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    Summary
    EudraCT Number:2021-000838-34
    Sponsor's Protocol Code Number:NOE-TSC-201
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2023-02-27
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2021-000838-34
    A.3Full title of the trial
    A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults with Seizures Associated with Tuberous Sclerosis Complex
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Basimglurant in children, adolescents, and young adults with Tuberous Sclerosis Complex
    A.3.2Name or abbreviated title of the trial where available
    Basimglurant in children, adolescents, and young adults with Tuberous Sclerosis Complex
    A.4.1Sponsor's protocol code numberNOE-TSC-201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05059327
    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 SponsorNoema Pharma
    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 supportNoema Pharma
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNoema Pharma
    B.5.2Functional name of contact pointVP, Pediatric Programs
    B.5.3 Address:
    B.5.3.1Street AddressBarfüsserplatz 3
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4051
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number41615511025
    B.5.6E-mailrLazarova@noemapharma.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 namebasimglurant
    D.3.2Product code NOE-101
    D.3.4Pharmaceutical form Capsule
    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 INNBASIMGLURANT    
    D.3.9.1CAS number 802906-73-6
    D.3.9.2Current sponsor codeNOE-101
    D.3.9.4EV Substance CodeSUB177915
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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 namebasimglurant
    D.3.2Product code NOE-101
    D.3.4Pharmaceutical form Capsule
    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 INNBASIMGLURANT    
    D.3.9.1CAS number 802906-73-6
    D.3.9.2Current sponsor codeNOE-101
    D.3.9.4EV Substance CodeSUB177915
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.0
    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 placeboCapsule
    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
    Tuberous Sclerosis Complex
    E.1.1.1Medical condition in easily understood language
    Tuberous Sclerosis Complex is a genetic disorder of uncontrolled growth of numerous benign tumors in many parts of the body including the brain, and one of the leading genetic causes of epilepsy.
    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 21.0
    E.1.2Level PT
    E.1.2Classification code 10080584
    E.1.2Term Tuberous sclerosis complex
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of a double-blind, daily basimglurant administration, adjunctive to ongoing anticonvulsive therapy compared with placebo adjunctive to ongoing anticonvulsive therapy in patients with Tuberous Sclerosis Complex (TSC).
    E.2.2Secondary objectives of the trial
    -To evaluate the impact of treatment on functioning at school and during social activities.
    -To determine the effect of basimglurant on the severity of symptoms of TSC.
    -To determine the longest seizure free interval (i.e., seizure free days).
    -To evaluate the number of patients considered treatment responders.

    Other Secondary Objectives:
    - To evaluate the safety of basimglurant in children, adolescents, and young adults with seizures associated with TSC.
    - To investigate the proportion of patients tolerating each dose during dose escalation.
    - To investigate long-term safety of basimglurant in children, adolescents, and young adults with seizures associated with TSC
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all the following criteria for study entry:
    1. Ability and willingness to provide informed assent or written consent or consent from their legal representative and willingness to comply with the study procedures.
    2. Fluency in the language of the investigator, study staff and the informed assent or consent form when applicable.
    3. Age 5 to 30 years at study entry
    4. A documented history of TSC, diagnosed according to the International Tuberous Sclerosis Complex diagnostic criteria of 2021 and including a record of either genetic test or MRI/CT scan documenting tumors.
    5. Continued seizures associated with TSC (including atypical absences, atonic, focal, tonic, tonic-clonic or myoclonic) despite adequate dosage of at least 1 or more appropriate AEDs, within approximately the previous year.
    6. Refractory seizure history, defined as 3 or more countable seizures per month on average over the last year 3 months and at least 3 countable seizures within the past 28 days.
    7. Currently receiving 1 or more AEDs with no change in doses in the 30 days prior to study entry and no anticipated dose changes during study participation.
    8. All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must be stable for 30 days prior to study entry and the patient must be willing to maintain a stable regimen throughout the study. The ketogenic diet and neurostimulation treatments are not considered AEDs for the purpose of this study.
    9. Patients or their caregiver must be willing to complete daily PRO assessments
    10. For female patients of childbearing potential:
    a. willingness to undergo serum or urinary pregnancy testing at screening and during the
    trial period.
    b. willingness to use contraception as defined in Appendix D.
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria will be excluded from study participation:
    1. Etiology of a patient’s seizures is a progressive neurologic disease other than TSC.
    2. Anoxic episode requiring resuscitation within 6 months of screening.
    3. Patient weight below 15kg.
    4. Clinically significant unstable medical conditions other than epilepsy including, but not limited to, cardiovascular, gastrointestinal, renal, hepatic, neurological, musculoskeletal, infectious, endocrine, metabolic, hematological, psychiatric, or other major physical impairment that is not stable in the opinion of the investigator and could affect the safety of the patient throughout the study, influence the findings of the study or their interpretation, or might impede the patient’s ability to complete the entire duration of the study.
    5. Any clinically significant abnormal findings in physical examination, vital signs, hematology, clinical chemistry, or urinalysis during screening which, in the opinion of the investigator, may put the patient at risk because of their participation in the study, or might influence the results of the study, or the patient’s ability to complete the entire duration of the study.
    6. Clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to screening or randomization, other than epilepsy.
    7. Current or past use of recreational or medicinal cannabis within the 3 months prior to study entry and unwillingness to abstain for the duration of the study or a positive result on a urine tetrahydrocannabinol (THC) panel test. (Cannabidoil is an AED and is therefore allowed for the treatment of TSC).
    8. Participation in a clinical trial involving another investigational product (IP) in the previous 6 months or at any time in a gene therapy clinical trial.
    9. Brain surgery ≤6 months prior to study entry for all ages, and for patients<12-year-old, brain surgery for epilepsy treatment prior to study entry).
    10. Patient has bipolar disorder.
    11. Patient is currently taking long-term systemic steroids (excluding inhaled medication for asthma treatment) or any other daily medication known to exacerbate epilepsy. An exception will be made for prophylactic medication such as medications for idiopathic nephrotic syndrome or asthma.
    12. Pregnancy or lactation.
    13. Participants with a history of an allergic reaction or hypersensitivity
    to constituents of the study drug (and its
    excipients) and/or other products in the same class.
    14. Current alcohol abuse/dependence.
    E.5 End points
    E.5.1Primary end point(s)
    Estimand 1a (Primary):
    - Ratio of geometric means of monthly seizure counts in Periods 2 and 4 between basimglurant and placebo in children, adolescents, and young adults with seizures associated with TSC, assuming hospitalization due to COVID-19 or death would not have occurred, irrespective of treatment discontinuation and use of prohibited medications

