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    Summary
    EudraCT Number:2012-005742-38
    Sponsor's Protocol Code Number:SANTA2012
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-04-05
    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 ConcernedUK - MHRA
    A.2EudraCT number2012-005742-38
    A.3Full title of the trial
    Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1
    A.3.2Name or abbreviated title of the trial where available
    SimvAstatin in Neurofibromatosis Type 1-Autism (SANTA)
    A.4.1Sponsor's protocol code numberSANTA2012
    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 SponsorCentral Manchester University Hospitals NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    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 supportCentral Manchester University Hospitals NHS Foundation Trust
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCentral Manchester University Hospitals NHS Foundation Trust
    B.5.2Functional name of contact pointResearch Secretary
    B.5.3 Address:
    B.5.3.1Street AddressResearch and Innovation, Postgraduate Centre, Oxford Road
    B.5.3.2Town/ cityManchester
    B.5.3.3Post codeM13 9WL
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+4401612763565
    B.5.5Fax number+4401612765766
    B.5.6E-mailresearch.secretary@cmft.nhs.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 Yes
    D.2.1.1.1Trade name Simvastatin 20mg/5ml Oral Suspension
    D.2.1.1.2Name of the Marketing Authorisation holderRosemont Pharmaceuticals Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameSimvastatin
    D.3.4Pharmaceutical form Oral suspension
    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 INNSimvastatin
    D.3.9.1CAS number 79902-63-9
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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 placeboOral suspension
    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
    Neurofibromatosis Type 1
    E.1.1.1Medical condition in easily understood language
    Neurofibromatosis Type1 (NF1) is a relatively common genetic condition of nerve tissue. It causes benign (non cancerous) lumps to grow on nerves.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10029270
    E.1.2Term Neurofibromatosis, type 1 (von Recklinghausen's disease)
    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
    The overall research aim is to determine whether treatment with statins improves the ASD phenotype in children with NF1 autism. This aim of this study is to test:
    -Acceptability and feasibility of involvement for families of children with NF1 ASD
    -The feasibility and acceptability of the assessment protocol
    -Treatment effects on intermediate and endpoint behavioural phenotype measures and imaging parameters

    The hypothesis is that treatment with statins in young children with NF1 autism will be:
    -Feasible, safe and acceptable to families
    -Associated with signals of change in brain imaging parameters
    -Associated with signals of change in autism and other behavioural symptoms
    E.2.2Secondary objectives of the trial
    None
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    There are two sub-studies along with the the proposed trial. The blood samples collected as part of the trial will be used to conduct the two sub-studies described below:

    1)P-MAPK level as a peripheral biomarker for cognitive deficits of NF1. The aim of this exploratory study is to ascertain pre and post MAPK levels as a potential biomarker for simvastatin treatment effects. Blood samples will be collected from participants at baseline and at 12 weeks trial end point.

    2)NF1 mutational analysis and evaluation of genotype-phenotype correlation. The aim of this study is to look at the mutations of the NF1 gene and correlate that with the clinical ASD phenotype. Blood samples for this study will be collected at baseline only.
    E.3Principal inclusion criteria
    Children between 5-8yrs meeting diagnostic criteria for
    1)Neurofibromatosis Type 1(National Institute of Health criteria)
    2)Autism Spectrum Disorder (ASD) using Collaborative Programme for Excellence in Autism (CPEA) criteria, based on ADI-R and ADOS-G, age and IQ assessments). 22
    3)Patients who are on a stable dose of methylphenidate and/or dexamphetamine for at least three month prior to screening and who will remain on the same dose for the duration of the study.
    4)Hepatic function: Patients with normal liver function defined as < 2 x upper limit of normal for age.
    5)Renal function: Patients must have adequate renal function defined as GFR >60ml/min/1.73m2 calculated by 40 x height (in cm) / plasma creatinine (in micromol/l).
    6)Informed consent.
    E.4Principal exclusion criteria
    1)Severe learning disability defined as verbal IQ<50 as measured by the WASI.
    2)Patients in active treatment for NF1 complication (e.g. chemotherapy for optic gliomas, Ilizarov frame for pseudarthrosis).
    3)Individuals with abnormal liver function or renal function at baseline as defined in the inclusion criteria.
    4)Parents of patients with insufficient English to complete the ASD screening assessments.
    5)Patients taking psychotropic medication other than methylphenidate and/or dextroamphetamines. These patients are eligible if, as clinically indicated, they discontinue medication for at least 30 days prior to screening and remain off these medications for the duration of the study.
    6)Patients who have received any investigational drug within 4 months of screening.
    7)Patients who have recently taken simvastatin or any other statins. These participants will be eligible after a washout period of at least three months.
    8)Patients with a clinically significant unrelated illness, which in the judgment of the principal or associate investigator, would compromise the participant’s ability to tolerate the medication or interfere with ability to participate in the required testing.
    9)Low cholesterol (defined as total cholesterol < 90mg/dl).
    10)Patients with planned surgery within 16 weeks of potential enrolment
    E.5 End points
    E.5.1Primary end point(s)
    The endpoint outcome measures for the study are:
    -ASD symptoms as assessed by the behavioural phenotype measures
    -Effect of 12 weeks of Simvastatin treatment on multi-parametric imaging findings including anatomical, physiological, spectroscopic and resting state functional connectivity in the default network as assessed at 3T MRI
    - Telephone interviews with parents on experience of trial, intervention, assessments including imaging procedures.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoints of evaluation for the endpoints are:
    - ASD behavioural phenotype measures at 0,4 and 12 weeks
    - Imaging measures at 0 and 12 weeks
    - Telephone interviews with parents at 16 weeks
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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 No
    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 No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Triple blind - clinician/patient/assessor
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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 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
    Endpoint is completion of 12 week dosing. The end of the trial will be when at the last follow-up visit of the last recruited participant. The end of the study will be when the biomarker data has been analysed. An end of trial notification will be submitted to the REC and Regulatory Authority within 90 days of this date.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 30
    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) No
    F.1.1.6.1Number of subjects for this age range: 0
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 0
    F.4.2.2In the whole clinical trial 0
    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)
    The statins will be stopped after 12 weeks as this is an experimental phase 2 study. If the intervention proves helpful for the participants then a larger phase 3 trial will be planned.
    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 Decision2013-05-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-11-30
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