- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05149898
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
Open-Label, Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome
Study Overview
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
-
-
Queensland
-
Brisbane, Queensland, Australia, 4101
- Lady Cilento Children's Hospital - South Brisbane
-
-
Victoria
-
Melbourne, Victoria, Australia, 3161
- Genetics Clinics Australia
-
-
-
-
South Carolina
-
Greenville, South Carolina, United States, 29605
- Greenwood Genetic Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Male or female children and adolescents aged 4 to less than 18 years, at the time of Screening.
- Judged by the Investigator to be in generally good health at Screening based upon the results of a medical history, physical examination, and clinical laboratory test results.
- Patients must have a diagnosis of 22q Deletion Syndrome (22qDS) confirmed by genetic testing.
- Patients have a Clinical Global Impression-Severity (CGI-S) score of 4 or higher at Screening and Visit 2.
- Patients must have a Pediatric Anxiety Rating Scale-Revised (PARS-R) severity score of 10 or higher at Screening and Visit 2.
- Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of one or two AEDs, or must be seizure-free for one year if not currently receiving AEDs.
- If patients are receiving non-pharmacological behavioral and/or dietary interventions, they must be stable for three months prior to Screening.
- Patient has demonstrated stable calcium levels for one year prior to Screening.
- Patients have a body mass index between 12-35 kg / m2 (inclusive).
- Females of childbearing potential must have a negative pregnancy test at the Screening Visit and a negative pregnancy test at all designated study visits.
- Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
- 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 study drug administration.
- In the Investigator's opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.
Exclusion Criteria:
- 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 for the duration of therapy and for three months after the last dose of study medication.
- History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
- Exposure to any investigational drug or device ≤ 30 days prior to Screening or at any time during the study.
- Patient has ALT, AST, or total bilirubin levels ≥ 2 times the ULN)or has alkaline phosphatase levels ≥ 3 times the ULN as determined from Screening safety laboratories.
- Use of cannabis or any THC or CBD-containing product within three months of Screening Visit or during the study.
- Patient has a positive drug screen.
- Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin or vigabatrin.
- Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4 including but not limited to the following medications: midazolam, 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.
- Patient with diagnosis of known genetic disorder, other than 22qDS.
- Patient has diagnosis of DiGeorge or Velocardiofacial syndrome without the presence of 22qDS.
- Patient has a primary psychiatric diagnosis other than 22qDS or anxiety, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder.
- Patient is on stable treatment of >6 months of not more than two psychoactive medications at screening or throughout the study (with the exception of one psychoactive medication prescribed for sleep).
- Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
- Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- Patient has an acute or progressive neurological disease, or any psychiatric disorder or severe mental abnormalities that are likely to require changes in drug therapy or interfere with the objectives of the study or ability to adhere to protocol requirements.
- Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.
- Patients at risk of needing cardiovascular surgical repair within the upcoming 12 months.
- Patient has unstable cardiovascular disease, such as advanced arteriosclerosis, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome), other serious or other clinically unstable cardiac problems as indicated by history, physical examination, or ECG.
- Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication.
- Any skin disease or condition that may affect treatment application, application site assessments, or absorption of the study drug.
- History of treatment for, or evidence of, drug abuse within the past year.
- Patient responds "yes" to Question '4' or '5' on the Columbia Suicide Severity Rating Scale Children (C-SSRS) during Screening or at any time on study.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Open-label
|
CBD Transdermal Gel pharmaceutically manufactured. Cannabidiol (CBD) formulated as a clear gel (transdermal delivery). Dose received is based on weight.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of treatment-emergent adverse events (safety and tolerability)
Time Frame: Adverse Event time frame is from admission to a study completion, an average of 9 months
|
Safety assessment will include collection of any treatment-emergent adverse events (safety and tolerability).
|
Adverse Event time frame is from admission to a study completion, an average of 9 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Aberrant Behavior Checklist (ABC-C) Pre-specified Subscale 1
Time Frame: Screening, Visit 2, Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period
|
ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
|
Screening, Visit 2, Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period
|
Clinical Global Impression-Severity and Improvement (CGI-S and CGI-I)
Time Frame: CGI-S is collected at Screening, Visit 2, Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period and CGI-I will be assessed at Visit 3 and Visit 4/EOS/ET, over 14 week period
|
CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. CGI-I is a 7-point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a Baseline state at the beginning of the intervention and rated as: 1 - very much improved; 2 - much improved; 3 - minimally improved; 4 - no change; 5 - minimally worse; 6 - much worse; or 7 - very much worse. |
CGI-S is collected at Screening, Visit 2, Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period and CGI-I will be assessed at Visit 3 and Visit 4/EOS/ET, over 14 week period
|
Anxiety, Depression and Mood Scale (ADAMS)
Time Frame: Screening, Visit 2, Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period
|
The ADAMS is comprised of 28 items, which are rated on a scale of "0 - not a problem" to "3 - severe problem."
