Clinical study of cannabidiol in children, adolescents and young adults with Fragile X syndrome (RECONNECT)
- Conditions
- Fragile X SyndromeHuman Genetics and Inherited Disorders - Other human genetics and inherited disordersNeurological - Other neurological disorders
- Registration Number
- ACTRN12621000874819
- Lead Sponsor
- Zynerba Pharmaceuticals Pty Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 204
1.Male or female children, adolescents, and young adults aged 3 to <23 years, at the time of Screening.
2.Patient resides with caregiver who will continue to provide consistent care throughout the study.
3.Judged by the Investigator to be in generally good health at Screening based upon the results of a medical history, physical examination, 12-lead ECG, and clinical laboratory test results. Laboratory results outside of the reference range must be documented as not clinically significant by both the Investigator and Sponsor.
4.Patients must have a diagnosis of FXS through molecular documentation of full mutation of the FMR1 gene documented through genetic testing at Screening.
5.Patients with an ABC-CFXS Pre-Specified Subscale 1 score at Screening and at Visit 2.
6.Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of no more than two AEDs for the four weeks preceding study Screening; or must be seizure-free for one year if not currently receiving AEDs.
7.Patients who are taking psychotropic medication(s) should be on a stable regimen of no more than three such medications for at least four weeks preceding study Screening and must maintain that regimen throughout the study. Psychotropic medications include (but are not limited to) antipsychotics, antidepressants, anxiolytics, attention-deficit / hyperactivity disorder (ADHD) medications, and medications for sleep.
8.If patients are receiving non-pharmacological behavioral and/or dietary interventions, they must be stable and have been doing so for three months prior to Screening.
9.Patients have a body mass index between 12–30 kg/m2 (inclusive).
10.Females of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative serum or urine pregnancy test at all designated visits.
11.Patients and parents/caregivers must be adequately informed of the nature and risks of the study and give written informed consent (and assent if applicable) prior to Screening. Parents/caregiver(s) must provide written consent to assist in trial drug administration.
12.Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures, and in the Investigator’s opinion, are reliable and willing and able to comply with all protocol requirements and procedures.
Any of the following is considered criterion for exclusion:
1.Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of study medication.
Standard acceptable methods of contraception include abstinence (defined as refraining from heterosexual intercourse from screening to three months after the last dose of study medication) or the use of a highly effective method of contraception, including hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, spermicide, vasectomy, or intrauterine device. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception.
2.Patient has transitioned to independent living or living in a residential facility such as a university setting or congregate care.
3.History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
4.Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study.
5.Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels greater than or equal to 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels greater than or equal to 3 times the ULN as determined from Screening safety laboratories.
6.Use of cannabis or any THC or CBD-containing product within 3 months of Screening Visit or during the study (aside from ZYN002).
7.Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines (except midazolam or comparable administered for blood draws and ECG collection), and opiates.
8.Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin, or vigabatrin.
9.Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4 including but not limited to the following medications: midazolam (except single doses administered for the purposes of obtaining blood samples and ECG’s), oral ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin, vincristine, vinorelbine, St. John’s Wort, and grapefruit juice/products.
10.Patients may not be taking any benzodiazepines (except single doses administered for the purposes of obtaining blood samples and ECGs) at screening or throughout the study.
11.Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
12.Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
13.Patient has an acute or progressive neurological disease, psychosis, schizophrenia, or any psychiatric
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in the Aberrant Behavior Checklist-Community FXS specific [ABC-C(FXS)] Pre-Specified Subscale 1 score in patients with complete methylation (100%) of the FMR1 gene.[Change from Baseline to Week 18.]
- Secondary Outcome Measures
Name Time Method