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HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease (DREPHAPLO)

Primary Purpose

Sickle Cell Disease

Status

Active

Phase

Phase 2

Locations

France

Study Type

Interventional

Intervention

bone marrow transplant

About this trial

This is an interventional other trial for Sickle Cell Disease focused on measuring sickle cell disease, haploidentical, graft, marrow

Eligibility Criteria

13 Years - 40 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria recipient:

Age: 13 years-40 years
Severe Sickle cell with at least one of the following criteria:
- Stenosing vasculopathy with abnormal MRA despite prolonged transfusion program
- PAH confirmed by right catheterization with mPAP> 25mmHg
- Systolic ejection fraction <55% and tricuspid regurgitation speed> 2.5m /s at distance from an acute episode
- No possibility of blood transfusion or very complicated blood transfusion
- Report albumin / creatinine> 30 mg / mmol, confirmed 3 times, away at distance from acute episode and persistent despite hydroxyurea or IEC
- GFR <80ml / min /1.73m2 (CKD-Epi without ethnic criterion)
- Previous history of acute liver sequestration with liver failure
- Acute chest syndrome or vaso-occlusive crises under hydroxyurea
- Complications of sickle cell transfusion imposing an exchange program with no possible withdrawal beyond a period of one year
Not having geno-identical donor, but a haploidentical major donor (parent, sibling, adult child, or HbAA AS)
Having red and understood the information letter and signed the informed consent
Patients affiliated to a social security system (Social Security or Universal Medical Coverage)

Exclusion Criteria recipient:

Patient with a geno-identical donor
Performans status: ECOG> 1
lung disease: FEV1 and FVC <50% predicted,
score of PAH NYHA≥2
Liver disease with bilirubin> 50 .mu.mol / L
heart failure defined by NYHA≥3 score ejection fraction <45% or shortening fraction <24%
anti HLA alloimmunization against the donor or against red cell antigens of the donor
Serology or HIV viral load positively
Patients who for family, social or geographical reasons, do not wish to be regularly monitored in consultation
severe uncontrolled infection at the time of inclusion or graft
pregnant woman (positive beta HCG) or during lactation
incapable adult patient, trust, guardianship, or safeguard justice

Inclusion criteria donor

Age> 18 years and <60 years
Viral serologic economy allows the graft
No contraindication for general anesthesia
No contraindication the administration of G-CSF (the existence of sickle cell trait is not a contraindication)
Lack antigens HLA recognized by the recipient antibody
Hemoglobin S <50%
When several donors are compatible: choose according to the ABO recipient: prefer ABO compatibility and major incompatibility and minor incompatibility, and finally major and minor incompatibility.
Signature of informed consent
Non-inclusion criteria donors: β HCG positive or known pregnancy

Sites / Locations

CHU Henri-Mondor
intercommunal hospital of Créteil
CHU La Timone
Hospital Necker
Hospital Robert-Debré
Saint-Louis hospital
CHU Strasbourg

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

bone marrow transplant

Arm Description

All the included patient will receive an haploidentical bone marrow transplant with the following protocol concerning the conditioning and GvHD prevention Conditioning THYMOGLOBULINE : 0.5mg/kg at D-9 and 2 mg/kg at D-8 and D-7 THIOTEPA: 10mg/kg/j at D-7 CYCLOPHOSPHAMIDE (Endoxan®):14.5mg/kg/j at D-6 and D-5 FLUDARABINE (Fludara®): 30mg/m2 per Day from D-6 to D-2 TBI : 2GY : D -1 Graft : Injection at D0 of G-CSF-stimulated bone marrow transplant. Prophylaxis of GvHD CYCLOPHOSPHAMIDE (Endoxan®): 50mg/Kg per Day from D+3 to D+4 Sirolimus and MycophénolateMofétil (MMP) from D+5. In the absence of acute GvHD (aGvHD), stop of MMP to D35 and pursuit of sirolimus 1 year after the graft.

Outcomes

Primary Outcome Measures

Survival rate

Survival without sickle cell survival rate (electrophoresis of hemoglobin similar to that from the donor, that is to say a percentage of HbS not exceeding 10% of that of distance donor transfusions and that of a stable manner and without GvHDc other than mild

Secondary Outcome Measures

Survival rate

haematologic reconstitution

defined as a neutrophil count> 500 / mm3 and platelets> 20000 / mm3, for three consecutive days.

