Clinical trials: Fanconi's Anemia
Ovarian Neoplasm
Study to Assess the Efficacy and Safety of a PARP Inhibitor for the Treatment of BRCA-positive Advanced Ovarian Cancer
The purpose of the study is to see if the drug KU 0059436 is effective and well tolerated in treating patients with measurable BRCA1- or BRCA2-positive advanced ovarian cancer and for whom no curative therapeutic option exists.
- Trial name
- ICEBERG 2 (NCT00494442)
- Testing
- KU-0059436 (AZD2281)(PARP inhibitor)
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 58 people
- Sponsor
- AstraZeneca
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
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
Advanced or Recurrent Solid Tumors, Breast Neoplasms
Study of Talazoparib, a PARP Inhibitor, in Patients With Advanced or Recurrent Solid Tumors
This is a single-arm, open-label study to assess the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of talazoparib in patients with advanced tumors with DNA-repair pathway deficiencies.
- Conditions
- Advanced or Recurrent Solid Tumors, Breast Neoplasms, Ovarian Cancer, Epithelial, Ewing Sarcoma
- Trial name
- NCT01286987
- Testing
- Talazoparib
- Where
- 15 sites in United States, United Kingdom
- Volunteers
- 113 people
- Sponsor
- Pfizer
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Fanconi Anemia Complementation Group A
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the…
- Trial name
- NCT03814408
- Testing
- RP-L102
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Ovarian Cancer
Assessment of Efficacy of AZD2281 in Platinum Sensitive Relapsed Serous Ovarian Cancer
The primary purpose of this study to determine if AZD2281 is effective and well tolerated in maintaining the improvement in your cancer after previous platinum-based chemotherapy
- Trial name
- NCT00753545
- Testing
- AZD2281
- Where
- 107 sites in United States, Australia and 15 more
- Volunteers
- 265 people
- Sponsor
- AstraZeneca
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
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
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
HIV Infections, Fanconi Syndrome
Fanconi Syndrome Due to ARVs in HIV-Infected Persons
Cross-sectional cohort study of participants with HIV with or without protocol-defined Fanconi syndrome (confirmed creatinine clearance [CLcr] decline and evidence of proximal tubulopathy).
- Conditions
- HIV Infections, Fanconi Syndrome, Kidney Disease, Renal Impairment
- Trial name
- NCT00499187
- Testing
- Blood Draws
- Where
- 13 sites in United States, Canada
- Volunteers
- 56 people
- Sponsor
- Gilead Sciences
Nephropathic Cystinosis
DFT383 in Pediatric Participants With Nephropathic Cystinosis
An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase.
- Trial name
- CYStem (NCT06910813)
- Testing
- DFT383
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Fanconi Anemia Complementation Group A
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
- Trial name
- NCT04069533
- Testing
- RP-L102
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Primary Peritoneal Cancer, Advanced Epithelial Ovarian Cancer
Single Arm Study of BSI-201 in Patients With BRCA-1 or BRCA-2 Associated Advanced Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
The goal of this study was to determine the efficacy of iniparib (BSI-201/SAR240550) in patients with breast cancer gene-associated (BRCA) ovarian cancer.
- Trial name
- NCT00677079
- Testing
- Iniparib
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Sanofi
Ovarian Neoplasms, BRCA1 Protein
A Study to Assess the Safety and Pharmacokinetics of an Inhibitor of Poly ADP-Ribose Polymerase-1 (PARP)
To determine the safety, tolerability, dose-limiting toxicity (DLT), pharmacokinetic-pharmacodynamic profile, and maximum tolerated dose (MTD) of KU-0059436 when administered orally to patients with advanced solid tumours.
- Conditions
- Ovarian Neoplasms, BRCA1 Protein, BRCA2 Protein
- Trial name
- NCT00516373
- Testing
- KU-0059436 (AZD2281)(PARP inhibitor)
- Where
- 5 sites in Belgium, Netherlands and 2 more
- Volunteers
- 98 people
- Sponsor
- AstraZeneca
Fanconi Anemia Complementation Group A
Gene Therapy for Fanconi Anemia, Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A).
- Trial name
- NCT04248439
- Testing
- RP-L102
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Breast Neoplasms
Study to Assess The Efficacy and Safety of a PARP Inhibitor For The Treatment of BRCA-positive Advanced Breast Cancer
The purpose of the study is to see if the drug KU-0059436 (olaparib) is effective and well tolerated in treating participants with measurable breast cancer gene (BRCA)1- or BRCA2-positive advanced breast cancer and for whom no…
- Trial name
- ICEBERG 1 (NCT00494234)
- Testing
- Olaparib
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 54 people
- Sponsor
- AstraZeneca
Advanced Solid Tumors
A Phase 1/2 Trial SRA737 in Combination With Gemcitabine and Cisplatin or Gemcitabine Alone in Advanced Cancer Subjects
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose (RP2D) and schedule of SRA737 in combination with low dose gemcitabine; and to…
- Trial name
- NCT02797977
- Testing
- SRA737, gemcitabine, cisplatin, SRA737, gemcitabine
- Where
- 20 sites in Spain, United Kingdom
- Volunteers
- 153 people
- Sponsor
- Sierra Oncology LLC - a GSK company