Exploratory study of pitolisant in children and adolescents with Autism Spectrum Disorders
Research type
Research Study
Full title
An Exploratory, Multicenter, Randomized, double-blind, placebo-controlled study evaluating the effect and safety of pitolisant in children and adolescents with Autism Spectrum Disorders.
IRAS ID
1007904
Contact name
Pascale Vernade
Contact email
Sponsor organisation
Bioprojet Pharma
Eudract number
2023-503678-18
Clinicaltrials.gov Identifier
Research summary
Research Summary
Rationale: Autism refers to a range of conditions that are characterised by challenges with social skills, speech and nonverbal communication, and repetitive and restrictive behaviours. Children and adolescent living with autism experience a quality-of-life decline, especially regarding their social and emotional functioning.
Despite autism being considered a common neurological condition, there has been few pharmacological advancements in the field. Pitolisant represents a potential option for the treatment of autism social features of autistic subjects as observed in autistic animal model experiments.
The aim of the present clinical study is to evaluate the effect of pitolisant on the social and communication impairment of autistic male children and adolescents.
The main objective of this study is to evaluate the impact of pitolisant on the autistic social communication and interaction features as measured by the Social Responsiveness Scale Second Edition (SRS-2) total score.
Trial design
This is a Phase II exploratory study. Treatment (either pitolisant or placebo) will be assigned like flipping a coin by a computer 1 to 1, and neither the patient nor the trial doctor will be aware of the treatment assigned to the participant (blinding). Subjects are participating for 15 weeks including 2 weeks of screening period, 12 weeks of treatment period and 1 week of follow-up after the end of treatment. A total of 5 visits and 2 phone calls are planned for each participant during the study. Unscheduled visit by phone call or at site may be performed at any time during the study for safety reason, in case of undesirable event for doctor decision on the treatment.
Trial population:
Autistic male children and adolescent aged between 6 and 17, with a moderate social impairment, no learning disability, normal cardiac and hepatic function and no features of severe depression or epilepsy are eligible to this clinical study.Summary of Results
"Who took part in this study?
Boys and girls aged 6 to 17 years with ASD took part in the study.
Children and teenagers could join if:
• They had a confirmed diagnosis of ASD.
• They did not have intellectual difficulties
• They could take part in the study tests, with help from a parent if needed.
• Their usual therapies for ASD symptoms had not changed before joining the study.
They could not join if they:
• Had certain serious medical or mental health conditions.
• Had heart, liver, or kidney problems that could make the study unsafe.
• Had recently taken therapies that could modify how pitolisant works.
In total, 63 children were assigned by chance to receive pitolisant or placebo. Most were boys.What happened during the study?
This was a double-blind, randomized study. Children were assigned by chance to receive pitolisant or placebo. Families and study doctors did not know which treatment each participant received.
Each participant took part for about 17 weeks. This included:
• A check-up to see if the child could join the study.
• Then 12 weeks of treatment, with one tablet each morning of either pitolisant or placebo.
• After that, there was a short follow-up period.
The dose of pitolisant depended on the child’s weight and how they tolerated the study drug.
During the study, children:
• Had their parents complete questionnaires about their social behavior, daily life skills, sleep, and attention.
• Had regular visits with study doctors.
Were checked for any health problems or side effects.What were the side effects ?
Side effects are unwanted health problems that can happen during a study. Study doctors checked whether these problems might be linked to the test drug.
In this study:
• 4 out of 10 children had side effects in both the pitolisant and placebo groups.
• Most side effects were mild or moderate.
• The most common side effects were sleep problems, irritability, insomnia, headache, and vomiting.
Side Effect Pitolisant Group Placebo Group
Sleep problems 4 of 30 children (13.3%) 3 of 33 children (9.1%)
Irritability 1 of 30 children (3.3%) 4 of 33 children (12.1%)
Insomnia 2 of 30 children (6.7%) 3 of 33 children (9.1%)
Headache 3 of 30 children (10.0%) 1 of 33 children (3.0%)
Vomiting 2 of 30 children (6.7%) 1 of 33 children (3.0%)
Serious side effects are those that cause death, threaten life or mean the person has to go to the hospital. No serious side effects occurred.
A small number of children stopped treatment early because of side effects. This happened only in the placebo group.What were the main results of the study ?
The main question was to see if pitolisant could help children and teenagers with ASD communicate and interact better.
To answer this question, the parents completed a questionnaire called the Social Responsiveness Scale (SRS-2). Lower scores mean fewer symptoms. The study was completed as planned.
After 12 weeks of treatment:
• 57 of 63 children (90%) completed the study
• Both the pitolisant and placebo groups showed some improvement.
The improvement with pitolisant was smaller than with placebo.How was this study useful?
Most children tolerated pitolisant well with no major problems. However, it did not improve the main autism symptoms more than placebo.
This summary shows the results of one study only. Usually, several studies are needed to fully understand how well a drug works and how safe it is.
Based on these results, pitolisant will not be studied further as a treatment for ASD.What does this mean for families?
The results suggest that pitolisant does not help the main symptoms of autism more than placebo. This means pitolisant is unlikely to help children and teenagers with ASD communicate or interact better.
Families are encouraged to:
• Talk with their child’s doctor about any questions on therapies.
Continue using therapies and support that are already recommended for ASD.Are there plans for further studies?
Based on the results of this study, no more studies of pitolisant in ASD are planned.Where can I find more information about this study?
More information is available on public clinical trial websites:
• EU Clinical Trials Information System: https://euclinicaltrials.eu (search for study number 2023-503678-18-00)
• ClinicalTrials.gov (search for NCT05953389)
The full title of the study is: An Exploratory, Multicenter, Randomized, Double-blind, Placebo-Controlled Study Evaluating the Effect and Safety of Pitolisant in Children and Adolescents with Autism Spectrum Disorders"REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
23/WM/0149
Date of REC Opinion
12 Sep 2023
REC opinion
Further Information Favourable Opinion