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Chimerism

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

hemoglobin electrophoresis

occurence of graft versus host disease

evaluated monthly from M1 to M6, and M9, M12, M24

grade of graft versus host disease

Incidence and grade of GvHD, toxic deaths and infectious complications and secondary cancer

occurrence of toxic deaths

occurrence of infectious complications

occurrence of secondary cancer

Lymphocyte immunophenotyping

Lymphocyte immunophenotyping T, B and NK + The Extended Phenotype including activation markers, assessment of naive people and memories, T reg populations etc) and plasma protein electrophoresis: 1 month, 3 months, 6 months, 12 months and 24 months post-transplantation.

ECOG score value

Index Trading ECOG complete physical examination with determination of weight

Assessment of sickle cell disease complications

Microalbuminuria, creatinuria; echocardiography for measurement of systolic ventricular ejection fraction (February), PAH research and measurement IT Vmax; respiratory function tests with measurement of DLCO; MRI brain with ARM and Cervical Pre-transplant anomalies; Radio of the pelvis

ferritin dosage

Evaluation of iron overload by ferritin

ferritin dosage

Evaluation of iron overload by ferritin

MRI iron overload

hepatic and cardiac MRI to assess the iron overload

Full Information

NCT ID

NCT03240731

First Posted

July 31, 2017

Last Updated

September 5, 2023

Sponsor

Centre Hospitalier Intercommunal Creteil

Collaborators

Keocyt, Association Clinique Thérapeutique Infantile du val de Marne

1. Study Identification

Unique Protocol Identification Number

NCT03240731

Brief Title

HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease

Acronym

DREPHAPLO

Official Title

Bone Marrow Transplantation HLA Haploidentical After a Reduced Intensity Conditioning and Prevention of GvHD Based on Post-transplant Cyclophosphamide Administration in Patients With Severe Sickle Cell Disease

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

August 10, 2017 (Actual)

Primary Completion Date

March 2024 (Anticipated)

Study Completion Date

September 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Centre Hospitalier Intercommunal Creteil

Collaborators

Keocyt, Association Clinique Thérapeutique Infantile du val de Marne

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

multicentric interventional biomedical research phase II, prospective, non-randomized evaluating a haploidentical marrow transplants after reduced-intensity conditioning and prevention of GvHD based on cyclophosphamide administration post transplantation in patients with severe sickle cell disease.

Detailed Description

Sickle cell disease is a severe disease with frequent occurrence of painful crises and progressive installation of a multi organ injuries. Despite the progress in its management, particularly since the introduction of hydroxycarbamide, the median age of death in sickle cell patients was about 40 years in a recent US study. Severe forms resistant to hydroxyurea or cerebral vasculopathy require transfusion programs throughout susceptible to risks of iron overload and alloimmunization. The bone marrow transplantation cures almost 95% of children and adolescents transplant from an HLA-identical siblings. In patients without HLA-identical donor, interesting results have been reported in haploidentical transplants marrow without ex vivo T cell depletion taken after non myeloablative conditioning regimen and GvHD prevention with cyclophosphamide high dose injection after bone marrow transplant . This approach performed in 14 patients was effective to cure 50% of the patients and 50% have rejected the transplant . No death or severe GvHD were related to the procedure. DREPHAPLO protocol aims to evaluate that approach in a population of sickle cell patients with severe complications of the disease, bringing direct benefit to patients with a cure of the disease in at least half of them.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Disease

Keywords

sickle cell disease, haploidentical, graft, marrow

7. Study Design

Primary Purpose

Other

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Model Description

Haploidentical Marrow Transplants

Masking

None (Open Label)

Allocation

N/A

Enrollment

18 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

bone marrow transplant

Arm Type

Experimental

Arm Description

Intervention Type

Biological

Intervention Name(s)

bone marrow transplant

Intervention Description

haploidentical bone marrow transplant

Primary Outcome Measure Information:

Title

Survival rate

Description

Time Frame

2 years

Secondary Outcome Measure Information:

Title

Survival rate

Description

Time Frame

1 year

Title

haematologic reconstitution

Description

defined as a neutrophil count> 500 / mm3 and platelets> 20000 / mm3, for three consecutive days.

Time Frame

2 years

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 1

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 2

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 3

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 4

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 5

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 6

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 9

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 12

Title

Chimerism

Description

Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers

Time Frame

at month 24

Title

hemoglobin electrophoresis

Time Frame

at 1 month

Title

hemoglobin electrophoresis

Time Frame

at 2 month

Title

hemoglobin electrophoresis

Time Frame

at 3 months

Title

hemoglobin electrophoresis

Time Frame

at 4 months

Title

hemoglobin electrophoresis

Time Frame

at 5 months

Title

hemoglobin electrophoresis

Time Frame

at 6 months

Title

hemoglobin electrophoresis

Time Frame

at 9 months

Title

hemoglobin electrophoresis

Time Frame

at 12 months

Title

hemoglobin electrophoresis

Time Frame

at 24 months

Title

occurence of graft versus host disease

Description

evaluated monthly from M1 to M6, and M9, M12, M24

Time Frame

at month 24

Title

grade of graft versus host disease

Description

Incidence and grade of GvHD, toxic deaths and infectious complications and secondary cancer

