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

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ClinicalTrials.gov Identifier: NCT03240731

Recruitment Status : Active, not recruiting

First Posted : August 7, 2017

Last Update Posted : August 18, 2022

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborators:

Keocyt

Association Clinique Thérapeutique Infantile du val de Marne

Information provided by (Responsible Party):

Nathalie Dhédin, Saint-Louis Hospital, Paris, France

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.

Condition or disease	Intervention/treatment	Phase
Sickle Cell Disease	Biological: bone marrow transplant	Phase 2

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	18 participants
Allocation:	N/A
Intervention Model:	Single Group Assignment
Intervention Model Description:	Haploidentical Marrow Transplants
Masking:	None (Open Label)
Primary Purpose:	Other
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
Actual Study Start Date :	August 10, 2017
Estimated Primary Completion Date :	March 2024
Estimated Study Completion Date :	September 2025

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Sickle cell disease

MedlinePlus related topics: Bone Marrow Transplantation

Genetic and Rare Diseases Information Center resources: Sickle Cell Anemia

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: bone marrow transplant 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.	Biological: bone marrow transplant haploidentical bone marrow transplant

Arm

Intervention/treatment

Experimental: bone marrow transplant

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.

Biological: bone marrow transplant

haploidentical bone marrow transplant

Outcome Measures

Primary Outcome Measures :

Survival rate [ Time Frame: 2 years ]
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 [ Time Frame: 1 year ]
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
haematologic reconstitution [ Time Frame: 2 years ]
defined as a neutrophil count> 500 / mm3 and platelets> 20000 / mm3, for three consecutive days.
Chimerism [ Time Frame: at month 1 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 2 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 3 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 4 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 5 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 6 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 9 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 12 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
Chimerism [ Time Frame: at month 24 ]
Chimerism in the peripheral blood of the total population and the CD3 + population with the techniques usually employed by the centers
hemoglobin electrophoresis [ Time Frame: at 1 month ]
hemoglobin electrophoresis [ Time Frame: at 2 month ]
hemoglobin electrophoresis [ Time Frame: at 3 months ]
hemoglobin electrophoresis [ Time Frame: at 4 months ]
hemoglobin electrophoresis [ Time Frame: at 5 months ]
hemoglobin electrophoresis [ Time Frame: at 6 months ]
hemoglobin electrophoresis [ Time Frame: at 9 months ]
hemoglobin electrophoresis [ Time Frame: at 12 months ]
hemoglobin electrophoresis [ Time Frame: at 24 months ]
occurence of graft versus host disease [ Time Frame: at month 24 ]
evaluated monthly from M1 to M6, and M9, M12, M24
grade of graft versus host disease [ Time Frame: at month 24 ]
Incidence and grade of GvHD, toxic deaths and infectious complications and secondary cancer
occurrence of toxic deaths [ Time Frame: at month 24 ]
occurrence of infectious complications [ Time Frame: at month 24 ]
occurrence of secondary cancer [ Time Frame: at month 24 ]
Lymphocyte immunophenotyping [ Time Frame: 2 years ]
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 [ Time Frame: 2 years ]
Index Trading ECOG complete physical examination with determination of weight
Assessment of sickle cell disease complications [ Time Frame: at 1 year ]
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 [ Time Frame: at month 3 ]
Evaluation of iron overload by ferritin
ferritin dosage [ Time Frame: at month 6 ]
Evaluation of iron overload by ferritin
MRI iron overload [ Time Frame: at 12 months ]
hepatic and cardiac MRI to assess the iron overload

Eligibility Criteria

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Ages Eligible for Study:	13 Years to 40 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03240731

Locations

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France
CHU Henri-Mondor
Créteil, France, 94000
intercommunal hospital of Créteil
Créteil, France, 94000
CHU La Timone
Marseille, France
Hospital Necker
Paris, France
Hospital Robert-Debré
Paris, France
Saint-Louis hospital
Paris, France
CHU Strasbourg
Strasbourg, France

