TerminatedPhase 3

Open-Label Extension Study of Trofinetide for Rett Syndrome

Stopped: As a result of marketing approval of trofinetide on 10 March 2023, the study was terminated by the Sponsor with the intent of switching patients to commercially available product.

United States77 participantsStarted 2020-11-08

Plain-language summary

To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome

Who can participate

Age range

5 Years – 21 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks)
. May benefit from continued 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
. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use 2 clinically acceptable methods of contraception (e.g., oral, intrauterine device \[IUD\], diaphragm plus spermicide, injectable, transdermal or implantable contraception) for the duration of the study and for at least 30 days thereafter. Subject must not be pregnant or breastfeeding.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs

Timeframe: Mean study drug exposure was 426 days, corresponding to 1.2 years

Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline

Timeframe: Mean study drug exposure was 426 days, corresponding to 1.2 years

Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline

Timeframe: Mean study drug exposure was 426 days, corresponding to 1.2 years

Number (%) of Patients With Potentially Clinically Important Changes in Body Weight Post-baseline

Timeframe: Mean study drug exposure was 426 days, corresponding to 1.2 years

Number (%) of Patients With Potentially Clinically Important Changes in Laboratory Parameters Post-baseline

Timeframe: Mean study drug exposure was 426 days, corresponding to 1.2 years

Trial details

NCT IDNCT04776746

SponsorACADIA Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-06-30

Results submitted2024-08-19

View on ClinicalTrials.gov More Rett Syndrome trials

Who can participate

Age range

5 Years – 21 Years

Sex

FEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks)
. May benefit from continued 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
. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use 2 clinically acceptable methods of contraception (e.g., oral, intrauterine device \[IUD\], diaphragm plus spermicide, injectable, transdermal or implantable contraception) for the duration of the study and for at least 30 days thereafter. Subject must not be pregnant or breastfeeding.