    Primary Endpoint:
    Monthly seizure counts of countable seizure types(per 28 days) during the 12-week treatment period in Period 2 and Period 4.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Monthly seizure counts (per 28 days) during the 12-week treatment period in Period 2 and Period 4. The Holm procedure
    (Holm 1979) will be used within the test family at the end of the hierarchy for the responses in monthly seizure counts.
    E.5.2Secondary end point(s)
    - Changes from baseline in Sheehan Disability Scale(SDS) score at Week 16 in Period 2 and at Week 30 in Period 4
    - Caregiver Global Impression of Change (CGIC) score at Week 16 in Period 2 and Week 30 in Period 4.
    - Monthly seizure free days (per 28 days) during the 12-week treatment period in Period 2 and Period 4.
    -Seizure 25% response defined as ≥25% reduction from baseline in monthly seizure counts of countable seizure types during the 12-week treatment period in Periods 2 and 4.
    -Seizure 50% response defined as ≥50% reduction from baseline in monthly seizure counts of countable seizure types during the 12-week treatment period in Periods 2 and 4.
    -Seizure 75% response defined as ≥75% reduction from baseline in monthly seizure counts of countable seizure types during the 12-week treatment period in Periods 2 and 4.

    Other Secondary Endpoints:
    - Adverse events,
    -Absolute values and changes from baseline in vital signs, physical examination, electrocardiograms, and clinical laboratory test parameters.
    - Treatment delays, dose reductions, and dose discontinuations.
    - S-STS score for suicidal ideation.
    - Seizure types.
    - Escalation (Yes/No) to each dose level during the dose escalation in Periods 2 and 4.
    - Toleration (Yes/No) of each dose level during the dose escalation in Periods 2 and 4.
    -Adverse events and other safety and tolerability parameters during the OLE period.
    E.5.2.1Timepoint(s) of evaluation of this end point
    -Changes from baseline in SDS score at Week 16 in Period 2 and at Week 30 in Period 4
    -CGIC score at Week 16 in Period 2 and at Week 30 in Period 4
    -Monthly seizure-free days during the 12-week treatment period in Periods 2 and 4 -Response in monthly seizure counts
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    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 EEA22
    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
    India
    Israel
    United Kingdom
    United States
    Italy
    Poland
    Spain
    Türkiye
    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
    End of study is defined as LSLV or the last scheduled procedure.
    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 months8
    E.8.9.1In the Member State concerned days0
    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 days0
    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: 45
    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: 22
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 25
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    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 No
    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
    Nearly all people with TSC demonstrate at least one mental health complication or learning disabilities. The severity of mental health manifestations varies widely from one individual to another. For this reason, some participants may require an LAR.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 42
    F.4.2.2In the whole clinical trial 67
    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)
    After completion of the 8-week run-in phase followed by a 12-week, prospective, parallel-group, double-blind, randomized withdrawal, placebo-controlled phase of the study, study treatment will be provided during an OLE of 52 weeks. If a patient is withdrawn from study treatment or completes the study, the patient will be treated as determined by the attending physician and no longer receive investigational medication basimglurant (NOE-101)
    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 Decision2023-06-06
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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