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    Summary
    EudraCT Number:2020-003136-25
    Sponsor's Protocol Code Number:CTD-TCNPC-301
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-10
    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 ConcernedFrance - ANSM
    A.2EudraCT number2020-003136-25
    A.3Full title of the trial
    A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl β cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1
    A.4.1Sponsor's protocol code numberCTD-TCNPC-301
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCyclo Therapeutics, Inc.
    B.1.3.4CountryUnited States
    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 supportCyclo Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCyclo Therapeutics, Inc.
    B.5.2Functional name of contact pointChief Scientific Officer
    B.5.3 Address:
    B.5.3.1Street Address16th Street, Suite B
    B.5.3.2Town/ cityGainesville
    B.5.3.3Post code6714 NW
    B.5.3.4CountryUnited States
    B.5.4Telephone number386-418-8060
    B.5.6E-mailsharon.hrynkow@cyclodex.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/11/895
    D.3 Description of the IMP
    D.3.1Product nameTrappsol® Cyclo™
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNA
    D.3.9.3Other descriptive nameHYDROXYPROPYLBETADEX
    D.3.9.4EV Substance CodeSUB32181
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number2000 to 2000
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Niemann Pick Disease Type C1
    E.1.1.1Medical condition in easily understood language
    Rare inherited lysosomal lipidosis resulting in a progressive and fatal neurological deterioration
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10029403
    E.1.2Term Niemann-Pick disease
    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
    This study has dual primary objectives, such that for the FDA and countries following the FDA guidance, the primary objective is to evaluate the effectiveness of Trappsol Cyclo and standard of care (SOC) compared to placebo and SOC as measured by an improvement in the 4 domain Niemann-Pick disease Type C-Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech, and Swallow]) composite score at either Week 48 or Week 96 and for the EU and countries following EMA guidance, the primary objective is to evaluate the effectiveness of Trappsol Cyclo and SOC compared to placebo and SOC as measured by an improvement in the 5 domain Niemann Pick disease Type C Severity Scale (5D NPC SS) (Ambulation, Fine Motor, Speech, Swallow, and Cognition) composite score at either Week 48 or Week 96.
    The objective that is not primary for a specific country will be an exploratory objective.
    E.2.2Secondary objectives of the trial
    •To determine the ability of Trappsol Cyclo and SOC compared to placebo and SOC to improve ataxia as assessed by the Spinocerebellar Ataxia Functional Index (SCAFI)
    •To evaluate the effectiveness of Trappsol Cyclo and SOC compared to placebo and SOC as assessed by the Vineland Adaptive Behavior Scale 2nd edition (Vineland 2) composite raw score, including the optional Motor Skills domain
    •To evaluate the effectiveness of Trappsol Cyclo and SOC compared to placebo and SOC with regards to the patient’s ability to swallow as assessed by the Penetration Aspiration Scale (PAS)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    A substudy in patients newborn to <3 years of age will be conducted the European Union (EU) and countries following the European Medicines Agency (EMA) guidelines and will not be conducted in the United States (US) or countries following Food and Drug Administration (FDA) guidelines; this substudy is presented in Section 14.4 of the full protocol. This substudy will enroll patients newborn to <3 years of age in an exploratory module to evaluate the safety of Trappsol Cyclo in these patients, and to obtain descriptive data regarding global severity and improvement in response to Trappsol Cyclo from both Investigator and patient perspective (CGI-S, CGI-C, CaGI-S, CaGI-C, and CaGI C24). These patients will not be randomized, rather, these patients will all receive open-label Trappsol Cyclo and SOC. These patients will not contribute to the efficacy or safety data sets addressing the principal hypotheses of the study. These patients are eligible for 192 weeks of treatment with Trappsol Cyclo.
