AzTrials
AzTrials

Sickle Cell DiseasePhase 3DREPA-RICNCT04046705

Comparison of Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease

A Phase 3 study testing Allogeneic matched related haematopoietic stem cell transplantation and Standard arm in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults. Moreover, many children and most adults develop a chronic debilitating condition due to organ damage. Read more about this study

Who is it for?
Ages 15 to 45 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
Allogeneic matched related haematopoietic stem cell transplantation and Standard arm
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
78 participants
Where
No sites listed yet
Study dates
15 Oct 2019to 15 Oct 2024
Phase
Phase 3
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 15 to 45 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • SCD patients (SS/Sβ0)
  • Aged :15 to 45 years
  • With at least one non-SCD sibling > 18 years from the same parental couple
  • Who presented at least one of the following criteria:
  • 3 VOC requiring hospitalization over one year within the past 2 years and at least a past history of an ACS
  • At least 1 ACS within the past 2 years requiring transfusions
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  • History of ischemic stroke or cerebral/cervical arterial stenosis > 50%
  • Pulmonary hypertension defined by mean pulmonary artery pressure ≥ 25 mmHg at rest, determined by right heart catherization
  • Requiring treatment with Hydroxyurea or chronic transfusion, or already treated by Hydroxyurea or transfusion program (TP) at inclusion.
  • Patients already receiving chronic transfusions for VOC or ACS not responding to hydroxyurea, will be eligible, provided at least 3 VOC requiring hospitalization/year within the 2 years before initiation of chronic transfusions, and at least past history of an ACS.
  • Contraception during all the study period by sirolimus for women of child bearing potential
  • Signed informed consent
  • Amenable to HLA typing, HSCT if an HLA-identical sibling is available.
  • Patients affiliated to the French health care insurance

Who cannot join

  • Performance status: ECOG scale>1
  • Pulmonary function: FEV1 et CVF < 50% of the theorical value
  • Post capillary and severe pre-capillary pulmonary hypertension with measured mean pulmonary artery pressure at rest >35 mmHg
  • Cardiac ejection fraction < 45%
  • Estimated glomerular fraction rate (GFR) <50ml/mn /1.73m2
  • Conjugate bilirubin >50 µmole/L, cirrhosis, ALT>4N
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  • Uncontrolled infection
  • Known hypersensitivity of alemtuzumab
  • Known hypersensitivity to murine proteins and to the following excepients: disodium edetate, polysorbate 80, potassium chloride, potassium phosphate monobasic, sodium chloride, dibasic sodium phosphate, water for injections
  • Positivity for HIV
  • Pregnancy or breast-feeding women
  • Alloimmunization or Delayed Hemolytic Transfusion Reaction precluding red cell transfusions

Trial locations and contacts

No sites are listed yet.

Nathalie Dhedin contact

+33142385127

nathalie.dhedin@aphp.fr

Sylvie Chevret contact

+33142499742

sylvie.chevret@paris7.jussieu.fr

About this study

Official title: A Prospective Multicenter Trial Comparing Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen, With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease

Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults. Moreover, many children and most adults develop a chronic debilitating condition due to organ damage. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the unique curative approach; it allows the cure of more than 95% of children transplanted from a matched related donor (MRD) after a myeloablative conditioning regimen.To date, few studies have addressed the role of HSCT in SCD adults, due to the risk of graft versus host disease (GVHD) and to the toxicity expected in older patients with a higher risk of organ damage. The development of safe, non-myeloablative conditioning regimens that allow stable mixed chimerism and avoid GVHD appears as an attractive option for HSCT to cure adults with severe SCD. The investigators design a prospective multicenter trial targeting patients over 15 years with severe SCD, and compare non-myeloablative transplant (when a matched related donor (MRD) is identified) versus no HSCT (for patients lacking MRD). The main objective is to assess the benefit of HSCT on the 2-year event free survival compared to standard care. The primary endpoint is the 2-year event free survival.

