CBDV Compound for Royer Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY
Royer Syndrome+1 More
CBDV Compound - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether cannabidivarin may have positive impacts for individuals with Prader-Willi syndrome.

See full description

Eligible Conditions

  • Royer Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Royer Syndrome

Study Objectives

This trial is evaluating whether CBDV Compound will improve 1 primary outcome and 8 secondary outcomes in patients with Royer Syndrome. Measurement will happen over the course of from Baseline to Week 12.

Week 12
Aberrant Behavior Checklist (ABC) subscales in lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech
Aberrant Behavior Checklist-Irritability Subscale (ABC-I)
ActiGraph GT9X-BT activity monitors
Caregiver Strain Questionnaire (CSQ)
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Clinical Global Impression Scale - Improvement (CGI-I)
Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
Montefiore Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS)
Repetative Behavior Scale- Revised (RBS-R)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Royer Syndrome

Trial Design

2 Treatment Groups

Cannabidivarin (CBDV)
1 of 2
Matched Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 26 total participants across 2 different treatment groups

This trial involves 2 different treatments. CBDV Compound is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Cannabidivarin (CBDV)
Drug
Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks
Matched Placebo
Drug
Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from baseline to week 12
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly from baseline to week 12 for reporting.

Who is running the study

Principal Investigator
E. H.
Eric Hollander, Director, Autism and Obsessive Compulsive Spectrum Program, Anxiety and Depression Program
Montefiore Medical Center

Closest Location

Montefiore Medical Center, Albert Einstein College of Medicine - Bronx, NY

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Male or Female outpatients aged 5 to 30 years.
Diagnosis of PWS confirmed by genetic testing and patient medical records and history.
Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the patient's development and behavior throughout the study. Child Assent will be obtained if the subject is 7 years of age or older and has the mental capacity to understand and sign a written assent form and/or give verbal assent.
Score on the Clinical Global Impression Scale Severity (CGI-S) ≥ 4 (moderate severity) at baseline.
Score of ≥18 on the Aberrant Behavior Checklist-Irritability (ABC-I) at baseline.
Agree not to drive or operate machinery.
Have a physical exam and laboratory results that are within the norms for PWS

Patient Q&A Section

What causes royer syndrome?

"It is rare but important to be aware of royer syndrome when diagnosing a baby with severe neonatal jaundice. The most important aspect of management of the fetal liver is to control the bilirubin levels." - Anonymous Online Contributor

Unverified Answer

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

"There is only one case occurring per every 2,000,000 live births in the United States each year. This low incidence is also due to the rarity of the condition. It is also important that the disorder be diagnosed early to prevent further complications such as congenital cleft palate and the heart's right coronary artery being compressed and blocked by the aorta." - Anonymous Online Contributor

Unverified Answer

What are common treatments for royer syndrome?

"The only treatment for RoS may be genetic counseling. Additionally, the only treatment for RoS may be symptomatic management, unless associated with other syndromes. Data from a recent study, treatment included intravenous immunoglobulin administration." - Anonymous Online Contributor

Unverified Answer

Can royer syndrome be cured?

"In a recent study, findings support the hypothesis that RRM2 deficiency does not lead to an acute RS-like phenotypic phenotype in most individuals. RRM2 remains a key player and is sufficient for maintaining cellular viability in a large portion of RRM2 -mutated patients. However, we can propose that RRM2 deficiency is not a sufficient cause for the clinical features of RRM2-associated disease. A careful, but often impossible, diagnosis approach based on clinical and laboratory parameters can identify RRM2 deficiency as the only underlying cause when this diagnosis is needed to implement a correct therapy. This could be one of the first breakthroughs for RDR, helping the medical community understand RS in a more accurate and comprehensive manner." - Anonymous Online Contributor

Unverified Answer

What are the signs of royer syndrome?

"The signs of RWS are generally mild and nonspecific, and tend to become evident at diagnosis. These signs may include growth retardation, short stature, delayed pubertal growth, cleft palate, small teeth, bony dysmorphic changes, polydactyly, clinodactyly or syndactyly, and other cutaneous malformations. The skeletal deformities tend to be less severe and less frequent than in other associated conditions. RWS is inherited almost exclusively in an X-linked recessive fashion, with a 95% confidence level of carrier probability. The penetrance of RWS seems to be extremely homogenous, both within families and in men from different ethnic groups." - Anonymous Online Contributor

Unverified Answer

What is royer syndrome?

"Although royer syndrome was first described in 1958, its significance was not recognized until 1989 following the publication of five new descriptions of royer syndrome. In recent decades royer syndrome has become more commonly recognised with the development of treatment strategies and a growing awareness of the physical, developmental and psychosocial disabilities. A high degree of awareness of the syndrome is needed by health professionals, parents and patients for early detection, appropriate and timely referral and to provide adequate support and information so that affected families and individuals can make a successful transition into adulthood. The treatment strategy described can be considered in these patients with royer syndrome and should be part of their routine clinical care." - Anonymous Online Contributor

Unverified Answer

Has cbdv compound proven to be more effective than a placebo?

"We did not see a significant and clinically observable difference between CBD (1st generation, CBD, 1 g) and A placebo in controlling the pain of RSD. However, the patient-reported average pain/activity score fell by 35% in the CBD group (n=27) compared to a placebo group (n=23, p=0.013). The number of patients achieving improvement or remission was higher and the number of patients receiving adjuvant pain-relievers (opioids) fell by approximately one third." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for royer syndrome?

"Ryer Syndrome should be considered in clinical trials if there are criteria for inclusion by a panel of specialists; there are many questions that still need to be elucidated. There are 3 sub-syndromes - Roberts Syndrome, Roberts-Liss Syndrome and ROYER Syndrome. The current study suggests that royer syndrome should also be included in clinical trials if a patient has Roberts Syndrome, Roberts-Liss Syndrome and Roer-Liss Syndrome and the patient is symptomatic." - Anonymous Online Contributor

Unverified Answer

What is the latest research for royer syndrome?

"Current research for royer syndrome has provided a significant amount of basic knowledge to help diagnose royer syndrome in its various forms. In addition to this, the advancements can also help the treatment of royer syndrome. Research is still being conducted to understand royer syndrome’s development and progression more fully. [power(http://www.withpower.com/d/royers-research)]; and it involves a genetic component and has inherited pattern, and can cause a spectrum of symptoms and severity. There is also a new link for royer syndrome to be included in the [myasthenia gravis spectrum disorder]." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets royer syndrome?

"The mean age of presentation with Roer Syndrome in a single-site referral base from the Australian And New Zealand Gastroenterology Association is 60 years and 6 months. Because there is no specific test for predicting the risk of developing Roer's syndrome after surgery for colorectal cancer (in a referral base with a relatively high proportion of Crohn's and colitis patients), Roer's syndrome is rarely suspected. However, the diagnosis is relatively common compared to the high level of mortality and morbidity in these groups." - Anonymous Online Contributor

Unverified Answer

Is cbdv compound safe for people?

"There is no safety issues associated with this compound. Clinically, it does not act on the dopamine system, nor on the serotonin or histamine system, therefore it does not exert side effects common to these classes of drugs. As the compound did not alter the normal pharmacokinetics of CBD when given in combination with cannabis extract, it is unlikely to have altered the pharmacokinetics of CBD. It is highly recommended that clinicians should be aware of the potential for adverse effects of CBDP when using it with cannabis. The combination of cocaine + CBDV caused more hypotension than cocaine alone." - Anonymous Online Contributor

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