Sponsors and Collaborators

Centre Hospitalier Intercommunal Creteil

Keocyt

Association Clinique Thérapeutique Infantile du val de Marne

More Information

Publications:

Yawn BP, Buchanan GR, Afenyi-Annan AN, Ballas SK, Hassell KL, James AH, Jordan L, Lanzkron SM, Lottenberg R, Savage WJ, Tanabe PJ, Ware RE, Murad MH, Goldsmith JC, Ortiz E, Fulwood R, Horton A, John-Sowah J. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014 Sep 10;312(10):1033-48. doi: 10.1001/jama.2014.10517. Erratum In: JAMA. 2014 Nov 12;312(18):1932. JAMA. 2015 Feb 17;313(7):729.

Habibi A, Arlet JB, Stankovic K, Gellen-Dautremer J, Ribeil JA, Bartolucci P, Lionnet F; centre de reference maladies rares << syndromes drepanocytaires majeurs >>. [French guidelines for the management of adult sickle cell disease: 2015 update]. Rev Med Interne. 2015 May 11;36(5 Suppl 1):5S3-84. doi: 10.1016/S0248-8663(15)60002-9. French.

Lanzkron S, Carroll CP, Haywood C Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Rep. 2013 Mar-Apr;128(2):110-6. doi: 10.1177/003335491312800206.

Bernaudin F, Socie G, Kuentz M, Chevret S, Duval M, Bertrand Y, Vannier JP, Yakouben K, Thuret I, Bordigoni P, Fischer A, Lutz P, Stephan JL, Dhedin N, Plouvier E, Margueritte G, Bories D, Verlhac S, Esperou H, Coic L, Vernant JP, Gluckman E; SFGM-TC. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007 Oct 1;110(7):2749-56. doi: 10.1182/blood-2007-03-079665. Epub 2007 Jul 2.

Kuentz M, Robin M, Dhedin N, Hicheri Y, Peffault de Latour R, Rohrlich P, Bordigoni P, Bruno B, Socie G, Bernaudin F. Is there still a place for myeloablative regimen to transplant young adults with sickle cell disease? Blood. 2011 Oct 20;118(16):4491-2; author reply 4492-3. doi: 10.1182/blood-2011-07-367490. No abstract available.

Angelucci E, Matthes-Martin S, Baronciani D, Bernaudin F, Bonanomi S, Cappellini MD, Dalle JH, Di Bartolomeo P, de Heredia CD, Dickerhoff R, Giardini C, Gluckman E, Hussein AA, Kamani N, Minkov M, Locatelli F, Rocha V, Sedlacek P, Smiers F, Thuret I, Yaniv I, Cavazzana M, Peters C; EBMT Inborn Error and EBMT Paediatric Working Parties. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel. Haematologica. 2014 May;99(5):811-20. doi: 10.3324/haematol.2013.099747.

Hsieh MM, Fitzhugh CD, Weitzel RP, Link ME, Coles WA, Zhao X, Rodgers GP, Powell JD, Tisdale JF. Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype. JAMA. 2014 Jul 2;312(1):48-56. doi: 10.1001/jama.2014.7192.

Bolanos-Meade J, Fuchs EJ, Luznik L, Lanzkron SM, Gamper CJ, Jones RJ, Brodsky RA. HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood. 2012 Nov 22;120(22):4285-91. doi: 10.1182/blood-2012-07-438408. Epub 2012 Sep 6.

Kamani NR, Walters MC, Carter S, Aquino V, Brochstein JA, Chaudhury S, Eapen M, Freed BM, Grimley M, Levine JE, Logan B, Moore T, Panepinto J, Parikh S, Pulsipher MA, Sande J, Schultz KR, Spellman S, Shenoy S. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant. 2012 Aug;18(8):1265-72. doi: 10.1016/j.bbmt.2012.01.019. Epub 2012 Feb 16.

