Clinical trials: Gene
Lymphoma
Biomarkers in Tissue Samples From Patients With Hodgkin Lymphoma Enrolled on ECOG-2496 Clinical Trial
RATIONALE: Studying samples of tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
- Trial name
- NCT01505712
- Testing
- gene expression analysis, laboratory biomarker analysis
- Volunteers
- 393 people
- Sponsor
- ECOG-ACRIN Cancer Research Group
Neuroblastoma
Biological Therapy and Gene Therapy in Treating Children With Recurrent or Refractory Neuroblastoma
RATIONALE: Biological therapies use different ways to stimulate the immune system and stop cancer cell from growing. Inserting genetic material made in the laboratory into a person's blood cells may make the body build an immune…
- Trial name
- NCT00006480
- Testing
- aldesleukin, therapeutic autologous lymphocytes, chemotherapy
- Where
- 2 sites in United States
- Sponsor
- Fred Hutchinson Cancer Center
Intellectual Disability, Congenital Abnormalities
Apparently Balanced Chromosomal Translocation/ Next-generation Sequencing/ Intellectual Disability
Apparently balanced chromosomal rearrangement (ABCR) associated with an abnormal phenotype is a rare but problematic event. It occurs in 6% of de novo reciprocal translocations and 9% of de novo inversions.
- Trial name
- ANI (NCT02451761)
- Testing
- Blood sampling
- Where
- France
- Volunteers
- 55 people
- Sponsor
- Hospices Civils de Lyon
Autism Spectrum Disorder
MRI Study of Subjects with ASD, Their Relatives and TD
This project is structured around a central study called "Study of genetic factors involved in autism and related conditions ("Genes and Autism" study, sponsor: INSERM).
- Trial name
- NCT05470088
- Testing
- Neuroimaging (MRI)
- Where
- 2 sites in France
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Attention Deficit Hyperactivity Disorder
PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations
This is PART A of a 2-part, 6-week, double-blind, dose-optimization, parallel-group study in children and adolescents (ages 6-17 years) with ADHD with and without CNVs in specific genes implicated in glutamatergic signaling and…
- Trial name
- NCT03265119
- Testing
- AEVI-001
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
Gene Therapy, Hemophilia B
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low.
- Trial name
- NCT05044845
- Testing
- Interview
- Where
- 6 sites in United States, Nepal and 4 more
- Volunteers
- 150 people
- Sponsor
- St. Jude Children's Research Hospital
Leukemia, Cancer
WT1 for the Detection of Minimal Residual Disease
The purpose of this study is to determine if WT1 is an adequate measurement of minimal residual disease in leukemic patients.
- Trial name
- NCT00179829
- Testing
- WT 1 Testing
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Infant, Very Low Birth Weight
L-arginine Concentrations and CPS Polymorphisms in VLBW Infants
Plasma L-arginine concentrations are decreased in premature infants with necrotizing enterocolitis (NEC). A carbamoyl-phosphate synthetase 1 (CPS1) polymorphism has been correlated with low plasma concentrations of L-arginine in…
- Trial name
- NCT00554866
- Testing
- blood sample and buccal swab sample
- Where
- 4 sites in Italy, Netherlands and 1 more
- Volunteers
- 477 people
- Sponsor
- Maastricht University Medical Center
Exercise Physiology
Performance Determinants Factors in Elite Endurance Athletes.
Several studies have investigated determinant factors for endurance performance among different athletes, untrained individuals and patients focusing on the impact of age, genes and training intensity.
- Trial name
- NCT03569566
- Testing
- Age, Genes, Training, Tickbourne Disease and Endurance
- Where
- Norway
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- University of South-Eastern Norway
Dentatorubral-Pallidoluysian Atrophy
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide…
- Trial name
- NCT06706388
- Testing
- nL-ATN1-002
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- n-Lorem Foundation
Congenital Vertical Talus, Familial Encephalopathy With Neuroserpin Inclusion Bodies
Genetic Disease Gene Identification
This is a a study to identify inherited disease genes. The study will use molecular techniques to map genetic diseases using techniques such as Affymetrix SNP chips.
