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    Summary
    EudraCT Number:2018-002502-31
    Sponsor's Protocol Code Number:RP-L102-0118
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-10-08
    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 ConcernedUK - MHRA
    A.2EudraCT number2018-002502-31
    A.3Full title of the trial
    A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A
    A.3.2Name or abbreviated title of the trial where available
    FANCOLEN-II
    A.4.1Sponsor's protocol code numberRP-L102-0118
    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 SponsorRocket Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    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 supportRocket Pharmaceuticals, Inc.
    B.4.2CountryUnited States Minor Outlying Islands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRocket Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointChief Medical Officer
    B.5.3 Address:
    B.5.3.1Street Address9 Cedar Brook Drive
    B.5.3.2Town/ cityCranbury
    B.5.3.3Post codeNJ 08512
    B.5.3.4CountryUnited States
    B.5.4Telephone number1646440-9100
    B.5.6E-mailjs@rocketpharma.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/3/10/822
    D.3 Description of the IMP
    D.3.2Product code RP-L102
    D.3.4Pharmaceutical form Suspension for injection
    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 INNCD34+CELLS
    D.3.9.1CAS number CD34+CELLS
    D.3.9.2Current sponsor codeRP-L102
    D.3.9.3Other descriptive nameCD34+CELLS
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number5E+5 cells/kg
    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 Yes
    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 numberGene therapy medicinal product, reference number H0005064
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fanconi anemia (subtype A)
    E.1.1.1Medical condition in easily understood language
    Fanconi anemia (subtype A)
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10055206
    E.1.2Term Fanconi's anemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of this trial is to assess the therapeutic efficacy of a haematopoietic gene therapy procedure with an orphan drug consisting of a LV carrying the FANCA gene in patients with FA-A.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. FA as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent
    2. Patients of the complementation group FA-A
    3. Minimum age: 1 year and a minimum weight of 8 kg.
    4. Maximum age: 17 years
    5. At least 30 CD34+ cells/μL are determined in one BM aspiration within 3 months prior to CD34+ cell collection OR (see subsequent criterion)
    6. If the number of CD34+ cells/μL in BM is in the range of 10–29, PB parameters should meet two of the three following criteria:
    • Hemoglobin: ≥11g/dL
    • Neutrophils: ≥900 cells/μL
    • Platelets: ≥60,000 cells/μL
    7. Provide informed consent in accordance with current legislation
    8. Women of childbearing age must have a negative urine pregnancy test at the baseline visit, specifically within 14 days prior to infusion of IMP and must accept the use of an effective contraception method during participation in the trial as defined in the note below.
    Note: Female patients of childbearing potential (e.g., are menstruating or could reach menarche during the study) should be counselled on the need for contraception should they be sexually active or, in the opinion of the Investigator, likely to be sexually active. Such subjects must agree to the use of contraception as defined in the subsequent paragraph. Male patients who are sexually active must agree to use a condom during intercourse for the duration of their participation in the study.
    Acceptable methods of contraception for female subjects of childbearing potential include the following:
    - male or female condom with or without spermicide;
    - cap, diaphragm or sponge with spermicide;
    - intrauterine device (IUD);
    - bilateral tubal occlusion;
    - vasectomised partner;
    - sexual abstinence.
    E.4Principal exclusion criteria
    1. Patients with an available and medically eligible HLA-identical sibling donor
    2. Evidence of myelodysplastic syndrome or leukaemia, or cytogenetic abnormalities other than those reported as variant(s) of normal in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the patient enters the clinical trial
    3. Patients with somatic mosaicism associated with stable or improved counts in all PB cell lineages (If T-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease in at least one blood lineage over time must be documented to enable eligibility)
    4. Lansky performance index ≤60%
    5. Any concomitant disease or condition that, in the opinion of the Principal Investigator, deems the patient unfit to participate in the trial
    6. Pre-existing sensory or motor impairment ≥grade 2 according to the criteria of the National Cancer Institute (NCI)
    7. Pregnant or breastfeeding women
    8. Hepatic dysfunction as defined by either:
    • Bilirubin > 3.0× the upper limit of normal (ULN)
    • Alanine aminotransferase (ALT) >5.0× ULN
    • Aspartate aminotransferase (AST) >5.0× ULN.
    For subjects with bilirubin, ALT or AST above ULN, a workup to identify the etiology of liver abnormality should be conducted prior to confirmation of eligibility as stipulated in exclusion criterion 5, including evaluation of viral hepatitis, iron overload, drug injury or other causes.
    9. Renal dysfunction requiring either hemodialysis or peritoneal dialysis
    10. Pulmonary dysfunction as defined by either:
    • Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection)
    • Oxygen saturation (by pulse oximetry) <90%.
    11. Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years
    12. Subject is receiving androgens (i.e., danazol, oxymetholone)
    13. Subject is receiving other investigational therapy for treatment/prevention of FA-associated bone marrow failure.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is the extent of phenotypic correction to mosaic or normal range as determined by MMC-resistance of bone marrow colony forming cells (also known as colony forming units or CFUs), targeting an increase of at least 10% BM CFUs resistant to 10 nM MMC at any timepoint at least 12 months subsequent to investigational infusion (progressive increases in the percentage of resistant cells over at least 2 points in time will be considered confirmatory).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 3 years after infusion
    E.5.2Secondary end point(s)
    1. Level of phenotypic correction of haematopoietic cells as determined by additional parameters (T-lymphocyte chromosomal fragility assay; targeting fewer than 50% aberrant cells following DEB-culture and a minimum decrease of at least 20% from baseline) after the infusion of the IMP in subjects with FA-A.
    2. Level of engraftment of gene-corrected haematopoietic cells after the infusion of the IMP in subjects with FA-A.
    3. Clinical response: prevention or rescue of BMF after the infusion of the IMP in subjects with FA-A.
    4. Ongoing evaluation of the short- and long-term safety of the infusion of the IMP in subjects with FA-A.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 3 years after infusion
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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 No
    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.8.2.4Number of treatment arms in the trial1
    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 EEA3
    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
    This trial will end when all recruited patients complete the scheduled assessments
    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 months6
    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 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: 5
    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: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 4
    F.1.1.6Adolescents (12-17 years) No
    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 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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Some patients may be below the age for giving consent and a legally authorized representative will provide informed consent and/or assent as mandated by local regulations
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 5
    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)
    Patients will be followed-up for 3 years. However, patients will be monitored outside the clinical trial for a total period of 15 years.
    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 Decision2019-12-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-07
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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