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    Summary
    EudraCT Number:2021-002542-33
    Sponsor's Protocol Code Number:ZYN2-CL-033
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-08-10
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedIreland - HPRA
    A.2EudraCT number2021-002542-33
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults with Fragile X Syndrome - RECONNECT
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effects and Safety of Test drug ZYN002, applied as a gel onto the skin of children, adolescents and young adults with a certain hereditary disease called Fragile X Syndrome
    A.3.2Name or abbreviated title of the trial where available
    RECONNECT
    A.4.1Sponsor's protocol code numberZYN2-CL-033
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04977986
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/081/2024
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorZynerba Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportZynerba Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationZynerba Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointVice President, Medical
    B.5.3 Address:
    B.5.3.1Street Address80 W. Lancaster Ave., Suite 300
    B.5.3.2Town/ cityDevon, PA
    B.5.3.3Post code19333
    B.5.3.4CountryUnited States
    B.5.4Telephone number001919271-1339
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/22/2583
    D.3 Description of the IMP
    D.3.2Product code ZYN002
    D.3.4Pharmaceutical form Transdermal gel
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTransdermal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNn/a
    D.3.9.1CAS number 13956-29-1
    D.3.9.2Current sponsor codeZYN002
    D.3.9.3Other descriptive nameCANNABIDIOL
    D.3.9.4EV Substance CodeSUB26600
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4.2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTransdermal gel
    D.8.4Route of administration of the placeboTransdermal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fragile X syndrome (FXS) is a rare genetic disorder caused by the deficiency or absence of Fragile X Messenger Ribonucleoprotein 1 (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.
    E.1.1.1Medical condition in easily understood language
    Fragile X syndrome (FXS) is a rare disease caused by lack of Fragile X Messenger Ribonucleoprotein 1 due to a faulty gene. It mainly causes learning difficulties in patients.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10017324
    E.1.2Term Fragile X syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of ZYN002 administered as a transdermal gel formulation in patients ages 3 to <23 years, in the treatment of behavioral symptoms of FXS
    E.2.2Secondary objectives of the trial
    To further evaluate the efficacy of ZYN002 in the treatment of symptoms of FXS.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    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 Social Avoidance score of ≥ 5 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.
    9. Patients have a body mass index between 12–30 kg/m2 (inclusive) and patients with a body mass index >30 kg/m2 and <40 kg/m2 with normal liver function laboratory values and with no immediate family history of fatty liver disease.
    E.4Principal exclusion criteria
    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 ≤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 ≥2 times the upper limit of normal (ULN) or has alkaline phosphatase levels ≥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).
    8. Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, 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, and St. John’s Wort.
    10. 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.
    13. 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.
    17. 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.

    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint:
    Change from Baseline to Week 18 in the ABC-CFXS Social Avoidance subscale score in patients with complete methylation of the FMR1 gene.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From screening up to Week 18
    E.5.2Secondary end point(s)
    Key Secondary endpoints:
    1. Change from Baseline to Week 18 in ABC-CFXS Irritability subscale score in patients with complete methylation of the FMR1 gene.
    2. Percent of patients with any improvement on the CaGI-C at Week 18 for Social Interactions among patients with complete methylation of the FMR1 gene.
    3. Percent of patients rated as improved on the CGI-I scale (minimally, much, very much improved) at Week 18 among patients with complete methylation of the FMR1 gene.
    4. Change from Baseline to Week 18 in the ABC-CFXS Social Avoidance subscale score among all randomized patients (complete and partial methylation of the FMR1 gene).

    Other Secondary Efficacy Endpoints will be evaluated in the patients with complete methylation of the FMR1 gene and in all randomized patients.
    -Percent of patients with a clinically meaningful within-subject improvement from Baseline in ABC-CFXS Social Avoidance subscale score at Week 18 among patients.
    -Percent of patients with a clinically meaningful within-subject improvement from Baseline in ABC-CFXS Irritability subscale score at Week 18 among patients.
    -Change from Baseline in ABC-CFXS Social Avoidance and Irritability subscale scores at Weeks 10 and 14 in patients.
    -Change from Baseline in ABC-CFXS Socially Unresponsive/Lethargic, Stereotypy, Inappropriate Speech, and Hyperactivity subscale scores at Weeks 10, 14, and 18 in patients.
    -Percent of patients with any improvement on the CaGI-C at Week 18 for Social Avoidance and Isolation, Irritable and Disruptive Behaviors, and Overall Behavior among patients.
    -Percent of patients with any improvement on the CaGI-C at Week 18 for Social Interactions among all randomized patients.
    -Percent of patients rated as improved on the CGI-I scale (minimally, much, very much improved) at Week 18 among all randomized patients.
    -Incidence of TEAEs and SAEs by relationship to treatment and severity; incidence of SAEs/AEs leading to treatment discontinuation.
    -Changes in vital signs, clinical laboratory tests, and ECG.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From screening up to Week 18
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last patient, last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 204
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 92
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 92
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Signed informed consent will be obtained at Screening from patient or parent/caregivers (as applicable) and, if applicable, assent will be obtained from patients where developmentally appropriate.
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Subjects are minors
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 204
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-10-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-22
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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