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Trial · Interventional

TerminatedPhase 3Results postedRegistry data

Open-Label Extension Study of Trofinetide for Rett Syndrome

NCTNCT04776746 (opens ClinicalTrials.gov in a new tab)Other idACP-2566-005
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Terminated
Phase
Phase 3
Enrollment
77 participants (actual)
Sponsor
ACADIA Pharmaceuticals Inc. · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

United States

Start
8 Nov 2020
Primary completion
30 Jun 2023
Completion
30 Jun 2023
First posted
2 Mar 2021
Last update posted
27 Sept 2024
Conditions
Rett Syndrome

Timeline

1 trial · 1 stopped early · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20202021202220232024
  1. Ph 3

    Open-Label Extension Study of Trofinetide for Rett Syndrome

    NCT04776746

    8 Nov 2020 to 30 Jun 2023

    Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

  • trofinetideDrug

Arms1

  • Experimental

    Drug - trofinetide

    trofinetide oral solution

Primary outcomes5

Outcomes the sponsor listed as primary.

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

    Time frame · Mean study drug exposure was 426 days, corresponding to 1.2 years

    Registry description

    Withdrawals due to AEs included both study drug discontinuation as well as study termination

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

    Time frame · Mean study drug exposure was 426 days, corresponding to 1.2 years

    Registry description

    Potentially clinically significant ECG changes were defined as QTcF \>500 ms or QTcF change from baseline (CFB) of \>60 ms

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

    Time frame · Mean study drug exposure was 426 days, corresponding to 1.2 years

    Registry description

    Potentially clinically important changes from baseline in vital signs were defined 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 rate (PR) ≥120 bpm and increased ≥15 bpm from baseline; PR≤50 bpm and decreased ≥15 bpm from baseline. Baseline was the baseline value of previous study ACP-2566-003.

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

    Time frame · Mean study drug exposure was 426 days, corresponding to 1.2 years

    Registry description

    Potentially clinically important changes from baseline in body weight were defined as: Weight increase ≥7% from baseline Weight decrease ≥7% from baseline

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

    Time frame · Mean study drug exposure was 426 days, corresponding to 1.2 years

    Registry description

    Potentially clinically important changes in laboratory parameters were defined 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 \>2.75 mmol/L; blood urea nitrogen ≥10.71 mmol/L; creatinine \>1.5× upper limit of normal (ULN); uric acid ≥505.75 μmol/L; lactate dehydrogenase (LDH) ≥3×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 transaminase (ALT) ≥3×ULN; aspartate transaminase (AST) ≥3×ULN; gamma glutamyl transferase (GGT) ≥3×ULN; alkaline phosphatase (ALP) ≥3×ULN; bilirubin ≥1.5×ULN

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· Mean study drug exposure was 426 days, corresponding to 1.2 years

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

GroupValue (Participants)
Trofinetide68
Primary outcomeCount of participants· Mean study drug exposure was 426 days, corresponding to 1.2 years

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

GroupValue (Participants)
Trofinetide0
Primary outcomeCount of participants· 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

GroupValue (Participants)
Trofinetide0
Primary outcomeCount of participants· 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

GroupValue (Participants)
Trofinetide45
Primary outcomeCount of participants· 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

GroupValue (Participants)
Trofinetide0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
TrofinetideTrofinetide oral solution was administered twice daily for up to approximately 32 months, orally or administered by gastrostomy (G) tube. The patient's assigned dose for this study was the final dose from the antecedent study (ACP-2566-004).4 / 7723 / 7757 / 77

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

PubMed tag: none · 2024

6 results

Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study.

Percy et al. 2024Med (New York, N.Y.)cited by 20

PMID 39025065DOI 10.1016/j.medj.2024.06.007PUB-39025065

Full text at the publisher6 results
Publisher pageSee results (6)

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedParent 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. PMID 37460385

Eligibility

Who could join, as posted.

Sex
Female
Age
from 5 Years up to 21 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks) 2. May benefit from continued treatment with open-label trofinetide in the judgment of the Investigator 3. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube 4. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments Childbearing Potential 5. 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. Exclusion Criteria: 1. Began treatment with growth hormone during the antecedent study 2. Began treatment with IGF-1 during the antecedent study 3. Began treatment with insulin during the antecedent study 4. 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) 5. 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 6. Has a clinically significant abnormality in vital signs at Baseline 7. Has an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study) 8. 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 pre-specified entry criteria).

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