University of Alabama at Birmingham

13 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Early Vigabatrin in people with Tuberous Sclerosis Complex, run by Martina Bebin at 13 sites in United States.

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 3 inclusion and 8 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 19 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in 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 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?

Yes. Results are posted in the registry (first posted 19 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Cognitive Assessment Scores and Developmental Impact (time frame: 24 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02849457, which also lists 2 linked publications.

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 19 Aug 2024; AzTrials last refreshed its copy on 30 Oct 2026.