Open-Label Extension Study of Trofinetide for Rett Syndrome

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Brief Title

Open-Label Extension Study of Trofinetide for Rett Syndrome

Official Title

An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome

Brief Summary

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


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Percentage of subjects with treatment-emergent adverse events (TAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects withdrawals due to adverse events (AEs)


Condition

Rett Syndrome

Intervention

trofinetide

Study Arms / Comparison Groups

 Drug - trofinetide
Description:  trofinetide oral solution

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

153

Start Date

November 8, 2020

Completion Date

January 2023

Primary Completion Date

January 2023

Eligibility Criteria

        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 screening 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).
      

Gender

Female

Ages

5 Years - 22 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04776746

Organization ID

ACP-2566-005


Responsible Party

Sponsor

Study Sponsor

ACADIA Pharmaceuticals Inc.


Study Sponsor

, , 


Verification Date

August 2022