Clinical trial • Phase IV • Psychiatry

ARIPIPRAZOLE for Autism spectrum disorder

Phase IV trial of ARIPIPRAZOLE for Autism spectrum disorder.

Overview

Trial Therapeutic Area
Psychiatry
Trial Disease
Autism spectrum disorder
Trial Stage
Phase IV
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
20-06-2024
First CTIS Authorization Date
16-07-2024

Trial design

Randomised, open-label, two-arm randomized comparison: therapeutic drug monitoring (tdm) arm — trough plasma levels measured (dbs) at weeks 4 and 10 with pk/pd model-based dosing advice provided to the treating psychiatrist (insightrx®); care as usual (cau) arm — no dosing recommendations. drugs used in participants are risperidone or aripiprazole per standard clinical care (dosing per local practice).-controlled Phase IV trial across 13 sites in Netherlands, Germany.

Randomised
Yes
Open Label
Yes
Comparator
Two-arm randomized comparison: Therapeutic drug monitoring (TDM) arm — trough plasma levels measured (DBS) at weeks 4 and 10 with PK/PD model-based dosing advice provided to the treating psychiatrist (InsightRX®); Care As Usual (CAU) arm — no dosing recommendations. Drugs used in participants are risperidone or aripiprazole per standard clinical care (dosing per local practice).
Real World Control
Yes
Target Sample Size
280
Trial Duration For Participant
365

Eligibility

Recruits 280 paediatric patients.

Pregnancy Exclusion
Pregnancy
Vulnerable Population
Vulnerable population selected: children and adolescents (age 6–18). Subject information sheets and informed consent forms are provided for age groups 6-11 years, 12-15 years and 16-18 years, and a parent information/consent form is provided; minors require parental consent and age-appropriate participant information/assent as indicated by the age-specific ICF documents.

Inclusion criteria

  • {"criterion_text":"-  Age 6 to 18 years"}
  • {"criterion_text":"-  Documented clinical diagnosis of autism spectrum disorder according to DSM IV or DSM V and comorbid behavioural problems"}
  • {"criterion_text":"-  To start treatment with risperidone or aripiprazole"}

Exclusion criteria

  • {"criterion_text":"-  Diabetes type I or II"}
  • {"criterion_text":"-  Congenital or acquired syndrome associated with changes in appetite, body weight or lipid profile (e.g. Prader Willi)"}
  • {"criterion_text":"-  Known history of Long QT syndrome (LQTS), cardiovascular disorders (myocardial infarction or ischaemic heart disease, heart failure, or conduction abnormalities), seizure disorder or oesophageal dysmotility"}
  • {"criterion_text":"-  Pregnancy"}
  • {"criterion_text":"-  Hypersensitivity to the active substance or to any of the excipients"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Difference between the groups in change in body mass index z-scores (BMI-Z) after 6 months of treatment","definition_or_measurement_approach":"Change in BMI z-scores (BMI-Z) measured after 6 months of treatment comparing the two randomized groups."}

Secondary endpoints

  • {"endpoint_text":"- Difference between the groups in change in body mass index z-scores (BMI-Z) after 12 months of treatment.","definition_or_measurement_approach":"Change in BMI z-scores (BMI-Z) measured after 12 months of treatment comparing the two groups."}
  • {"endpoint_text":"- Aberrant Behavior Checklist, a parental symptom checklist for assessing problem behaviour in children. The gold standard for measuring the effect of treatment on aggression and irritability in children with autism spectrum disorder","definition_or_measurement_approach":"Aberrant Behavior Checklist (parent-rated) to assess severity of irritability and aggression; used to compare drug effectiveness between groups."}
  • {"endpoint_text":"- EuroQol-5D Youth. A descriptive system for health-related quality of life states in five dimensions, plus a visual analogue scale.","definition_or_measurement_approach":"EQ-5D-Y descriptive system and VAS to assess health-related quality of life in children."}
  • {"endpoint_text":"- Pediatric Quality of Life Inventory. A modular instrument aimed at measuring healthrelated quality of life in children and adolescents. It showed excellent reliability and validity in individuals with autism spectrum disorder","definition_or_measurement_approach":"PedsQL instruments to measure health-related quality of life in children and adolescents."}
  • {"endpoint_text":"- Clinical Global Improvement","definition_or_measurement_approach":"Clinician-rated global improvement scale to assess overall clinical change."}
  • {"endpoint_text":"- Treatment Inventory of Costs in Patients with psychiatric disorders. A modular questionnaire aimed at mapping healthcare utilization and the repercussions of psychosocial problems, allowing for the assessment of associated costs.","definition_or_measurement_approach":"TIC-P questionnaire to capture healthcare utilization and associated costs."}
  • {"endpoint_text":"- Levels of glucose, cholesterol, lipoproteins and triglycerides; the hormones ghrelin, prolactin and leptin.","definition_or_measurement_approach":"Laboratory measurements of metabolic parameters (glucose, cholesterol, lipoproteins, triglycerides) and hormone levels (ghrelin, prolactin, leptin) to compare safety profiles between groups."}
  • {"endpoint_text":"- Abnormal Involuntary Movement Scale, a clinician administered observational scale aimed at detecting extrapyramidal side effects.","definition_or_measurement_approach":"AIMS clinician-administered observational scale to detect extrapyramidal side effects."}
  • {"endpoint_text":"- Blood pressure","definition_or_measurement_approach":"Measurement of blood pressure as a safety parameter."}

