Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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12 December 2020 |
Main ID: |
NCT02099747 |
Date of registration:
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06/03/2014 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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hATG+CsA vs hATG+CsA+Eltrombopag for SAA
RACE |
Scientific title:
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A 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. |
Date of first enrolment:
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July 2015 |
Target sample size:
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202 |
Recruitment status: |
Active, not recruiting |
URL:
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https://clinicaltrials.gov/show/NCT02099747 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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France
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Germany
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Italy
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Netherlands
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Spain
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Switzerland
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United Kingdom
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Contacts
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Name:
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Regis Peffault de Latour, MD, PhD |
Address:
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Telephone:
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Email:
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Affiliation:
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St. Louis Hospital, Haematology Division, Paris |
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Name:
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Antonio Risitano, MD, PhD |
Address:
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Telephone:
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Email:
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Affiliation:
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Federico II Medical School, Haematology Division, Napels |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Diagnosis of severe or very severe aplastic anemia, defined by [29]:
- At least two of the following:
- Absolute neutrophil counts <0.5 x 109/L (severe) or <0.2 x 109/L (very
severe)
- Platelet counts <20 x 109/L
- Reticulocyte counts <60 x 109/L
- Hypocellular bone marrow (<30% cellularity), without evidences of fibrosis or
malignant cells
2. Male or female age > 14 years;
3. Written informed consent
4. Willing and able to comply with all of the requirements and visits in the protocol
5. Understands that they can be randomised to either treatment arm
6. Negative pregnancy test for women of child bearing age
7. Written acceptance to use contraception (hormonal or barrier method of birth control;
abstinence) for the entire duration of study participation.
Exclusion Criteria:
1. Prior immunosuppressive therapy with ATG (horse of rabbit) or any other lymphocyte
depleting agent (i.e., alemtuzumab)
2. Eligibility to a sibling allogeneic stem cell transplantation
3. Evidence of a myelodysplastic syndrome, defined by the presence of myelodysplastic
features, excess of blasts or karyotypic abnormalities typical of MDS (according to
revised WHO 2008 criteria) [30],, as well as other primitive marrow disease. Patients
with diagnosis of AA with cytogenetic abnormalities which are recurrent in MDS
(according to revised WHO 2008 criteria) [30] should be included in this category, and
are not eligible for the study; patients with del(20q), +8 and -Y are not included in
this category, and thus are eligible for this study. The list of karyotypic
abnormalities which qualifies for the diagnosis of MDS are listed in the Appendix.
4. History or clinical suspect of constitutional aplastic anemia (i.e. Fanconi Anemia
with positive DEB/MMC test or Dyskeratosis Congenita)
5. History of malignant tumors with active disease within 5 years from enrollment, and/or
previous chemo-radiotherapy
6. Previous history of stem cell transplantation
7. Treatment with cyclosporin A unless
- <4 weeks of cyclosporin A treatment before enrolement and
- wash out period of 2 weeks before enrollment
8. CMV viremia, as defined by positive PCR or pp65 test
9. WHO performance status =3
10. Pregnant or breast feeding patients
11. Patients with hepatic, renal or cardiac failure, or any other life- threatening
concurrent disease
12. Patients with HIV infection
13. Patients without social health care assistance
14. Participation in another clinical trial within 1 month before the start of this trial
15. Patients and/or female partners of male patients not using highly effective method of
birth control i.e. intrauterine device (IUD), hormonal (oral pill, injection,
implants), tubal ligation or partner's vasectomy
16. subjects with known hypersensitivity to any of the component medications
The presence of a Paroxysmal Nocturnal Hemoglobinuria clone is not an exclusion criterion.
Age minimum:
15 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Severe Aplastic Anemia
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Intervention(s)
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Drug: CsA
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Drug: Eltrombopag
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Drug: hATG
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Primary Outcome(s)
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CR rate
[Time Frame: 3 months]
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Secondary Outcome(s)
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Comparison of number of SAEs between the two arms
[Time Frame: 2 year]
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Overall survival
[Time Frame: 2 year]
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Time to best heamatological response
[Time Frame: 2 year]
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Cumulative incidence of relapse rate
[Time Frame: 2 year]
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Heamatological Response at 6, 12, 18 and 24 months
[Time Frame: 2 year]
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Rate of CsA-independent hematological response at 24 months
[Time Frame: 2 year]
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Cumulative incidence of discontinuation of immunosuppressive therapy
[Time Frame: 2 year]
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Need for any supportive care
[Time Frame: 2 year]
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Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence
[Time Frame: 2 year]
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Cumulative incidences of clonal evolution
[Time Frame: 2 year]
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Cumulative incidence of response
[Time Frame: 2 year]
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Need for transfusions and number of transfusions required from treatment
[Time Frame: 2 year]
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Event-free survival
[Time Frame: 2 year]
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Secondary ID(s)
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2014-000363-40
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EBMT-RACE
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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