Clinical trials: DNA Damage Repair Deficiency
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Chronic Lymphocytic Leukemia
RP-3500 and Olaparib in DNA Damage Repair Pathway Deficient Relapsed/Refractory Chronic Lymphocytic Leukemia
This is an open-label, multicenter, phase Ib/II study of the combination of RP-3500 and olaparib in Relapsed/Refractory Chronic Lymphocytic Leukemia (R/R CLL) patients with DDR deficiencies.
- Trial name
- CORONADO CLL (NCT05405309)
- Testing
- RP-3500, Olaparib
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Utah
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Lynch Syndrome
Colonoscopy Check-up in People With Lynch Syndrome
Lynch syndrome, an inherited condition, increases bowel cancer risk. People with Lynch syndrome are recommended to have regular colonoscopies where a camera in a tube is used to look inside the bowel for cancer and for polyps…
- Trial name
- NCT06126120
- Where
- United Kingdom
- Volunteers
- 231 people
- Sponsor
- Imperial College London
Colorectal Cancer, Rectal Cancer
PeLear CCC: Proyecto Latino Contra Cancer Colorrectal
The study aims to recruit 60 Spanish speaking individuals who identify as Latinos, are 18 years or older and attend the Saint Thomas More (STM) Church in Chapel Hill.
- Conditions
- Colorectal Cancer, Rectal Cancer, Colon Cancer, Colon Rectal Cancer
- Trial name
- PeLear CCC (NCT06426927)
- Testing
- Colorectal Cancer Educational Videos in Spanish
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Metastatic Castration Resistant Prostate Cancer
A Study of Rucaparib in Patients With Metastatic Castration-resistant Prostate Cancer and Homologous Recombination Gene Deficiency
The purpose of this study is to determine how patients with metastatic castration-resistant prostate cancer, and evidence of a homologous recombination gene deficiency, respond to treatment with rucaparib.
- Trial name
- TRITON2 (NCT02952534)
- Testing
- Rucaparib
- Where
- 149 sites in United States, Australia and 10 more
- Volunteers
- 277 people
- Sponsor
- pharmaand GmbH
ATM Gene Mutation, BRCA1 Gene Mutation
Docetaxel, Carboplatin, and Rucaparib Camsylate in Treating Patients With Metastatic Castration Resistant Prostate Cancer With Homologous Recombination DNA Repair Deficiency
This phase II trial studies how well docetaxel with carboplatin followed by rucaparib camsylate works in treating patients with metastatic castration resistant prostate cancer (spread outside of prostate and resistant to…
- Conditions
- ATM Gene Mutation, BRCA1 Gene Mutation, BRCA2 Gene Mutation, Castration Levels of Testosterone
- Trial name
- NCT03442556
- Testing
- Carboplatin, Docetaxel, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of Washington
Hereditary Cancer Syndrome
Cancer Health Assessments Reaching Many
The CHARM (Cancer Health Assessment Reaching Many) study will assess the utility of clinical exome sequencing and how it affects care in diverse populations.
- Trial name
- CHARM (NCT03426878)
- Testing
- Modified genetic counseling, Traditional genetic counseling
- Where
- 2 sites in United States
- Volunteers
- 967 people, healthy volunteers welcome
- Sponsor
- Kaiser Permanente
Fanconi Anemia
TBI Dose De-escalation for Fanconi Anemia
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
- Trial name
- NCT00352976
- Testing
- Cyclophosphamide, Fludarabine, Total Body Irradiation
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fanconi Anemia
Stem Cell Transplantation for Fanconi Anemia
The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched…
- Trial name
- NCT00167206
- Testing
- Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Decision Aid
Using Visual Arrays to Support Understanding of Genetic Risk
The goal of this study is to understand how people understand risk from genetic testing. The investigators want to understand what visual aid best helps people accurately assess risk.
- Trial name
- NCT06994832
- Testing
- Visual Array, Narrative Visual Array, Choice
- Where
- United States
- Volunteers
- 1,045 people, healthy volunteers welcome
- Sponsor
- Stephanie B. Seminara, MD
Metastatic Malignant Solid Neoplasm, Refractory Malignant Solid Neoplasm
Avelumab and M6620 for the Treatment of DDR Deficient Metastatic or Unresectable Solid Tumors
This phase I/II trial studies the side effects and best dose of avelumab with M6620 in treating patients with deoxyribonucleic acid (DNA) damage repair (DDR) deficient solid tumors that have spread to other places in the body…
- Conditions
- Metastatic Malignant Solid Neoplasm, Refractory Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm
- Trial name
- NCT04266912
- Testing
- Avelumab, Berzosertib
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- M.D. Anderson Cancer Center
Mesothelioma, Uveal Melanoma
A Trial of Niraparib in BAP1 and Other DNA Damage Response (DDR) Deficient Neoplasms (UF-STO-ETI-001)
This open-label, non-randomized study will investigate the use of niraparib in patients with tumors known to have mutations in BAP1 and other select DNA damage response pathway genes.
- Conditions
- Mesothelioma, Uveal Melanoma, Renal Cell Carcinoma, Cholangiocarcinoma
- Trial name
- NCT03207347
- Testing
- Niraparib
- Where
- 3 sites in United States
- Volunteers
- 37 people
- Sponsor
- University of Florida
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
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
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by…
- Trial name
- NCT01380990
- Testing
- Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Lynch Syndrome
Overcoming Barriers to the Uptake of Cascade Screening for Lynch Syndrome: Workbook Feasibility Study
The investigators will evaluate the feasibility of an intervention to improve Lynch syndrome cascade screening uptake. The investigators will conduct a pilot study among 15 patients diagnosed with Lynch Syndrome and 5 genetic…
- Trial name
- NCT04978350
- Testing
- Educational Workbook
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
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
Pain
Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film
The primary aim of this study is to determine if chronic pain subjects on around-the-clock opioids who are receiving 100 to 220 mg oral morphine sulfate equivalent (MSE) can be safely transitioned on to buprenorphine…
- Trial name
- NCT01871285
- Testing
- Buprenorphine, Oxycodone, Morphine sulfate
- Where
- 3 sites in United States
- Volunteers
- 39 people
- Sponsor
- BioDelivery Sciences International