Effects and Safety of Test drug ZYN002, applied as a gel onto the skin of children and adolescents with a certain hereditary disease called Fragile X Syndrome

Phase 1

• Conditions: Fragile X syndrome (FXS) is a rare genetic disorder caused by the deficiency or absence of Fragile X mental retardation protein (FMRP), an RNA-binding protein and the gene product of the FMR1 gene. FXS is most commonly caused by silencing of the FMR1 gene due to a trinucleotide repeat expansion. In FXS patients, the relative absence of functional FMRP is associated with critical impairments in neurodevelopment and learning, as well as disruption to other neuronal and non-neuronal functions.; MedDRA version: 20.1Level: PTClassification code 10017324Term: Fragile X syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2021-002542-33-IE
• Lead Sponsor: Zynerba Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-10
• Last Update Posted: 3 November 2021

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 204

Inclusion Criteria

1. Male or female children and adolescents aged 3 to <18 years, at the time of Screening.
2. 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.
3. Patients must have a diagnosis of FXS through molecular documentation of full mutation of the FMR1 gene documented through genetic testing at Screening.
4. Patients with an ABC-CFXS Social Avoidance score of = 5 at Screening and at Visit 2.
5. 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.
6. 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. Patients have a body mass index between 12–30 kg/m2 (inclusive).
Are the trial subjects under 18? yes
Number of subjects for this age range: 204
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

2. History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
3. Exposure to any investigational drug or device =30 days prior to Screening or at any time during the study.
4. Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels =2 times the upper limit of normal (ULN) or has alkaline phosphatase levels =3 times the ULN as determined from Screening safety laboratories.
5. 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 is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, or vigabatrin.
8. 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, and St. John’s Wort.
9. Patients may not be taking any benzodiazepines (except single doses administered for the purposes of obtaining blood samples and ECG’s) at screening or throughout the study.
12. Patient has an acute or progressive neurological disease, psychosis, schizophrenia, or any psychiatric disorder or severe mental abnormalities (other than FXS) that are likely to require changes in drug therapy or interfere with the objectives of the study or the ability to adhere to protocol requirements.
16. Any skin disease or condition, including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration, that may affect treatment application, application site assessments, or absorption of the trial drug.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified