Clinical trials: Congenital Diseases
Hereditary Hemorrhagic Telangiectasia (HHT)
Phase 1b Study of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT).
- Trial name
- NCT07863609
- Testing
- TX002100
- Volunteers
- 20 people
- Sponsor
- Tectonic Operating Company, Inc.
Hyper Bilirubinemia, Phototherapy
The Effect of Abdominal Massage on Jaundice and Defecation in Newborn Babies
Hyperbilirubinemia is a common condition in term newborns and is often treated with phototherapy. It is thought that abdominal massage may increase bowel movements, thereby increasing the frequency of defecation and supporting…
- Conditions
- Hyper Bilirubinemia, Phototherapy, Jaundice, Neonatal
- Trial name
- NCT07388732
- Testing
- Massage once a day for 3 days., Massage twice a day for 3 days.
- Where
- Turkey (Türkiye)
- Volunteers
- 35 people
- Sponsor
- Kutahya Health Sciences University
Rett Syndrome, Family Caregivers
Adapting a Psychosocial Support Intervention for the Rett Syndrome Community
This study seeks to adapt Caregiver Speaks, a psychosocial supportive intervention delivered through Facebook, for parent caregivers of children living with Rett Syndrome.
- Conditions
- Rett Syndrome, Family Caregivers, Social Support, Psychological Support
- Trial name
- NCT07730112
- Testing
- Those who have patients prescribed Daybue, Caregiver Speaks + Daybue
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Colorado State University
Lynch Syndrome
The Yield of Artificial Intelligence (GI Genius) in Lynch Syndrome - A Randomized Tandem-colonoscopy Trial
We aim to investigate the incremental detection rate of polyps using AI- assisted colonoscopy compared with high-definition white-light endoscopy (HD-WLE).
- Trial name
- NCT06654128
- Testing
- artificial intelligence
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Sheba Medical Center
Polycystic Liver Disease
A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD…
- Trial name
- POSITANO (NCT05281328)
- Testing
- CAM2029
- Where
- 11 sites in United States, Belgium and 2 more
- Volunteers
- 71 people
- Sponsor
- Camurus AB
Bicuspid Aortic Valve Disease
TAVR vs SAVR in Severe Bicuspid Aortic Stenosis
The study is a multicenter, randomized superiority trial of standard of care therapies for severe aortic stenosis (AS) in patients with a bicuspid aortic valve (BAV).
- Trial name
- BELIEVERS (NCT07413965)
- Testing
- Transcatheter Aortic Valve Replacement, the open chest surgery to replace the aortic valve
- Where
- United States
- Volunteers
- 1,200 people
- Sponsor
- Cedars-Sinai Medical Center
MPS IV A, Mucopolysaccharidosis IVA
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA).
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Trial name
- NCT01242111
- Testing
- BMN 110
- Where
- 4 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Developmental Dysplasia of the Hip, Hip Dysplasia
Sequelae of Developmental Dysplasia of the Hip
The aim of this study is to assess long term effects of developmental dysplasia of the hip (DDH) to overall health and quality of life later in life.
- Trial name
- NCT02431871
- Where
- Finland
- Volunteers
- 340 people
- Sponsor
- University of Oulu
Hereditary Cancer Syndrome
Feasibility Trial of Combination of Obstetrical Carrier Screening and Hereditary Cancer Screening
The investigators hypothesize that preconception and pregnancy may be a feasible and effective time to offer inherited cancer risk screening.
- Trial name
- FOCUS (NCT07195071)
- Testing
- Natera empower comprehensive hereditary cancer panel, Obstetrical carrier screening
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Periodontal Diseases, Gingivitis
Asprosin, Adropin, Irisin Levels in Periodontitis
The recently discovered association of the hormones 'asprosin, adropin and irisin' with obesity and metabolic status, as well as the fact that periodontal diseases are affected by obesity and nutrition bilaterally, have led us to…
- Conditions
- Periodontal Diseases, Gingivitis, Obesity
- Trial name
- NCT06250764
- Testing
- Clinical periodontal measuremenets, ELISA test from gingival crevicular fluid (GCF)
- Where
- Turkey (Türkiye)
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- AYSAN LEKTEMUR ALPAN
Infant, Newborn, Diseases, Intubation Complication
Noninvasive Positive Pressure Ventilation During Neonatal Nasal Intubation
The goal of this study is to perform a quality control assessment of our local intubation protocol in any newborn infant requiring endotracheal intubation in the delivery room or neonatal intensive care unit at the Department of…
- Trial name
- IntubAID (NCT06139809)
- Testing
- Intubation
- Where
- Switzerland
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Facioscapulohumeral Muscular Dystrophy
Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy.
