Clinical trials: Genetic Change
Gastrointestinal Stromal Tumors
A Study of Sunitinib In Young Patients With Advanced Gastrointestinal Stromal Tumor
Children and young adults with gastrointestinal stromal tumors (GIST) will be treated with sunitinib. The safety (including pharmacokinetics) and tolerability of sunitinib will be studied in these patients.
- Trial name
- NCT01396148
- Testing
- sunitinib malate dose escalation, sunitinib malate
- Where
- 9 sites in United States, Czechia and 1 more
- Volunteers
- 6 people
- Sponsor
- Pfizer
Hereditary Angioedema
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
- Trial name
- CHANGE 3 (NCT00462709)
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 48 sites in United States
- Volunteers
- 146 people
- Sponsor
- Shire
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)
Leiden Factor V Deficiency,Preeclampsia, Doppler Examination
Prediction of Outcome in Severe Preeclampsia
this summary studied the predictive values of factor V Leiden mutation , doppler indices , placental pathology to unfavorable fetal and maternal outcomes
- Conditions
- Leiden Factor V Deficiency,Preeclampsia, Doppler Examination, Palental Pathology
- Trial name
- NCT03871764
- Testing
- blood sample , doppler of uterine vessel and placenta
- Volunteers
- 100 people
- Sponsor
- Cairo University
DFNB9
An Investigator Initiated Study for OTOV101N+OTOV101C Injection
This study is an investigator initiated study to evaluate the safety, tolerability, and efficacy of OTOV101N+OTOV101C injection in treating patients with OTOF mutation-related deafness.
- Trial name
- NCT05901480
- Testing
- OTOV101N+OTOV101C Injections
- Where
- 23 sites in China
- Volunteers
- 25 people
- Sponsor
- Otovia Therapeutics
X-Linked Retinitis Pigmentosa
Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
- Trial name
- NCT05874310
- Testing
- FT-002
- Where
- 2 sites in China
- Volunteers
- 18 people
- Sponsor
- Frontera Therapeutics
Obesity, Childhood
Mutation of the LEPR Gene at Reunion Island: Involvement in Pediatric Obesity
The objective of this study is to estimate the proportion of the recurrent deletion Δ6-8 of the LEPR gene in the homozygous and heterozygous state in pediatric cases with severe and early ( before the age of 6) obesity (BMI ≥…
- Trial name
- GREOL (NCT04329260)
- Volunteers
- 250 people
- Sponsor
- Centre Hospitalier Universitaire de la Réunion
Cystic Fibrosis
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance…
- Trial name
- NCT02516410
- Testing
- VX-661 plus ivacaftor combination, Ivacaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 168 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Osteosarcoma
Cancer Genome Study Using Samples From Patients Treated on Clinical Trial SHR1020-SHR-1210-II-OS
RATIONALE: Studying samples of tumor tissue from patients with advanced osteosarcoma refractory to chemotherapy in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to…
- Trial name
- CSSG-02 (NCT03997747)
- Testing
- cytology specimen
- Where
- 2 sites in China
- Volunteers
- 38 people
- Sponsor
- Peking University People's Hospital
Amyotrophic Lateral Sclerosis (ALS)
Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
- Trial name
- NCT07743268
- Testing
- nL-TARDB-006
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- n-Lorem Foundation
Zellweger Spectrum Disorder
Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder
The goal of this observational study is to define the course of the retinal degeneration in a ZSD patient cohort. The objective of this study is to gather information so the investigators can: 1.
- Trial name
- ZSDvision (NCT06190626)
- Where
- 3 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Germline Mutation, Myelodysplastic Syndromes
Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants
Background: Hereditary hematopoietic malignancy (HHM) syndromes are a group of inherited disorders that raises the risk of blood cancers.
