A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy

NCT ID: NCT03839771

Last Updated: 2024-10-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

968 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-01

Study Completion Date

2034-09-19

Brief Summary

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AML and MDS-EB2 are malignancies of the bone marrow. The standard treatment for these diseases is chemotherapy. Patients participating have a special type of this disease because the leukemia cells (blasts) have developed an error in the genetic material (DNA). This error is called an IDH1 mutation or an IDH2 mutation (a mutation is a change in the DNA), which leads to changes in specific substances in the leukemia cells. This trial will investigate whether the addition of the new drugs Ivosidenib (for patients with IDH1 mutation) or Enasidenib (for patients with IDH2 mutation) to the standard treatment of chemotherapy controle the disease more effectively and for a longer period.

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Detailed Description

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Conditions

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Acute Myeloid Leukemia Myelodysplastic Syndrome With Excess Blasts-2

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Arm A: Placebo

Cycle 1: day 1-start cycle 2 \| Cycle 2: day 1 - start consolidation treatment \| Consolidation treatment: day 1 - start maintenance \| Maintenance treatment: day 1- day 730 (2 years) \|

The dosage for Placebo for AG-120 (IDH1): 500 mg dose/day

The dosage for Placebo for AG-221 (IDH2): 100mg dose/day

Group Type PLACEBO_COMPARATOR

Placebo for AG-120

Intervention Type DRUG

250mg tablets

Placebo for AG-221

Intervention Type DRUG

100mg tablets

Arm B: Ivosidenib (IDH1) or Enasidenib (IDH2)

Cycle 1: day 1-start cycle 2 \| Cycle 2: day 1 - start consolidation treatment \| Consolidation treatment: day 1 - start maintenance \| Maintenance treatment: day 1- day 730 (2 years) \|

The dosage for AG-120 (IDH1): 500 mg dose/day

The dosage for AG-221 (IDH2): 100mg dose/day

Group Type EXPERIMENTAL

AG-120

Intervention Type DRUG

250mg tablets

AG-221

Intervention Type DRUG

100mg tablets

Interventions

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AG-120

250mg tablets

Intervention Type DRUG

Placebo for AG-120

250mg tablets

Intervention Type DRUG

AG-221

100mg tablets

Intervention Type DRUG

Placebo for AG-221

100mg tablets

Intervention Type DRUG

Other Intervention Names

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Ivosidenib Enasidenib

Eligibility Criteria

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Inclusion Criteria

* Age ≥18 years
* Newly diagnosed AML or MDS-EB2 defined according to WHO criteria, with a documented IDH1 or IDH2 gene mutation (as determined by the clinical trial assay) at a specific site (IDH1 R132, IDH2 R140, IDH2 R172). AML may be secondary to prior hematological disorders, including MDS, and/or therapy-related (in which prior disease should have been documented to have existed for at least 3 months). Patients may have had previous treatment with hypomethylating agents (HMAs) for MDS. HMAs have to be stopped at least four weeks before registration
* Patients with dual mutant FLT3 and IDH1 or IDH2 mutations may be enrolled only if, for medical or other reasons, treatment with a FLT3 inhibitor is not considered.
* Considered to be eligible for intensive chemotherapy.
* ECOG/WHO performance status ≤ 2
* Adequate hepatic function as evidenced by:

* Serum total bilirubin ≤ 2.5 × upper limit of normal (ULN) unless considered due to Gilbert's disease (e.g. a mutation in UGT1A1) (only for patients in IDH2 cohort), or leukemic involvement of the liver - following written approval by the (Co)Principal Investigator.
* Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤ 3.0 × ULN, unless considered due to leukemic involvement of the liver, following written approval by the Principal Investigator.
* Adequate renal function as evidenced by creatinine clearance \> 40 mL/min based on the Cockroft-Gault formula for glomerular filtration rate (GFR).
* Able to understand and willing to sign an informed consent form (ICF).
* Written informed consent

Female patient must either:

o Be of nonchildbearing potential: Postmenopausal (defined as at least 1 year without any menses) prior to screening, or Documented surgically sterile or status posthysterectomy (at least 1 month prior to screening)

o Or, if of childbearing potential: Agree not to try to become pregnant during the study and for 6 months after the final study drug administration And have a negative urine or serum pregnancy test at screening And, if heterosexually active, agree to consistently use highly effective\* contraception per locally accepted standards in addition to a barrier method starting at screening and throughout the study period and for 6 months after the final study drug administration.

