Haematological MalignancyNCT03750994
Economic Evaluation of Innovative Molecular Analyses in Onco-haematology
An observational study of Haematological Malignancy, run by Assistance Publique - Hôpitaux de Paris at 26 sites in France.
Trial summary
To evaluate the impact of innovative molecular diagnostics on the clinical management of patients with haematological malignancies via updated Appropriate-Prescribing-Guides including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and optimised use of costly novel therapeutics and… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haematological Malignancy
- How many people
- 3,960 participants
- Where
- 26 sitesFrance
- Study dates
- 18 Oct 2018to Oct 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients referred for molecular diagnosis at one of the certified molecular onco-haematology platforms in France.
Who may be able to join
- Patients with haematological malignancies referred for molecular diagnosis workup. RuBIH2 will focus on 5 clinical situations in onco-haematology:
- Myelodysplasia (MDS)
- Acute lymphocytic leukemia (T) (ALL)
- Lymphoproliferative disorders (LPD)
- Acute myeloblastic leukemia (AML)
- Myeloproliferative disorders (MPD)
Who cannot join
- Other haematological diseases not included in the list above.
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
France26 sites, 26 recruiting
- Recruiting
CHU Angers
Angers
Dr Odile Blanchet
- Recruiting
Hôpital Avicenne AP-HP
Bobigny
Professor Fanny Baran-Marszak
- Recruiting
CHU de Bordeaux
Bordeaux
Dr Audrey Bidet
- Recruiting
CHRU Brest
Brest
Dr Eric Lippert
- Recruiting
CHU Estaing
Clermont-Ferrand
Professor Marc Berger
- Recruiting
Hôpital Henri Mondor AP-HP
Créteil
Dr Dominique Bories
- Recruiting
CHRU Dijon Bourgogne
Dijon
Professor Mary Callanan
- Recruiting
Centre hospitalier régional universitaire de Lille
Lille, Hauts-de-France, 59000
Professor Claude PREUDHOMME, Claude.PREUDHOMME@chru-lille.fr
- Recruiting
CHU Limoges
Limoges
Dr David Rizzo
- Recruiting
CHU Lyon Sud Pierre Bénite
Lyon
Dr Pierre Sujobert
- Recruiting
CHU Montpellier
Montpellier
Dr Melissa Alamé
- Recruiting
CHU Hôtel Dieu
Nantes
Dr Yannick LE BRIS
- Recruiting
CHU Nice
Nice
Professor Sophie Raynaud
- Recruiting
Hôpital Pitié-Salpêtrière AP-HP
Paris, 75004
Professor Frédéric Davi
- Recruiting
Hôpital Robert Debré
Paris, 75004
Professor Hélène Cave
- Recruiting
Hôpital St Louis AP-HP
Paris, 75004
Dr Jean-Michel Cayuela
- Recruiting
Hôpital Cochin AP-HP
Paris
Professor Olivier Kosmider
- Recruiting
Hôpital Necker AP-HP
Paris
Professor Elizabeth Macintyre, elizabeth.macintyre@aphp.fr
- Recruiting
Hôpital Saint Antoine AP-HP
Paris
Professor François Delhommeau
- Recruiting
CHU Robert Debré Reims
Reims
Dr Pascale Cornillet-Lefebvre
- Recruiting
CHU Pontchaillou
Rennes
Professor Thierry Fest
- Recruiting
Centre Henri-Becquerel
Rouen
Dr Martine Becker
- Recruiting
Centre hospitalier universitaire de Saint-Étienne
Saint-Etienne
Dr Pascale Flandrin Gresta
- Recruiting
Hôpitaux Universitaires Strasbourg
Strasbourg
Dr Laurent Miguet
- Recruiting
CHU Toulouse
Toulouse
Professor Eric Delabesse
- Recruiting
Institut Gustave Roussy
Villejuif
Dr Christophe Marzac
About this study
Official title: Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)
To evaluate the impact of innovative molecular diagnostics on the clinical management of patients with haematological malignancies via updated Appropriate-Prescribing-Guides including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and optimised use of costly novel therapeutics and risk-adapted treatment. A micro-costing approach will be used to develop flat fee tarifs for NGS analyses.
Read the detailed description
The 12 somatic genetic cancer tests that have received temporary authorisation in France form the basis of this study. These tests are not yet in the national biology reimbursement nomenclature but are supported by the ministry of health in a temporary list "Le référentiel des actes innovants hors nomenclature de biologie et d'anatomocytopathologie" (RIHN).
The PRME RuBIH2 will focus on 5 clinical situations in onco-haematology:
- Myelodysplasia (MDS)
- Acute lymphocytic leukemia (T) (ALL)
- Lymphoproliferative disorders (LPD)
- Acute myeloblastic leukemia (AML)
- Myeloproliferative disorders (MPD)
The project is organised in 4 complementary work packages (WP): WP1 Cost evaluation, WP2 Prescription Guidelines, WP3 Clinical Validation and WP4 Budget Impact and Organisation.
