A Safety Study of NNZ-2566 in Patients With Rett Syndrome...

A Safety Study of NNZ-2566 in Patients With Rett Syndrome


Verified by: Neuren Pharmaceuticals Limited, October 2012

First Received: October 4, 2012 | Last Updated: October 11, 2012

Phase: Phase 2 | Start Date: December 2012

Overall Status: Not yet recruiting | Estimated Enrollment: 42

Brief Summary

? Skip to Participation Criteria

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Official Title: “A Phase IIa Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in the Treatment of Adolescent and Adult Females With Rett Syndrome”

The purpose of this study is to determine whether NNZ-2566 is safe in the treatment of Rett Syndrome in adolescent and adult females.
?Study Type: Interventional
?Study Design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
?Study Primary Completion Date: December 2013

Detailed Clinical Trial Description

Rett Syndrome is a developmental disorder primarily if not exclusively 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 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 and tolerability of treatment with oral administration of NNZ-2566 at 35 mg/kg or 70 mg/kg BID in adolescent or adult females with Rett Syndrome. The study also will also investigate measures of efficacy during treatment.

Interventions Used in this Clinical Trial
?Drug: NNZ-2566?Glycyl-L-2-Methylprolyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder (2g in 50mL vials) for reconstitution with lemon flavored cordial in Water for Injection.

?Drug: Placebo?Lemon flavored cordial and Water for Injection


Arms, Groups and Cohorts in this Clinical Trial
?Experimental: NNZ-2566?Glycyl-L-2-Methylprolyl-L-Glutamic Acid

?Placebo Comparator: Placebo (lemon flavored cordial)?Lemon flavored cordial and Water for Injection


Outcome Measures for this Clinical Trial

Primary Measures
?Adverse events?Time Frame: Through to Day 33
Safety Issue?: Yes

?Physiological changes?Time Frame: Through to Day 33
Safety Issue?: Yes


Secondary Measures
?EEG and video monitoring?Time Frame: Baseline, daily during first five days of dosing, Days 11, 23 and 33
Safety Issue?: No

?Behavior?Time Frame: Baseline and Day 23
Safety Issue?: No

?Physiological changes?Time Frame: Baseline and Days 2, 4, 11, 23 and 33.
Safety Issue?: No


Criteria for Participation in this Clinical Trial

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Inclusion Criteria
?Diagnosis of Rett Syndrome with proven mutation of the MECP2 gene
?Age 16 to 40 years
?Severity rating of between 14 and 30 (Rett Syndrome Natural History/Clinical Severity Scale)
?Concomitant medications must be stable for >6 weeks prior to enrollment.
?Ability to swallow study medication provided as a liquid solution, or via gastrostomy tube

Exclusion Criteria
?No detectable abnormality of the EEG at baseline
?Actively undergoing regression
?QTc exclusions
?Current treatment with insulin
?Abnormal HbAIC values
?Current/past treatment with IGF-1
?Current/past treatment with growth hormone
?Current treatment with NMDA antagonists
?Planned or recent (<6 weeks)non-medication based interventional therapy
?Current clinically significant cardiovascular, renal , hepatic or respiratory disease
?gastrointestinal disease
?Cerebrovascular disease or brain trauma
?Significant endocrine disorder (hypo/hyperthyroidism or diabetes mellitus
?History of, or current malignancy
?Clinically significant safety laboratory, vital signs or ECG abnormalities
?Significant hearing and/or visual impairment
?Enrollment in another clinical trial within the previous 30 days

Gender Eligibility for this Clinical Trial: Female

Minimum Age for this Clinical Trial: 16 Years

Maximum Age for this Clinical Trial: 40 Years

Are Healthy Volunteers Accepted for this Clinical Trial: No

Clinical Trial Investigator Information
?Lead Sponsor?Neuren Pharmaceuticals Limited

?Collaborator?Baylor College of Medicine

?Provider of Information About this Clinical Study ?Sponsor

?Overall Official(s)?Daniel G Glaze, M.D., Principal Investigator, Baylor College of Medicine
?Jeffrey L Neul, M.D., Ph.D., Principal Investigator, Baylor College of Medicine

?Overall Contact(s)?Joseph Horrigan, M.D., [email protected]



Additional Information on this Clinical Trial

Information obtained from ClinicalTrials.gov on October 21, 2012

Link to the current ClinicalTrials.gov record. – http://clinicaltrials.gov/show/NCT01703533

Study ID Number: Neu-2566-RETT-001

ClinicalTrials.gov Identifier: NCT01703533

Health Authority: United States: Food and Drug Administration
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Source

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The URL of this page is:
http://clinicaltrialsfeeds.org/clinical-trials/show/NCT01703533