Clinical trials: Multiple Anomalies
Type 1 Diabetes, Diabetes Insipidus
Tracking Neurodegeneration in Early Wolfram Syndrome
The goal of this study is to determine the pattern of early neurodegenerative changes in WFS (Wolfram Syndrome). The investigator will perform cross-sectional and longitudinal assessments of youth with WFS, targeting sensitive…
- Conditions
- Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
- Trial name
- TRACK (NCT02455414)
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Quality of Life
Impact of Different Carbohydrate Feedings on a Hydrogen Breath Curve and Self-reported Gastrointestinal Complaints
GI distress is widely prevalent among athletes, ranging from 30 to 90% in recreationally and professionally active athletes. As the recipient of nutrients, the gastrointestinal (GI) tract plays an important role in athleticism.
- Trial name
- NCT06648967
- Testing
- Supplementation with a multi-ingredient whey supplement
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Arizona State University
Infant, Newborn, Infant, Low Birth Weight
The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Very Low Birth Weight Infants
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would…
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature
- Trial name
- EPO (NCT01203514)
- Testing
- Erythropoietin
- Where
- 9 sites in United States
- Volunteers
- 318 people, healthy volunteers welcome
- Sponsor
- NICHD Neonatal Research Network
Down Syndrome (Trisomy 21)
Vascular Function in Adults With Down Syndrome
Adults with Down syndrome (Ds) are often thought to have a lower risk of heart and blood vessel disease because they tend to have lower blood pressure (BP) and fewer heart attacks than people without Ds.
- Trial name
- VDS (NCT07630207)
- Where
- United States
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- University of Nevada, Las Vegas
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Calorie Deficiency, Fasting
Effects of Calorie Restriction and Cold Stimuli on Health-related Indicators, Cognitive and Motor Functions
The goal of this clinical trial was to identify if duration of acute calorie restriction (CR)/fasting or combination CR with cold stimuli have any effects on mental and physical health-related markers, and to clarify if different…
- Conditions
- Calorie Deficiency, Fasting, Cold Exposure
- Trial name
- NCT05545943
- Testing
- 2-days of zero calorie restriction and whole-body cold-water immersion, 6-days of calorie restriction, 2-days of zero calorie restriction without whole-body cold-water immersion
- Where
- Lithuania
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Lithuanian Sports University
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
Photoaging, Wrinkle
Evaluating Procedure Pairing of a Post-Procedure Cream Versus a Comparator in Patients Treated With a Hybrid-Fractional Laser for Facial Rejuvenation
This randomized, multi-center, double-blinded, split-face, controlled clinical trial was conducted to investigate the tolerability, safety, and efficacy of a post-procedure cream when used immediately after hybrid-fractional…
- Conditions
- Photoaging, Wrinkle, Skin Laxity
- Trial name
- NCT06436261
- Testing
- Hybrid Fractional Laser, Facial Cleanser, Facial Moisturizer
- Where
- 2 sites in United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Revision Skincare
Obesity, Gender
Work-Related Effects of Heat, Activity, and Fat in Middle Aged Men
Obesity is associated with many undesirable health effects and disease, and middle age is associated with increased risk for disease.
- Conditions
- Obesity, Gender, Problem of Aging
- Trial name
- NCT02532725
- Testing
- Cardiopulmonary Exercise Stress Test, Climatic-Controlled Cardiopulmonary Exercise Stress Test, Body Composition Evaluation
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Freeman-Sheldon Research Group, Inc.
Down Syndrome, Occupational Therapy
Occupational Therapy Home Programs in Children With Down Syndrome
Children with Down Syndrome frequently encounter difficulties with sensory functions and feeding, such as oral sensory processing issues and developmental disorders.
- Conditions
- Down Syndrome, Occupational Therapy, Feeding Problem
- Trial name
- NCT06244108
- Testing
- Occupational therapy home program
- Where
- Turkey (Türkiye)
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Bezmialem Vakif University
Healthy Participants, Atopic Dermatitis
FIH Study to Evaluate the Tolerability of PF-07832837 in Healthy Adults and Patients
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetics of escalating single and repeat doses of PF-07832837 in healthy participants and in participants with moderate to severe atopic dermatitis.
- Trial name
- NCT06564389
- Testing
- PF-07832837
- Where
- 8 sites in United States, Poland
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- Pfizer
Primary Ciliary Dyskinesia
Screening for Primary Ciliary Dyskinesia Using Nasal Nitric Oxide
Background: Primary ciliary dyskinesia (PCD) is a rare genetic disease characterised by recurrent respiratory infections and subfertility due to dysfunction of cilia (brushes) of the lining cells.
- Trial name
- NCT00739817
- Where
- Israel
- Volunteers
- 6,000 people, healthy volunteers welcome
- Sponsor
- Ziv Hospital
Cystic Fibrosis (CF)
Safety, Tolerability and Pharmacokinetics Study of AIR DNAse Administered by Inhalation to Healthy Adult Volunteers
This study will be performed to assess the safety, tolerability and PK of escalating inhaled AIR-DNase doses administered as a single dose followed by multiple doses for 5 consecutive days in healthy subjects.
- Trial name
- NCT02605590
- Testing
- AIR DNase
- Where
- Israel
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Protalix
Primary Ciliary Dyskinesia
Muscle Function, Exercise Capacity and Physical Activity Level in Primary Ciliary Dyskinesia and Kartagener Syndrome
Studies evaluating respiratory and peripheral muscle functions in PCD patients and comparing them with healthy children are limited in the literature.
- Trial name
- NCT05816876
- Where
- Turkey (Türkiye)
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Gazi University
Angelman Syndrome, Prader-Willi Syndrome
Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome
The overall purpose of this project is to establish the capability of screening for Angelman syndrome (AS) and Prader-Willi syndrome (PWS) in public health newborn screening (NBS) programs, with an aim of developing and…
- Trial name
- NCT05783791
- Testing
- Newborn Screening Assay
- Where
- United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
Atopic Dermatitis
Safety and Tolerability of LEO 138559 in Healthy Subjects and Subjects With Atopic Dermatitis
The primary objective of this trial is to investigate the safety and tolerability of LEO 138559 in healthy subjects and subjects with moderate to severe atopic dermatitis.
- Trial name
- NCT03514511
- Testing
- LEO 138559
- Where
- 3 sites in United Kingdom
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- LEO Pharma
Healthy
Study of Glucagon, Ghrelin and Growth Hormone as Counterregulatory Hormones
Glucagon has been used for decades as a test of growth hormone (GH) reserve. The pathway by which GH is stimulated by glucagon is not established.
- Trial name
- NCT01795235
- Testing
- Saline, Glucagon, Atenolol
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University of Virginia
Down Syndrome
Effects of Karate in Adolescents With Down Syndrome
This randomized control study will investigate the health outcomes of adolescents with Down Syndrome who participate in a karate class as measured by ALPHA fitness testing and various PROMIS parent proxy questionnaires to assess…
- Trial name
- NCT03058640
- Testing
- PKSA karate class
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants
The purpose of the study is to evaluate the effect of VX-407 on the pharmacokinetics of levonorgestrel (LNG) and ethinyl estradiol (EE), norgestimate (NGM) and EE, norethindrone (NET) and EE and drospirenone (DRSP) and EE.
- Trial name
- NCT07074327
- Testing
- VX-407, LNG/EE, NGM/EE
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated