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    Summary
    EudraCT Number:2017-002545-30
    Sponsor's Protocol Code Number:IRAS220495
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-06-07
    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 number2017-002545-30
    A.3Full title of the trial
    Autologous Stem cell Transplantation In refractory Crohn's disease - Low Intensity Therapy Evaluation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Controlled trial to assess low intensity autologous stem cell transplant in refractory Crohn's Disease
    A.3.2Name or abbreviated title of the trial where available
    ASTIClite
    A.4.1Sponsor's protocol code numberIRAS220495
    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 SponsorBarts Health NHS 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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity of Sheffield
    B.5.2Functional name of contact pointLizzie Swaby (Trial Manager)
    B.5.3 Address:
    B.5.3.1Street AddressRoom 2.15, Innovation Centre, 217 Portobello
    B.5.3.2Town/ citySheffield
    B.5.3.3Post codeS1 4DP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01142224023
    B.5.6E-maile.a.swaby@sheffield.ac.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 Cyclophosphamide
    D.2.1.1.2Name of the Marketing Authorisation holderSandoz Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameCyclophosphamide
    D.3.4Pharmaceutical form Powder for solution 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 INNCyclophosphamide monohydrate
    D.3.9.1CAS number 50-18-0
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit gm/m2 gram(s)/square meter
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCyclophosphamide monohydrate
    D.3.9.1CAS number 50-18-0
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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.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 Yes
    D.2.1.1.1Trade name Filgrastim
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen Europe
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameFilgrastim
    D.3.4Pharmaceutical form Solution 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 INNFilgrastim
    D.3.9.1CAS number 121181-53-1
    D.3.9.4EV Substance CodeAS3
    D.3.10 Strength
    D.3.10.1Concentration unit µg/kg microgram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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) Yes
    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.IMP: 3
    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 Fludarabine
    D.2.1.1.2Name of the Marketing Authorisation holderEBEWE Pharma
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameFludarabine
    D.3.4Pharmaceutical form Concentrate for solution for injection/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 INNFludarabine phosphate
    D.3.9.1CAS number 75607-67-9
    D.3.9.4EV Substance CodeAS4
    D.3.10 Strength
    D.3.10.1Concentration unit mg/m2 milligram(s)/square meter
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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.IMP: 4
    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 Thymoglobuline
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe B.V
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameThymoglobuline
    D.3.4Pharmaceutical form Powder for 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 INNRabbit anti-human thymocyte immunoglobulin
    D.3.9.4EV Substance CodeAS5
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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) Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Refractory Crohn's Disease
    E.1.1.1Medical condition in easily understood language
    Crohn's Disease that has not responded to previous treatment
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011401
    E.1.2Term Crohn's disease
    E.1.2System Organ Class 10017947 - Gastrointestinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of this trial is to assess whether autologous haematopoietic stem call transplantation (HSCT) with a low intensity treatment regimen (lite) is safe and effective in inducing regression of ileo-colonic ulceration in Crohn's Disease, where previous treatments have been unsuccessful, compared with standard care.

    Definitions:
    Autologous - cells or tissue obtained from the same individual
    Hematopoietic - cells that give rise to blood cells
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    1. To assess the impact of HSCTlite on clinical disease activity, quality of life and adverse events compared to standard care.

    2. To assess whether HSCTlite is a safe regimen for patients with refractory Crohn's Disease.

    3. To assess the safety and efficacy of anti-TNF therapy in patients who demonstrate endoscopic disease recurrence at week 24 after HSCTlite.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Mechanistic substudy embedded within the main trial

    HSCT is thought to induce regression of autoimmune diseases by altering the diversity of the T cell receptor repertoire and generating functional renewal of regulatory T cells. Neither the mechanism of action nor the time-course of response is known in CD.

