An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

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Brief Title

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Official Title

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Brief Summary

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


Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Safety and tolerability of treatment with oral trofinetide


Condition

Rett Syndrome

Intervention

Trofinetide

Study Arms / Comparison Groups

 Drug - trofinetide
Description:  Oral dose of trofinetide

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

15

Start Date

September 22, 2021

Completion Date

July 2023

Primary Completion Date

July 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Female subject

               1. 2 to 4 years of age and body weight ≥9 kg and <20 kg at Screening OR

               2. 5 years of age and body weight ≥9 kg and <12 kg at Screening

          -  Can 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

          -  Has classic/typical Rett syndrome (RTT) or possible RTT according to the Rett Syndrome
             Diagnostic Criteria

          -  Has a documented disease-causing mutation in the MECP2 gene

          -  Has a stable pattern of seizures, or has had no seizures, within 8 weeks prior to
             Screening

          -  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 4 weeks prior to
             Screening

        Exclusion Criteria:

          -  Has been treated with insulin within 12 weeks of Baseline

          -  Has current 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

          -  Has a history of, or current, cerebrovascular disease or brain trauma

          -  Has significant, uncorrected visual or uncorrected hearing impairment

          -  Has a history of, or current, malignancy

          -  Has any of the following:

               1. QTcF interval of >450 ms at Screening or Baseline

               2. History of a risk factor for torsades de pointes (e.g., heart failure or family
                  history of long QT syndrome)

               3. History of clinically significant QT prolongation that is deemed to put the
                  subject at increased risk of clinically significant QT prolongation

               4. Other clinically significant finding on ECG at Screening or Baseline

        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).
      

Gender

Female

Ages

2 Years - 5 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04988867

Organization ID

ACP-2566-009


Responsible Party

Sponsor

Study Sponsor

ACADIA Pharmaceuticals Inc.


Study Sponsor

, , 


Verification Date

May 2022