Recruitment

Planned Sample Size
280
Recruitment Window Months
48
Consent Approach
Age-specific subject information and informed consent forms are provided: ICFs/SIS for 6-11 years, 12-15 years, 16-18 years, and a parent information/consent form. Minors require parental consent and age-appropriate participant information/assent as per the provided ICF documents.

Geography

Total Number Of Sites
13
Total Number Of Participants
280

Netherlands

Earliest CTIS Part Ii Submission Date
10-07-2024
Latest Decision Or Authorization Date
16-07-2024
Processing Time Days
6
Number Of Sites
10
Number Of Participants
250

Sites

Site Name
Mondriaan
Department Name
KJPP
Contact Person Name
T. van Amelsvoort
Contact Person Email
info@mondriaan.eu
Site Name
Accare
Department Name
KJPP
Contact Person Name
P. Hoekstra
Contact Person Email
SPACe@erasmusmc.nl
Site Name
GGz Breburg
Department Name
KJPP
Contact Person Name
C. van Looy
Contact Person Email
info@ggzbreburg.nl
Site Name
Erasmus MC
Department Name
KJPP
Principal Investigator Name
B. Dierckx
Principal Investigator Email
b.dierckx@erasmusmc.nl
Contact Person Name
B. Dierckx
Contact Person Email
SPACe@erasmusmc.nl
Site Name
GGZ Delfland
Department Name
KJPP
Contact Person Name
D. van Altena
Contact Person Email
SPACe@erasmusmc.nl
Site Name
Karakter
Department Name
KJPP
Contact Person Name
G. van de Loo
Contact Person Email
SPACe@erasmusmc.nl
Site Name
LUMC Curium
Department Name
KJPP
Contact Person Name
R. Vermeieren
Contact Person Email
SPACe@erasmusmc.nl
Site Name
's Heeren Loo
Department Name
KJPP
Contact Person Name
A. van Eeghen
Contact Person Email
SPACe@erasmusmc.nl
Site Name
Yulius GGZ
Department Name
KJPP
Contact Person Name
D. Bastiaansen
Contact Person Email
SPACe@erasmusmc.nl
Site Name
Accare (additional / duplicate listing as per CTIS site list)
Department Name
KJPP

Germany

Earliest CTIS Part Ii Submission Date
10-07-2024
Latest Decision Or Authorization Date
31-07-2024
Processing Time Days
21
Number Of Sites
3
Number Of Participants
30

Sites

Site Name
Wurzburg University Hospital
Department Name
Kinder- und Jugendpsychiatrie
Principal Investigator Name
Marcel Romanos
Principal Investigator Email
romanos_M@ukw.de
Contact Person Name
Marcel Romanos
Contact Person Email
romanos_M@ukw.de
Site Name
KJP Mariaberg
Department Name
Kinder- und Jugendpsychiatrie
Principal Investigator Name
Martin Menzel
Principal Investigator Email
m.menzel@mariaberg.de
Contact Person Name
Martin Menzel
Contact Person Email
m.menzel@mariaberg.de
Site Name
Tübingen University Hospital
Department Name
Kinder- und Jugendpsychiatrie
Principal Investigator Name
Gottfried Barth
Principal Investigator Email
gottfried.barth@med.uni-tuebingen.de
Contact Person Name
Gottfried Barth

Sponsor

Primary sponsor

Full Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Netherlands

Third parties

  • {"country":"","full_name":"ZonMw","duties_or_roles":"Funding","organisation_type":""}
  • {"country":"","full_name":"Stichting de Merel","duties_or_roles":"Funding","organisation_type":""}
  • {"country":"","full_name":"Erasmus MC","duties_or_roles":"Funding","organisation_type":""}

Investigational products

Investigational Product Name
ARIPIPRAZOLE
Active Substance
ARIPIPRAZOLE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
marketingAuthNumber: '-' ; prodAuthStatus: 2
Maximum Dose
10 mg (maxDailyDoseAmount 10 mg)
Investigational Product Name
RISPERIDONE
Active Substance
RISPERIDONE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
marketingAuthNumber: '-' ; prodAuthStatus: 2
Maximum Dose
0.08 mg/kg (maxDailyDoseAmount 0.08 mg/kg)

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