Being tested
Allogeneic matched related haematopoietic stem cell transplantation, Standard arm
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Allogeneic matched related haematopoietic stem cell transplantationProcedure or surgery

    Allogeneic matched related haematopoietic stem cell transplantation after a reduced intensity conditioning regimen

  • Standard armOther

    In the standard arm, patients who will not be transplanted, will receive the best standard care according to their situation and their previous treatment: initiation of hydroxyurea, continuation or optimization of the dose of hydroxyurea, initiation or continuation of transfusion program, initiation of a new drug proved to improve SCD and having authorization to use in France

Study groups in NCT04046705
Study groupRoleWhat they receive
HLA matched haematopoietic stem cell transplantationExperimentalPeripheral blood stem cell from matched HLA related donor.
Receives: Procedure: Allogeneic matched related haematopoietic stem cell transplantation
Control armOtherBest standard care : Patients will receive the best standard care according to their situation and their previous treatment: initiation of Hydroxyurea, continuation or optimization of the dose of Hydroxyurea, initiation or continuation of TP, initiation of a new drug proved to improve SCD and having authorization to use in France.
Receives: Other: Standard arm

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 2 year event-free survival

    An event will be defined as :

    • death from any cause
    • or acute grade II-IV GVHD according to the Magic consortium 2016 classification or a moderate or severe chronic GVHD according to the NIH classification
    • or 3 hospitalizations for VOC defined according to usual criteria
    • or one ACS defined by usual clinical criteria and a pulmonary infiltrate on chest film and/or thoracic computed-tomography (CT) scan
    • or a stroke defined as a clinical event confirmed by an MRI
    • or a cerebral or cervical stenosis >25% in a new territory, or increase >25% of previous stenosis evaluated MRI and MRI
    • or a increased of at least +10% of tricuspid regurgitation velocity, (confirmed by 2 echocardiography performed with a delay of at least 3 months) compared with pre-inclusion value for patients with TRV≥2.7 at inclusion

    Measured: 2 years post-inclusion

Secondary outcomes (132)

  1. Overall survival

    Measured: 2 years post-inclusion

  2. Number of days requiring hospitalization

    Number of days requiring hospitalization at 1 year post-inclusion with exclusion of the 5 first months post-inclusion

    Measured: 1 year

  3. Number of days requiring hospitalization

    Number of days requiring hospitalization at 2 years post-inclusion with exclusion of the 5 first months post-inclusion

    Measured: 2 years post-inclusion

  4. Number of vaso-occlusive crisis (VOC) requiring hospitalization

    Measured: 1 year post-inclusion

  5. Number of vaso-occlusive crisis (VOC) requiring hospitalization

    Measured: 2 years post-inclusion

  6. Number of acute chest syndrome (ACS) requiring hospitalization

    Measured: 1 year post-inclusion

  7. Number of acute chest syndrome (ACS) requiring hospitalization

    Measured: 2 years post-inclusion

  8. Number of hospitalizations in intensive care unit

    Measured: 1 year post-inclusion

  9. Number of hospitalizations in intensive care unit

    Measured: 2 years post-inclusion

  10. Number of priapism

    Measured: 1 year post-inclusion

  11. Number of priapism

    Measured: 2 years post-inclusion

  12. Number of stroke episodes

    Measured: 1 year post-inclusion

  13. Number of stroke episodes

    Measured: 2 years post-inclusion

  14. LDH count

    Changes in LDH

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  15. Percentage of patients with an aminotransferase value higher than five times the normal value

    Changes in aminotransferase

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  16. Percentage of patients with a gamma-GT value higher than five times the normal value

    Changes in gamma-GT

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  17. Percentage of patients with an Alkaline phosphatase value higher than five times the normal value

    Changes in alkaline phosphatase

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  18. Percentage of patients with a bilirubin value higher than three times the normal value

    Changes in bilirubin

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  19. Percentage of patients with a prothrombin value less than 70%

    Changes in TP

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  20. Activated partial thromboplastin time higher than 1.5 times the normal value

    Changes in TCK

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  21. Rate of hemoglobin

    Changes in hemoglobin level

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  22. Hematocrit

    Changes in hematocrit

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  23. Mean corpuscular volume

    Changes in mean corpuscular volume

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  24. Hemoglobin variants

    Changes of percentage of hemoglobin variants

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  25. Reticulocyte count

    Changes in percentage of reticulocyte

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  26. White blood cells count

    Changes in white blood cells

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  27. Platelets counts

    Changes in platelet counts

    Measured: every 3 months (from inclusion to 24 months) and at Month 1, Month 2 and Month 3 in the transplant arm

  28. Microalbuminuria/creatininuria ratio

    Measured: at 3 months

  29. Microalbuminuria/creatininuria ratio

    Measured: at 6 months

  30. Microalbuminuria/creatininuria ratio

    Measured: at 12 months

  31. Microalbuminuria/creatininuria ratio

    Measured: at 24 months

  32. Ferritin level

    Measured: at 3 months

  33. Ferritin level

    Measured: at 6 months

  34. Ferritin level

    Measured: at 12 months

  35. Ferritin level

    Measured: at 24 months

  36. Transferrin saturation level

    Measured: at 3 months

  37. Percentage of transferrin saturation

    Measured: at 6 months

  38. Percentage of transferrin saturation

    Measured: at 12 months

  39. Percentage of transferrin saturation

    Measured: at 24 months

  40. LH count

    Gonadic function will be measured using LH

    Measured: at 24 months

  41. FSH count

    Gonadic function will be measured using FSH

    Measured: at 24 months

  42. Testosterone count

    Gonadic function will be measured using testosterone level in men

    Measured: at 24 months

  43. Spermogram

    Gonadic function will be measured using spermogram in men

    Measured: at 24 months

  44. Oestrogen count

    Gonadic function will be measured using oestrogen level in women

    Measured: at 24 months

  45. AMH count

    Gonadic function will be measured using AMH level in women

    Measured: at 24 months

  46. Incidence of amenorrhea

    Gonadic function will be measured using incidence of amenorrhea in women

    Measured: at 24 months

  47. Number of parity

    Measured: at 24 months

  48. Percentage of patients with a proliferative retinopathy

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 12 months

  49. Percentage of patients with a proliferative retinopathy

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 24 months

  50. Percentage of patients with a hemorrhagic retinopathy

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 12 months

  51. Percentage of patients with a hemorrhagic retinopathy

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 24 months

  52. Percentage of patients with retinal detachment

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 12 months

  53. Percentage of patients with retinal detachment

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 24 months

  54. Proportion of patients with keratitis

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 12 months

  55. Proportion of patients with keratitis

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 24 months

  56. Proportion of patients with uveitis

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 12 months

  57. Proportion of patients with uveitis

    Changes in retinopathy status (appearance, disappearance, improvement, aggravation)

    Measured: at 24 months

  58. Tricuspid regurgitant jet velocity

    Heart function will be assessed by a transthoracic echocardiography and using tricuspid régurgitant jet velocity

    Measured: at 12 months

  59. Tricuspid regurgitant jet velocity

    Heart function will be assessed by a transthoracic echocardiography and using tricuspid régurgitant jet velocity

    Measured: at 24 months

  60. Left atrial dimension

    Heart function will be assessed by a transthoracic echocardiography using left atrial dimension indexed to body surface

    Measured: at 12 months

  61. Left atrial dimension

    Heart function will be assessed by a transthoracic echocardiography using left atrial dimension indexed to body surface

    Measured: at 24 months

  62. Left ventricular dimension

    Heart function will be assessed by a transthoracic echocardiography using left ventricular dimension indexed to body surface

    Measured: at 12 months

  63. Left ventricular dimension

    Heart function will be assessed by a transthoracic echocardiography using left ventricular dimension indexed to body surface

    Measured: at 24 months

  64. Ventricular mass index value

    Heart function will be assessed by a transthoracic echocardiography using ventricular mass index

    Measured: at 12 months

  65. Ventricular mass index value

    Heart function will be assessed by a transthoracic echocardiography using ventricular mass index

    Measured: at 24 months

  66. Left ventricular ejection fraction

    Heart function will be assessed by a transthoracic echocardiography using left ventricular ejection fraction

    Measured: at 12 months

  67. Left ventricular ejection fraction

    Heart function will be assessed by a transthoracic echocardiography using left ventricular ejection fraction

    Measured: at 24 months

  68. Forced Expiratory Volume in one second (FEV)

    Lung function will be evaluated Forced Expiratory Volume in one second (FEV) , %

    Measured: at 12 months

  69. Forced Expiratory Volume in one second (FEV)

    Lung function will be evaluated Forced Expiratory Volume in one second (FEV) , %

    Measured: at 24 months

  70. DLCO

    Lung function will be evaluated using DLCO the diffusion capacity of carbon monoxide

