Q10 Ubiquinol in Autism Spectrum Disorder and in Phelan-McDermid Syndrome. (NCT04312152) | Clinical Trial Compass
UnknownNot Applicable
Q10 Ubiquinol in Autism Spectrum Disorder and in Phelan-McDermid Syndrome.
Italy200 participantsStarted 2019-03-09
Plain-language summary
This double-blind, cross-over, randomized, controlled trial (RCT) has the aim of evaluating the effectiveness of a metabolic support therapy in two cohorts of patients with idiopathic Autism Spectrum Disorder or Phelan-McDermid syndrome, commonly associated with syndromic autism. Each patient will receive Q10 ubiquinol + Vit. E and B for 4 months and only Vit. E and B for 4 months in a double-blind, cross-over design. Primary outcome measures of efficacy include Vineland Adaptive Behavior Scales, Childhood Autism Rating Scale, Clinical Global Impression-Improvement and Visual Analog Scales; secondary outcome measures include several questionnaires and tests of autism, cognitive function, problem behaviors, quality of life, communication and comorbid disorders, as well as measures of oxidative stress.
Who can participate
Age range
2 Years – 40 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Both parents or a legally authorized patient representative (LAR) must provide written informed consent. The parents and guardian must be able to understand and comply with the experimental protocol;
. Subjects of both sexes, aged between 2 and 40 years old, may be included in the study;
. The subject must meet DSM-5 criteria for a primary diagnosis of Autism Spectrum Disorder (idiopathic autism) or carry a documented deletion of human chromosome 22q13.33 or mutation in the SHANK3 gene (Phelan-McDermid Syndrome);
. Subjects with idiopathic autism must pass the threshold score for Autism of the Autism Diagnostic Observation Schedule;
. Baseline Children's Global Assessment Scale score must be between 45 and 59;
. Patients treated with psychoactive drugs (neuroleptics, antiepileptics, etc.) are enrolled only if the treatment and dosage of these drugs has been constant for at least 3 months prior to enrollment in the trial and is kept constant throughout the 8-month duration of the trial;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Vineland Adaptive Behavior Scales scores
Timeframe: At 0, 4 and 8 months (pre- and post-treatment after each arm)
2
Change in Childhood Autism Rating Scale score
Timeframe: At 0, 4 and 8 months (pre- and post-treatment after each arm)
3
Change in Clinical Global Impression of Improvement scale scores between experimental and active comparator arms.
Timeframe: 4 and 8 months (record once at the end of each arm)
4
Change in Visual Analog Scales scores
Timeframe: At 0, 4 and 8 months (pre- and post-treatment after each arm)
. Patients undergoing any kind of behavioral intervention must have must have started the intervention at least 3 months prior to enrollment and the intervention must remain unchanged throughout the 8-month duration of the trial;
. The patient is able to swallow the capsule or his/her parents are available to open it and administer immediately its content in a small quantity of juice or soft-drink.
Exclusion criteria
. Patients with autism secondary to known genetic syndromes other than Phelan-McDermid syndrome (for example, Rett syndrome, fragile-X syndrome, etc.);
. Presence of brain malformations or major structural anomalies visible by magnetic resonance imaging;
. Patients with autism secondary to epileptic encephalopathy or with idiopathic autism comorbid with seizures more frequent than one episode every 6 months despite ongoing antiepileptic drug therapy;
. Patients with autism accompanied by marked facial dysmorphism and/or congenital malformations;
. Patients treated with anticoagulants;
. Patients with serious medical illnesses (chronic renal disease, severe liver disease, cardiovascular disorders, uncontrolled hypertension with systolic pressure values\> 170 and diastolic pressure\> 100 mm Hg, malignant tumors, HIV infection);
. Patients with a history of acute cerebrovascular episodes;
. Patients with a history of stomach bleeding or active peptic ulcer;