Clinical trials: Congenital Malformation
Chronic Granulomatous Disease
Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease
This study will investigate the safety and effectiveness of a new stem cell transplant procedure for treating chronic granulomatous disease (CGD) in patients with active infection.
- Trial name
- NCT00006417
- Testing
- Stem cell transplantation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Atopic Dermatitis, Healthy Participants
A Study of BYN-001 in Healthy Adults and in Adults With Moderate to Severe Atopic Dermatitis
This first-in-human study evaluates BYN-001, a humanized IgG1 monoclonal antibody targeting interleukin-25 (IL-25), a cytokine implicated in atopic dermatitis.
- Trial name
- BYN-001-101 (NCT07746817)
- Testing
- BYN-001
- Where
- Australia
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Bionyra Pharma
Congenital Disorders
Outcomes of Children With Congenital Single Ventricle Heart
Congenital heart disease affects 1 in 100 newborn babies each year and more than 2,000,000 Americans have a congenital heart defect.
- Trial name
- NCT00266968
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Children's Healthcare of Atlanta
Sickle Cell Disease, Pain Management
Hypnosis to Manage Pain and Symptoms in Patients With Sickle Cell Disease
This study will examine whether hypnosis can reduce the frequency and intensity of pain in patients with sickle cell disease. Patients 18 years of age and older with sickle cell disease and a history of pain associated with their…
- Trial name
- NCT00393250
- Testing
- Hypnosis, Control
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Down Syndrome
Drumming Lessons' Influence on Children With Down Syndrome
The goal of this clinical trial is to learn if drumming lessons can increase self-control in children with Down syndrome. The main question it aims to answer is whether 2 months of drumming lessons can improve the behavioral…
- Trial name
- NCT07156318
- Testing
- 2 months of drumming lessons given by a professional drummer with extensive experience working with children with Down syndrome, Lessons after measurement
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt University
Phenylketonurias
Impact of Fruit and Vegetables in PKU
In the UK, some fruits and vegetables are permitted without measurement in the diets of people with phenylketonuria (PKU). It is proven that fruits and vegetables containing phenylalanine (Phe) up to 75mg/100g (e.g.
- Trial name
- NCT05249218
- Testing
- Phase B - Extra 1 Phe exchange from fruits/vegetables containing Phe from 76-100mg/100g, Phase C - Extra 2 Phe exchanges from fruits/vegetables containing Phe from 76-100mg/100g, Phase D - Extra 1 Phe exchange from milk or yoghurt
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- Alex Pinto
Smith-Magenis Syndrome (SMS)
Natural History Study of Smith-Magenis Syndrome
This study will examine how a rare disease called Smith-Magenis syndrome (SMS) affects people and how they change over time. SMS is caused by a small chromosome 17p11.2 deletion (missing piece).
- Trial name
- NCT00013559
- Where
- United States
- Volunteers
- 593 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Chronic Kidney Disease, Pain, Procedural
Comparing Mirror and Music for Dialysis Needle Pain
Inserting needles into the vascular access (fistula) is a major source of pain and anxiety for many hemodialysis patients. This study compared two simple, non-drug techniques to reduce this pain: listening to music and looking…
- Conditions
- Chronic Kidney Disease, Pain, Procedural, Arteriovenous Fistula
- Trial name
- MIRROR-MUSIC (NCT07320859)
- Testing
- Mirror Therapy (Visual Illusion Therapy), Music Therapy
- Where
- Turkey (Türkiye)
- Volunteers
- 75 people
- Sponsor
- Izmir Kavram Vocational School
Recurrent Childhood Kidney Neoplasm, Stage I Kidney Wilms Tumor
Gene Analysis in Studying Susceptibility to Wilms Tumor
This clinical trial studies gene analysis in studying susceptibility to Wilms tumor. Finding genetic markers for Wilms tumor may help identify patients who are at risk of relapse.
- Conditions
- Recurrent Childhood Kidney Neoplasm, Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor
- Trial name
- NCT01808079
- Testing
- Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Oncology Group
Friedreich Ataxia, Cardiomyopathy, Secondary
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy.
- Trial name
- NCT05445323
- Testing
- Low dose LX2006, Mid Dose LX2006, High Dose LX2006
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Lexeo Therapeutics
Cognitive Impairment, Hereditary Spastic Paraplegia
Studying Cognition in SPG4
Comparing the cognitive levels of patients with SPG4 mutations to healthy controls.
- Trial name
- NCT03104088
- Testing
- CANTAB, MOCA
- Where
- Germany
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen
Atopic Dermatitis (AD)
A Study Evaluating BFB759 in Moderate to Severe Atopic Dermatitis
This is a double-blind, placebo-controlled study where subjects are participating for approximately 36 to 40 weeks. The study compares how well BFB759 works and how safe it is compared with a placebo.
- Trial name
- COMPASS 2-AD (NCT07105488)
- Testing
- BFB759
- Where
- 88 sites in United States, Bulgaria and 5 more
- Volunteers
- 225 people
- Sponsor
- Bluefin Biomedicine, Inc.
Cystic Fibrosis
Intestine-lung Axis of Cystic Fibrosis Patients Treated With the Combination Elexacaftor/Tezacaftor/Ivacaftor
Cystic fibrosis is a systemic disease, which affects in particular the respiratory and digestive systems of patients, sites of chronic inflammation.
- Trial name
- KAF-BIOTA (NCT05937815)
- Testing
- Sample collection
- Where
- 15 sites in France
- Volunteers
- 253 people
- Sponsor
- University Hospital, Bordeaux
Cryopyrin-Associated Periodic Syndromes
Kineret CAPS Post Authorisation Study
A non-interventional, post authorization safety study to evaluate the safety of Kineret in the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) in routine clinical care with regard to serious infections, malignancies…
- Trial name
- NCT02326376
- Testing
- anakinra (Kineret)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Swedish Orphan Biovitrum
Atopic Dermatitis (AD)
A Study of NOR-101 in Healthy Adults and Adults With Moderate-to-Severe Atopic Dermatitis
This Phase 1 study will assess NOR-101, an investigational study drug, in two parts. Part 1 is a randomized, blinded study comparing single and multiple doses of NOR-101 with placebo in healthy adults.
- Trial name
- POLARIS-1 (NCT07904689)
- Testing
- NOR-101
- Where
- Australia
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- North Immunology Inc.
Constipation Chronic Idiopathic, Cystic Fibrosis
Maralixibat in Patients With Cystic Fibrosis and Constipation
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions.
- Trial name
- NCT06413368
- Testing
- Maralixibat 9.5 MG/ML [Livmarli]
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
Cystic Fibrosis
Human MesenchymAl Stem Cells Infusion in Patients With Cystic Fibrosis
A Safety Run-In will be followed by a Double-Blinded Randomized Phase. All subjects shall meet the inclusion/exclusion criteria, and will be evaluated prior to the scheduled infusion to establish baseline.
- Trial name
- HAPI (NCT03058068)
- Testing
- Allo-hMSCs
- Sponsor
- Joshua M Hare
Atopic Dermatitis
Study to Assess the Safety and Efficacy of Lebrikizumab (LY3650150) in Adolescent Participants With Moderate-to-Severe Atopic Dermatitis
This is an open-label, single arm study of 52 weeks duration. The study will assess the safety and efficacy of lebrikizumab in adolescent participants (≥12 to <18 years weighing ≥40 kilograms) with moderate-to-severe atopic…
- Trial name
- ADore (NCT04250350)
- Testing
- Lebrikizumab
- Where
- 70 sites in United States, Australia and 2 more
- Volunteers
- 206 people
- Sponsor
- Eli Lilly and Company
Genu Varum, Genu Valgum
Retrospective Evaluation of Guided Growth Plate System Plus's Safety and Clinical Performance for the Treatment of Lower Limbs Deformities.
Orthofix is conducting this retrospective Post-market clinical follow up (PMCF) study to assess the safety and clinical performance of the Guided Growth Plate System Plus (GGPSP) device, which is a new version of the Guided…
- Conditions
- Genu Varum, Genu Valgum, Joint Diseases, Knee Deformity
- Trial name
- NCT05241691
- Testing
- Guided Growth Plate System Plus
- Where
- Italy
- Volunteers
- 69 people
- Sponsor
- Orthofix s.r.l.
Cystic Fibrosis, Pediatrics
A Prospective, Multicenter, Collaborative Study to Determine the Prevalence of Nontuberculous Mycobacteria (NTM) in Pediatric Patients With Cystic Fibrosis in Florida
To determine the prevalence of nontuberculous mycobacterial infection (NTM) in pediatric patients with cystic fibrosis in the State fo Florida.
- Trial name
- NCT02198079
- Testing
- Sputum
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- University of Miami
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