WP1 will provide costing information on molecular tests and will build on previous studies conducted in France.
WP2 will update existing prescription guidelines based on evidence from the literature and evidence from the WP3. These prescription guidelines will in turn be valued and provide recommendations for a flat fee bundle for pre-specified clinical situations.
WP3 will provide evidence on the clinical impact of molecular diagnosis (in particular NGS) in the 5 pre-specified conditions. Changes in patient management will be measured using a prospective questionnaire for an estimated 3960 molecular tests. The impact of the test on the patient clinical pathway will be analysed. The impact of molecular tests on patient outcome will not be measured.
WP4 will use information from WP1 and WP2 to estimate the budget impact and to provide scenario analyses on the territorial organisation of molecular biology platforms. Based on the estimation of the national activity of molecular onco-haematology platforms the annual functioning budget required to implement molecular diagnosis in France will be estimated.
- Conditions
- Haematological Malignancy
- Medical subject headings
- Hematologic Neoplasms
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies.
Measured: 2 years
Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres.
Measured: 2 years
Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician.
Measured: 2 years
Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response
Measured: 2 years
Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient.
Measured: 2 years
Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2018
- Study start18 Oct 2018
- Primary completionOct 2020 (estimated)
- Study completionOct 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haematological MalignancyStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 3,960 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, France
- Responsible party
- Sponsor
- Investigators
- Claude Preudhomme, Professor, Principal investigator, DRCI AP-HP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03750994
- Sponsor's protocol ID
- RuBIH2
- Status last verified
- Nov 2018
- First submitted
- 11 Aug 2017
- Last updated
- 23 Nov 2018
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)
- ClinicalTrials.gov ID
- NCT03750994
- Acronym / sponsor ID
- RuBIH2
- Conditions
- Haematological Malignancy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 3,960 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Ministry of Health, France
- Study start
- 18 Oct 2018 (actual)
- Primary completion
- Oct 2020 (estimated)
- Study completion
- Oct 2020 (estimated)
- First posted
- 23 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the impact of innovative molecular diagnostics on the clinical management of patients with haematological malignancies via updated Appropriate-Prescribing-Guides including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and optimised use of costly novel therapeutics and risk-adapted treatment. A micro-costing approach will be used to develop flat fee tarifs for NGS analyses.
Detailed description
The 12 somatic genetic cancer tests that have received temporary authorisation in France form the basis of this study. These tests are not yet in the national biology reimbursement nomenclature but are supported by the ministry of health in a temporary list "Le référentiel des actes innovants hors nomenclature de biologie et d'anatomocytopathologie" (RIHN).
The PRME RuBIH2 will focus on 5 clinical situations in onco-haematology:
- Myelodysplasia (MDS)
- Acute lymphocytic leukemia (T) (ALL)
- Lymphoproliferative disorders (LPD)
- Acute myeloblastic leukemia (AML)
- Myeloproliferative disorders (MPD)
The project is organised in 4 complementary work packages (WP): WP1 Cost evaluation, WP2 Prescription Guidelines, WP3 Clinical Validation and WP4 Budget Impact and Organisation.
WP1 will provide costing information on molecular tests and will build on previous studies conducted in France.
WP2 will update existing prescription guidelines based on evidence from the literature and evidence from the WP3. These prescription guidelines will in turn be valued and provide recommendations for a flat fee bundle for pre-specified clinical situations.
WP3 will provide evidence on the clinical impact of molecular diagnosis (in particular NGS) in the 5 pre-specified conditions. Changes in patient management will be measured using a prospective questionnaire for an estimated 3960 molecular tests. The impact of the test on the patient clinical pathway will be analysed. The impact of molecular tests on patient outcome will not be measured.
WP4 will use information from WP1 and WP2 to estimate the budget impact and to provide scenario analyses on the territorial organisation of molecular biology platforms. Based on the estimation of the national activity of molecular onco-haematology platforms the annual functioning budget required to implement molecular diagnosis in France will be estimated.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients referred for molecular diagnosis at one of the certified molecular onco-haematology platforms in France.
Key inclusion criteria (6 listed)
- Patients with haematological malignancies referred for molecular diagnosis workup. RuBIH2 will focus on 5 clinical situations in onco-haematology:
Key exclusion criteria (1 listed)
- Other haematological diseases not included in the list above.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies.
Time frame: 2 years
Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres.
Time frame: 2 years
Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician.
Time frame: 2 years
Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response
Time frame: 2 years
Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient.
Time frame: 2 years
Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment.
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Claude Preudhomme, Professor principal investigator
DRCI AP-HP
26 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haematological Malignancy, run by Assistance Publique - Hôpitaux de Paris at 26 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Claude Preudhomme, Professor (+ 33 3 20 44 47 82, claude.preudhomme@chru-lille.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies. (time frame: 2 years). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Nov 2018.