Clinical trials: Genetics Disease
Retinoschisis, Retinal Disease
Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis
The goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS.
- Conditions
- Retinoschisis, Retinal Disease, Retinal Degeneration, Eye Diseases
- Trial name
- NCT06289452
- Testing
- IVB102 Injection
- Where
- China
- Volunteers
- 18 people
- Sponsor
- InnoVec Biotherapeutics Inc.
Lynch Syndrome, Colon Cancer
Mesalamine for Colorectal Cancer Prevention Program in Lynch Syndrome
Multicenter, multinational, randomized, 2-arm, double-blind, phase II clinical study with 2000mg mesalamine, or placebo for prevention of colorectal neoplasia in Lynch Syndrome patients during and following daily intake for 2…
- Conditions
- Lynch Syndrome, Colon Cancer, Colon Neoplasm
- Trial name
- MesaCAPP (NCT04920149)
- Testing
- Mesalamine
- Where
- 8 sites in Denmark, Sweden
- Volunteers
- 150 people
- Sponsor
- Ann-Sofie Backman
Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.
- Trial name
- NCT01434511
- Testing
- OBI-1
- Where
- 3 sites in United States, South Africa and 1 more
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire
Severe Hemophilia
Individualized Exercise Combined With Manual Therapy in Severe Hemophilia Patients With Polyarthropathy
The aim of this study is to investigate the effect of individualized physical therapy, combined manual therapy and exercise intervention, for pain perception, range of motion (ROM), muscle strength, joint health, cardiopulmonary…
- Trial name
- NCT06535971
- Testing
- Manual therapy combined exercise
- Where
- Taiwan
- Volunteers
- 10 people
- Sponsor
- Far Eastern Memorial Hospital
Hemophilia A, Hemophilia B
An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the…
- Conditions
- Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT07644832
- Testing
- SR604
- Where
- 9 sites in China
- Volunteers
- 76 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Cardiomyopathies, Myocarditis
CMP-MYTHiC Trial and Registry - CardioMyoPathy With MYocarditis THerapy With Colchicine
Two-parallel groups randomized, single-blinded, multi-center phase III controlled trial in patients with chronic inflammatory cardiomyopathy to assess the efficacy of colchicine and associated prospective registry to assess the…
- Conditions
- Cardiomyopathies, Myocarditis, Inflammatory Cardiomyopathy, Heart Failure
- Trial name
- CMP-MYTHiC (NCT06158698)
- Testing
- Colchicine
- Where
- 10 sites in Italy
- Volunteers
- 80 people
- Sponsor
- Niguarda Hospital
Solid Tumor
Tuvusertib (M1774) Human Mass Balance and Absolute Bioavailability Study (DDRIVER Solid Tumors 303)
This is a single sequence 2-period open label study in participants with advanced solid tumors. The purpose of Period 1 of this study is to assess the mass balance to determine drug-related entities present in circulation and…
- Trial name
- NCT06308263
- Testing
- Tuvusertib [14C]Tuvusertib microtracer, Tuvusertib, Tuvusertib + [14C]Tuvusertib microdose bolus injection
- Where
- Hungary
- Volunteers
- 12 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Fabry Disease
Registry of Fabry Disease - A Multicenter Observational Study
The purpose of this study is to compile a registry of patients with Fabry disease, an inherited metabolic disorder. In this disease, an enzyme called a-galactosidase A, which normally breaks down a lipid (fatty substance) called…
- Trial name
- NCT00055016
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cystic Fibrosis
ECMO for Lung Transplantation in Cystic Fibrosis Patients
Cystic fibrosis (CF) is the most common genetically inherited disease in the Caucasian population. Bilateral lung transplantation (LUTX) is a viable option for these patients.
- Trial name
- RetroLUTX (NCT03919604)
- Testing
- Extracorporeal Life Support
- Where
- Italy
- Volunteers
- 80 people
- Sponsor
- Policlinico Hospital
Healthy Volunteer, Sjogren's Syndrome
Characterization of Diseases With Salivary Gland Involvement
Background: Salivary glands in and around the mouth and throat make saliva. Salivary gland disorders can affect a person s quality of life.