Time Frame

at month 24

Title

occurrence of toxic deaths

Time Frame

at month 24

Title

occurrence of infectious complications

Time Frame

at month 24

Title

occurrence of secondary cancer

Time Frame

at month 24

Title

Lymphocyte immunophenotyping

Description

Time Frame

2 years

Title

ECOG score value

Description

Index Trading ECOG complete physical examination with determination of weight

Time Frame

2 years

Title

Assessment of sickle cell disease complications

Description

Time Frame

at 1 year

Title

ferritin dosage

Description

Evaluation of iron overload by ferritin

Time Frame

at month 3

Title

ferritin dosage

Description

Evaluation of iron overload by ferritin

Time Frame

at month 6

Title

MRI iron overload

Description

hepatic and cardiac MRI to assess the iron overload

Time Frame

at 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

13 Years

Maximum Age & Unit of Time

40 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion criteria recipient: Age: 13 years-40 years Severe Sickle cell with at least one of the following criteria: Stenosing vasculopathy with abnormal MRA despite prolonged transfusion program PAH confirmed by right catheterization with mPAP> 25mmHg Systolic ejection fraction <55% and tricuspid regurgitation speed> 2.5m /s at distance from an acute episode No possibility of blood transfusion or very complicated blood transfusion Report albumin / creatinine> 30 mg / mmol, confirmed 3 times, away at distance from acute episode and persistent despite hydroxyurea or IEC GFR <80ml / min /1.73m2 (CKD-Epi without ethnic criterion) Previous history of acute liver sequestration with liver failure Acute chest syndrome or vaso-occlusive crises under hydroxyurea Complications of sickle cell transfusion imposing an exchange program with no possible withdrawal beyond a period of one year Not having geno-identical donor, but a haploidentical major donor (parent, sibling, adult child, or HbAA AS) Having red and understood the information letter and signed the informed consent Patients affiliated to a social security system (Social Security or Universal Medical Coverage) Exclusion Criteria recipient: Patient with a geno-identical donor Performans status: ECOG> 1 lung disease: FEV1 and FVC <50% predicted, score of PAH NYHA≥2 Liver disease with bilirubin> 50 .mu.mol / L heart failure defined by NYHA≥3 score ejection fraction <45% or shortening fraction <24% anti HLA alloimmunization against the donor or against red cell antigens of the donor Serology or HIV viral load positively Patients who for family, social or geographical reasons, do not wish to be regularly monitored in consultation severe uncontrolled infection at the time of inclusion or graft pregnant woman (positive beta HCG) or during lactation incapable adult patient, trust, guardianship, or safeguard justice Inclusion criteria donor Age> 18 years and <60 years Viral serologic economy allows the graft No contraindication for general anesthesia No contraindication the administration of G-CSF (the existence of sickle cell trait is not a contraindication) Lack antigens HLA recognized by the recipient antibody Hemoglobin S <50% When several donors are compatible: choose according to the ABO recipient: prefer ABO compatibility and major incompatibility and minor incompatibility, and finally major and minor incompatibility. Signature of informed consent Non-inclusion criteria donors: β HCG positive or known pregnancy

Facility Information:

Facility Name

CHU Henri-Mondor

City

Créteil

ZIP/Postal Code

94000

Country

France

Facility Name

intercommunal hospital of Créteil

City

Créteil

ZIP/Postal Code

94000

Country

France

Facility Name

CHU La Timone

City

Marseille

Country

France

Facility Name

Hospital Necker

City

Paris

Country

France

Facility Name

Hospital Robert-Debré

City

Paris

Country

France

Facility Name

Saint-Louis hospital

City

Paris

Country

France

Facility Name

CHU Strasbourg

City

Strasbourg

Country

France

12. IPD Sharing Statement

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Citation

Serody JS, Sparks SD, Lin Y, Capel EJ, Bigelow SH, Kirby SL, Gabriel DA, Wiley JM, Brecher ME, Schell MJ, Folds J, Shea TC. Comparison of granulocyte colony-stimulating factor (G-CSF)--mobilized peripheral blood progenitor cells and G-CSF--stimulated bone marrow as a source of stem cells in HLA-matched sibling transplantation. Biol Blood Marrow Transplant. 2000;6(4A):434-40. doi: 10.1016/s1083-8791(00)70035-8.

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HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease

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