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 range2 Years – 40 Years
SexALL
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;
✓. 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;
✓
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)
. 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;