Clinical trials: Metabolic Repair
Fanconi Anemia
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene.
- Trial name
- NCT03351868
- Testing
- Gene-modified autologous stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
Pancreas Cancer, Pancreatitis
Pancreas Registry and High Risk Registry
The purpose of this study is to establish a registry of patients with pancreatic diseases. Patients included in the registry may include those with: pancreatic cancer, precancerous lesions of the pancreas, inflammatory lesions of…
- Conditions
- Pancreas Cancer, Pancreatitis, Chronic Pancreatitis, Pancreatic Cyst
- Trial name
- NCT02775461
- Where
- 2 sites in United States
- Volunteers
- 1,368 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Ventral Hernia
Bariatric SUrgery With Mesh REpair of Ventral Hernia: a Randomized Controlled Trial
The primary objective of the study is to demonstrate that hernia recurrence (VH) repair with non absorbable mesh concomitant to Bariatric Surgery (BS) decreases the risk of VH, with or without surgical repair, during the first…
- Trial name
- BeSURE (NCT05488288)
- Testing
- Mesh repair, Suture repair
- Where
- France
- Volunteers
- 354 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Colorectal Cancer
FITting Non-invasive Tests in Lynch Syndrome Surveillance
The purpose of this clinical study is to evaluate the accuracy of the fecal immunochemical test (FIT) in comparison to colonoscopy in patients with Lynch Syndrome (LS) who are undergoing colonoscopy surveillance.
- Trial name
- NCT06898996
- Testing
- Fecal immunochemical test
- Where
- 3 sites in United States
- Volunteers
- 400 people
- Sponsor
- University of Chicago
Complications Wounds, Negative Pressure Wound Therapy
Incisional Negative Pressure Wound Therapy in High Risk Patients Undergoing Panniculectomy: A Prospective Randomized Controlled Trial
Incisional negative pressure therapy (INPWT) has previously been shown in certain patient populations to decrease wound healing complications, decrease the rate of hematomas and seromas, as well as have better scar quality.
- Conditions
- Complications Wounds, Negative Pressure Wound Therapy, Wound Healing Delayed, Incisional
- Trial name
- NCT03010137
- Testing
- Incisional Negative Pressure Wound Therapy, Standard Closure with Skin Glue, PICO (Smith&Nephew)
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of California, Davis
Cesarean Section, Surgical Wound Infection
Timing of Cesarean Wound Dressing Removal on Wound Healing in Women With BMI ≥ 30 kg/m²
Cesarean delivery is one of the most common surgical procedures in obstetric care. Women with obesity (body mass index [BMI] of 30 kg/m² or higher) are at increased risk for postoperative wound complications, including surgical…
- Conditions
- Cesarean Section, Surgical Wound Infection, Wound Healing, Obesity
- Trial name
- NCT07846930
- Volunteers
- 726 people
- Sponsor
- Assiut University
Fanconi Anemia, Unspecified Adult Solid Tumor, Protocol Specific
Tissue Sample Collection From Patients With Fanconi Anemia
RATIONALE: Collecting and storing samples of tumor tissue from patients with Fanconi anemia to test in the laboratory may help the study of cancer in the future.
- Conditions
- Fanconi Anemia, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00899522
- Testing
- biologic sample preservation procedure, biopsy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- OHSU Knight Cancer Institute
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Insulin Resistance, Hypermetabolism
Mechanisms of Improved Wound Healing and Protein Synthesis of Insulin and Metformin
Massive pediatric burns are associated with a persistent and sustained hypermetabolic response characterized by elevated levels of circulating catecholamine's, cortisol, and glucagon's, which can cause extreme muscle wasting…
- Conditions
- Insulin Resistance, Hypermetabolism, Hyperglycemia
- Trial name
- NCT01666665
- Testing
- Metformin, Sugar pill
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Lynch Syndrome, Li Fraumeni Syndrome
Video Capsule Examination in Patients With Lynch Syndrome
Surveillance for colorectal cancer (CRC) in patients predisposed to develop CRC during their lifetime has been impacted by access to colonoscopy suites and endoscopy specialists in the past couple of years.
- Conditions
- Lynch Syndrome, Li Fraumeni Syndrome, PTEN Hamartoma Syndrome, FAP
- Trial name
- PILLCAM (NCT06712095)
- Testing
- Video capsule investigation
- Where
- United Kingdom
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Royal Marsden NHS Foundation Trust
Lynch Syndrome
CAD-EYE System for the Detection of Neoplastic Lesions in Patients With Lynch Syndrome
Lynch syndrome (LS) is the most common genetic predisposition syndrome for colorectal cancer (CRC), responsible for around 2-4% of cancers.
- Trial name
- CADLYNCH (NCT05963191)
- Testing
- Colorectal cancer screening with CAD EYE colonoscopy
- Where
- France
- Volunteers
- 272 people
- Sponsor
- PERROD Guillaume
Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7)
Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia
This research study is studying identification of de novo Fanconi anemia in younger patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT01146210
- Testing
- laboratory biomarker analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Oncology Group
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
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
Li-Fraumeni Syndrome
A Pilot Study of Metformin in Patients With a Diagnosis of Li-Fraumeni Syndrome
Background: Li Fraumeni Syndrome (LFS) is a highly penetrant, autosomal dominant cancer predisposition disorder. Four main cancer types including sarcoma, adrenocortical carcinoma, breast cancer, and malignant brain tumors…
- Trial name
- NCT01981525
- Testing
- Metformin
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- National Cancer Institute (NCI)
Diabetes Mellitus, Type 1, Diabetes Mellitus, Type 2
Three-dimensional Measurements of Diabetic Foot Ulcers
Diabetic foot ulcers constitute an increasing health problem in Denmark concurrent with an ageing population and an increase in diabetes prevalence. Diabetic foot ulcers belong to the most serious and costly complications.
- Conditions
- Diabetes Mellitus, Type 1, Diabetes Mellitus, Type 2, Diabetes Complications, Diabetic Foot
- Trial name
- NCT02423850
- Testing
- 3D Wound Assessment Monitor (WAM) camera
- Where
- Denmark
- Volunteers
- 150 people
- Sponsor
- Odense University Hospital
Prostate Cancer, BRCA Mutation
The IMPACT Study - Identification of Men With a Genetic Predisposition to ProstAte Cancer
The IMPACT study is an international targeted prostate screening study of men at increased prostate cancer risk due to the presence of known pathogenic mutations in BRCA1 and BRCA2 genes and Mis-Match Repair genes (MLH1, MSH6…
- Conditions
- Prostate Cancer, BRCA Mutation, Mismatch Repair Gene Mutation, Genetic Predisposition to Disease
- Trial name
- NCT00261456
- Testing
- PSA test, Prostate Biopsy
- Where
- United Kingdom
- Volunteers
- 3,500 people
- Sponsor
- Institute of Cancer Research, United Kingdom