    The objectives of this mechanistic substudy are:
    1. Intestinal MRI will be performed to determine the early impact on mucosal disease, at week 4 post-HSCT (intervention arm only).
    2. Immune profiling of peripheral blood (samples at week 8, 14, 24, 32 and 48) and mucosal biopsies (samples at week 24 and 48) will:
    a. Characterise immune re-constitution after HSCT, and assess impact of HSCT on disease activity
    b. Assess immunological events that precede disease recurrence post HSCT
    c. Assess the mechanism of restoration of responsiveness to anti-TNF therapies
    d. Confirm appropriate responses to vaccination post HSCT
    E.3Principal inclusion criteria
    A participant is eligible for the study if the following criteria are met:
    1.Participant of any gender, aged between 18 – 60
    2.Participants must be willing and able to provide full informed consent.
    3.Participants should be well nourished and of healthy weight in the opinion of the PI (typically BMI >18.5).
    4.Diagnosis of CD using colonoscopy, histology and/or radiology.
    5.Disease duration of at least six months.
    6.Disease distribution accessible to endoscopic assessment (ileal, ileo-caecal, or colonic).
    7.Active clinical CD activity with impaired quality of life at any time within 3 months prior to randomisation into the trial, as assessed by a gastroenterology clinician.
    8.Participants will be refractory or intolerant to azathioprine, mercaptopurine or methotrexate.
    9.Participants will be refractory or intolerant to at least two classes of biologic therapy (currently anti-TNF therapy, Vedolizumab or Ustekinumab) despite dose optimisation.
    10.Participants where surgery is considered not appropriate or has been declined.
    11.Endoscopic evidence of active disease in screening (SES CD ulceration sub-score of 2 or more in at least one segment).
    12.Satisfactory EBMT Autoimmune Disease Working Party (ADWP) recommended screening assessment prior to HSCT.
    13.Willingness to discontinue all immunosuppressant medication after randomisation if allocated to HSCT arm.
    14.Participants, who, in the opinion of the TMG, are fit enough to undergo treatment.
    E.4Principal exclusion criteria
    A participant is not eligible for the study if any of the following criteria are met:
    1.Diagnosis of ulcerative colitis or indeterminate colitis.
    2.No evidence of active CD on screening ileocolonoscopy.
    3.Inability to assess for active disease at ileocolonoscopy due to strictures.
    4.Undrained perianal fistulae (patients with previous perianal disease or perianal disease adequately drained with a seton in situ are eligible).
    5.Presence of undrained perianal sepsis on screening pelvic MRI.
    6.Evidence of intra-abdominal sepsis on abdominal MRI.
    7.Active or latent mycobacterial infection.
    8.Prior exposure to Hepatitis B, Hepatitis C or HIV.
    9.Evidence of an enteric or systemic infection.
    10.Participant is currently pregnant or breastfeeding, or planning pregnancy within the study duration. Current pregnancy will be confirmed with a pregnancy test at screening assessment.
    11.Unwilling to use adequate contraception (if appropriate) during duration of 48 week follow up.
    12.Participants with significant medical co-morbidity that precludes HSCT adjudicated by the TMG.
    13.Participants with significant psychiatric co-morbidity.
    14.Significant language barriers, which are likely to affect the participant’s understanding of the study, or ability to complete outcome questionnaires.
    15.Concurrent participation in another interventional clinical trial.
    16.Participants who are not considered medically fit for HSCT defined by any of the following. Participants who meet one or more of the below criteria, who, in the opinion of the local investigator are medically fit, may be discussed at TMG for potential inclusion, and this will be documented in the patient’s notes, and the site file:
    a.Renal: creatinine clearance <40ml/min (measured or estimated)
    b.Cardiac: clinical evidence of refractory congestive heart failure, left ventricular ejection fraction <45% by multigated radionuclide angiography (MUGA) or cardiac echo; uncontrolled ventricular arrhythmia; pericardial effusion with haemodynamic consequences as evaluated by an experienced echo cardiographer
    c.Hepatic: AST > two times the upper limit of normal
    d.Concurrent neoplasms or myelodysplasia
    e.Bone marrow insufficiency defined as neutropenia with an absolute neutrophil count <1x109/l, or thrombocytopenia with a platelet count <50x109/l, or anaemia with a haemoglobin <80g/l
    f.Uncontrolled hypertension, defined as resting systolic blood pressure >= 140ml and/or resting diastolic pressure >= 90ml mercury despite at least 2 anti-hypertensive agents (subject to discussion at TMG).
    g.Uncontrolled acute or chronic infection with HIV, HTLV – 1 or 2, hepatitis viruses or any other infection the investigator or TMG consider a contraindication to participation.
    h.Other chronic disease causing significant organ failure, including established cirrhosis with evidence of impaired synthetic function on biochemical testing and known respiratory disease causing resting arterial oxygen tension <8kPa or carbon dioxide tension >6.7kPa. FEV1/FVC <50%. Patients not known to have respiratory disease need not have blood gas measurements.
    E.5 End points
    E.5.1Primary end point(s)
    Treatment success at week 48 defined as mucosal healing (no endoscopic ulceration (SES CD ulcer sub score = 0, assessed by adjudication panel blind to allocation and time of assessment)) without surgery or death. Patient who do not complete the week 48 colonoscopy will be categorised as treatment failures.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 48 post stem cell transplant in the intervention arm, and week 48 (plus 49 days) post randomisation for the control arm.
    E.5.2Secondary end point(s)
    Clinical endpoints
    1.Clinical remission (CDAI <150)
    2.Steroid free clinical remission (CDAI <150)
    3.Clinical remission (Harvey Bradshaw Index ≤4)
    4.Clinical remission (PRO2 – mean scores – abdominal pain ≤1, stool frequency ≤1.5)
    5.Absolute CDAI at week 48
    6.Absolute SES CD at week 48
    7.Change in CDAI and SES CD between baseline and week 48
    8.Proportion of patients in complete endoscopic remission (SES CD score of 0)