- Conditions
- Healthy Volunteer, Sjogren's Syndrome, Salivary Gland Disease
- Trial name
- NCT02327884
- Where
- United States
- Volunteers
- 1,150 people, healthy volunteers welcome
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Sickle Cell Disease
Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in MSD HSCT in Adult SCD Patients
In this study the investigators will prospectively investigate whether the addition of a 3-months long preconditioning with azathioprine to the alemtuzumab/TBI non-myeloablative conditioning results in improved disease-free…
- Trial name
- NCT05249452
- Testing
- Preconditioning with azathiprine and hydroxyurea (3 months)
- Where
- Netherlands
- Volunteers
- 20 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Severe Trauma
Predictors of Hypofibrinogenemia in Severe Trauma
In this retrospective and prospective study, the aim is to determine a score for predicting a fibrinogen <1.5g/L (corresponding to European threshold of Directors recommendations of fibrinogen concentrates)
- Trial name
- NCT03020849
- Where
- 2 sites in France
- Volunteers
- 1,038 people
- Sponsor
- Hospices Civils de Lyon
Heart Disease, Physical Frailty
Genetics and Epidemiology of Aging Associated Conditions in the Sardinian Population
Background: The island of Sardinia, an Italian region, has a population that is highly interrelated. Researchers have developed the SardiNIA longitudinal study to examine the possible genetic factors that predispose individuals…
- Conditions
- Heart Disease, Physical Frailty, Exaggerated Personality Traits
- Trial name
- NCT00182949
- Where
- Italy
- Volunteers
- 6,926 people, healthy volunteers welcome
- Sponsor
- National Institute on Aging (NIA)
Trisomy 21
Diagnosis Accuracy of Noninvasive Screening by PCR Digital for Down Syndrom
The main objective of this study is to estimate the intrinsic diagnostic qualities of the digital dPCR in the screening of T21 from a multicentrique sample of patients with pregnancies at high risk of T21 (risk > 1/250).
- Trial name
- DIgiT21 (NCT02872948)
- Testing
- Diagnostic
- Where
- France
- Volunteers
- 1,260 people
- Sponsor
- University Hospital, Grenoble
Cystic Fibrosis, Postural Defect
The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
The study was conducted in the age range of 18-45 December, who were diagnosed with Cystic fibrosis.
- Conditions
- Cystic Fibrosis, Postural Defect, Pulmonary Disease, Physical Inactivity
- Trial name
- NCT06088485
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Amyloidosis, Amyloid Cardiomyopathy
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- ATTRibute-CM (NCT03860935)
- Testing
- acoramidis
- Where
- 104 sites in United States, Australia and 16 more
- Volunteers
- 632 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Behcet Syndrome
Gut Microbiota and Behcet's Syndrome: a Dietary Intervention Trial (MAMBA Study)
Behçet's syndrome (BS) is an idiopathic, chronic, multi-systemic inflammatory disorder characterized by ocular disease, skin lesions, vascular, neurological and gastrointestinal involvement.
- Trial name
- NCT03962335
- Testing
- Vegetarian diet, Mediterranean diet with butyrate, Mediterranean diet
- Where
- Italy
- Volunteers
- 90 people
- Sponsor
- Azienda Ospedaliero-Universitaria Careggi
Laxity; Skin
Long-Term Efficacy and Duration of MFU-V at Multiple Depths and at 1.5 mm
The goal of this clinical trial is to evaluate the long-term safety and effectiveness of Ultherapy Prime (MFU-V) for improving lines, wrinkles, laxity, and crepiness of the lower face, submentum, and neck.
- Trial name
- NCT07224308
- Testing
- Ultherapy Prime
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- The Levine Center for Plastic Surgery
Diabetes Mellitus
Defining the Molecular and Physiological Mechanisms of Pancreatic Islet Cell Dysfunction Which Lead to Type 2 Diabetes
Defects in insulin secretion are central to the pathogenesis of type 2 diabetes (T2D) but the molecular basis and physiological consequences of those defects are poorly understood, impeding efforts to develop novel therapeutic…
- Trial name
- DIVA-Exeter (NCT02505308)
- Testing
- Glucose-Potentiated Arginine-Induced Insulin Secretion
- Where
- 2 sites in United Kingdom
- Volunteers
- 328 people, healthy volunteers welcome
- Sponsor
- Royal Devon and Exeter NHS Foundation Trust
Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101…
- Trial name
- NCT03389308
- Testing
- diacerein 1% ointment
- Where
- 3 sites in United States
- Volunteers
- 51 people
- Sponsor
- Castle Creek Pharmaceuticals, LLC