Luznik L, O'Donnell PV, Symons HJ, Chen AR, Leffell MS, Zahurak M, Gooley TA, Piantadosi S, Kaup M, Ambinder RF, Huff CA, Matsui W, Bolanos-Meade J, Borrello I, Powell JD, Harrington E, Warnock S, Flowers M, Brodsky RA, Sandmaier BM, Storb RF, Jones RJ, Fuchs EJ. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008 Jun;14(6):641-50. doi: 10.1016/j.bbmt.2008.03.005.

McCurdy SR, Kanakry JA, Showel MM, Tsai HL, Bolanos-Meade J, Rosner GL, Kanakry CG, Perica K, Symons HJ, Brodsky RA, Gladstone DE, Huff CA, Pratz KW, Prince GT, Dezern AE, Gojo I, Matsui WH, Borrello I, McDevitt MA, Swinnen LJ, Smith BD, Levis MJ, Ambinder RF, Luznik L, Jones RJ, Fuchs EJ, Kasamon YL. Risk-stratified outcomes of nonmyeloablative HLA-haploidentical BMT with high-dose posttransplantation cyclophosphamide. Blood. 2015 May 7;125(19):3024-31. doi: 10.1182/blood-2015-01-623991. Epub 2015 Mar 26.

Bashey A, Zhang X, Sizemore CA, Manion K, Brown S, Holland HK, Morris LE, Solomon SR. T-cell-replete HLA-haploidentical hematopoietic transplantation for hematologic malignancies using post-transplantation cyclophosphamide results in outcomes equivalent to those of contemporaneous HLA-matched related and unrelated donor transplantation. J Clin Oncol. 2013 Apr 1;31(10):1310-6. doi: 10.1200/JCO.2012.44.3523. Epub 2013 Feb 19.

Bashey A, Zhang X, Jackson K, Brown S, Ridgeway M, Solh M, Morris LE, Holland HK, Solomon SR. Comparison of Outcomes of Hematopoietic Cell Transplants from T-Replete Haploidentical Donors Using Post-Transplantation Cyclophosphamide with 10 of 10 HLA-A, -B, -C, -DRB1, and -DQB1 Allele-Matched Unrelated Donors and HLA-Identical Sibling Donors: A Multivariable Analysis Including Disease Risk Index. Biol Blood Marrow Transplant. 2016 Jan;22(1):125-33. doi: 10.1016/j.bbmt.2015.09.002. Epub 2015 Sep 7.

Ciurea SO, Mulanovich V, Saliba RM, Bayraktar UD, Jiang Y, Bassett R, Wang SA, Konopleva M, Fernandez-Vina M, Montes N, Bosque D, Chen J, Rondon G, Alatrash G, Alousi A, Bashir Q, Korbling M, Qazilbash M, Parmar S, Shpall E, Nieto Y, Hosing C, Kebriaei P, Khouri I, Popat U, de Lima M, Champlin RE. Improved early outcomes using a T cell replete graft compared with T cell depleted haploidentical hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2012 Dec;18(12):1835-44. doi: 10.1016/j.bbmt.2012.07.003. Epub 2012 Jul 11.

Raiola AM, Dominietto A, Ghiso A, Di Grazia C, Lamparelli T, Gualandi F, Bregante S, Van Lint MT, Geroldi S, Luchetti S, Ballerini F, Miglino M, Varaldo R, Bacigalupo A. Unmanipulated haploidentical bone marrow transplantation and posttransplantation cyclophosphamide for hematologic malignancies after myeloablative conditioning. Biol Blood Marrow Transplant. 2013 Jan;19(1):117-22. doi: 10.1016/j.bbmt.2012.08.014. Epub 2012 Aug 29.

Kanakry CG, Ganguly S, Zahurak M, Bolanos-Meade J, Thoburn C, Perkins B, Fuchs EJ, Jones RJ, Hess AD, Luznik L. Aldehyde dehydrogenase expression drives human regulatory T cell resistance to posttransplantation cyclophosphamide. Sci Transl Med. 2013 Nov 13;5(211):211ra157. doi: 10.1126/scitranslmed.3006960.

Ganguly S, Ross DB, Panoskaltsis-Mortari A, Kanakry CG, Blazar BR, Levy RB, Luznik L. Donor CD4+ Foxp3+ regulatory T cells are necessary for posttransplantation cyclophosphamide-mediated protection against GVHD in mice. Blood. 2014 Sep 25;124(13):2131-41. doi: 10.1182/blood-2013-10-525873. Epub 2014 Aug 18.

Stem Cell Trialists' Collaborative Group. Allogeneic peripheral blood stem-cell compared with bone marrow transplantation in the management of hematologic malignancies: an individual patient data meta-analysis of nine randomized trials. J Clin Oncol. 2005 Aug 1;23(22):5074-87. doi: 10.1200/JCO.2005.09.020.

Yang JZ, Zhang JQ, Sun LX. Mechanisms for T cell tolerance induced with granulocyte colony-stimulating factor. Mol Immunol. 2016 Feb;70:56-62. doi: 10.1016/j.molimm.2015.12.006. Epub 2015 Dec 17.

Jun HX, Jun CY, Yu ZX. In vivo induction of T-cell hyporesponsiveness and alteration of immunological cells of bone marrow grafts using granulocyte colony-stimulating factor. Haematologica. 2004 Dec;89(12):1517-24.

Deotare U, Al-Dawsari G, Couban S, Lipton JH. G-CSF-primed bone marrow as a source of stem cells for allografting: revisiting the concept. Bone Marrow Transplant. 2015 Sep;50(9):1150-6. doi: 10.1038/bmt.2015.80. Epub 2015 Apr 27.

Couban S, Messner HA, Andreou P, Egan B, Price S, Tinker L, Meharchand J, Forrest DL, Lipton J. Bone marrow mobilized with granulocyte colony-stimulating factor in related allogeneic transplant recipients: a study of 29 patients. Biol Blood Marrow Transplant. 2000;6(4A):422-7. doi: 10.1016/s1083-8791(00)70033-4.

Isola L, Scigliano E, Fruchtman S. Long-term follow-up after allogeneic granulocyte colony-stimulating factor--primed bone marrow transplantation. Biol Blood Marrow Transplant. 2000;6(4A):428-33. doi: 10.1016/s1083-8791(00)70034-6.

Ji SQ, Chen HR, Wang HX, Yan HM, Pan SP, Xun CQ. Comparison of outcome of allogeneic bone marrow transplantation with and without granulocyte colony-stimulating factor (lenograstim) donor-marrow priming in patients with chronic myelogenous leukemia. Biol Blood Marrow Transplant. 2002;8(5):261-7. doi: 10.1053/bbmt.2002.v8.pm12064363.

Morton J, Hutchins C, Durrant S. Granulocyte-colony-stimulating factor (G-CSF)-primed allogeneic bone marrow: significantly less graft-versus-host disease and comparable engraftment to G-CSF-mobilized peripheral blood stem cells. Blood. 2001 Dec 1;98(12):3186-91. doi: 10.1182/blood.v98.12.3186.

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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Responsible Party:	Nathalie Dhédin, Principal Investigator, Saint-Louis Hospital, Paris, France
ClinicalTrials.gov Identifier:	NCT03240731
Other Study ID Numbers:	DREPHAPLO
First Posted:	August 7, 2017 Key Record Dates
Last Update Posted:	August 18, 2022
Last Verified:	August 2022

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Studies a U.S. FDA-regulated Drug Product:	No
Studies a U.S. FDA-regulated Device Product:	No

Keywords provided by Nathalie Dhédin, Saint-Louis Hospital, Paris, France:


sickle cell disease haploidentical graft marrow

Additional relevant MeSH terms:

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Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia	Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn

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