    Safety endpoints
    1.Toxicity of chemotherapy using NCI CTCAE criteria version 4.03
    2.Adverse events (AEs) and Serious Adverse Events (SAEs), including mortality

    Patient-reported endpoints
    1.Disease specific quality of life using the IBDQ
    2.Disease specific quality of life using the IBD Control
    3.Quality of life using the EQ-5D-5L
    4.Health care resource utilisation questionnaire

    Exploratory secondary endpoints
    1.Efficacy of re-introduction of anti-TNF therapy in patients with disease recurrence post-HSCT (change in CDAI at 6 weeks and change in SES CD at 22 weeks after initiation)
    2.Safety of re-introduction of anti-TNF therapy in patients with disease recurrence post-HSCT
    3.Presence of any of the late side effects of HSCT
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 48 post stem cell transplant in the intervention arm, and week 48 (plus 49 days) post randomisation for the control arm.
    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 No
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3.1Comparator description
    Can include steroids, immunomodulators, licensed/approved biologic therapy or enteral/IV nutrition
    E.8.2.4Number of treatment arms in the trial1
    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 concerned8
    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 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. The end of the trial is defined as the date of the last recruited participant's week 48 follow up visit. Sites will be closed once data cleaning is completed and the regulatory authority and ethics committee will be informed.
    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 months7
    E.8.9.1In the Member State concerned days31
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days31
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    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) No
    F.1.1.5.1Number of subjects for this age range: 0
    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) Yes
    F.1.2.1Number of subjects for this age range: 99
    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 state99
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 99
    F.4.2.2In the whole clinical trial 99
    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 continue to be monitored as per normal clinical practice after the trial is complete, and may have other treatment restarted if required.

    When the research is complete, the haematopoietic stem cell transplantation with low intensity treatment regimen may be offered to participants who did not receive it during the trial. After the main trial, all participants will be followed for a further 4 to 9 years by the European Society of Blood and Marrow Transplantation.
    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 CRN: North Thames
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-10-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-11-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-11-29
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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