Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44132   clinical trials with a EudraCT protocol, of which   7324   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2021-003791-13
    Sponsor's Protocol Code Number:BP42992
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2023-01-16
    Trial results View 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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-003791-13
    A.3Full title of the trial
    A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN PARTICIPANTS AGED 2 TO 14 YEARS OLD WITH DUP15Q SYNDROME FOLLOWED BY A 2-YEAR OPTIONAL OPEN-LABEL EXTENSION.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of 52 weeks of Treatment with Basmisanil in Participants Aged 2 to 14 Years Old with Dup15q Syndrome Followed by a 2-Year Optional Open-Label Extension.
    A.4.1Sponsor's protocol code numberBP42992
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05307679
    A.5.4Other Identifiers
    Name:EU Trial NumberNumber: 2022-502165-20-00
    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 SponsorF. Hoffmann La-Roche Ltd
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportF.Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.com
    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 namebasmisanil
    D.3.2Product code Ro 518-6582/F13-01
    D.3.4Pharmaceutical form Granules in sachet
    D.3.4.1Specific paediatric formulation Yes
    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 INNbasmisanil
    D.3.9.1CAS number 1159600-41-5
    D.3.9.2Current sponsor codeRO5186582
    D.3.9.3Other descriptive nameGABAA a5 negative allosteric modulator, previously referred to as GABAA a5 inverse agonist
    D.3.9.4EV Substance CodeSUB31464
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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.IMP: 2
    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 namebasmisanil
    D.3.2Product code Ro 518-6582/F15-01
    D.3.4Pharmaceutical form Granules in sachet
    D.3.4.1Specific paediatric formulation Yes
    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 INNbasmisanil
    D.3.9.1CAS number 1159600-41-5
    D.3.9.2Current sponsor codeRO5186582
    D.3.9.3Other descriptive nameGABAA a5 negative allosteric modulator, previously referred to as GABAA a5 inverse agonist
    D.3.9.4EV Substance CodeSUB31464
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    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 placeboGranules in sachet
    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
    Dup15q Syndrome
    E.1.1.1Medical condition in easily understood language
    Dup15q Syndrome is a neurodevelopmental disorder, caused by the partial duplication of Chromosome 15, that confers a strong risk for autism spectrum disorder, epilepsy, and intellectual disability.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level LLT
    E.1.2Classification code 10083952
    E.1.2Term Dup15q syndrome
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effects of 52 weeks of treatment with basmisanil on core symptom domains of Dup15q syndrome (language and social skills) and daily functioning (Part 1).
    E.2.2Secondary objectives of the trial
    • To evaluate the effects of 52 weeks of treatment with basmisanil treatment on Motor function, Cognition, Language, Social skills, Clinical global impression of severity and change, Challenging behaviors (Part 1)
    • To evaluate the tolerability and safety of 52 weeks of treatment with basmisanil (Part 1)
    • To characterize the PK of basmisanil and its metabolite M1 and determine the dose that will deliver target exposure in study participants (Part 1)
    • To evaluate potential relationships between selected covariates and exposure to basmisanil (Part 1)
    • To evaluate the effects of basmisanil treatment on the characteristic Dup15q EEG phenotype acutely and at steady state (Part 1).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Participants aged 2 to 14 years inclusive at the time the caregiver signs the informed consent
    • Documented maternal duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2-q13.1 region that includes the Prader-Willi/Angelman critical region defined as [BP2-BP3] segment
    • Dup15q syndrome Clinician Global Impression of Severity scale (Dup15q CGI-S) overall severity score ≥ 4 (at least moderately ill)
    • Stage 1 specific inclusion criterion: Participants aged 6 to 14 years with epilepsy
    • Body weight equal to or above the third percentile for age
    • Male and female participants: Some of the provisions that follow may have limited applicability based on the age range of study participants (i.e., up to the age of 14) and the nature of the disease under study
    • Female Participants: A female participant is eligible to participate if she is not pregnant, not breastfeeding
    • Male Participants: Male contraception is not required in this study because of the minimal seminal dose transmitted through sexual intercourse
    • The participant has a parent, caregiver, or legally authorized representative (hereinafter “caregiver”) of at least 18 years of age, who is fluent in the local language at the site, and capable and willing to provide written informed consent for the participant according to International Council for Harmonisation and local regulations
    • The participant’s caregiver must be living with the participant and, in the opinion of the Investigator, able and willing to reliably assess the participant’s ongoing condition, to accompany the participant to all clinic visits, and ensure compliance to study treatment throughout the study. The same caregiver is able and willing to complete the caregiver assessments and is available to the Investigational Site by telephone or email if needed
    • The participant’s caregiver is able and willing to use electronic devices to record information on the participant’s condition and to complete assessments at home and agrees to home nursing visits, if local regulations allow for it and if home nursing service is available in the country/region.
