DDrare: Database of Drug Development for Rare Diseases

Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = AMD3100; AP1903; AP1903 A594; ATG; Abatacept; Alefacept; Anti-CD45; Anti-Thymocyte Globulin; Anti-Thymocyte Globulin (Rabbit); Anti-thymocyte globulin; BPX-501; BPX-501 T cells; BPX-501 cells; Busulfan; Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.; CAMPATH-1H; CD34+ CELLS; CD34+ Cells; CD34+ selected cells; CD34+CELLS; Campath; Campath 1h; Chemotherapy; Cyclophosphamide; Cyclophosphamide (CY) (Plan 1); Cyclophosphamide (CY) (Plan 2); Cyclophosphamide 30; Cyclophosphamide 40; Cyclophosphamide, Fludarabine; Cyclosporine; Danazol; Device: AmCell CliniMACs; Device: CliniMACS device; Device: CliniMACs device; Device: Donor mobilized PBSC infusion; Device: The CliniMACS device; ENBREL*SC 4FL 25MG+4SIR 1ML; Eltrombopag; Enbrel; Etanercept; FP-045; Filgrastim; Fludarabine; Fludarabine (FLU); Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Genetically Engineered Hematopoietic Stem Progenitor Cells; Genetically Engineered Hematopoietic Stem/Progenitor Cells; Graft-versus-tumor induction therapy; Hematopoietic stem cell transplantation; Melphalan; Metformin; Metformin HCl; Methotrexate; Methylprednisolone; Methylprednisolone (MP); Miltenyi CliniMACS; Mozobil; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Neupogen; Olaparib; Other: Laboratory Biomarker Analysis; Oxandrolone; Pembrolizumab; Peripheral blood stem cell; Phosphate; Plerixafor; Plérixafor; Prednisone; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Transplantation; Procedure: CD34 selected haploidentical PBSCT; Procedure: Hematopoietic Stem Cell Transplant; Procedure: Hematopoietic Stem Cell Transplantation; Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs); Procedure: Leukapheresis; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Stem cell infusion; Procedure: Thymic Shielding During Radiation; Procedure: Total Body Irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation; Quercetin; Quercetin (dietary supplement); RP-L102; Rabbit; Rabbit ATG; Radiation: Total body irradiation (TBI); Radiation: Total-Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Rimiducid; Rituximab; Rivogenlecleucel; Rivogenleucleucel; Sargramostim; Sirolimus; T cell infusion; Tacrolimus; Therapeutic allogeneic lymphocytes; Total Body Irradiation (TBI) (Plan 1); Transduced CD34+ Cells; Zarzio

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02678533 (ClinicalTrials.gov)	February 10, 2017	5/2/2016	Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor	Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy	Fanconi Anemia	Drug: G-CSF;Drug: Plerixafor	Assistance Publique - Hôpitaux de Paris	EuroFancolen	Recruiting	2 Years	17 Years	All	8	Phase 1;Phase 2	France
2	NCT03157804 (ClinicalTrials.gov)	January 7, 2016	26/4/2017	Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A	Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)	Fanconi Anemia	Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow Aspiration	Hospital Infantil Universitario Niño Jesús, Madrid, Spain	Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de Barcelona	Active, not recruiting	1 Year	21 Years	All	9	Phase 1;Phase 2	Spain
3	EUCTR2014-004272-29-GB (EUCTR)	30/03/2015	15/07/2015	Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia	Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia	Fanconi Anaemia;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Great Ormond Street Hospital NHS foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	France;Spain;United Kingdom
4	NCT02931071 (ClinicalTrials.gov)	September 2013	6/9/2016	Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1	Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient	Fanconi Anemia	Drug: filgrastim;Drug: plerixafor	Hospital Universitari Vall d'Hebron Research Institute	CIEMAT;CIBERER	Completed	2 Years	64 Years	All	13	Phase 2	Spain
5	NCT01331018 (ClinicalTrials.gov)	February 22, 2012	16/3/2011	Gene Therapy for Fanconi Anemia	Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)	Fanconi Anemia	Procedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: Prednisone	Fred Hutchinson Cancer Research Center	National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited	Active, not recruiting	4 Years	N/A	All	3	Phase 1	United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT02678533
(ClinicalTrials.gov)

February 10, 2017

5/2/2016

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor

Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy

Fanconi Anemia

Drug: G-CSF;Drug: Plerixafor

Assistance Publique - Hôpitaux de Paris

EuroFancolen

Recruiting

2 Years

17 Years

All

Phase 1;Phase 2

France

NCT03157804
(ClinicalTrials.gov)

January 7, 2016

26/4/2017

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)

Fanconi Anemia

Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow Aspiration

Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de Barcelona

Active, not recruiting

1 Year

21 Years

All

Phase 1;Phase 2

Spain

EUCTR2014-004272-29-GB
(EUCTR)

30/03/2015

15/07/2015

Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia

Fanconi Anaemia;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Great Ormond Street Hospital NHS foundation Trust

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

France;Spain;United Kingdom

NCT02931071
(ClinicalTrials.gov)

September 2013

6/9/2016

Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient

Fanconi Anemia

Drug: filgrastim;Drug: plerixafor

Hospital Universitari Vall d'Hebron Research Institute

CIEMAT;CIBERER

Completed

2 Years

64 Years

All

Phase 2

Spain

NCT01331018
(ClinicalTrials.gov)

February 22, 2012

16/3/2011

Gene Therapy for Fanconi Anemia

Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)

Fanconi Anemia

Procedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: Prednisone

Fred Hutchinson Cancer Research Center

National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited

Active, not recruiting

4 Years

N/A

All

Phase 1

United States