- Trial name
- ReSolve (NCT03458832)
- Testing
- FSHD-specific functional rating scale, Electrical Impedance Myography
- Where
- 14 sites in United States, France and 4 more
- Volunteers
- 324 people
- Sponsor
- University of Kansas Medical Center
Kidney Transplant; Complications, Polycystic Kidney Diseases
Evaluation of Iliac and Renal Artery for Mechanism of Intracranial Aneurysm in ADPKD
ADPKD is the most common form of hereditary kidney disease and is known to occur in 1 of 400 to 1000 population in the U.S. ADPKD consists of 2.8% of patients receiving kidney transplantation in our center.
- Conditions
- Kidney Transplant; Complications, Polycystic Kidney Diseases, Aneurysm, Brain
- Trial name
- NCT03726463
- Testing
- Kidney transplantation
- Where
- South Korea
- Volunteers
- 100 people
- Sponsor
- Asan Medical Center
Urethral Obstruction
Standardized Prenatal Clinical Care for LUTO
The investigators propose a standardized prenatal management, based on the scientific evidence published in the literature, to manage prenatally fetuses with lower urinary tract obstruction (LUTO).
- Trial name
- NCT02315521
- Testing
- Fetal Intervention for LUTO
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Baylor College of Medicine
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn How Different Levels of Decreased Liver Function Influence Blood Levels of Elinzanetant Compared to Normal Liver Function in Male and Female Participants
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, like those that happen in women during menopause. These symptoms can include vasomotor symptoms.
- Trial name
- NCT04903821
- Testing
- BAY3427080
- Where
- 2 sites in Germany
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Bayer
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Acute Myeloid Leukemia, Down Syndrome
Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome
This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome.
- Conditions
- Acute Myeloid Leukemia, Down Syndrome, Myelodysplastic Syndrome, Myeloid Leukemia Associated With Down Syndrome
- Trial name
- NCT02521493
- Testing
- Asparaginase, Asparaginase Erwinia chrysanthemi, Cytarabine
- Where
- 191 sites in United States, Australia and 4 more
- Volunteers
- 280 people
- Sponsor
- Children's Oncology Group
Neonatal Sepsis
Effect of Recombinant Human Granulocyte Colony- Stimulating Factor on CD11b in Septic Neonates
The investigators aimed to evaluate the effect of G-CSF administration in septic neonates on neutrophil production and CD11b expression.
- Trial name
- NCT01479114
- Testing
- rh-GCSF
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Ain Shams University
Aortic Coarctation
Comparison Between Serratus Anterior Plane Block and Erector Spinae Plane Block in Coarctectomy
Pain is considered to be subjective, however, in children, it is believed to be felt rather than expressed because they often depend on the caregiver for their safety and well-being.
- Trial name
- NCT06567275
- Testing
- Erector Spinae Plane Block, Serratus Anterior Plane Block
- Where
- Egypt
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Cairo University
Atopic Dermatitis
LEO 29102 Cream in the Treatment of Atopic Dermatitis
This is a proof of concept and dose finding Phase II trial comparing 5 dose strengths with vehicle and an active comparator (Elidel cream 10 mg/g) in a 4 week, twice daily treatment regimen in mild to moderate atopic dermatitis…
- Trial name
- NCT01037881
- Testing
- LEO 29102, Elidel®
- Where
- 3 sites in Canada, Finland and 1 more
- Volunteers
- 183 people
- Sponsor
- LEO Pharma
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