Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

Learn more about:
Related Clinical Trial
The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome Development of the ORCA Communication Measure for Rett Syndrome The Rett Syndrome Global Registry Open-Label Extension Study of Trofinetide for Rett Syndrome ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME Predictors of Caregiver Adaptation to Pervasive Developmental Disorders A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD) New Genes Involved in Molecular Etiology of Rett Syndrome Through DNA Microarray Comparative Genomic Hybridization An Open-label Extension Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome ANAVEX2-73 Study in Patients With Rett Syndrome Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome Nutritional Aspects of Rett Syndrome Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW Study of the Pathogenesis of Rett Syndrome Advanced Grandparental Age as a Risk Factor for Autism A Safety Study of NNZ-2566 in Pediatric Rett Syndrome Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin Biobanking of Rett Syndrome and Related Disorders Telehealth Support to Increase Physical Activity in Rett Syndrome Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome Natural History of Rett Syndrome & Related Disorders Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome Study of ANAVEX2-73 in Patients With Rett Syndrome Trial of Dextromethorphan in Rett Syndrome Pharmacological Treatment of Rett Syndrome With Statins An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome Analysis of the Glutathione Cycle in Children With Rett Syndrome Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome ‘Uptime’ Participation Intervention in Girls and Women With Rett Syndrome Functional Abilities in Rett Syndrome Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection) Placebo Controlled Trial of Dextromethorphan in Rett Syndrome Effects of Creatine Supplementation in Rett Syndrome Metabolic Evaluation of Nutrition in Rett Syndrome Treatment of Rett Syndrome With Recombinant Human IGF-1 A Study to Evaluate Ketamine for the Treatment of Rett Syndrome Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms A Safety Study of NNZ-2566 in Patients With Rett Syndrome Genetic and Physical Characteristics of Rett Syndrome Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™) The Role of Family Functioning in Adaptation to Being a Caregiver of an Individual With Rett Syndrome A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome The Findings of MR Imaging in Rett Syndrome Phase 2 Study of EPI-743 for Treatment of Rett Syndrome Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome Patients With RETT Syndrome Creatine Metabolism in Rett Syndrome

Brief Title

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

Official Title

A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

Brief Summary

      To investigate the safety and tolerability of 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)

Secondary Outcome

 Rett Syndrome Behaviour Questionnaire (RSBQ) total score - change from Baseline to Week 40

Condition

Rett Syndrome

Intervention

Trofinetide

Study Arms / Comparison Groups

 Trofinetide
Description:  

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

180

Start Date

January 29, 2020

Completion Date

October 2022

Primary Completion Date

October 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study
             ACP-2566-003

          2. Met all entry criteria for the antecedent study

          3. May benefit from long-term treatment with open-label trofinetide in the judgment of
             the Investigator

          4. Can still swallow the study medication provided as a liquid solution or can take it by
             gastrostomy tube

          5. The subject's caregiver is English-speaking and has sufficient language skills to
             complete the caregiver assessments

          6. 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

        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) or has major surgery planned during the study

          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 a QTcF interval of >450 ms on the Baseline ECG performed before the first dose of
             trofinetide is given in the present 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 prespecified entry criteria).
      

Gender

Female

Ages

5 Years - 21 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04279314

Organization ID

ACP-2566-004


Responsible Party

Sponsor

Study Sponsor

ACADIA Pharmaceuticals Inc.


Study Sponsor

, , 


Verification Date

November 2021