* Highly effective forms of birth control include:

* Consistent and correct usage of established hormonal contraceptives that inhibit ovulation,
* Established intrauterine device (IUD) or intrauterine system (IUS),
* Bilateral tubal occlusion,
* Vasectomy (A vasectomy is a highly effective contraception method provided the absence of sperm has been confirmed. If not, an additional highly effective method of contraception should be used.)
* Male is sterile due to a bilateral orchiectomy.
* Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual activity during the entire period of risk associated with the study drug. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the patient.
* List is not all inclusive. Prior to enrollment, the investigator is responsible for confirming patient will utilize highly effective forms of birth control per the requirements of the CTFG Guidance document 'Recommendations related to contraception and pregnancy testing in clinical trials', September 2014 (and any updates thereof) during the protocol defined period.

* Female patient must agree not to breastfeed starting at screening and throughout the study period, and for 2 months and 1 week after the final study drug administration.
* Female patient must not donate ova starting at screening and throughout the study period, and for 6 months after the final study drug administration.

* Male patient and their female partners who are of childbearing potential must be using highly effective contraception per locally accepted standards in addition to a barrier method starting at screening and continue throughout the study period and for 4 months and 1 week after the final study drug administration
* Male patient must not donate sperm starting at screening and throughout the study period and for 4 months and 1 week after the final study drug administration.

* Subject agrees not to participate in another interventional study while on treatment

Exclusion Criteria

* Prior chemotherapy for AML or MDS-EB2 (with the exception of HMA). Hydroxyurea is allowed for the control of peripheral leukemic blasts in patients with leukocytosis (e.g., white blood cell \[WBC\] counts \> 30x109/L).
* Dual IDH1 and IDH2 mutations.
* Acute promyelocytic leukemia (APL) with PML-RARA or one of the other pathognomonic variant fusion genes/chromosome translocations.
* Blast crisis after chronic myeloid leukemia (CML).
* Known allergy or suspected hypersensitivity to Ivosidenib or Enasidenib and/or any exipients.
* Taking medications with narrow therapeutic windows with potential interaction with investigational medication (see Appendix I), unless the patient can be transferred to other medications prior to enrolling or unless the medications can be properly monitored during the study.
* Taking P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) transporter-sensitive substrate medications (see Appendix J) unless the patient can be transferred to other medications within ≥ 5 half-lives prior to administration of ivosidenib or enasidenib, or unless the medications can be properly monitored during the study.
* Breast feeding at the start of study treatment.
* Active infection, including hepatitis B or C or HIV infection that is uncontrolled at randomization. An infection controlled with an approved or closely monitored antibiotic/antiviral/antifungal treatment is allowed.
* Patients with a currently active second malignancy. Patients are not considered to have a currently active malignancy if they have completed therapy and are considered by their physician to be at \< 30% risk of relapse within one year. However, patients with the following history/concurrent conditions are allowed:

* Basal or squamous cell carcinoma of the skin
* Carcinoma in situ of the cervix
* Carcinoma in situ of the breast
* Incidental histologic finding of prostate cancer
* Significant active cardiac disease within 6 months prior to the start of study treatment, including New York Heart Association (NYHA) Class III or IV congestive heart failure (appendix G); myocardial infarction, unstable angina and/or stroke; or left ventricular ejection fraction (LVEF) \< 40% by ultrasound or MUGA scan obtained within 28 days prior to the start of study treatment.
* QTc interval using Fridericia's formula (QTcF) ≥ 450 msec or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure,family history of long QT interval syndrome). Prolonged QTc interval associated with bundle branch block or pacemaking is permitted with written approval of the Principal Investigator.
* Taking medications that are known to prolong the QT interval (see Appendix K), unless deemed critical and without a suitable alternative. In those cases, they may be administered, but with proper monitoring (see section 10.2, Table 13).
* Dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of orally administered drugs.
* Clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid (CSF) during screening is only required if there is a clinical suspicion of CNS involvement by leukemia during screening.
* A known medical history of progressive multifocal leukoencephalopathy (PML).
* Immediately life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or severe disseminated intravascular coagulation
* Any other medical condition deemed by the Investigator to be likely to interfere with a patient's ability to give informed consent or participate in the study.
* Any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Deutsch-Österreichische Studiengruppe Akute Myeloische Leukämie (AMLSG)

UNKNOWN

Sponsor Role collaborator

Stichting Hemato-Oncologie voor Volwassenen Nederland

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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B.J. Wouters

