Clinical trials: Fanconi's Anemia
Fanconi Anemia
Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi…
- Trial name
- FANCOLEN-1 (NCT03157804)
- Testing
- IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs), Genetically Engineered Hematopoietic Stem/Progenitor Cells, Laboratory Biomarker Analysis
- Where
- 2 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Fanconi Anemia
Quercetin in Children With Fanconi Anemia; a Pilot Study
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
- Trial name
- NCT01720147
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Fanconi Anemia
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated…
- Trial name
- NCT05910853
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sanguine Biosciences
FANCONI ANEMIA
Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT
While stem cell transplantation has proven an effective means of treating a wide variety of diseases involving hematopoietic stem cells and their progeny, a shortage of donors has proved a major impediment to the widest…
- Trial name
- RAFHAS (NCT00586274)
- Testing
- CD34 selected haploidentical PBSCT, Fludarabine, T cell infusion
- Where
- 2 sites in United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Renal Fanconi Syndrome, Photophobia
Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Cystinosis disease from the blood
- Conditions
- Renal Fanconi Syndrome, Photophobia, Hypophosphatemia
- Trial name
- BioCystinosis (NCT02837523)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Fallopian Tube, Primary Peritoneal Cancer
Study of ABT-767 in Subjects With Breast Cancer 1 and Breast Cancer 2 (BRCA 1 and BRCA 2) Mutations and Solid Tumors or High Grade Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
This is a Phase 1, dose escalation trial evaluating the tolerability, pharmacokinetics, and pharmacodynamics of ABT-767 in subjects with advanced Breast Cancer 1 or 2 gene (BRCA1 or BRCA2)-mutated solid tumors and high grade…
- Conditions
- Fallopian Tube, Primary Peritoneal Cancer, Solid Tumors (e.g. Breast, Ovarian, Prostate, or Pancreatic) and Ovarian
- Trial name
- NCT01339650
- Testing
- ABT-767
- Where
- 3 sites in Netherlands
- Volunteers
- 93 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Fanconi Anemia
MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients
This study being done to learn more about the use of medical Magnetic Resonance Imaging (mMRI) and dedicated dental MRI (ddMRI) as a non-invasive diagnosing tool when evaluating potential oral cancerous and precancerous lesions…
- Trial name
- NCT07649031
- Testing
- MRIs
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of Minnesota
Advanced Cancers (Breast, Ovarian, mCRPC, Pancreatic Ductal Adenocarcinoma (PDAC), Brain Mets) With Specific Mutations (Homologous Recombination Deficiency)
Study to Assess Safety, Tolerability and Activity of DSB2455 in Participants With Advanced Malignancies
Open label, multi-centre, Phase Ia/b adaptive design study with an initial 2-stage Dose Escalation Phase followed by a Dose Expansion Phase.
- Trial name
- NCT06458712
- Testing
- DSB2455
- Where
- 21 sites in United States, France and 3 more
- Volunteers
- 180 people
- Sponsor
- Duke Street Bio Ltd
Fanconi Anemia, Squamous Cell Carcinoma
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous…
- Trial name
- NCT03476330
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Ovarian Cancer, Hereditary Ovarian Cancer Syndrome
C-PATROL - Non-interventional Study (NIS) to Collect Clinical and Patient Reported Outcome Data in an Olaparib Treated BRCAm+ PSR Ovarian Cancer Population
The main objective of the proposed non-interventional study is to obtain real-world effectiveness, safety and treatment patterns data of patients with BRCAm+ (Breast Cancer Gene(s) mutation positive) platinum sensitive relapsed…
- Trial name
- C-PATROL (NCT02503436)
- Where
- 68 sites in Germany
- Volunteers
- 276 people
- Sponsor
- AstraZeneca
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)
A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose and schedule of SRA737; and to evaluate the efficacy of SRA737 in…
- Trial name
- NCT02797964
- Testing
- SRA737
- Where
- 15 sites in United Kingdom
- Volunteers
- 107 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Aplastic Anemia, Leukemia
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
This is a genetic disease (transmitted through the parents' genes) called Fanconi Anemia. Because of that genetic disease, the bone marrow has changed and now has failed, or has given rise to a preleukemia called myelodysplastic…
- Conditions
- Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00987480
- Testing
- Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
- Where
- 6 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Ovarian Epithelial Cancer, Carcinoma in Situ
Lavage of the Uterine Cavity for the Diagnosis of Serous Tubal Intraepithelial Carcinoma
The current study aims at answering the scientific question, whether exfoliated cells from STICs get transported into the uterine cavity via the fallopian tube, and whether it is possible to detect those cells in the lavage fluid…
- Conditions
- Ovarian Epithelial Cancer, Carcinoma in Situ, Ovarian Cancer
- Trial name
- LUSTIC (NCT02039388)
- Testing
- Lavage of the Cavum uteri and proximal Fallopian tubes, performed in the luteal phase of the female cycle
- Where
- 14 sites in Austria, Belgium and 6 more
- Volunteers
- 701 people, healthy volunteers welcome
- Sponsor
- Medical University of Vienna
Fanconi Anemia
Corrective Exercise and Rehabilitation in Fanconi Anemia: A Case Study
Introduction: Fanconi anemia (FA) is a rare autosomal recessive DNA repair disorder characterized by congenital malformations, progressive bone marrow failure, and reduced quality of life.
- Trial name
- NCT06958380
- Testing
- Corrective exercise and rehabilitation
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Pardis Specialized Wellness Institute
Fanconi Syndrome
Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease
This observational protocol will allow for an independent, prospective evaluation of the improvement in survival of patients with Fanconi disease in hematological deadlock due to the absence of an HLA-identical donor and having…
- Trial name
- HAPLO-FANCONI (NCT05903365)
- Testing
- Blood sampling
- Volunteers
- 18 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease
- Trial name
- NCT00290628
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fanconi Anemia Complementation Group A, Fanconi Anemia
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial.
- Trial name
- NCT04437771
- Testing
- Safety and efficacy assessments
- Where
- Spain
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Pancreatic Cancer Metastatic
Vilastobart+Retifanlimab in BRCA or PALB2 Deficient PC
This is a single arm, open-label, non-randomized multi-institution phase II trial of a Fc enhanced CTLA4 antibody, vilastobart, in combination with PD-1 antibody, retifanlimab, in patients with BRCA1, BRCA2 or PALB2 deficient…
- Trial name
- EKLAVYA-HRD (NCT07765836)
- Testing
- vilastobart (XTX101), Retifanlimab
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Massachusetts General Hospital
Fanconi Anemia
Gene Therapy for Fanconi Anemia
This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA).
- Trial name
- NCT01331018
- Testing
- Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Fred Hutchinson Cancer Center