- Conditions
- Germline Mutation, Myelodysplastic Syndromes, Acute Myeloid Leukemia
- Trial name
- NCT07019155
- Where
- United States
- Volunteers
- 510 people
- Sponsor
- National Cancer Institute (NCI)
Medulloblastoma Recurrent, Medulloblastoma, Childhood, Recurrent
Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study
The purpose of this study is to create a biobank for patients diagnosed with Sonic Hedgehog Medulloblastoma at Baylor College of Medicine/Texas Children's Cancer Center.
- Conditions
- Medulloblastoma Recurrent, Medulloblastoma, Childhood, Recurrent, Medulloblastoma, SHH-activated and TP53 Mutant, Medulloblastoma, SHH-activated and TP53 Wildtype
- Trial name
- NCT07242963
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- Mohammad H. Abu Arja
Dengue (Virus); Fever, Sandfly, Sickle Cell Disease
Molecular and Cellular Basis of Severe Forms of Dengue in Sickle Cell Patients
Dengue virus (DENV) belongs to the genus of Flavivirus transmitted by the mosquito Aedes aegypti and is responsible for an infectious disease associated with different forms and severities such as dengue hemorrhagic fever or…
- Trial name
- DREPADENGUE (NCT07000747)
- Where
- Guadeloupe
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Bladder Cancer
Cost-Effectiveness in Bladder Cancer
This randomized clinical multicentre trial aims to evaluate the efficacy of microsatellite analysis on voided urine to detect tumour recurrences in the follow-up of patients with superficial urothelialcell carcinoma (UCC).
- Trial name
- NCT00126958
- Testing
- urinary analysis
- Volunteers
- 500 people
- Sponsor
- Erasmus Medical Center
Sickle Cell Disease
Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE)
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the…
- Trial name
- DREPAGLOBE (NCT03964792)
- Testing
- DREPAGLOBE drug product
- Where
- France
- Volunteers
- 6 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Muscular Dystrophy, Duchenne
Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
Placebo-controlled, multi-centre, randomized, double-blind dose escalation study. The aim is to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a…
- Trial name
- NCT02383511
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Astrocytoma, IDH-Mutant, Grade 2, Astrocytoma, IDH-Mutant, Grade 3
Telaglenastat With Radiation Therapy and Temozolomide in Treating Patients With IDH-Mutated Diffuse Astrocytoma or Anaplastic Astrocytoma
This phase 1b trial studies the side effects and best dose of telaglenastat in combination with radiation therapy and temozolomide in treating patients with IDH-mutated diffuse or anaplastic astrocytoma.
- Trial name
- NCT03528642
- Testing
- Questionnaire Administration, Radiation Therapy, Telaglenastat Hydrochloride
- Where
- 25 sites in United States
- Volunteers
- 23 people
- Sponsor
- National Cancer Institute (NCI)
Brain Tumor, Cancer
000860: A Phase I/II Study of Zotiraciclib for Recurrent Malignant Gliomas With Isocitrate Dehydrogenase 1 or 2 (IDH1 or IDH2) Mutations
Protocol # 000860 Background: Diffuse gliomas are tumors that affect the brain and spinal cord. Gliomas that develop in people with certain gene mutations (IDH1 or IDH2) are especially aggressive. Better treatments are needed.
- Trial name
- NCT05588141
- Testing
- Zotiraciclib
- Where
- 2 sites in United States
- Volunteers
- 96 people
- Sponsor
- National Cancer Institute (NCI)
Atopic Dermatitis (Eczema), Atopic Dermatitis, Probiotics
Prebiotic Effects of Mulberry Fruit in Children and Adolescents With Atopic Dermatitis
This project proposes a randomized controlled human study to explore the prebiotic effects of consuming mulberry juice on atopic dermatitis in children and adolescents.
- Trial name
- NCT06636240
- Testing
- Mulberry fruit juice, Mometasone furoate 0.1% cream
- Where
- 2 sites in Taiwan
- Volunteers
- 120 people
- Sponsor
- Taipei Medical University Shuang Ho Hospital