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A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Primary Purpose

Rett Syndrome

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

NNZ-2566

Placebo

About this trial

This is an interventional treatment trial for Rett Syndrome focused on measuring Autism, Rett's syndrome, Rett disorder, Rett's disorder, Ataxia

Eligibility Criteria

5 Years - 15 Years (Child)FemaleDoes not accept healthy volunteers

Inclusion Criteria:

Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene.
Age 5 - 15 years.
Weight at Screening and Baseline between 15.0 kg-100.0 kg (at least 15.0 kg and no greater than 100.0 kg).
Each subject must be able to swallow the study medication provided as a liquid solution, or via gastrostomy tube.

Exclusion Criteria:

Actively undergoing neurological regression
Abnormal QT interval, prolongation or significant cardiovascular history.
Current treatment with insulin.
Anti-convulsants with liver enzyme inducing effects.
Unstable seizure profile.
Excluded concomitant medications.
Current clinically significant (as determined by the investigator). cardiovascular, renal, hepatic, or respiratory disease.
Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication.
History of, or current cerebrovascular disease or brain trauma.
History of, or current clinically significant endocrine disorder, e.g. hypo- or hyperthyroidism, or diabetes mellitus.
History of, or current, malignancy.
Significant hearing and/or visual impairments that may affect ability to complete the test procedures.
Allergy to strawberry.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

NNZ-2566

Placebo (strawberry flavored solution)

Arm Description

Glycyl-L-2-Methylpropyl-L-Glutamic Acid

Strawberry flavored solution and Water for Injection

Outcomes

Primary Outcome Measures

Adverse events

Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the three NNZ-2566 doses and placebo. SAEs and AEs will be examined throughout the study.

Secondary Outcome Measures

Motor Behaviour Assessment Scale (MBA)

Clinical Global Impression of Improvement (CGI-I)

Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS)

Full Information

NCT ID

NCT02715115

First Posted

February 21, 2016

Last Updated

August 6, 2020

Sponsor

Neuren Pharmaceuticals Limited

Collaborators

rettsyndrome.org

1. Study Identification

Unique Protocol Identification Number

NCT02715115

Brief Title

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Official Title

A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome

Study Type

Interventional

2. Study Status

Record Verification Date

August 2020

Overall Recruitment Status

Completed

Study Start Date

March 2016 (Actual)

Primary Completion Date

January 5, 2017 (Actual)

Study Completion Date

January 5, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Neuren Pharmaceuticals Limited

Collaborators

rettsyndrome.org

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett syndrome in children and adolescents.

Detailed Description

Rett syndrome is a neurodevelopmental disorder primarily affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of regression with onset of systemic and neurological signs. The CNS symptoms of Rett syndrome include learning disability, autism symptomatology and epilepsy and these can be severe and highly debilitating. Affected individuals also show signs of autonomic dysfunction, reflected in cardiovascular and respiratory abnormalities. There is no currently effective treatment for Rett syndrome. This study will investigate the safety, tolerability and blood pharmacokinetics of treatment with oral administration of NNZ-2566 at 50 mg/kg, 100 mg/kg, 200 mg/kg BID, or placebo BID, in children and adolescent females with Rett syndrome. The study also will also investigate measures of efficacy and biomarkers during treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Rett Syndrome

Keywords

Autism, Rett's syndrome, Rett disorder, Rett's disorder, Ataxia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

82 (Actual)

8. Arms, Groups, and Interventions

Arm Title

NNZ-2566

Arm Type

Experimental

Arm Description

Glycyl-L-2-Methylpropyl-L-Glutamic Acid

Arm Title

Placebo (strawberry flavored solution)

Arm Type

Placebo Comparator

Arm Description

Strawberry flavored solution and Water for Injection

Intervention Type

Drug

Intervention Name(s)

NNZ-2566

Other Intervention Name(s)

trofinetide

Intervention Description

Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Strawberry flavoring

Intervention Description

Strawberry flavored solution and Water for Injection

Primary Outcome Measure Information:

Title

Adverse events

Description

Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the three NNZ-2566 doses and placebo. SAEs and AEs will be examined throughout the study.

Time Frame

Through study completion, an average of 11 weeks

Secondary Outcome Measure Information:

Title

Motor Behaviour Assessment Scale (MBA)

Time Frame

Through study completion, an average of 11 weeks

Title

Clinical Global Impression of Improvement (CGI-I)

Time Frame

Through study completion, an average of 11 weeks

Title

Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS)

Time Frame

Through study completion, an average of 11 weeks

10. Eligibility

Sex

Female

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

15 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene. Age 5 - 15 years. Weight at Screening and Baseline between 15.0 kg-100.0 kg (at least 15.0 kg and no greater than 100.0 kg). Each subject must be able to swallow the study medication provided as a liquid solution, or via gastrostomy tube. Exclusion Criteria: Actively undergoing neurological regression Abnormal QT interval, prolongation or significant cardiovascular history. Current treatment with insulin. Anti-convulsants with liver enzyme inducing effects. Unstable seizure profile. Excluded concomitant medications. Current clinically significant (as determined by the investigator). cardiovascular, renal, hepatic, or respiratory disease. Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication. History of, or current cerebrovascular disease or brain trauma. History of, or current clinically significant endocrine disorder, e.g. hypo- or hyperthyroidism, or diabetes mellitus. History of, or current, malignancy. Significant hearing and/or visual impairments that may affect ability to complete the test procedures. Allergy to strawberry.

Overall Study Officials: