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    The EU Clinical Trials Register currently displays   38499   clinical trials with a EudraCT protocol, of which   6324   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2011-001953-10
    Sponsor's Protocol Code Number:ALD-102
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2017-12-11
    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 ConcernedGermany - PEI
    A.2EudraCT number2011-001953-10
    A.3Full title of the trial
    A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy
    A.4.1Sponsor's protocol code numberALD-102
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01896102
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/290/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorbluebird bio, 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 supportbluebird bio, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationbluebird bio, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address60 Binney Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number001 339 4999300
    B.5.6E-mailclinicaltrials@bluebirdbio.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/3/12/1003
    D.3 Description of the IMP
    D.3.1Product nameLenti-D Drug Product
    D.3.4Pharmaceutical form Dispersion 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.3.9.3Other descriptive nameAUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA
    D.3.9.4EV Substance CodeSUB127987
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number5000000
    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 numberClassification: Gene Therapy Medicinal Product Reference Number: EMA/CAT/988313/2011
    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 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
    Cerebral Adrenoleukodystrophy (CALD)
    E.1.1.1Medical condition in easily understood language
    See above.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10051260
    E.1.2Term Adrenoleukodystrophy
    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
    • Efficacy
    • Safety
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local IRB/Independent Ethics Committee (IEC) approved consent (informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements).
    2. Males aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent.
    3. Active cerebral ALD as defined by:
    -Elevated VLCFA values, and
    -Active CNS disease established by central radiographic review
    of brain MRI demonstrating:
    i. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
    ii. Gadolinium enhancement on MRI of demyelinating lesions.
    4. Neurological Function Score (NFS) ≤ 1.
    E.4Principal exclusion criteria
    1. Receipt of an allogeneic transplant or gene therapy.
    2. Availability of a willing 10/10 HLA-matched sibling donor (excluding female heterozygotes).
    3. Use of statins, Lorenzo's Oil, or dietary regimens used to lower VLCFA of consent.
    4. Receipt of an investigational study drug or procedure within 3 months before Screening that might confound study outcomes. Use of investigational study drugs is prohibited throughout the course of the study.
    5. Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
    6. Hematological compromise as evidenced by:
    - Peripheral blood ANC count < 1500 cells/mm3,
    - Platelet count < 100,000 cells/mm3, or
    - Hemoglobin < 10 g/dL.
    - Uncorrected bleeding disorder.
    7. Hepatic compromise as evidenced by:
    - Aspartate transaminase (AST) value > 2.5×ULN
    - Alanine transaminase (ALT) value > 2.5×ULN
    - Total bilirubin value > 3.0 mg/dL, except if there is a diagnosis of Gilbert's Syndrome and the subject is otherwise stable
    8. Renal compromise as evidenced by abnormal renal function (actual or calculated creatinine clearance < 50 mL/min)
    9. Cardiac compromise as evidenced by left ventricular ejection fraction <40%
    10. Immediate family member with a known or suspected familial cancer syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome, and familial adenomatous polyposis).
    11. Clinically significant active bacterial, viral, fungal, parasitic, or prionassociated infection
    12. Positive for human immunodeficiency virus type 1 or 2 (HIV-1, HIV-2); hepatitis B; hepatitis C; human T lymphotrophic virus 1 (HTLV-1). (Note that subjects who have been vaccinated against hepatitis B [hepatitis B surface antibody-positive] who are negative for other markers of prior hepatitis B infection [eg, negative for hepatitis B core antibody (Ab)] are eligible. Subjects with past exposure to HBV [HBcAb positive and/or HBeAb positive] are also eligible for the study provided they have a negative test for HBV DNA. Also note that subjects who are positive for anti-hepatitis C antibody are eligible as long as they have a negative hepatitis C viral load).
    13. Any clinically significant cardiovascular or pulmonary disease, or other disease or condition that would be contraindicated for any of the other study procedures.
    14. Absence of adequate contraception for fertile subjects. Male subjects and their female partners are required to use two different effective methods of contraception from Screening through at least 6 months after drug product infusion.
    15. Any contraindications to the use of G-CSF during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and cyclophosphamide, including known hypersensitivity to the active substances or to any of the excipients.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is:
    • Proportion of subjects who are alive and have none of the 6 MFDs at Month 24 (i.e. Month 24 MFD-free survival). MFDs are:
    o loss of communication
    o cortical blindness
    o tube feeding
    o total incontinence
    o wheelchair dependence
    o complete loss of voluntary movement

    The primary safety endpoint is:
    • The proportion of subjects who experience either acute (≥ Grade II) or chronic GVHD by Month 24.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months post-transplant
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include:
    • Proportion of subjects who demonstrate resolution of gadolinium positivity on MRI (i.e., GdE-) at Month 24
    • Time to sustained resolution of gadolinium positivity on MRI (i.e., GdE -).
    • Change in total NFS from Baseline to Month 24
    • MFD-free survival over time
    • Overall survival

    The secondary safety endpoints :
    • Proportion of subjects with neutrophil engraftment by 42 days post-drug product infusion
    • Time to neutrophil engraftment post-drug product infusion
    • Proportion of subjects with platelet engraftment by Month 24
    • Time to platelet engraftment post-drug product infusion
    • Proportion of subjects with loss of engraftment post-drug product infusion by Month 24
    • Proportion of subjects who undergo a subsequent HSC infusion by Month 24
    • Proportion of subjects with transplant-related mortality through 100 and 365 days post-drug product infusion
    • Proportion of subjects with and severity of clinical ≥ Grade 3 AEs, all drug-product related AEs, all SAEs, ≥ Grade 3 infections, and changes in laboratory parameters by Month 24
    • Proportion of subjects with ≥ Grade II acute GVHD by Month 24
    • Proportion of subjects with chronic GVHD by Month 24
    • Number of emergency room visits (post-neutrophil engraftment) by Month 24
    • Number and duration of in-patient hospitalizations (post-neutrophil engraftment) by Month 24
    • Number and duration of ICU stays (post-neutrophil engraftment) by Month 24
    • Incidence of vector-derived RCL at Month 24
    • The number of subjects with insertional oncogenesis (myelodysplasia, leukemia, lymphoma, etc.) by Month 24
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 months post-transplant
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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) 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 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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    France
    United Kingdom
    United States
    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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months8
    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: 32
    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: 28
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) No
    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 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
    children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 32
    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 ALD-102 study, subjects will be followed for long term safety and efficacy under a separate protocol (LTF-304; EudraCT number: 2015-002805-13).
    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 Decision2018-06-18
    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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