Clinical trials: Gene Therapy
Postmenopause, Osteoporosis
Effects of Tibolone Treatment on the Endometrium
Tibolone, a tissue-selective compound with a combination of estrogenic, progestogenic and androgenic properties, is used as an alternative for estrogen or estrogen plus progesterone hormone therapy for the treatment of symptoms…
- Trial name
- NCT00294463
- Testing
- Tibolone, Estradiol, Estradiol + Medroxy Progesterone Acetate
- Where
- Netherlands
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Erasmus Medical Center
Nephronophthisis, NPH1
Research of Therapeutic Targets in the Frame of Nephronophthisis and Renal Associated Ciliopathies
Nephronophthisis (NPH) is an autosomal recessive, genetically heterogeneous disease, with mutations identified in over 20 genes (notably NPHP1 and NPHP4).
- Conditions
- Nephronophthisis, NPH1, Autosomal, Recessive
- Trial name
- NPH1 (NCT06648044)
- Testing
- Description: Research of therapeutic targets in the frame of nephronophthisis and renal associated ciliopathie
- Where
- France
- Volunteers
- 310 people, healthy volunteers welcome
- Sponsor
- Imagine Institute
Amino Acid Metabolism, Inborn Errors
Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
RATIONALE: The urea cycle is the process in which nitrogen is removed from the blood and converted into urea, a waste product found in urine .
- Trial name
- NCT00004307
- Testing
- Protein and calorie controlled diet, Ornithine transcarbamylase vector
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- National Center for Research Resources (NCRR)
Any Conditions That Are Genetic or Suspected to be Genetic in Origin
UCSF Center for Genome Surgery Biobank and Registry
Gene editing approaches (including but not limited to: CRISPR, LNP, AAV, RNAi etc.) for the medical specialty of interventional genomics require (1) determination of the clinical relevance of genomic variants and (2) systematic…
- Trial name
- NCT07396285
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Obesity, Cardiovascular Risk
Long-term Effects of Lifestyle Intervention in Obesity and Genetic Influence
The purpose of the study is to determine the genetic influence on short-, middle- and longterm effects of an inpatient lifestyle therapy program in overweight and obese children and adolescents.
- Trial name
- LOGIC (NCT01067157)
- Testing
- LOGIC
- Where
- Germany
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Technical University of Munich
Neuroblastoma
A Pilot Study on ALK Gene Mutations in Neuroblastoma
Neuroblastoma (NB) originates from the sympathetic nervous system and is one of the most common cancers in infants and children.
- Trial name
- NCT01986595
- Where
- Taiwan
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Vitiligo
5-fluorouracil for Treatment of Stable Vitiligo
Vitiligo, a significant dermatologic challenge affecting 0.5 to 2% of the global population. Despite various existing medical approaches, current vitiligo treatments are still far from optimal.
- Trial name
- NCT06209138
- Testing
- 5 fluorouracil, Saline
- Where
- Egypt
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Al-Azhar University
Peutz-Jeghers Syndrome
Study of Accurate Diagnosis and Treatment of Peutz-Jeghers Syndrome
The mutation of STK11 has been regcognized to be the major cause of Peutz-Jeghers syndrome (PJS).The aim of this study was to confirm the mutation rate of gene associated with gastrointestinal malignancies,including STK11…
- Trial name
- NCT03806075
- Testing
- Second generation sequencing, 16s rRNA gene sequencing
- Where
- China
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Yiqi Du
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
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)
Psoriasis, Psoriatic Arthritis
IKBKB Gene Polymorphism (Single Nucleotide Polymorphism) (SNP) in Psoriatic and Psoriatic Arthritis Patients
Psoriasis is a chronic inflammatory skin disease characterized by scaly indurated erythema. It impairs patients' quality of life enormously. Psoriasis refers to a persistent inflammatory illness of the skin and joints.
- Trial name
- NCT06729463
- Testing
- Humira
- Where
- Egypt
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- South Valley University
Acute LHON, Onset Within Three Months
Efficacy Study of Gene Therapy for The Treatment of Acute LHON Onset Within Three Months
Efficacy Study of Gene Therapy for The Treatment of Acute Leber's Hereditary Optic Neuropathy (LHON) onset within three months
- Conditions
- Acute LHON, Onset Within Three Months, Onset Between 3 to 6 Months, Onset Between 6 to 12 Months
- Trial name
- LHON (NCT03428178)
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Bin Li
Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR…
- Trial name
- NCT06360289
- Testing
- Standard of Care
- Where
- France
- Volunteers
- 346 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
BRCA Mutation, Ovarian Cancer
Prediction of Germline BRCA 1/2 Genes From Healthy Ovaries
The project aims at enhancing performance metrics and prospectively validating a radiogenomics model based on ovarian US images for predicting germline breast cancer susceptibility gene 1 and/or 2 (BRCA) status in women with…
- Conditions
- BRCA Mutation, Ovarian Cancer, Ultrasound Therapy; Complications
- Trial name
- PROBE-II (NCT05769517)
- Testing
- Germinal BRCA
- Where
- Italy
- Volunteers
- 6,465 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sickle Cell Disease
Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic…
- Trial name
- NCT04416178
- Where
- United States
- Volunteers
- 352 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Reading Disorder
Gene x Environment Interplay in Developmental Dyslexia Treatment: A Round-trip Translation Between Humans and Animal
Developmental dyslexia affects 7% of school-age children (Male:Female ratio of 1.5:1) and incurs disadvantages in education and occupation.
- Trial name
- Gedys (NCT06186882)
- Testing
- Action Video-Game
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Congenital Hearing Loss, Gene Therapy
Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss
This study aims to establish a novel multimodal evaluation and precision rehabilitation system for patients with congenital hearing loss who have received hearing aids, cochlear implantation, or emerging gene therapy.
- Conditions
- Congenital Hearing Loss, Gene Therapy, Speech Perception, Rehabilitation
- Trial name
- NCT07024524
- Testing
- Rehabilitation training
- Where
- China
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Eye & ENT Hospital of Fudan University
Congenital Central Hypoventilation Syndrome
Late-onset Congenital Central Hypoventilation Syndrome and the Mutation of Phox2B Gene
Congenital central hypoventilation syndrome (CCHS) is a rare disorder of respiratory control characterized by ventilatory impairment that results in arterial hypoxemia.
- Trial name
- CCHS (NCT01225679)
- Testing
- positive airway pressure, Non-invasive mechanic ventilation
- Where
- Brazil
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Associação Fundo de Incentivo à Pesquisa
Retroviridae Infections, Cancer
Long-Term Follow-Up of Recipient of Gene Transfer Research
The goal of this clinical research study is to collect information about the long-term safety of gene transfer therapy. This study is also designed to create a database of medical information about patients who have received gene…
- Trial name
- NCT01492036
- Testing
- Blood Tests, Questionnaires
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center
Short Stature
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
With this study we want to investigate the pharmacokinetic (PK) effect of a single injection of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy controls.
- Trial name
- NCT02636270
- Testing
- Increlex
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati