NCT01894958

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Fixed-Dose Study of NNZ-2566 in Fragile X Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 72 (actual)
• Sponsor: Neuren Pharmaceuticals Limited · Industry
• Sex: Male
• Age: 12 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Fragile X Syndrome in adolescent and adult males.

Detailed description

Fragile X Syndrome is a genetically determined neurological disorder in which affected individuals are intellectually handicapped to varying degrees and display a variety of associated psychiatric symptoms. Clinically, Fragile X Syndrome is characterized by intellectual handicap, hyperactivity and attentional problems, autism spectrum symptoms, emotional lability and epilepsy. The epilepsy seen in Fragile X Syndrome is most commonly present in childhood, but then gradually remits towards adulthood. Physical features such as prominent ears and jaw, and hyper-extensibility of joints are frequently present but are not diagnostic. Intellectual handicap is the most common feature defining the phenotype. Treatment for the disorder is symptomatic - focusing on the management of symptoms - and supportive, requiring a multidisciplinary approach. 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 males with Fragile X Syndrome. The study also will also investigate measures of efficacy during treatment.

Conditions

• Fragile X Syndrome

Interventions

Type	Name	Description
DRUG	NNZ-2566	Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder (2g in 50mL vials or 3g in 30mL bottles) for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.
DRUG	Placebo	Strawberry flavored solution

Timeline

• Start date: 2014-01-01
• Primary completion: 2015-09-01
• Completion: 2015-10-01
• First posted: 2013-07-10
• Last updated: 2018-02-05

Locations

17 sites across 1 country: United States