    Measured: at 12 months

  71. DLCO

    Lung function will be evaluated using DLCO the diffusion capacity of carbon monoxide

    Measured: at 24 months

  72. Forced vital capacity

    Lung function will be evaluated using forced vital capacity (FVC)

    Measured: at 12 months

  73. Forced vital capacity

    Lung function will be evaluated using forced vital capacity (FVC)

    Measured: at 24 months

  74. 6 minutes walk test

    Lung function will be evaluated using 6 minutes walk test

    Measured: at 12 months

  75. 6 minutes walk test

    Lung function will be evaluated using 6 minutes walk test

    Measured: at 24 months

  76. Number of new episodes of avascular osteonecrosis

    Measured: at 24 months

  77. Number of patients for each location of new episodes of avascular osteonecrosis

    Location of new episodes of avascular osteonecrosis will be assessed using radiography and magnetic resonance imaging

    Measured: at 24 months

  78. Fractures

    Number of new episodes of fractures

    Measured: at 24 months

  79. Central nervous system function

    Central nervous system function will be assessed using magnetic resonance Imaging with ARM/MRI

    Measured: at 12 months

  80. Central nervous system function

    Central nervous system function will be assessed using magnetic resonance Imaging with ARM/MRI

    Measured: at 24 months

  81. Iron overload

    Iron overload will be assessed using liver and heart magnetic resonance Imaging in patients with ferritin > 1000 microg/L

    Measured: at inclusion

  82. Iron overload

    Iron overload will be assessed using liver and heart magnetic resonance Imaging in patients with ferritin > 1000 microg/L

    Measured: at 12 months

  83. Iron overload

    Iron overload will be assessed using liver and heart magnetic resonance Imaging in patients with ferritin > 1000 microg/L

    Measured: at 24 months

  84. Red blood cell packed transfused

    Number of red blood cell packed transfused from 6 months post-inclusion (pre and early post-transplant transfusion are a standard of care and may not be counted)

    Measured: at 24 months

  85. Number of delayed hemolytic transfusion reaction (DHTR)

    DHTR will be defined as associated with hemoglobinuria/dark urine in the month following transfusion +/- bone pain with increased hemolytic markers and drop in HbA or new RBC allo-Ab

    Measured: at 3 months

  86. Number of delayed hemolytic transfusion reaction (DHTR)

    DHTR will be defined as associated with hemoglobinuria/dark urine in the month following transfusion +/- bone pain with increased hemolytic markers and drop in HbA or new RBC allo-Ab

    Measured: at 6 months

  87. Number of delayed hemolytic transfusion reaction (DHTR)

    DHTR will be defined as associated with hemoglobinuria/dark urine in the month following transfusion +/- bone pain with increased hemolytic markers and drop in HbA or new RBC allo-Ab

    Measured: at 12 months

  88. Number of delayed hemolytic transfusion reaction (DHTR)

    DHTR will be defined as associated with hemoglobinuria/dark urine in the month following transfusion +/- bone pain with increased hemolytic markers and drop in HbA or new RBC allo-Ab

    Measured: at 24 months

  89. Proportion of patients with new RBC alloantibodies

    New RBC alloantibodies will be assessed using blood test

    Measured: at 3 months

  90. Proportion of patients with new RBC alloantibodies

    New RBC alloantibodies will be assessed using blood test

    Measured: at 6 months

  91. Proportion of patients with new RBC alloantibodies

    New RBC alloantibodies will be assessed using blood test

    Measured: at 12 months

  92. Proportion of patients with new RBC alloantibodies

    New RBC alloantibodies will be assessed using blood test

    Measured: at 24 months

  93. Percentage of patients with an oral opioid consumption

    Measured: at 3 months

  94. Percentage of patients with an oral opioid consumption

    Measured: at 6 months

  95. Percentage of patients with an oral opioid consumption

    Measured: at 12 months

  96. Percentage of patients with an oral opioid consumption

    Measured: at 24 months

  97. Quality of life evaluated using MOS SF36 questionnaire

    Quality of life evaluated using MOS SF36 questionnaire (Medical Outcomes Study - 36-Item Short Form Health Survey). SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting. Items are grouped into three categories: functional status, well-being, overall health assessment. In two dimensions, the answer is binary (yes / no) and in the other 6 in ordinal quality (3 to 6 possible answers). For each dimension, the scores for the different items are coded and then summed and transformed linearly on a scale ranging from 0 to 100. A physical composite score and a mental composite score can be calculated according to an established algorithm

