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BELIEVE 1

Clinical study of Cannabidiol in children and adolescents with Developmental and Epileptic Encephalopathy

Recruitment closed. Enrolment target met.

Primary Objective: This is a sequential, multi-stage, open-label, multi-national, multiple-center, multiple-dose study to assess the long-term safety and tolerability of ZYN002 in child and adolescent epilepsy patients 3 to <18 years of age having seizures associated with developmental and epileptic encephalopathies (DEE).

Principal Investigator: Lynette Sadlier

Study Coordinator:  Natasha Eagle

Trial Website: https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=374807

Central Precocious Puberty Study

Pharmacokinetic Study of Leuprolide Acetate Injectable Suspension in the Treatment of Central Precocious Puberty

Recruitment closed. Enrolment target met.

Primary Objective: This study determines the effectiveness of leuprolide acetate 45 mg for injectable suspension for treatment of children (ages 2-9) with Central Precocious Puberty. Leuprolide acetate is a GnRH agonist that inhibits pituitary gonadotropin secretion by binding to the GnRH receptors and blocking downstream hormone synthesis. The steady decrease in hormone synthesis (LH and FSH) leads to a suppression of testicular and ovarian steroidogenesis. In children with CPP, this steady decrease in hormone synthesis disrupts the progression of puberty.

Principal Investigator: Esko Wiltshire

Study Coordinator: Jonathan Barrett

Trial Website: https://clinicaltrials.gov/ct2/show/NCT02452931

CONNECT-FX (FRAGILE X)

Clinical Study of Cannabidiol in Children and Adolescents with Fragile X (CONNECT-FX)

This study is currently recruiting participants.

Primary Objective: This study will evaluate the efficacy and safety of ZYN002, a clear cannabidiol (CBD) gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioural symptoms of Fragile X Syndrome (FXS). Eligible participants will then participate in up to a 14 week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to < 18 years will be eligible to participate.

Principal Investigator: Andrew Marshall

Study Coordinator: Natasha Eagle

Trial Website: https://clinicaltrials.gov/ct2/show/NCT03614663

CONNECT-FX (FRAGILE X) Open Label Extension (OLE)

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents with FXS

Must be enrolled in the primary CONNECT-FX study.

Primary Objective: ZYN002 is a pharmaceutically manufactured cannabidiol (CBD) that is developed as a clear gel that can be applied to the skin (called transdermal delivery). Only participants from the ZYN2-CL-016 study who meet the inclusion criteria and none of the exclusion criteria for study ZYN2-CL-017 are eligible.

Principal Investigator: Andrew Marshall

Study Coordinator: Natasha Eagle

Trial Website: https://clinicaltrials.gov/ct2/show/NCT03802799

REviral

A phase 2a Study in Infants with RSV lower respiratory tract infection, to evaluate the safety, tolerability, pharmacokinetics and antiviral effect of RV521

This study is on hold. Estimated start up is in 2020.

Primary Objective: This study is designed to evaluate the safety and tolerability of single (Part A) and multiple (Part B) oral doses of RV521 in infants (≥ 1 month and ≤ 24 months) hospitalised with Respiratory Syncytial Virus (RSV) lower respiratory tract infection (LRTI).

Principal Investigator: Thorsten V Stanley

Study Coordinator: Marina Dzhelali

Trial Website: https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-001010-15/PL

T2NOW

Study to Evaluate Safety and Efficacy of Dapagliflozin and Saxagliptin in Patients with Type 2 Diabetes Mellitus Aged 10 to Below 18 Years Old

This study is currently recruiting participants.

Primary Objective: Dapagliflozin and saxagliptin are both approved for use in patients with Type 2 Diabetes aged 18 years or older. This study will assess how well dapagliflozin and saxagliptin work by finding out how these treatments affect blood glucose (sugar) levels compared to placebo (a pill that contains no active drug), in children and adolescents (ages 10-18). Dapagliflozin and saxagliptin are considered investigational products in this study since while they have been approved for use in adults (patients 18 years or older), they haven't been approved for children and adolescents due to lack of clinical studies in this specific population.

Principal Investigator: Esko Wiltshire

Study Coordinator: Jonathan Barrett

Trial Website: https://clinicaltrials.gov/ct2/show/NCT03199053

Last updated 25 November 2019.