Role: PRINCIPAL_INVESTIGATOR

Erasmus MC / HOVON

Locations

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AU-Adelaide-FLINDERS

Adelaide, , Australia

Site Status

AU-Adelaide-RAH

Adelaide, , Australia

Site Status

AU-Brisbane-PAH

Brisbane, , Australia

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AU-Camperdown-RPA

Camperdown, , Australia

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AU-Canberra-CANBERRAHOSPITAL

Canberra, , Australia

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AU-Douglas-TOWNSVILLE

Douglas, , Australia

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AU-Hobart TAS-RHOBART

Hobart, , Australia

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AU-Launceston TAS-LAUNCESTON

Launceston, , Australia

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AU-Melbourne-ALFRED

Melbourne, , Australia

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AU-Melbourne-AUSTIN

Melbourne, , Australia

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AU-Melbourne-MONASH

Melbourne, , Australia

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AU-Melbourne-RMELBOURNE

Melbourne, , Australia

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AU-Melbourne-SVHM

Melbourne, , Australia

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AU-Perth-FSH

Perth, , Australia

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AU-Perth-RPH

Perth, , Australia

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AU-Perth-SCGH

Perth, , Australia

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AU-Sydney-CONCORD

Sydney, , Australia

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AU-Sydney-RNSH

Sydney, , Australia

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AU-Sydney-SVHS

Sydney, , Australia

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AU-Sydney-WSAH

Sydney, , Australia

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AU-Waratah-CALVARYMATER

Waratah, , Australia

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AT-Graz-MEDUNIGRAZ

Graz, , Austria

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AT-Innsbruck-IMED

Innsbruck, , Austria

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AT-Linz-ORDENSKLINIKUM

Linz, , Austria

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AT-Vienna-HANUSCH

Vienna, , Austria

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BE-Antwerpen-ZNASTUIVENBERG

Antwerp, , Belgium

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BE-Brugge-AZBRUGGE

Bruges, , Belgium

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BE-Brussel-BORDET

Brussels, , Belgium

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BE-Brussel-UZBRUSSEL

Brussels, , Belgium

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BE-Bruxelles-STLUC

Brussels, , Belgium

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BE-Gent-UZGENT

Ghent, , Belgium

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BE-Haine-Saint-Paul-JOLIMONT

Haine-Saint-Paul, , Belgium

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BE-Hasselt-VIRGAJESSE

Hasselt, , Belgium

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BE-Leuven-UZLEUVEN

Leuven, , Belgium

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BE-Liege-CHRCITADELLE

Liège, , Belgium

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BE-Liege-CHULIEGE

Liège, , Belgium

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BE-Roeselare-AZDELTA

Roeselare, , Belgium

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BE-Yvoir-MONTGODINNE

Yvoir, , Belgium

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EE-Tartu-TARTU

Tartu, , Estonia

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FI-Helsinki-HUS

Helsinki, , Finland

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FI-Tampere-TAYS

Tampere, , Finland

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FR-Amiens-CHUAMIENS

Amiens, , France

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FR-Angers-CHUANGERS

Angers, , France

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FR-Argenteuil-CHARGENTEUIL

Argenteuil, , France

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FR-Bayonne-CHCOTEBASQUE

Bayonne, , France

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FR-Besançon Cedex-JEANMINJOZ

Besançon, , France

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FR-Bobigny-AVICENNE

Bobigny, , France

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FR-Chambery-CHMETROPOLESAVOIE

Chambéry, , France

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FR-Le Chesnay cedex-CHVERSAILLES

Chesnay, , France

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FR-Clamart-HIAPERCY

Clamart, , France

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FR-Clermont-Ferrand-ESTAING

Clermont-Ferrand, , France

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FR-Créteil cedex-CHUMONDOR

Créteil, , France

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FR-Grenoble cedex 9-CHUGRENOBLE

Grenoble, , France

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FR-Lens-CHLENS

Lens, , France

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FR-Lille-CHULILLE

Lille, , France

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FR-Limoges-CHULIMOGES

Limoges, , France

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FR-Lyon Pierre Benite cedex-LYONSUD

Lyon, , France

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FR-Lyon-LEONBERARD

Lyon, , France

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FR-Marseille-IPC

Marseille, , France

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FR-Montpellier-STELOI

Montpellier, , France

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FR-Mulhouse-GHRMSA

Mulhouse, , France

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FR-Nantes-CHUNANTES

Nantes, , France

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FR-Nice-CAL

Nice, , France

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FR-Nice-LARCHET

Nice, , France

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FR-Orléans-CHORLEANS

Orléans, , France

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FR-Paris cedex 10-SAINTLOUIS

Paris, , France

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FR-Paris cedex 12-SAINTANTOINE

