Clinical trials: Birth Defect, open to volunteers
Neurofibromatosis Type 2, Vestibular Schwannoma
Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
This is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with NF2-related schwannomatosis (NF2-SWN, formerly known as neurofibromatosis type 2) with…
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma
- Trial name
- INTUITT-NF2 (NCT04374305)
- Testing
- Brigatinib, Neratinib, Retifanlimab
- Where
- 6 sites in United States
- Volunteers
- 109 people
- Sponsor
- Scott R. Plotkin, MD, PhD
Hemoglobinopathy (Disorder), Severe Aplastic Anemia
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition.
- Conditions
- Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
- Trial name
- NCT04356469
- Testing
- Haploidentical Hematopoietic Cell Transplantation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins All Children's Hospital
Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID)
Evaluation of Patients With Immune Function Abnormalities
This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation.
- Conditions
- Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
- Trial name
- NCT00128973
- Where
- United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Total Colonic Aganglionosis, Hirschsprung Disease
Robot/Laparoscopic-Assisted Transanal Transection Duhamel Versus Modified Soave Pull-Through for TCA
Total colonic Hirschsprung disease (TCA) is the most severe form of Hirschsprung disease and is commonly managed with neonatal enterostomy followed by delayed definitive pull-through.
- Conditions
- Total Colonic Aganglionosis, Hirschsprung Disease, Duhamel, Soave
- Trial name
- NCT07343622
- Testing
- Transanal Transection Duhamel operation, Modified Soave Pull-Through
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Zunyi Medical College
Dental Implants, TNF-alpha Inhibitors
Dental Implant Healing With TNF-Alpha Inhibitors
The goal of this pilot study is to learn about healing after dental implant placement in patients taking a class of biologic drug called Tumor Necrosis Factor or TNF-alpha antagonist or inhibitor.
- Conditions
- Dental Implants, TNF-alpha Inhibitors, Edentulism
- Trial name
- NCT06538870
- Testing
- Placement of dental implants
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Lung Cancer
Detection of Genetic Markers of Lung Cancer
The purpose of this research study is to determine the genetic changes and immunologic changes that are involved in the development and progression of bronchogenic lung cancer.
- Trial name
- NCT00280202
- Testing
- Biopsy of the major carinal area, Biopsy of abnormal & suspicious areas of the bronchial tree, Evaluation of the tumor for DNA mutations
- Where
- United States
- Volunteers
- 6,000 people
- Sponsor
- University of Pittsburgh
Aggressive Behavior in Fragile X Syndome
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
The goal of this clinical trial is to learn if drug SRX246 works to treat irritability, agitation, aggression and self-injury (IAAS) behaviors in adult males with Fragile X Syndrome (FXS).
- Trial name
- NCT07328529
- Testing
- SRX246
- Where
- 3 sites in United States
- Volunteers
- 80 people
- Sponsor
- Azevan Pharmaceuticals
Sanfilippo Syndrome (MPS III)
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
The goal of this clinical trial is to learn if one infusion of rAAV9-CMV-hNAGLUop can treat patients with Sanfilippo syndrome (MPS III) 6 months or older.
- Trial name
- NCT07818759
- Testing
- rAAV9-CMV-hNAGLUop
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- NeuroGT
Transfusion Dependent Beta-Thalassemia
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced…
- Trial name
- NCT05762510
- Testing
- GMCN-508B (LentiRed)
- Where
- China
- Volunteers
- 5 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Macrosomia, Fetal, Gestational Diabetes Mellitus in Pregnancy
New Markers of Glycation to Predict Gestational Diabetes Mellitus and Macrosomia.
Gestational diabetes mellitus (GDM) increases the risk of macrosomia and other adverse pregnancy outcomes. Screening strategies are debated: universal vs.
- Trial name
- GLYCAGEST (NCT06048510)
- Testing
- Pregnant women
- Where
- France
- Volunteers
- 800 people
- Sponsor
- University Hospital, Bordeaux
Bowel Dysfunction, Dysmotility
Comparison of a New Non-invasive Device With the Invasive Gold-standard Investigation to Assess Function of the Large Bowel in Children
The goal of this clinical trial is to learn if the new non-invasive device (Body Surface Colonic Mapping) works to accurately assess colonic movement and function in children.The main questions it aims to answer are: Are Body…
- Conditions
- Bowel Dysfunction, Dysmotility, Hirschsprung Disease, Anorectal Malformations
- Trial name
- NCT07789782
- Testing
- Body Surface Colonic Mapping
- Where
- Australia
- Volunteers
- 50 people
- Sponsor
- Murdoch Childrens Research Institute
Familial Mediterranean Fever, Non-celiac Gluten Sensitivity
Can Gluten/Wheat or Other Foods be Responsible for FMF Attacks
Familial Mediterranean Fever (FMF) is a chronic hereditary autoinflammatory disease caused by mutations in the MEditerranean FeVer (MEFV) gene which codes for pyrin.
- Trial name
- NCT06338891
- Testing
- Main questionnaire, Secondary questionnaire
- Where
- Italy
- Volunteers
- 60 people
- Sponsor
- University of Palermo
Congenital Heart Disease, Heart Diseases
IPSC Repository of Pediatric Cardiovascular Disease
Obtain blood samples for generation and maintenance of induced pluripotent stem cells (iPSCs) and genomic/DNA sequencing for biomedical research that will improve the understanding and treatment of pediatric cardiovascular disease
- Trial name
- NCT05550324
- Testing
- Blood draw
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Mingtao Zhao
Dermatitis, Atopic
A First-in-human Study to Investigate Single Doses of DCY636 in Healthy Volunteers and Multiple Doses in Participants With Moderate to Severe Atopic Dermatitis
The purpose of this first-in-human (FIH) study is to assess the safety and tolerability, pharmacokinetics (PK), immunogenicity (IG) and pharmacodynamics (PD) of DCY636.
- Trial name
- NCT07604324
- Testing
- DCY636
- Where
- Japan
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Brugada Syndrome
UZ Brussel HRMC Registry of Brugada Syndrome
The monocentric UZB registry for Brugada registry is intended to collect all data on patients affected by Brugada syndrome at UZ Brussel hospital (UZB).
- Trial name
- HRMCBrS (NCT05283759)
- Testing
- No intervention because it is a patient registry
- Where
- Belgium
- Volunteers
- 2,000 people
- Sponsor
- Universitair Ziekenhuis Brussel
Vitiligo Vulgaris, Psoriasis
PALLAS Laser for Skin Diseases
The primary objective of this study is to get clinical experiences with the PALLAS laser in the treatment of skin conditions that respond well to UV light (vitiligo, psoriasis, alopecia areata, atopic dermatitis).
- Conditions
- Vitiligo Vulgaris, Psoriasis, Alopecia Areata, Atopic Dermatitis
- Trial name
- PALLAS-UV (NCT07250997)
- Testing
- UVB laser treatment
- Where
- Hungary
- Volunteers
- 50 people
- Sponsor
- Szeged University
no Obvious Abnormalities, Diabetic Retinopathy (DR)
Retinal Clinical Assessment With AI-derived Quantitative Information
This randomized controlled trial evaluates whether providing clinicians with AI-derived quantitative retinal information improves the quality and efficiency of retinal clinical assessment.
- Conditions
- no Obvious Abnormalities, Diabetic Retinopathy (DR), AMD, Cup-to-disc Ratio Bigger Than 0.5
- Trial name
- NCT07291960
- Testing
- AI-derived retinal quantitative information-assisted reporting
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- Beijing Tongren Hospital
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
Natural History Study of Patients With Excess Androgen
This study will evaluate and gather information in patients with genetic causes of too much androgen (male-like hormone) in order to better understand the effects of too much androgen and describe problems associated with it.
- Trial name
- NCT00250159
- Where
- 2 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hemophilia A
Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients
Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug.
- Trial name
- AFEEL (NCT06938659)
- Testing
- Emicizumab Injection, Factor VIII (FVIII)
- Where
- Bangladesh
- Volunteers
- 20 people
- Sponsor
- Dhaka Medical College
Congenital Heart Disease
A Study of Hemodynamic Cardiopulmonary Exercise for CHD
The purpose of this study is to compare the difference in the ability to detect hemodynamic abnormalities between invasive hemodynamic assessments performed at rest versus exercise, to assess the correlation between invasive and…
- Trial name
- NCT06566196
- Testing
- Cardiac Cath
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Mayo Clinic
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