    E.4Principal exclusion criteria
    • Uncontrolled epilepsy at screening as indicated by: Use of rescue medication(s) to treat more than one seizure episode or seizure cluster per month on average in the past 6 months or concomitant chronic use of more than 4 anti-epileptic medications or status epilepticus within the past 6 months requiring hospitalization for treatment of the status epilepticus, or any implanted devices to treat drug-resistant epilepsy
    • Lymphoma, leukemia, or any malignancy within the past 5 years, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years
    • Clinically significant ECG abnormalities at Screening, including an average triplicate QTcF > 450 ms for participants > 10 years or QTcB > 450 ms for children up to and including age 10 years
    • Clinically significant abnormalities in laboratory test results at screening (including positive results for HIV, hepatitis B and/or hepatitis C). ALT values > 1.5 × the upper limit of normal. GFR < 90 mL/min per 1.73 m2 (Grade 1 CKD) as estimated using Schwarz formula.
    • Allowed prior existing medication should be on a stable regimen (or frequency of intervention) for at least 6 weeks, and at least 8 weeks for anti-epileptic treatment, prior to screening
    • Non-pharmacological / behavioral therapies should not be stopped or newly started at least 6 weeks prior to screening and are expected to remain stable for the entire study duration (excluding changes related to standard age and educational interventional programs and minor interruptions such as illness or vacation
    • Concomitant use of prohibited medications
    • Participation in an investigational drug study within one month or within 6 × the elimination half-life, whichever is longer, prior to dosing in the study
    • Significant risk for suicidal behavior, as assessed through the suicidal behavior question adapted from the Columbia Classification Algorithm for Suicide Assessment (C-CASA) (participants ≥ 6 years of age only)
    • Known sensitivity to any of the study treatments or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates the participation in the study, including severe lactose intolerance
    • Concomitant clinically relevant disease or condition or any clinically significant finding at screening that could interfere with, or for which, the treatment might interfere with, the conduct of the study or that would pose an unacceptable risk to the participants in this study
    • Known active or uncontrolled bacterial, viral, or other infection or any major clinically significant episode of infection or hospitalization (relating to the completion of the course of antibiotics) within 6 weeks prior to the start of drug administration.
    E.5 End points
    E.5.1Primary end point(s)
    1. Vineland Adaptive Behavior Scale–Third Edition (Vineland-3): Adaptive Behavior Composite.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Baseline, Day 183, Day 365.
    E.5.2Secondary end point(s)
    1. Vineland-3: gross and fine motor, expressive and receptive communication language, play and leisure time and interpersonal relationships subdomains
    2. Mullen Scales of Early Learning (MSEL): gross and fine motor domains, visual reception domain, expressive and receptive language subdomains
    3. Dup15q syndrome Clinical Global Impression Severity scale (Dup15q CGI-S)
    4. Dup15q syndrome Clinical Global Impression Change scale (Dup15q CGI-C)
    5. Aberrant Behavior Checklist - Second Edition – Community Version (ABC-2-C) domain scores
    6. Incidence, nature, and severity of AEs and SAEs
    7. Incidence of treatment discontinuations due to AEs
    8. Incidence of laboratory abnormalities based on hematology, clinical chemistry, and urinalysis test results
    9. ECG changes from baseline; incidence of abnormal ECG assessments
    10. Change from baseline in all seizure frequency, duration, and type of seizure as reported in a seizure diary by caregivers
    11. Abnormal changes in EEG recordings compared to baseline with a focus on treatment-emergent epileptiform abnormalities
    12. Systolic and diastolic blood pressure and heart rate measurements
    13. Suicidality as assessed through questions from selected items adapted from the Columbia Classification Algorithm for Suicide (C-CASA) in participants aged 6 years and above
    14. Height, weight, head circumference
    15. Tanner staging over time (in participants aged 9 years and above)
    16. Plasma concentration of basmisanil and its major metabolite M1 at specified timepoints
    17. Plasma concentration ratio of M1 to basmisanil at trough
    18. qEEG beta-band power.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-2. Baseline, Day 183, Day 365
    3. Screening, Baseline, Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up visit (Day 395)
    4. Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up visit (Day 395)
    5. Baseline, Day 28, Day 92, Day 183, Day 274, Day 365
    6-8. Up to Follow-up visit (Day 395)
    9. Screening, Baseline, Day 1, Day 2, Day 14, Day 183, Day 365
    10. Baseline, Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up visit (Day 395)
    11. Baseline, Day 1, Day 2, Day 14, Day 183, Day 365
    12-13. Up to Follow-up visit (Day 395)
    14. Screening, Baseline, Day 183, Day 365
    15. Baseline, Day 365
    16-17. Day 1, Day 2, Day 14, Day 92, Day 183, Day 274, Day 365
    18. Baseline, Day 1, Day 2, Day 14, Day 183, Day 365.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 Yes
    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 trial1
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Russian Federation
    United Kingdom
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    E.8.7Trial has a data monitoring committee No
    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
    A participant is considered to have completed the study if he/she has completed all phases of the study, including the last scheduled procedure per SoA or withdrawal of consent.
    The end of the study is defined as the date when the last participant’s last observation occurs.
    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 months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months1
    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: 90
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 72
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 18
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Participants aged 2 to 14 years inclusive at the time the caregiver signs the informed consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 90
    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)
    All participants who complete the 52 weeks of study treatment in Part 1 will be offered the option to roll over into an OLE to receive basmisanil treatment for a duration of approximately 2 years (Part 2). Participants will be required to sign a separate ICF for participation in Part 2. As per study protocol section 6.7.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2023-01-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-05-23
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA