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    Summary
    EudraCT Number:2018-000942-21
    Sponsor's Protocol Code Number:AIMS-2-CT1
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-06-12
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-000942-21
    A.3Full title of the trial
    A Phase II Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of Arbaclofen Administered for the Treatment of Social Function in Children and Adolescents with Autism Spectrum Disorders.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    STAR-AIMS: Does treatment with arbaclofen help improve social function in autism?
    A.3.2Name or abbreviated title of the trial where available
    Does treatment with arbaclofen help improve social function in autism?
    A.4.1Sponsor's protocol code numberAIMS-2-CT1
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03682978
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelso Arango
    B.1.3.4CountrySpain
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportEU IMI programme
    B.4.2CountryEuropean Union
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInstitute of Psychiatry, Psychology and Neuroscience, King's College London
    B.5.2Functional name of contact pointProfessor Andre Strydom
    B.5.3 Address:
    B.5.3.1Street AddressIoPPN, 16 De Crespigny Park
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE5 8AF
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+447894551353
    B.5.6E-mailandre.strydom@kcl.ac.uk
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameArbaclofen
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNR-Baclofen
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5 to 20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Autism Spectrum Disorder
    E.1.1.1Medical condition in easily understood language
    Autism
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003805
    E.1.2Term Autism
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003805
    E.1.2Term Autism
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10063844
    E.1.2Term Autism spectrum disorder
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The overall goal of this project is to improve patient outcomes. For this study the primary objective is to examine the effect of Arbaclofen compared to placebo on social function in children and adolescents (age 5 - 17) with Autism spectrum disorders.
    E.2.2Secondary objectives of the trial
    Secondary research objectives are:
    -To examine the effect of Arbaclofen vs. placebo on measures of global (general) function
    -To explore the effect of Arbaclofen vs. placebo on specific daily living skills, including communication, and motor skills
    -To explore the effect of Arbaclofen vs. placebo on social communication.
    -To explore the effect of Arbaclofen on other measures of core autism symptoms.
    -To explore the effect of Arbaclofen on associated behavioural problems
    -To examine the safety and tolerability of Arbaclofen in children and adolescents with ASD

    - In addition, the study will explore whether measures of electrophysiology (EEG) and sensory discrimination are associated with treatment response
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.1) Signed Written Informed Consent

    Participants or their legal representative (LAR) must have signed and dated an approved written informed consent form in accordance with regulatory and institutional guidelines. This must be obtained before the performance of any protocol related procedures that are not part of normal participant care. Participants who do not have the capacity to consent will give developmentally appropriate assent.

    Participants must be willing and able to comply with scheduled visits, treatment schedule, and laboratory testing.

    The participant’s parent/caregiver/LAR must be able to speak and understand the local language where the study is conducted sufficiently to understand the nature of the study and to allow for the completion of all study assessments. The same parent/caregiver/LAR must be capable of providing reliable information about the subject’s condition, agree to oversee the administration of study drug, and accompany the subject to all clinic visits.

    Participants must be able to speak and understand the local language where the study is conducted sufficiently to understand the nature of the study and to allow for the completion of all study assessments.

    2) Type of Participant and Target Disease Characteristics

    Diagnosis of an Autism Spectrum Disorder according to the DSM-5 criteria

    Complex language (i.e. fluent language abilities) as defined in ADOS-2 to qualify for a Module 3 or 4, which would exclude individuals with significant cognitive impairments.

    Current pharmacological treatment regimen affecting behaviour has been stable for at least 6 weeks prior to screening and is expected to be stable during the duration of the study

    Current psychotherapeutic/psychosocial interventions affecting behaviour stable for 3 months prior to screening and are expected to be stable during the duration of the study (standard regular school breaks and/or annual teacher/classroom change do not qualify for intervention change).

    Participants with a history of seizure disorder must currently be receiving stable treatment with anticonvulsant medication and must have been seizure free for 6 months prior to screening, or must be seizure free for 3 years prior to screening if not currently on a stable (>3 months) dose of antiepileptics.

    3) Age, Residential and Reproductive Status

    Male or female participants 5 to 17 years of age at the time of providing consent, inclusive.

    Reside with the parent/carer who is interviewed for the Vineland.

    Negative pregnancy test for females of childbearing potential (subject has experienced onset of menses) within 24h prior to study treatment onset.

    Females of childbearing potential who are sexually active must agree to use an accepted form of contraception (i.e., existing surgical sterilization, abstinence or or a combination of two effective forms of contraception, such as for example, condoms plus hormonal treatment).