    E.3Principal inclusion criteria
    1.Diagnosis of NPC1 confirmed by:
    a.Genetically confirmed (deoxyribonucleic acid sequence analysis) by mutations in both alleles of NPC1, OR
    b.Mutation in only 1 allele of NPC1 and either positive filipin staining in skin or vertical supranuclear gaze palsy (VSGP)
    2.Patients with an ASIS score between 0.5 to 2.0 at Screening using the 17-Domain Niemann-Pick Type C Severity Scale (17D NPC SS) composite score
    3.Treated or not treated with miglustat
    a.If a patient is receiving treatment with miglustat, the dose must have been stable for at least 3 continuous months prior to the first Screening Visit
    b.If a patient has been discontinued from prescribed treatment with miglustat, she/he must have been discontinued for at least 3 continuous months prior to the first Screening Visit
    4.Body weight >4.5 to ≤125 kg
    5.Presenting at least 1 neurological symptom of the disease (including, but not limited to, hearing loss, VSGP, ataxia, dementia, dystonia, history of seizures, dysarthria, or dysphagia)
    6.Willing and capable to participate in all aspects of trial design, including blood sampling (efficacy, PK, blood biomarkers, and safety laboratory tests). Adequate compliance with the assessments to obtain complete data can become a discussion between the Investigator and the medical monitor prior to randomization at the Baseline Visit
    7.Ability to travel to the corresponding clinical trial site at the scheduled visit times for evaluation and follow-up
    8.Contraception requirements:
    a.All sexually active WOCBP (post menarche) must use highly effective contraception during the study and until 3 months after the last dose of study treatment
    b.Highly effective birth control methods include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; or vasectomized partner
    c.All sexually active male patients with WOCBP partners (post menarche) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of study treatment
    d.Sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the study and for 3 months after the last dose of study treatment for WOCBP and for male patients with WOCBP partners. The reliability of sexual abstinence needs to be evaluated by the Investigator in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient
    9.The patient or legally authorized representative has read and signed the informed consent (or assent, as applicable) form prior to any study-related procedures
    10.The legally authorized representative (if applicable) agrees for the patient to participate in all aspects of the trial
    11.The patient’s caregiver (as applicable) agrees to participate in all of the protocol-specified assessment scales, questionnaires, and interviews for the duration of the trial
    Criteria to Switch to Open label Trappsol Cyclo Treatment Prior to Week 96
    1.Received double-blind treatment for ≥36 weeks, AND
    2.CGI-S Overall Disease deterioration of ≥2 levels from Baseline at 2 consecutive assessment visits 12 weeks apart per SoA
    Note that based on these criteria the earliest timepoint that a patient may be switched to open-label Trappsol Cyclo due to deterioration is following completion of Week 48 assessments. Trappsol Cyclo will be administered at subsequent visits.
    Criteria to Participate in the Open-label Extension
    1.Completed double-blind treatment through Week 92 and completed Week 96 assessments, OR
    2.Discontinued early from double-blind treatment for reasons other than safety but completed all assessments through Week 96
    E.4Principal exclusion criteria
    1.Recipient of a liver transplant or planned liver transplantation
    2.Patients with active liver disease from any cause other than NPC1 or prolonged icterus or malformation of organs other than NPC1
    3.Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalized ratio >1.8
    4.Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2. In patients aged ≤18 years, eGFR is calculated according to the Schwartz equation (Schwartz and Work, 2009), and in patients aged >18 years eGFR is calculated using the Modification of Diet in Renal Disease equation
    5.Use of curcumin or fish oil within 12 weeks prior to enrollment
    6.Known or suspected allergy or intolerance to the study treatment
    7.In the opinion of the Investigator, the patient's clinical condition does not allow for the blood collection required as per the protocol specified procedures
    8.Treatment with any investigational drug during the 3 months prior to entering the study, including leucine in a clinical trial; however, leucine as a nutraceutical is allowed
    9.Treatment with HPβCD prior to entering the study
    10.Treatment with any other investigational drug during the study
    11.Pregnancy or breastfeeding
    12.Current participation in another trial is not permitted unless it is a noninterventional study and the sole purpose of the trial is for long term follow up describing clinical features or survival data (registry)
    13.Patients with uncontrolled, severe epileptic seizure periods (at least 3 consecutive severe epileptic seizures that required medication) within 2 months prior to completion of informed consent (or assent, as applicable). This includes patients with ongoing seizures that are not stable in frequency or type or duration over a 2-month period prior to enrollment, requiring change in dose of antiepileptic medication (other than adjustment for weight) over a 2-month period prior to enrollment, or requiring 3 or more antiepileptic medications to control seizures over a 2-month period prior to enrollment
    14.Neurologically asymptomatic patients
    15.Inability to participate in the primary study assessment (4D-NPC-SS or 5D NPC SS, depending on jurisdiction) as determined by the Investigator

    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoints of this study for the EMA and countries following the EMA guidelines are:
    •Mean change in the 5D NPC SS composite score (Ambulation, Fine Motor, Speech, Swallow, and Cognition) from Baseline (Week 0) to 48 weeks
    •Mean change in the 5D NPC SS composite score (Ambulation, Fine Motor, Speech, Swallow, and Cognition) from Baseline (Week 0) to 96 weeks
    The primary endpoints of this study for the FDA and countries following the FDA guidelines are:
    •Mean change in the 4D-NPC-SS (Ambulation, Fine Motor, Speech, and Swallow) composite score from Baseline (Week 0) to 48 weeks
    •Mean change in the 4D-NPC-SS (Ambulation, Fine Motor, Speech, and Swallow) composite score from Baseline (Week 0) to 96 weeks
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 and 96 weeks
    E.5.2Secondary end point(s)
    •Mean change from Baseline in the total SCAFI composite score at 48 and 96 weeks
    •Mean change from Baseline in the Vineland 2 composite raw score, including the optional Motor Skills domain, at 48 and 96 weeks
    •Mean change from Baseline in the patient’s ability to swallow as assessed by the PAS, at 48 and 96 weeks (at select sites)
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 and 96 weeks
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Eligible patients may enter an Open-label extension upon completion of blinded treatment period
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Israel
    United States
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    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: 42
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 19
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 17
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 63
    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
    children and newborn: 0-10 years old
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 105
    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)
    not indicated
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation name
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-18
    P. End of Trial
    P.End of Trial StatusOngoing
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