The ADAMS yields a total score as well as five subscale scores: "Manic/Hyperactive Behavior," "Depressed Mood," "Social Avoidance," "General Anxiety," and "Compulsive Behavior."
|
Screening, Visit 2, Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period
|
Qualitative Caregiver Reported Behavioral Problems Survey
Time Frame: Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period
|
The parent/caregiver will be asked the following question "What are the three behavioral, emotional, or social problems that most impacted your son/daughter and his/her family in approximately the past year?"
At each study visit the parent/caregiver will be reminded of their responses from the Screening Visit in order to rate the three questions for improvement or worsening.
|
Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period
|
Pediatric Anxiety Rating Scale-Revised (PARS-R)
Time Frame: Screening, Weeks 14, 22, 30, and Week 38
|
The PARS-R is a clinician-rated caregiver interview that covers 61 behaviors related to anxiety.
The PARS-R provides broad coverage of separation anxiety, social phobia, and generalized anxiety.
Symptoms are further categorized into Social Interactions or Performance Situations, Separation, Generalized, Specific Phobia, Panic Symptoms/Physical Signs, Obsessive-Compulsive, Health/Illness Concerns, and Other.
|
Screening, Weeks 14, 22, 30, and Week 38
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Carol O'Neill, VP, Development
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Heart Diseases
- Cardiovascular Diseases
- Lymphatic Diseases
- Endocrine System Diseases
- Disease
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Parathyroid Diseases
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Craniofacial Abnormalities
- Musculoskeletal Abnormalities
- Abnormalities, Multiple
- Chromosome Disorders
- 22q11 Deletion Syndrome
- Lymphatic Abnormalities
- Hypoparathyroidism
- Syndrome
- DiGeorge Syndrome
- Anticonvulsants
- Cannabidiol
Other Study ID Numbers
- ZYN2-CL-031
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on 22Q Deletion Syndrome
-
Xiao-dong ZhuangSun Yat-sen UniversityUnknownTelomere Length, Mean Leukocyte | Telomere Shortening | 22q Telomere Deletion Syndrome
-
Cindy CisnerosCompleted
-
Washington University School of MedicineDickson, Patricia I., M.D.; Milbrandt, Jeffrey, MD, PhD; Mitra, Rob, PhD; Turner...RecruitingChromosome 9P Deletion Syndrome | 9p Minus Syndrome | Alfi Syndrome | 9P Monosomy | 9P Partial Monosomy SyndromeUnited States
-
Clinical Research Associates, LLCRecruiting16P11.2 Deletion SyndromeUnited States
-
Icahn School of Medicine at Mount SinaiNational Institute of Mental Health (NIMH)CompletedPhelan-McDermid Syndrome | 22q13 Deletion SyndromeUnited States
-
Centre Hospitalier Universitaire de NīmesCompleted22q11 Deletion Syndrome Di George SyndromeFrance
-
Simons SearchlightBoston Children's Hospital; Geisinger Clinic; Simons FoundationRecruitingSMARCA4 Gene Mutation | DDX3X | 16P11.2 Deletion Syndrome | 16p11.2 Duplications | 1Q21.1 Deletion | 1Q21.1 Microduplication Syndrome (Disorder) | ACTL6B | ADNP | AHDC1 | ANK2 | ANKRD11 | ARID1B | ASH1L | BCL11A | CHAMP1 | CHD2 | CHD8 | CSNK2A1 | CTBP1 | CTNNB1 Gene Mutation | CUL3 | DNMT3A | DSCAM | DYRK1A | FOXP1 | GRIN2A | GRIN2B | HIVEP2-Related Intellectual... and other conditionsUnited States
-
The Champ FoundationChildren's Hospital of Philadelphia; The Cleveland ClinicRecruitingPearson Syndrome | Single Large Scale Mitochondrial DNA Deletion Syndromes (SLSMDS)United States
-
Stephan EliezRecruitingtACS | 22Q11 Deletion SyndromeSwitzerland
-
UMC UtrechtChildren's Hospital of Philadelphia; Netherlands Brain FoundationUnknownChromosome 22q11.2 Deletion SyndromeUnited States, Netherlands
Clinical Trials on ZYN002
-
Zynerba Pharmaceuticals, Inc.Enrolling by invitationFragile X SyndromeUnited States, United Kingdom, New Zealand, Australia
-
Zynerba Pharmaceuticals, Inc.CompletedFragile X SyndromeUnited States, New Zealand, Australia
-
Zynerba Pharmaceuticals, Inc.RecruitingFragile X SyndromeUnited States, Australia, United Kingdom, Ireland