Severe Aplastic AnemiaRACE2NCT05049668
RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
An observational study of Severe Aplastic Anemia, run by European Society for Blood and Marrow Transplantation at 1 site in France.
Trial summary
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- ATGAM plus CsA with or without Eltrombopag
- How many people
- 197 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2021to Dec 2034
- How long will I take part?
- 15 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
- Child, Adult, Older adult
Study population: Patients who participated in the initial RACE 1 trial
Who may be able to join
- Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.
- Subject has provided informed consent to participate in long-term data collection
Who cannot join
- None
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Long-term Follow-up of Patients Participating in RACE: the Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
Read the detailed description
Patients will be followed up annually, according to standard of care.
All diagnostic and therapeutic intervention will be performed according to standard of care, at discretion of the treating physician. In particular, during the study no extra Peripheral blood or Bone Marrow sampling will be performed, in addition to routine sampling for morphology and karyotype surveillance.
Molecular analysis by Next Generation Sequencing (NGS) will also be collected if the centre is doing this on a routine basis.
No Investigational Medicinal Product (IMP) or Non-Investigational Medicinal Product (NIMP) will be given to the patients.
- Conditions
- Severe Aplastic Anemia
- Being tested
- ATGAM plus CsA with or without Eltrombopag
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATGAM plus CsA with or without EltrombopagDrug
Also called hATG+CsA +/- Revolade
Standard treatment with or without Eltrombopag
| Study group | Role | What they receive |
|---|---|---|
| RACE 1 patients | After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study Receives: Drug: ATGAM plus CsA with or without Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Failure Free Survival
Failure Free Survival, where treatment failure is defined as one or more of the following: death, relapse, malignant clonal evolution, need for further (e.g. transplant)
Measured: 15 years
Secondary outcomes (12)
Response Rate: number of patients who reach a hematological response
Response Rate
Measured: 15 years
Overall Survival
Overall Survival
Measured: 15 years
Cumulative incidence of relapse after response
Cumulative incidence of relapse after initial hematological response (complete or partial)
Measured: 15 years
Cumulative incidence of clonal evolution
Cumulative incidence of clonal evolution: Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or karyotypic abnormalities qualifying for the diagnosis of MDS (see World Health Organization (WHO) 2016)
Measured: 15 years
Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)
Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment
Measured: 15 years
Cumulative incidence of Solid Tumors
Cumulative incidence of solid tumours
Measured: 15 years
Number of patients who need a Human Stem Cell Transplantation (HSCT)
Need for HSCT
Measured: 15 years
Number of patients who need additional IST
Need for additional intensive Imune Suppressive Therapy (IST) (e.g. ATG, alemtuzumab or cyclophosphamide-based, or any other lymphocyte-depleting agent)
Measured: 15 years
Number of patients who need Maintenance IST (e.g. CsA beyond 2 years)
Need for maintenance intensive IST (e.g. CsA beyond 2 years)
Measured: 15 years
Number of patients who need additional Eltrombopag (EPAG)
Need for additional EPAG
Measured: 15 years
Number of patients who need any other approved Aplastic Anemia (AA) treatment
Need for any other approved Aplastic Anemia (AA) treatment
Measured: 15 years
Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)
As tracked by somatic mutations in genes associated with myeloid disorders
Measured: 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2021
- Study startOct 2021 (estimated)
- Primary completionDec 2033 (estimated)
- Study completionDec 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic AnemiaStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 197 participants (estimated)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- European Society for Blood and Marrow Transplantation
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Regis Peffault de Latour, Prof, MD, Principal investigator, Hospital St. Louis, Paris, FranceAntonio M Risitano, MD, PhD, Principal investigator, Hospital Avellino, Napels, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05049668
- Sponsor's protocol ID
- EBMT-RACE 2
- Acronym
- RACE2
- Status last verified
- Sep 2021
- First submitted
- 13 Sep 2021
- Last updated
- 27 Sep 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Patients Participating in RACE: the Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients
- ClinicalTrials.gov ID
- NCT05049668
- Acronym / sponsor ID
- RACE2 / EBMT-RACE 2
- Conditions
- Severe Aplastic Anemia
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 197 participants (estimated)
- Lead sponsor
- European Society for Blood and Marrow TransplantationResearch network
- Study start
- Oct 2021 (estimated)
- Primary completion
- Dec 2033 (estimated)
- Study completion
- Dec 2034 (estimated)
- First posted
- 20 Sep 2021 (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.
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
Detailed description
Patients will be followed up annually, according to standard of care.
All diagnostic and therapeutic intervention will be performed according to standard of care, at discretion of the treating physician. In particular, during the study no extra Peripheral blood or Bone Marrow sampling will be performed, in addition to routine sampling for morphology and karyotype surveillance.
Molecular analysis by Next Generation Sequencing (NGS) will also be collected if the centre is doing this on a routine basis.
No Investigational Medicinal Product (IMP) or Non-Investigational Medicinal Product (NIMP) will be given to the patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 15 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RACE 1 patients | – | Drug: ATGAM plus CsA with or without EltrombopagAfter exiting the RACE trial (NCT02099747) patient will be invited to participate in this study |
Who may qualify
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who participated in the initial RACE 1 trial
Key inclusion criteria (2 listed)
- Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.
- Subject has provided informed consent to participate in long-term data collection
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ATGAM plus CsA with or without EltrombopagAlso: hATG+CsA +/- RevoladeStandard treatment with or without Eltrombopag | Drug | RACE 1 patients |
Primary outcome
Failure Free Survival
Time frame: 15 years
Failure Free Survival, where treatment failure is defined as one or more of the following: death, relapse, malignant clonal evolution, need for further (e.g. transplant)
12 secondary outcomes
Response Rate: number of patients who reach a hematological response
Time frame: 15 years
Overall Survival
Time frame: 15 years
Cumulative incidence of relapse after response
Time frame: 15 years
Cumulative incidence of clonal evolution
Time frame: 15 years
Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)
Time frame: 15 years
Cumulative incidence of Solid Tumors
Time frame: 15 years
Number of patients who need a Human Stem Cell Transplantation (HSCT)
Time frame: 15 years
Number of patients who need additional IST
Time frame: 15 years
Number of patients who need Maintenance IST (e.g. CsA beyond 2 years)
Time frame: 15 years
Number of patients who need additional Eltrombopag (EPAG)
Time frame: 15 years
Number of patients who need any other approved Aplastic Anemia (AA) treatment
Time frame: 15 years
Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)
Time frame: 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2021.
- Individual participant dataSharing planned: No
Related trials for Severe Aplastic Anemia
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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.
Regis Peffault de Latour, Prof, MD principal investigator
Hospital St. Louis, Paris, France
Antonio M Risitano, MD, PhD principal investigator
Hospital Avellino, Napels, Italy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Severe Aplastic Anemia, run by European Society for Blood and Marrow Transplantation at 1 site 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 2 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: enrolling by invitation, as of the sponsor's last update on 27 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Failure Free Survival (time frame: 15 years). 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 27 Sep 2021.