    Measured: at 3 months

  98. Quality of life evaluated using MOS SF36 questionnaire

    Quality of life evaluated using MOS SF36 questionnaire (Medical Outcomes Study - 36-Item Short Form Health Survey). SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting. Items are grouped into three categories: functional status, well-being, overall health assessment. In two dimensions, the answer is binary (yes / no) and in the other 6 in ordinal quality (3 to 6 possible answers). For each dimension, the scores for the different items are coded and then summed and transformed linearly on a scale ranging from 0 to 100. A physical composite score and a mental composite score can be calculated according to an established algorithm

    Measured: at 6 months

  99. Quality of life evaluated using MOS SF36 questionnaire

    Quality of life evaluated using MOS SF36 questionnaire (Medical Outcomes Study - 36-Item Short Form Health Survey). SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting. Items are grouped into three categories: functional status, well-being, overall health assessment. In two dimensions, the answer is binary (yes / no) and in the other 6 in ordinal quality (3 to 6 possible answers). For each dimension, the scores for the different items are coded and then summed and transformed linearly on a scale ranging from 0 to 100. A physical composite score and a mental composite score can be calculated according to an established algorithm

    Measured: at 12 months

  100. Quality of life evaluated using MOS SF36 questionnaire

    Quality of life evaluated using MOS SF36 questionnaire (Medical Outcomes Study - 36-Item Short Form Health Survey). SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting. Items are grouped into three categories: functional status, well-being, overall health assessment. In two dimensions, the answer is binary (yes / no) and in the other 6 in ordinal quality (3 to 6 possible answers). For each dimension, the scores for the different items are coded and then summed and transformed linearly on a scale ranging from 0 to 100. A physical composite score and a mental composite score can be calculated according to an established algorithm

    Measured: at 24 months

  101. Depression and Anxiety status

    Depression and anxiety will be assessed using Hospital Anxiety and Depression Scale (HADS) questionnaire. The HAD scale is a self-assessment scale for detecting states of depression and anxiety in the setting of an hospital medical outpatient clinic.

    HADS is a self-administered scale of 14 items which assessed levels of depression and anxiety, divided into 2 subscales of 7 items (Anxiety or HADS-A, Depression or HADS-D). Each item is scored on a scale of 0 to 3. A score is generated for each of the two sub-scales (sum of the 7 items, ranging from 0 to 21). Limit scores, for each of the scores, distinguish: non-cases or asymptomatic ones (score ≤ 7); probable or borderline cases (score 8-10); clearly or clinically symptomatic cases (score ≥ 11).

    Measured: at 3 months

  102. Depression and Anxiety status

    HADS is a self-administered scale of 14 items which assessed levels of depression and anxiety, divided into 2 subscales of 7 items (Anxiety or HADS-A, Depression or HADS-D). Each item is scored on a scale of 0 to 3. A score is generated for each of the two sub-scales (sum of the 7 items, ranging from 0 to 21). Limit scores, for each of the scores, distinguish: non-cases or asymptomatic ones (score ≤ 7); probable or borderline cases (score 8-10); clearly or clinically symptomatic cases (score ≥ 11).

    Measured: at 6 months

  103. Depression and Anxiety status

    Depression and anxiety will be assessed using Hospital Anxiety and Depression Scale (HADS) questionnaire. The HAD scale is a self-assessment scale for detecting states of depression and anxiety in the setting of an hospital medical outpatient clinic.

    HADS is a self-administered scale of 14 items which assessed levels of depression and anxiety, divided into 2 subscales of 7 items (Anxiety or HADS-A, Depression or HADS-D). Each item is scored on a scale of 0 to 3. A score is generated for each of the two sub-scales (sum of the 7 items, ranging from 0 to 21). Limit scores, for each of the scores, distinguish: non-cases or asymptomatic ones (score ≤ 7); probable or borderline cases (score 8-10); clearly or clinically symptomatic cases (score ≥ 11).