Paris, , France

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FR-Paris cedex 15-NECKER

Paris, , France

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FR-Pessac Cedex-CHUBORDEAUX

Pessac, , France

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FR-Poitiers-CHUPOITERS

Poitiers, , France

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FR-Reims-CHREIMS

Reims, , France

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FR-Rennes cedex 9-CHURENNES

Rennes, , France

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FR-Rouen cedex-BECQUEREL

Rouen, , France

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FR-Strasbourg cedex-HAUTEPIERRE

Strasbourg, , France

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FR-Toulouse-CHUTOULOUSE

Toulouse, , France

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FR-Tours cedex-BRETONNEAU

Tours, , France

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FR-Vandoeuvre Les Nancy-CHRUNANCY

Vandœuvre-lès-Nancy, , France

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FR-Villejuif-GUSTAVEROUSSY

Villejuif, , France

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DE-Bad Saarow-HELIOSBADSAAROW

Bad Saarow, , Germany

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DE-Berlin-CAMPUSBENFRANKLIN

Berlin, , Germany

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DE-Berlin-CAMPUSVIRCHOW

Berlin, , Germany

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DE-Berlin-VIVANTESNEUKOLLN

Berlin, , Germany

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DE-Berlin-VIVANTESURBAN

Berlin, , Germany

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DE-Bochum-RUB

Bochum, , Germany

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DE-Bonn-UNIBONN

Bonn, , Germany

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DE-Braunschweig-KLINIKUMBRAUNSCHWEIG

Braunschweig, , Germany

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DE-Bremen-KBM

Bremen, , Germany

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DE-Dortmund-JOHODORTMUND

Dortmund, , Germany

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DE-Düsseldorf-MEDUNIDUESSELDORF

Düsseldorf, , Germany

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DE-Essen-KEM

Essen, , Germany

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DE-Esslingen-KLINIKUMESSLINGEN

Esslingen am Neckar, , Germany

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DE-Flensburg-MALTESER

Flensburg, , Germany

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DE-Giessen-UKGM

Giessen, , Germany

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DE-Goch-KKLE

Goch, , Germany

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DE-Hamburg-ASKLEPIOSSTGEORG

Hamburg, , Germany

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DE-Hamburg-ASKLEPIOS

Hamburg, , Germany

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DE-Hamburg-UKE

Hamburg, , Germany

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DE-Hamm-EVKHAMM

Hamm, , Germany

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DE-Hanau-KLINIKUMHANAU

Hanau, , Germany

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DE-Hannover-MHHANNOVER

Hanover, , Germany

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DE-Hannover-SILOAHKRH

Hanover, , Germany

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DE-Herne-MARIENHOSPITALHERNE

Herne, , Germany

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DE-Homburg-UNIKLINIKSAARLAND

Homburg, , Germany

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DE-Karlsruhe-KLINIKUMKARLSRUHE

Karlsruhe, , Germany

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DE-Lebach-CARITASKHLEBACH

Lebach, , Germany

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DE-Lemgo-KLINIKUMLIPPE

Lemgo, , Germany

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DE-Ludwigshafen-KLILU

Ludwigshafen, , Germany

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DE-Luedenscheid-KLINIKUMLUEDENSCHEID

Lüdenscheid, , Germany

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DE-Magdeburg-OVGU

Magdeburg, , Germany

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DE-Mainz-KLINKUNIMAINZ

Mainz, , Germany

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DE-Mainz-UNIMEDIZINMAINZ

Mainz, , Germany

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DE-Meschede-HOCHSAUERLAND

Meschede, , Germany

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DE-Minden-MUEHLENKREISKLINKEN

Minden, , Germany

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DE-München-IRZTUM

München, , Germany

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DE-München-MEDUNIMUNCHIN

München, , Germany

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DE-Offenburg-ORTENAUKLINIKUM

Offenburg, , Germany

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DE-Oldenburg-KLINIKUMOLDENBURG

Oldenburg, , Germany

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DE-Passau-KLINIKUMPASSAU

Passau, , Germany

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DE-Stuttgart-DIAKSTUTTGART

Stuttgart, , Germany

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DE-Stuttgart-KLINIKUMSTUTTGART

Stuttgart, , Germany

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DE-Traunstein-TSSOB

Traunstein, , Germany

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DE-Trier-MUTTERHAUS

Trier, , Germany

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DE-Tübingen-MEDUNITUEBINGEN

Tübingen, , Germany

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DE-Ulm-UNIKLINKULM

Ulm, , Germany

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DE-Villingen-Schwenningen-SBKVS

