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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   41232   clinical trials with a EudraCT protocol, of which   6757   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 in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
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    Summary
    EudraCT Number:2017-003104-42
    Sponsor's Protocol Code Number:NSR-CHM-OS2(273CH201)
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-07-01
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2017-003104-42
    A.3Full title of the trial
    A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2- REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study
    A.4.1Sponsor's protocol code numberNSR-CHM-OS2(273CH201)
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03584165
    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 SponsorNightstaRx Ltd (A Biogen Company)
    B.1.3.4CountryUnited Kingdom
    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 supportNightstaRx Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNightstaRx Ltd (A Biogen Company)
    B.5.2Functional name of contact pointClinical Operations Department
    B.5.3 Address:
    B.5.3.1Street Address70 Norden Road
    B.5.3.2Town/ cityMaidenhead
    B.5.3.3Post codeSL6 4AY
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44016 285 12604
    B.5.6E-mailenquiries@nightstartx.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/3/14/1290
    D.3 Description of the IMP
    D.3.1Product nameAAV2-REP1
    D.3.2Product code AAV2-REP1
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraocular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNtimrepigene emparvovec
    D.3.9.1CAS number 1905415-02-2
    D.3.9.2Current sponsor codeAAV2-REP1
    D.3.9.3Other descriptive nameAdeno-associated virus type 2 vector encoding Rab escort protein 1
    D.3.9.4EV Substance CodeSUB182470
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.0 x 10e12DRP/mL
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberATMP scientific recommendation: EMA/H0005418 Purified recombinant adeno-associated viral vector serotype 2 (AAV2) encoding the complementary DNA (cDNA) of human Rab escort protein type 1 (REP1)
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/18/1975
    D.3 Description of the IMP
    D.3.1Product nameAAV8-RPGR
    D.3.2Product code AAV8-RPGR
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraocular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCotoretigene Toliparvovec
    D.3.9.1CAS number 2246424-95-1
    D.3.9.2Current sponsor codeAAV8-RPGR
    D.3.9.3Other descriptive nameAAV8-RPGR
    D.3.9.4EV Substance CodeSUB184227
    D.3.10 Strength
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5.0 x 10e11 gp/ml
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Choroideremia (CHM)
    X-Linked Retinitis Pigmentosa (XLRP)
    E.1.1.1Medical condition in easily understood language
    Choroideremia (CHM)
    X-Linked Retinitis Pigmentosa (XLRP)
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10008791
    E.1.2Term Choroideremia
    E.1.2System Organ Class 100000004853
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10038914
    E.1.2Term Retinitis pigmentosa
    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
    The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 (AAV2-REP1) in participants with Choroideremia (CHM) who have been previously treated with BIIB111 (AAV2-REP1) and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 (AAV8-RPGR) in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 (AAV8-RPGR) and who have exited an antecedent study
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Key Inclusion Criteria:
    - CHM Participants
    a. Have participated in and exited from an
    interventional study that investigated the safety and efficacy of a subretinal injection of BIIB111 for CHM.

    - XLRP Participants
    a. Have received a sub-retinal injection of BIIB112 for XLRP and have
    exited an antecedent study
    E.4Principal exclusion criteria
    Key Exclusion Criteria:
    Participants are not eligible for study participation if they meet the following exclusion criterion.
    a. In the opinion of the investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.
    NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
    E.5 End points
    E.5.1Primary end point(s)
    1. Percentage of Participants with Adverse Events (AEs)
    2. Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
    3. Ophthalmic Examination Assessment: Abnormal Slit Lamp
    Examination
    4. Ophthalmic Examination Assessment: Lens Opacity Grading
    5. Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
    6. Ophthalmic Examination Assessment: Indirect Ophthalmoscopy
    7. Immunogenicity: Percentage of Participants with Antibodies Against Viral Vector BIIB111 and BIIB112
    8. Immunogenicity: Percentage of Participants with Cell Based Responses Against the Viral Vector BIIB111 and BIIB112
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-8: Up to 5 years
    E.5.2Secondary end point(s)
    1. Change from Baseline in Best-Corrected Visual Acuity (BCVA)
    2. Percentage of Participants with no Decrease from Baseline in BCVA or
    a Decrease from Baseline in BCVA of <5 ETDRS Letters in Choroideremia (CHM) Participants
    3. Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants
    4. Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants
    5. Change from Baseline in Fundus Autofluorescence (AF)
    6. Change from Baseline in Fundus Photography
    7. Change from Baseline in Spectral Domain Optical Coherence
    Tomography (SD-OCT)
    8. Change from Baseline in Microperimetry
    9. Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25)
    10. Change from Baseline in Visual Field
    11. Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants
    12. Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112- treated Participants
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-10: Up to 5 years
    11-12: 18 Months to 60 Months, Post-Day 0 Visits
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    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
    Long term Efficacy and Safety follow-up. No administration of treatment included
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Brazil
    Canada
    Denmark
    Finland
    France
    Germany
    Netherlands
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months0
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 440
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 145
    F.4.2.2In the whole clinical trial 440
    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)
    None
    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 Decision2020-07-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-08
    P. End of Trial
    P.End of Trial StatusOngoing
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