Clinical trials: Genes
Cutaneous Melanoma
5-year Study to Track Clinical Application of DecisionDx-Melanoma Gene Expression Profile Assay Results
The INTEGRATE Registry Study follows patients with cutaneous melanoma who have had the DecisionDx-Melanoma gene expression assay performed.
- Trial name
- INTEGRATE (NCT02355574)
- Where
- 3 sites in United States
- Volunteers
- 301 people
- Sponsor
- Castle Biosciences Incorporated
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Autoinflammatory Disease, Type 1 Interferonopathies
Combining Gene Editing and iPS to Study and Treat Monogenic Autoinflammatory Diseases
Type I interferonopathies (T1I) are rare autoinflammatory diseases characterized by dysregulated type I interferon signaling, leading to severe multisystem inflammation, including skin and lung involvement.
- Trial name
- MAGIC (NCT07889947)
- Where
- 2 sites in Italy
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Istituto Giannina Gaslini
Congenital Diaphragmatic Hernia
Diaphragmatic Hernia Research & Exploration, Advancing Molecular Science
The goal of this study is to identify genes that convey susceptibility to congenital diaphragmatic hernia in humans. The identification of such genes, and examination of their structure and function, will enable a delineation of…
- Trial name
- DHREAMS (NCT00950118)
- Where
- 14 sites in United States, Egypt
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Gene Amplification
LAMP Assay Versus PCR for Detection of blaNDM-1 and blaKPC Genes
The aim of this work is Detection of gram-negative isolates from different clinical samples,determination the antimicrobial susceptibility pattern of gram-negative isolates to various antimicrobial agents, Molecular Detection of…
- Trial name
- NCT04479189
- Volunteers
- 100 people
- Sponsor
- Assiut University
Malignant Mesothelioma
Vaccine Therapy and Ganciclovir in Treating Patients With Mesothelioma
RATIONALE: Ganciclovir may ease some of the side effects of cancer treatment. Vaccines made from a person's modified malignant mesothelioma cells may make the cancer more sensitive to ganciclovir.
- Trial name
- NCT00006216
- Testing
- PA-1-STK ovarian carcinoma vaccine, ganciclovir
- Where
- United States
- Sponsor
- Louisiana State University Health Sciences Center in New Orleans
Hearing Loss, Genetic Disease
Auditory Restoration and Outcomes After Gene Therapy and Cochlear Implantation: a PRospective Cohort Analysis (AURORA)
This cohort study aims to explore the trends and differences in multidimensional perceptual levels of patients after cochlear implants or gene therapy, as well as to comprehensively assess the efficacy of gene therapy for…
- Conditions
- Hearing Loss, Genetic Disease, Speech Perception
- Trial name
- NCT06237790
- Where
- 6 sites in China
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Eye & ENT Hospital of Fudan University
Rare Diseases
Finding Genes for Rare Diseases
This study will help the investigator understand the pathogenesis of different rare genetic conditions and to establish database of rare genetic databases.
- Trial name
- NCT02724995
- Where
- United States
- Sponsor
- University of Kentucky
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma
Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with brain tumors, solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have come back (relapsed) or do not respond to…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma
- Trial name
- NCT03213665
- Testing
- Tazemetostat
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Graft Rejection, Lung Disease
Lung Allograft Rejection Gene Expression Observational (LARGO) Study
The objective of the LARGO Study protocol is to collect peripheral blood samples, select associated lung biopsy pathology slides, and clinical data from lung transplant recipients to perform molecular analyses in association with…
- Trial name
- LARGO (NCT00751309)
- Testing
- Non-interventional
- Where
- 20 sites in United States, Austria and 3 more
- Volunteers
- 2,044 people, healthy volunteers welcome
- Sponsor
- XDx
Glaucoma, Healthy
Cause of Pigment Dispersion Syndrome
The purpose of this study is to learn how pigment is released from the iris (the colored part of the eye) in patients with pigment dispersion syndrome.
- Conditions
- Glaucoma, Healthy, Pigment Dispersion Syndrome
- Trial name
- NCT00005919
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- National Eye Institute (NEI)
Peri-Implantitis
Peri-implantitis and MMP-8
Peri-implantitis is defined as the pathological condition around dental implants characterized by inflammation in the peri-implant mucosa and progressive bone loss, eventually leading to implant loss.
- Trial name
- NCT05711407
- Testing
- Gene Polymorphisms of MMP-8
- Where
- Greece
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Aristotle University Of Thessaloniki
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to…
- Trial name
- CHORD (NCT05788536)
- Testing
- DB-OTO
- Where
- 17 sites in United States, Germany and 3 more
- Volunteers
- 36 people
- Sponsor
- Regeneron Pharmaceuticals
Inflammatory Bowel Diseases
Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for IBD Caused by IL-10R Gene Deficiency
Very early onset inflammatory bowel disease (VEO-IBD) is a special subtype of children's inflammatory bowel disease (IBD). VEO-IBD is mostly caused by single-gene defects and can be cured by allo-hematopoietic stem cell…
- Trial name
- NCT04170192
- Testing
- Cord Blood Stem Cell Transplantation
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Fudan University
Anovulation, Hyperandrogenism
Whole Genome Analysis for the Detection of Key Genes in the Polycystic Ovary Syndrome
Background: Infertility affects up to 15% of married couples. About half are attributable to female factors, among which anovulation is the leading cause.
- Conditions
- Anovulation, Hyperandrogenism, Polycystic Ovary Syndrome, Single Nucleotide Polymorphism
- Trial name
- NCT00665171
- Where
- Israel
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Maaynei Hayesha Medical Center
Gaucher's Disease
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Gaucher's disease is a lysosomal storage disease resulting from glycocerebroside GLUCOCEREBROSIDE (1) accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages.
- Trial name
- NCT00001234
- Testing
- human glucocerebrosidase cDNA
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Child Language
Genetic Biomarkers Associated With Child Language Development in Taiwan
A study of the relation between genetic biomarkers and child language development in Taiwan.
- Trial name
- NCT04518787
- Where
- Taiwan
- Volunteers
- 596 people, healthy volunteers welcome
- Sponsor
- Chang Gung Memorial Hospital
Kidney Diseases, Transplant Failure
Evaluation of APOL1 Gene Variants in Kidney Donors and Their Impact on Long-term Renal Function in Donors and Recipients
Evaluation of the frequency of APOL1 gene variants in kidney donors and the impact of these variants on the long-term renal function of kidney transplant donors and recipients.
- Trial name
- NCT06116721
- Where
- Brazil
- Volunteers
- 2,450 people
- Sponsor
- Hospital do Rim e Hipertensão
MPS IIIA, Sanfilippo Syndrome
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT02716246
- Testing
- UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 36 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Spinal Muscular Atrophy
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
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