Villingen-Schwenningen, , Germany

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DE-Wuppertal-HELIOSGESUNDHEIT

Wuppertal, , Germany

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IE-Cork-CUH

Cork, , Ireland

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IE-Dublin 8-STJAMES

Dublin, , Ireland

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IE-Dublin 9-BEAUMONT

Dublin, , Ireland

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IE-Galway-UHGALWAY

Galway, , Ireland

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LT-Vilnius-SANTA

Vilnius, , Lithuania

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LU-Luxembourg-CHL

Luxembourg, , Luxembourg

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NL-Den Bosch-JBZ

's-Hertogenbosch, , Netherlands

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NL-Amersfoort-MEANDERMC

Amersfoort, , Netherlands

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NL-Amsterdam-AMC

Amsterdam, , Netherlands

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NL-Amsterdam-OLVG

Amsterdam, , Netherlands

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NL-Amsterdam-VUMC

Amsterdam, , Netherlands

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NL-Arnhem-RIJNSTATE

Arnhem, , Netherlands

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NL-Breda-AMPHIA

Breda, , Netherlands

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NL-Delft-RDGG

Delft, , Netherlands

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NL-Dordrecht-ASZ

Dordrecht, , Netherlands

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NL-Eindhoven-MAXIMAMC

Eindhoven, , Netherlands

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NL-Enschede-MST

Enschede, , Netherlands

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NL-Groningen-UMCG

Groningen, , Netherlands

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NL-Leeuwarden-MCL

Leeuwarden, , Netherlands

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NL-Leiden-LUMC

Leiden, , Netherlands

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NL-Maastricht-MUMC

Maastricht, , Netherlands

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NL-Nieuwegein-ANTONIUS

Nieuwegein, , Netherlands

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NL-Nijmegen-RADBOUDUMC

Nijmegen, , Netherlands

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NL-Rotterdam-ErasmusMC

Rotterdam, , Netherlands

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NL-Den Haag-HAGA

The Hague, , Netherlands

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NL-Utrecht-UMCUTRECHT

Utrecht, , Netherlands

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NL-Zwolle-ISALA

Zwolle, , Netherlands

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NO-Bergen-HELSEBERGEN

Bergen, , Norway

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NO-Oslo-OSLOUH

Oslo, , Norway

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NO-Stavanger-HELSESTAVANGER

Stavanger, , Norway

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NO-Tromsø-NORTHNOORWEGEN

Tromsø, , Norway

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NO-Trondheim-STOLAV

Trondheim, , Norway

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ES-Barcelona-CLINICUB

Barcelona, , Spain

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ES-Barcelona-GERMANTRIALS

Barcelona, , Spain

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ES-Barcelona-ICODURANREYNALS

Barcelona, , Spain

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ES-Barcelona-MUTUATERRASSA

Barcelona, , Spain

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ES-Barcelona-PARCDESALUTMAR

Barcelona, , Spain

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ES-Barcelona-SANTPAU

Barcelona, , Spain

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ES-Barcelona-VHEBRON

Barcelona, , Spain

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ES-Girona-ICSTRUETA

Girona, , Spain

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ES-Madrid-CSGREGORIOMARANON

Madrid, , Spain

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ES-Palma-SSIB

Palma, , Spain

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ES-Tarragona-JOAN

Tarragona, , Spain

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ES-Valencia-MALVARROSA

Valencia, , Spain

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SE-Lund-SUH

Lund, , Sweden

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SE-Stockholm-KAROLINSKAHUDDINGE

Stockholm, , Sweden

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SE-Uppsala-UPPSALAUH

Uppsala, , Sweden

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CH-Basel-USB

Basel, , Switzerland

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CH-Bellinzona-IOSI

Bellinzona, , Switzerland

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CH-Bern-INSEL

Bern, , Switzerland

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CH-Fribourg-HFR

Fribourg, , Switzerland

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CH-Geneve (14)-HCUGE

Geneva, , Switzerland

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CH-Luzern-LUKS

Lucerne, , Switzerland

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CH-St. Gallen-KSSG

Sankt Gallen, , Switzerland

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CH-Zürich-USZ

Zurich, , Switzerland

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Countries

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Australia Austria Belgium Estonia Finland France Germany Ireland Lithuania Luxembourg Netherlands Norway Spain Sweden Switzerland

Related Links

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http://www.hovon.nl

HOVON website

Other Identifiers

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2018-000451-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

HO150

Identifier Type: -

Identifier Source: org_study_id

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