    Measured: at 12 months

  104. Weight

    Evolution of weight

    Measured: at 3 months

  105. Weight

    Evolution of weight

    Measured: at 6 months

  106. Weight

    Evolution of weight

    Measured: at 12 months

  107. Weight

    Evolution of weight

    Measured: at 24 months

  108. Number of severe infections

    A severe infection will be defined as a CTAE score of grade 3 or 4

    Measured: at 24 months

  109. GvHD incidence

    Measured: at 12 months

  110. GvHD incidence

    Measured: at 24 months

  111. Grading of GvHD

    Grading of GvHD will be assessed using magic consortium 2016 and NIH classification

    Measured: at 12 months

  112. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 1 month

  113. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 2 months

  114. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 3 months

  115. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletionchimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant.

    Measured: at 6 months

  116. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 9 months

  117. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 12 months

  118. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 18 months

  119. Chimerism in HSCT

    Chimerism in HSCT will be assessed on total blood population and on T subset. In patients transplanted in Paris area, a more extensive centralized chimerism will be performed if donor T lymphocyte chimerism will be under 70% at 3 months post-transplant. Chimerism analysis by PCR microsatellite or by quantitative real-time PCR of insertion/deletion

    Measured: at 24 months

  120. Number of days of hospitalization

    Number of days of hospitalization from inclusion

    Measured: Number of days of hospitalization from inclusion at M24

  121. RBC and WBC adherence

    Study of RBC and WBC adherence on lymphocytes subpopulation (including NK-T cells). Modulation of WBC and RBC surface markers by allograft.

    Measured: at inclusion

  122. RBC and WBC adherence

    Study of RBC and WBC adherence on lymphocytes subpopulation (including NK-T cells). Modulation of WBC and RBC surface markers by allograft.

    Measured: at 12 months

  123. RBC and WBC adherence

    Study of RBC and WBC adherence on lymphocytes subpopulation (including NK-T cells). Modulation of WBC and RBC surface markers by allograft.

    Measured: at 24 months

  124. Expression of RBC and WBC surface markers

    Expression of RBC and WBC surface markers on lymphocytes subpopulation

    Measured: at inclusion

  125. Expression of RBC and WBC surface markers

    Expression of RBC and WBC surface markers on lymphocytes subpopulation

    Measured: at 12 months

  126. Expression of RBC and WBC surface markers

    Expression of RBC and WBC surface markers on lymphocytes subpopulation

    Measured: at 24 months

  127. Mast cell mediator release

    Master cell mediator release will be assessed using plasma sample. Modulation of mast cell mediators release by allograft (tryptase, substance P and histamine)

    Measured: at inclusion

  128. Mast cell mediator release

    Master cell mediator release will be assessed using plasma sample. Modulation of mast cell mediators release by allograft (tryptase, substance P and histamine)

    Measured: at 12 months

  129. Mast cell mediator release

    Master cell mediator release will be assessed using plasma sample. Modulation of mast cell mediators release by allograft (tryptase, substance P and histamine)

    Measured: at 24 months

  130. Inflammatory cytokines

    Inflammatory cytokines will be measured using serum sample. Modulation of inflammatory cytokine (TNF-alpha, IL-1, IL-6...) by allograft

    Measured: at inclusion

  131. Inflammatory cytokines

    Inflammatory cytokines will be measured using serum sample. Modulation of inflammatory cytokine (TNF-alpha, IL-1, IL-6...) by allograft

    Measured: at 12 months

  132. Inflammatory cytokines

    Inflammatory cytokines will be measured using serum sample. Modulation of inflammatory cytokine (TNF-alpha, IL-1, IL-6...) by allograft

    Measured: at 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Aug 2019
  2. Study start15 Oct 2019 (estimated)
  3. Primary completion15 Oct 2024 (estimated)
  4. Study completion15 Oct 2024 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
78 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04046705
Sponsor's protocol ID
K170912J
Acronym
DREPA-RIC
Status last verified
Jul 2019
First submitted
15 Jul 2019
Last updated
23 Sep 2019
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Allogeneic matched related haematopoietic stem cell transplantation and Standard arm in people with Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris.

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 14 inclusion and 12 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 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.

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.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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 Nathalie Dhedin (+33142385127, nathalie.dhedin@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

2 year event-free survival (time frame: 2 years post-inclusion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04046705.

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 Sep 2019.

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