Rett SyndromePhase 3LILAC™NCT04279314
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
A Phase 3 study testing Trofinetide in people with Rett Syndrome, run by ACADIA Pharmaceuticals Inc. at 21 sites in United States.
Trial summary
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome Read more about this study
- Who is it for?
- Ages 5 to 21 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- Trofinetide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 154 participants
- Where
- 21 sitesUnited States
- Study dates
- 29 Jan 2020to 19 Aug 2022
- Phase
- Phase 3
- 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 5 to 21 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
- Met all entry criteria for the antecedent study
- May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
- Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
- The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
- Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Baseline
Who cannot join
- Began treatment with growth hormone during the antecedent study
- Began treatment with IGF-1 during the antecedent study
- Began treatment with insulin during the antecedent study
- Has developed a clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory, or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
- Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
- Has a clinically significant abnormality in vital signs at Baseline
Show all 9Show fewer
- Has a QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
- Has developed a clinically significant ECG finding during the antecedent study
- Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all prespecified entry criteria).
Trial locations and contacts
21 sites in 1 country.
21 sites in 1 country
United States21 sites
Emory Genetics Clinical Trial Center
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute - Clinical Trials Unit
Baltimore, Maryland, 21205
University of Alabama at Birmingham
Birmingham, Alabama, 35223
Boston Children's Hospital
Boston, Massachusetts, 02115
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Cleveland Clinic
Cleveland, Ohio, 44195
Greenwood Genetic Center
Greenwood, South Carolina, 29626
Texas Children's Hospital
Houston, Texas, 77030
University of California, San Diego
La Jolla, California, 92093
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Translational Genomics Research Institute (TGen)
Phoenix, Arizona, 85012
UC Davis MIND Institute
Sacramento, California, 95817
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Seattle Children's
Seattle, Washington, 98105
Washington University School of Medicine, St. Louis Children's Hospital
St Louis, Missouri, 63110
Children Medical Services
Tampa, Florida, 33606
Montefiore Medical Center, Children's Hospital at Montefiore
The Bronx, New York, 10467
About this study
Official title: A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome
- Conditions
- Rett Syndrome
- Being tested
- Trofinetide
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrofinetideDrug
Trofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
| Study group | Role | What they receive |
|---|---|---|
| Trofinetide | Experimental | Not described Receives: Drug: Trofinetide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs
Percentage of subjects with treatment-emergent adverse events (TEAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects with withdrawals due to AEs
Measured: 40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG
Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval >500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of >60 ms
Measured: 40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs
Potentially clinically important changes in vital signs were defined in the study protocol as:
systolic blood pressure (SBP) ≥180 mmHg and increased ≥20 mmHg from baseline; SBP ≤90 mmHg and decreased ≥20 mmHg from baseline; diastolic blood pressure (DBP) ≥ 105 mmHg and increased ≥15 mmHg from baseline; DBP ≤50 mmHg and decreased ≥15 mmHg from baseline; Pulse ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline
Measured: 40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight
Potentially clinically important changes in body weight were defined in the study protocol as:
Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline
Measured: 40 Weeks Treatment Duration
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes
Potentially clinically important changes in laboratory parameters were defined in the study protocol as:
Sodium ≤125 mmol/L; Sodium ≥155 mmol/L; Potassium ≤3.0 mmol/L; Potassium ≥5.5 mmol/L; Chloride ≤85 mmol/L; Chloride ≥120 mmol/L; Calcium <2.0 mmol/L; Calcium >2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine >1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x ULN; Glucose ≤2.48 mmol/L; Glucose ≥11 mmol/L; Albumin ≤26 g/L; Albumin ≥60 g/L; Protein ≤50 g/L; Protein ≥100 g/L; Alanine aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN
Measured: 40 Weeks Treatment Duration
Secondary outcomes (11)
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40
The RSBQ is a 45-item caregiver-completed rating scalescale includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behaviour.
Measured: 40 Weeks Treatment Duration
Clinical Global Impression-Improvement (CGI-I) Score at Week 40
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse. Higher CGI-I scores denote more severe illness and less improvement in the illness.
Measured: 40 Weeks Treatment Duration
Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40
Scale to assess communication and pre-linguistic skills in children 12-24 months (or older children with developmental delay). The Checklist consists of 24 questions ranging from 0 to 4 points within each of 7 Clusters. 0 points are given for"Not Yet", 1 point for "Sometimes", or 2 points for "Often". For items describing a series of numbers or ranges, 0 points are given for "None" and 1 to 4 points for items containing numbered choices.
The Social Composite score is one of 3 composite scores. It comprises 13 items in skill areas "Emotion and Eye Gaze" (items 1 to 4), "Communication" (items 5 to 8), and "Gestures" (items 9 to 13). The Social Composite raw score (items 1 to 13), ranging from 0 to 26, is calculated as the sum of the item scores. Higher Social Composite raw scores indicate better social communication development.
Measured: 40 Weeks Treatment Duration
Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40
The overall quality of life score rating of the ICND ranges from 1 ("Poor") to 6 ("Excellent"); lower overall quality of life scores indicate lower quality of life.
Measured: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40
The RTT-HF is a clinician completed clinical assessment of the subject's ability to use her hands for functional purposes. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment
Measured: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) Change From Baseline to Week 40
The RTT-AMB is a clinician completed clinical assessment of the subject's ability to sit, stand, and ambulate. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) Change From Baseline to Week 40
The RTT-COMC is a clinician completed clinical assessment of the subject's ability to communicate her choices or preferences, which can include the use of nonverbal means such as eye contact or gestures. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) Change From Baseline to Week 40
The RTT-VCOM is a clinician completed clinical assessment of the subject's ability to communicate verbally. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 40 Weeks Treatment Duration
Change From Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S)
The CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on severity of illness at the time of rating: 1=normal, not at all ill; 2=borderline ill; 3=mildly ill; 4= moderately ill; 5=markedly ill; 6=severely ill; or 7=extremely ill. Higher CGI-S scores denote more severe illness and less improvement in the illness.
