Clinical trials: Genetic Condition, open to volunteers
Cystic Fibrosis (CF), Bronchiectasis Adult
Novel Sensors and Artificial Intelligence for Detection of Acute Pulmonary Exacerbations in Cystic Fibrosis and Bronchiectasis
The goal of this observational study is to learn about new sensors that measure changes in the body that happen during a chest infection, such as breathing rate and heart rate.
- Trial name
- NOVEL (NCT07289100)
- Testing
- Non-Interventional Study
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Papworth Hospital NHS Foundation Trust
Acute Coronary Syndrome, Gender
Prospective Multicenter Registry of Gender, Diversity and Inclusion (GEDI) of Women With Acute Coronary Syndrome
Create a multicenter prospective registry that collects information from women affected by acute coronary syndrome (ACS). This registry aims to understand the diversity in the presentation of women with ACS.
- Conditions
- Acute Coronary Syndrome, Gender, Genetic Predisposition
- Trial name
- ACS GEDI (NCT06441942)
- Where
- 4 sites in Italy
- Volunteers
- 100 people
- Sponsor
- IRCCS San Raffaele
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Brow Ptosis, Dermatochalasis
Enhancing the Durability of Thread-Assisted Brow Suspension Through Upper Blepharoplasty-Assisted Ligament Release
This prospective clinical study aims to investigate whether combining upper blepharoplasty with limited release of the lateral brow-retaining ligaments improves the long-term effectiveness of barbed thread brow suspension.
- Conditions
- Brow Ptosis, Dermatochalasis, Periorbital Aging
- Trial name
- NCT07879456
- Testing
- Upper Blepharoplasty, Barbed Thread Brow Suspension, Lateral Brow-Retaining Ligament Release
- Where
- Turkey (Türkiye)
- Volunteers
- 90 people
- Sponsor
- Muhammed Mustafa Demirok
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Acute Lymphoblastic Leukemia
Treatment of Breakpoint Cluster Region-Abelson (BCR-ABL) Negative ALL in Adults
After consolidation therapy adult patients (≥18 yr) with Ph-negative ALL will be treated with continuation chemotherapy or allogeneic hematopoietic stem cell transplantation (alloHSCT) according to both measurable residual…
- Trial name
- NCT04179929
- Testing
- Pediatric-type of chemotherapy, allogeneic HSCT
- Where
- 108 sites in Spain
- Volunteers
- 300 people
- Sponsor
- PETHEMA Foundation
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Refractory Hypercholesterolemia, Familial Hypercholesterolemia
Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing…
- Trial name
- NCT06293729
- Testing
- NGGT006
- Volunteers
- 9 people
- Sponsor
- Suzhou Municipal Hospital
Neurofibromas, Cutaneous, Neurofibromatosis 1
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs).
- Trial name
- NCT06300502
- Testing
- Kybella
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Massachusetts General Hospital
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT)
Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts
This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific T-cell (NST) therapy for chronic norovirus infection in participants following hematopoietic stem cell transplantation (HSCT) or who are…
- Conditions
- Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
- Trial name
- ATLANTIC (NCT04691622)
- Testing
- Norovirus -specific T-cell (NST) therapy
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Children's National Research Institute
Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema
Phase 2 Randomized Double-blind Study to Assess Topical Zabalafin Hydrogel Vs Vehicle in Mild to Moderate AD.
A Phase 2b study investigating the efficacy of zabalafin in people with mild to moderate atopic dermatitis (eczema).
- Conditions
- Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema, Atopic Dermatitis, Atopic Dermatitis (AD)
- Trial name
- CLEAR-AD1 (NCT06855745)
- Testing
- zabalafin
- Where
- 12 sites in Australia
- Volunteers
- 72 people
- Sponsor
- Alphyn Biologics
Down Syndrome
Rebound Therapy on Ventilatory Functions in Down Syndrome
The purpose of this study is to examine the effects of rebound therapy on ventilatory functions in children with Down Syndrome.
- Trial name
- NCT07425353
- Testing
- Chest Physical Therapy Protocol, Rebound Therapy Protocol
- Volunteers
- 40 people
- Sponsor
- Beni-Suef University
Tuberculosis
Paradoxical Tuberculosis Reactions in Patients Without HIV Infection
Background: Most people with tuberculosis (TB) feel better after starting treatment. But for some people, the opposite happens. They may feel better at first, but then suddenly get worse. This is a paradoxical reaction.
- Trial name
- NCT04052022
- Where
- 3 sites in United States, Mexico
- Volunteers
- 140 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Glucose Metabolism Disorders in Cystic Fibrosis
Reproducibility of Continuous Glucose Monitoring (CGM) in the Assessment of Glucose Metabolism Disorders in Children With Cystic Fibrosis
Glucose metabolism disorders in cystic fibrosis (CF-GMD) are common and have a negative clinical impact. Screening currently relies on the oral glucose tolerance test (OGTT); however, this procedure is burdensome and complex for…
- Trial name
- CGMUCODIAB (NCT07851831)
- Testing
- Continuous Glucose Monitoring, Eating Habits Questionnaire, Use of an actigraphy watch
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
A Study to Investigate the Effects of JMKX003142 on QTc Interval in Healthy Adults
The primary objective of this study is to assess the effect of a therapeutic and supratherapeutic oral dose of JMKX003142 on the corrected cardiac QT interval relative to a placebo in healthy participants
- Trial name
- NCT07470177
- Testing
- JMKX003142 tablets
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Jemincare
Huntington Disease
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Trial name
- NCT06826612
- Testing
- SPK-10001
- Where
- 5 sites in United States
- Volunteers
- 53 people
- Sponsor
- Hoffmann-La Roche
Atopic Dermatitis
Changes in Skin Myco- and Microbiome After Lebrikizumab Treatment
The goal of this observational study is to characterize the skin myco- and microbiome of patients with atopic dermatitis before and after initiation of treatment with lebrikizumab.
- Trial name
- NCT07807228
- Where
- Denmark
- Volunteers
- 60 people
- Sponsor
- University Hospital Bispebjerg and Frederiksberg
Eczema/Atopic Dermatitis, Otitis Externa
Nigella Sativa Oil for Eczematous Otitis Externa
Eczematous otitis externa is a common inflammatory condition of the external auditory canal characterized by itching, discomfort, discharge, and pain.
- Trial name
- NCT07525505
- Testing
- Mometasone Furoate (MF), Nigella sativa oil
- Where
- Turkey (Türkiye)
- Volunteers
- 85 people
- Sponsor
- Bezmialem Vakif University
Fragile X Syndrome (FXS), Autism Spectrum Disorder
Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder
Fragile X Syndrome (FXS) is a rare genetic syndrome, caused by a mutation in the FMR1 gene located on the X chromosome. It is considered the leading hereditary cause of intellectual disability (ID) and the primary cause of Autism…
- Trial name
- NCT06677866
- Testing
- Cognitive behavioral therapy, Waiting List
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
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