- Conditions
- Congenital Vertical Talus, Familial Encephalopathy With Neuroserpin Inclusion Bodies, Idiopathic Generalised Epilepsy, Familial Dementia
- Trial name
- NCT00916903
- Where
- United States
- Volunteers
- 176 people, healthy volunteers welcome
- Sponsor
- State University of New York - Upstate Medical University
Melanoma, Solid Tumor
Tovorafenib (DAY101) or in Combination With Pimasertib for Participants With Melanoma and Other Solid Tumors
This is a subprotocol of Master Protocol DAY101-102 and is a Phase 1b/2, multi-center, open label subprotocol of participants ≥12 years of age, with recurrent or progressive solid tumors with alterations in the key proteins of…
- Conditions
- Melanoma, Solid Tumor, Pilocytic Astrocytoma, Non Small Cell Lung Cancer
- Trial name
- NCT07121829
- Testing
- Tovorafenib, Tovorafenib Drug: Pimasertib
- Where
- 10 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Day One Biopharmaceuticals, Inc.
Neuroblastoma
3RD GENERATION GD2 SPECIFIC CHIMERIC ANTIGEN RECEPTOR TRANSDUCED AUTOLOGOUS NATURAL KILLER T-CELLS FOR NEUROBLASTOMA
This research study is for patients that have a cancer called Neuroblastoma that has either come back after treatment or did not respond to the standard medicines used to treat it.
- Trial name
- GINAKIT (NCT02439788)
- Testing
- cyclophosphamide, Fludarabine, GINAKIT Cells
- Where
- United States
- Sponsor
- Baylor College of Medicine
Glucocorticoid Resistance, Gene
Association Between the Effect of Glucocorticoid Pulse Therapy on Neuromyelitis Optica (NMO) and Gene Polymorphism
Aims of this study are to observe the correlation between ABCB1、VDR、TBX21、NR3C1 and other gene polymorphisms and the effect of glucocorticoid shock therapy on neuromyelitis optica.
- Conditions
- Glucocorticoid Resistance, Gene, Neuromyelitis Optica
- Trial name
- NCT04601142
- Testing
- Methylprednisolone
- Where
- China
- Volunteers
- 350 people
- Sponsor
- Beijing Tongren Hospital
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon
Brain and Central Nervous System Tumors
Studying Gene Expression and Location in Samples From Patients With Brain Tumors
RATIONALE: Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to brain tumors.
- Trial name
- NCT01585389
- Testing
- gene expression analysis, genetic linkage analysis, microarray analysis
- Volunteers
- 10 people
- Sponsor
- Children's Oncology Group
Inflammatory Bowel Diseases, Crohn Disease
Exploration of the Activity of DNA Located Outside of Cellular Nucleus to Amplify Inflammation in Inflammatory Bowel Disease in Children Through Biological Pathway Cyclic GMP-AMP Synthase (cGAS) - Stimulator of Interferon Genes (STING)
Frequency of Inflammatory Bowel Diseases in children (IBD)-Crohn's disease (CD), Ulcerative colitis (UC) is constantly increasing.
- Conditions
- Inflammatory Bowel Diseases, Crohn Disease, Ulcerative Colitis
- Trial name
- ROXANE (NCT05916274)
- Testing
- Blood and fecal samples, Coolonic biopsies
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Centre Hospitalier Régional d'Orléans
Cancer
Immunotherapy Based on Tumor Associated Antigen-specific Immune Effector Cells
The primary objectives are to evaluate the safety and efficacy of infusion of autologous tumor associated antigen-specific engineered immune effector cells (EIE).
- Trial name
- NCT03535246
- Testing
- Engineered Immune Cells
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)
WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to <84 months. The study follows an open-label, dose-escalation design.
- Trial name
- NCT06112275
- Testing
- ETX101
- Where
- Australia
- Volunteers
- 4 people
- Sponsor
- Encoded Therapeutics
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