Measured: 40 Weeks Treatment Duration
Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) Change From Baseline to Week 40
The RTT-CBI consists of 24 negatively worded items (Items 1 through 24). Frequency ratings are on a 5-point Likert scale including: 0-never; 1-rarely; 2-sometimes; 3-frequently and 4-nearly always. The RTT-CBI also includes 2 positively worded items (items 25 and 26) that comprise the Optimism Index; this index will not be used for analysis. The total score ranging from 0 to 96 is calculated as the sum of the scores for Items 1-24.Higher scores signify higher caregiver burden.
Measured: 40 Weeks Treatment Duration
Impact of Childhood Neurologic Disability Scale (ICND) Total Score Change From Baseline to Week 40
The ICND scale evaluates the effect of 4 health problems on 11 aspects of the child's or the family's life scored 0 ("Not at all"), 1 ("A little"), 2 ("Some"), 3 ("A lot"), or "Does not apply". The 4 health problems are 1) inattentiveness, impulsivity, or mood, 2) ability to think and remember, 3) neurologic or physical limitations, and 4) epilepsy. For each health problem, the score is calculated as the sum of the item scores. The ICND total score will be calculated as the sum of the average of each problem score multiplied by 11. The ICND total score ranges from 0 to 132. Higher ICND total scores indicate worse health problems. The ICND total score does not include the Overall Quality of Life Rating.
Measured: 40 Weeks Treatment Duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2020
- Study start29 Jan 2020
- Primary completion19 Aug 2022
- Study completion19 Aug 2022
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 Rett 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 154 participants (actual)
Sponsor and investigators
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04279314
- Sponsor's protocol ID
- ACP-2566-004
- Acronym
- LILAC™
- Status last verified
- Mar 2024
- First submitted
- 19 Feb 2020
- Last updated
- 11 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study. Med. 2024 Sep 13;5(9):1178-1189.e3. doi: 10.1016/j.medj.2024.05.018. Epub 2024 Jun 24.
PubMed 38917793Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
PubMed 37460385
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
- ClinicalTrials.gov ID
- NCT04279314
- Acronym / sponsor ID
- LILAC™ / ACP-2566-004
- Conditions
- Rett Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 154 participants (actual)
- Lead sponsor
- ACADIA Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Jan 2020 (actual)
- Primary completion
- 19 Aug 2022 (actual)
- Study completion
- 19 Aug 2022 (actual)
- First posted
- 21 Feb 2020 (actual)
- Results
- Posted 11 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Trofinetide | Experimental | Drug: Trofinetide |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
- Met all entry criteria for the antecedent study
- May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
- Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
- The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
Key exclusion criteria (9 listed)
- Began treatment with growth hormone during the antecedent study
- Began treatment with IGF-1 during the antecedent study
- Began treatment with insulin during the antecedent study
- Has developed a clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory, or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
- Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
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 |
|---|---|---|
| TrofinetideTrofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) | Drug | Trofinetide |
Primary outcome
Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs
Time frame: 40 Weeks Treatment Duration
Percentage of subjects with treatment-emergent adverse events (TEAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects with withdrawals due to AEs
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG
Time frame: 40 Weeks Treatment Duration
Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval >500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of >60 ms
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs
Time frame: 40 Weeks Treatment Duration
Potentially clinically important changes in vital signs were defined in the study protocol as: systolic blood pressure (SBP) ≥180 mmHg and increased ≥20 mmHg from baseline; SBP ≤90 mmHg and decreased ≥20 mmHg from baseline; diastolic blood pressure (DBP) ≥ 105 mmHg and increased ≥15 mmHg from baseline; DBP ≤50 mmHg and decreased ≥15 mmHg from baseline; Pulse ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight
Time frame: 40 Weeks Treatment Duration
Potentially clinically important changes in body weight were defined in the study protocol as: Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes
Time frame: 40 Weeks Treatment Duration
Potentially clinically important changes in laboratory parameters were defined in the study protocol as: Sodium ≤125 mmol/L; Sodium ≥155 mmol/L; Potassium ≤3.0 mmol/L; Potassium ≥5.5 mmol/L; Chloride ≤85 mmol/L; Chloride ≥120 mmol/L; Calcium <2.0 mmol/L; Calcium >2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine >1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x ULN; Glucose ≤2.48 mmol/L; Glucose ≥11 mmol/L; Albumin ≤26 g/L; Albumin ≥60 g/L; Protein ≤50 g/L; Protein ≥100 g/L; Alanine aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN
11 secondary outcomes
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Clinical Global Impression-Improvement (CGI-I) Score at Week 40
Time frame: 40 Weeks Treatment Duration
Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Change From Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S)
Time frame: 40 Weeks Treatment Duration
Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Impact of Childhood Neurologic Disability Scale (ICND) Total Score Change From Baseline to Week 40
Time frame: 40 Weeks Treatment Duration
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38917793Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study. Med. 2024 Sep 13;5(9):1178-1189.e3. doi: 10.1016/j.medj.2024.05.018. Epub 2024 Jun 24.
- Derived from this study: PubMed 37460385Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Trofinetide in people with Rett Syndrome, run by ACADIA Pharmaceuticals Inc. at 21 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 6 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 11 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 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. 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 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?
Yes. Results are posted in the registry (first posted 11 Apr 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?
Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs (time frame: 40 Weeks Treatment Duration). 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?
On the ClinicalTrials.gov record for NCT04279314, 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 11 Apr 2024.