    Male participants with female partners of childbearing potential are eligible to participate if they agree to the conditions stated in Appendix 4 of the protocol.
    E.4Principal exclusion criteria
    1) Medical Conditions

    Subjects with any condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. This includes, but is not limited to impairment of renal function, evidence or history of malignancy or any significant haematological, endocrine, respiratory, hepatic, cardiovascular or gastrointestinal disease, including any clinically significant abnormalities on ECG. In general, any co-morbid conditions that may interact with study procedures.

    2) Prior/Concomitant Therapy

    Subjects who are currently receiving treatment with racemic baclofen, vigabatrin, tiagabine, gabapentin, pregabalin or riluzole or other GABA-related medications. Only regular benzodiazepine use (PM, i.e. at night) will be allowed.

    Subjects who are currently receiving pharmacologic treatment affecting behaviour (see concomitant medication section) need to have a stable dose during the 6 weeks prior to the screening visit and for the duration of the study.

    Participating in programs including non-pharmacologic educational, behavioural, and/or dietary interventions affecting behaviour, participation in these programs must have been continuous during the 3 months prior to screening and participants or their parent/caregiver/LAR may not electively initiate new or modify ongoing interventions for the duration of the study. Typical school vacations are not considered modifications of stable programming.

    Potential participants who have taken another investigational drug within the last 30 days.


    3) Physical and Laboratory Test Findings

    Patients with evidence of any significant hematological, endocrine, cardiovascular (including uncorrected symptomatic congenital heart disease), respiratory, renal, hepatic, or gastrointestinal disease, not including mild common paediatric diseases in these areas that are stable (e.g. mild asthma, constipation, etc.), as judged by the investigator.



    4) Study-medication related

    Subjects who are not able to take oral medications.

    Subjects who have a history of hypersensitivity to racemic baclofen.

    Subjects with rare hereditary problems of galactose intolerance, lactase deficiency or glucose-galactose malabsorption should not take this medicine.

    Active peptic ulceration as Baclofen stimulates gastric acid secretion.

    Porphyria.

    5) Other Exclusion Criteria

    Subjects who are currently engaged in illicit drug use or alcohol abuse, according to DSM-5 criteria.

    Subjects who have previously participated in a clinical trial of Arbaclofen.

    Women who are breastfeeding.
    E.5 End points
    E.5.1Primary end point(s)
    Socialization domain of the Vineland-3 (Vineland Adaptive Behavior Scales, Third Edition, clinician interview).
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 0 (baseline), and repeated at week 16 (endpoint)
    E.5.2Secondary end point(s)
    Measures of Global Function:
    - Clinician’s Global Impression – Improvement (CGI-I; Busner and Targum 2007).
    - Clinician’s Global Impression – Severity (CGI-S; Busner and Targum 2007).

    Adaptive domains (other) - communication, daily living skills and motor skills:
    - Vineland-3 (Sparrow, Cicchetti et al. 2016)

    Social Communication behaviours:
    - The BOSCC - Brief Observation of Social Communication Change (BOSCC) (Kim et al 2018)
    - Social Responsiveness Scale (SRS-2, Second Edition, WPS, Constantino and Gruber 2012).

    Associated Problem Behaviours:
    - Aberrant Behaviour Checklist - Community Version (ABC-C)-(Aman and Singh 1994) - Child Behaviour Checklist (Achenbach and Rescorla 2001)

    Core Symptoms of Autism (other):
    - Autism Impact Measure (AIM) (Kanne et al 2014).

    Safety and Tolerability:
    - Safety Monitoring Uniform Report Form (SMURF) (Greenhill et al, 2004)
    - Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) (Janssen, Phillipson et al. 2017)
    - Suicidality assessment Columbia Suicide Severity Rating Scale (C-SSRS) (Posner, Brown et al. 2011).
    - Vital signs (pulse, blood pressure).
    - Height and weight.
    - Safety blood work and urine test.

    Exploratory:
    We will include EEG measures to obtain pilot evidence for predictive biomarker(s) in future trials of GABA modulating agents.
    E.5.2.1Timepoint(s) of evaluation of this end point
    For clinical outcomes, except CGI-I and CGI-S: week 0 (baseline) and week 16 (endpoint)

    For CGI-I and CGI-S, and safety: every 2 weeks from week 0.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of the trial is the last visit of the last subject.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days31
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days31
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 60
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.6.1Details of subjects incapable of giving consent
    Children and adolescents
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state58
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 130
    F.4.2.2In the whole clinical trial 130
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    At present, the study drug is unlicensed for the indication (social impairments in individuals with ASD). As such, it would not be possible to offer the drug to participants following the conclusion of the trial. We have informed participants of this in the information sheets.

    Should the drug trial show that the medication has positive effects, and if it is licensed, individuals who have participated in the trial will potentially have access to the drug through routine care.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-07-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-28
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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