Clinical trials: Multiple Anomalies
Osteogenesis Imperfecta
A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers
To understand if AGA2115 is safe and well tolerated in healthy adult volunteers.
- Trial name
- NCT06086613
- Testing
- AGA2115
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Angitia Incorporated Limited
Muscle Spasticity, Hypertonia, Muscle
Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals
To assess the safety and tolerability of single and multiple doses of MTR-601 in normal healthy volunteers under fed and fasted conditions. To evaluate the plasma and urine pharmacokinetics (PK) of MTR-601.
- Conditions
- Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis
- Trial name
- NCT06117020
- Testing
- MTR-601
- Where
- United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Motric Bio
Healthy Volunteer, Cystic Fibrosis
Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis
This trial will consist of two parts: Part 1 and Part 2. Part 1 will enroll adult healthy volunteers (HV) into four treatment groups.
- Trial name
- NCT03140527
- Testing
- PTI-801, PTI-808
- Where
- 38 sites in United States, Canada and 3 more
- Volunteers
- 171 people, healthy volunteers welcome
- Sponsor
- Proteostasis Therapeutics, Inc.
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Phase 1/2 Study of KRRO-121 is a randomized, placebo-controlled study in Healthy Volunteers (Phase 1 SAD and MAD) and Study Participants with Urea Cycle Disorders (Phase 2).
- Conditions
- Urea Cycle Disorders, Inborn, Urea Cycle Disorders, Ornithine Transcarbamylase (OTC) Deficiency, Arginase 1 Deficiency
- Trial name
- ANCHOR (NCT07773246)
- Testing
- KRRO-121
- Where
- Australia
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Korro Bio, Inc.
Zellweger Spectrum
Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders
The purpose of this study is to characterize the symptoms of Zellweger Spectrum Disorder (ZSD) and related peroxisome disorders, and to assess the quality of life of family caregivers (parents, stepparents, legal guardians) of…
- Trial name
- NCT03440905
- Where
- United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- University of South Florida
Autism Spectrum Disorders
Social Interactions: Ocular Explorations and Pupillometry in Autism
The primary objective of this work will first to characterize in typical childhood, visual exploratory behavior and pupillary response associated with salience of human social stimuli (faces and body movements), and then to…
- Trial name
- ISEOP (NCT01647295)
- Where
- France
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Autosomal Dominant Polycystic Kidney Disease
Assessment of Longitudinal Changes in Endothelial Function and Oxidative Stress in Normotensive Patients With ADPKD
The purpose of this study is to determine whether patients with autosomal dominant polycystic kidney disease (ADPKD) present with abnormal endothelial function, increased levels of NOX4 activity and mitochondrial abnormalities…
- Trial name
- NCT03493802
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Ichthyosis, Netherton Syndrome
Defining the Skin and Blood Biomarkers of Ichthyosis
Ichthyosis is a group of genetic skin disorders that present with dry, thickened, scaly, or flaky skin. As of today, there is no cure or treatment.
- Trial name
- NCT03417856
- Where
- 4 sites in United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Northwestern University
Gout
A Clinical Trial of HEC93077 in Chinese Healthy Subjects and HUA Patients With Single or Multiple Dosing
A phase 1 clinical trial of HEC93077,to evaluate the safety ,PK,PD and food effect in Chinese healthy subjects and HUA patients after single or multiple dosing.
- Trial name
- NCT05127772
- Testing
- HEC93077
- Where
- China
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Sunshine Lake Pharma Co., Ltd.
Atopic Dermatitis (AD), Eczema
Ph 1a/1b Single Ascending Dose and Multiple Ascending Dose Study of ARQ-234
This is a first-in-human, Phase 1, double-blind, randomized, placebo-controlled, dose-escalation study evaluating ARQ-234. The study is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD)…
- Trial name
- NCT07453602
- Testing
- ARQ-234
- Where
- 7 sites in United States
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Arcutis Biotherapeutics, Inc.
Diastolic Dysfunction, Neonatal Encephalopathy
Assessment of Diastolic Function During the Transitional Period and Infancy Using Serial Echocardiography
The goal of this single-centre longitudinal observational study is to create reference values for diastolic function parameters in neonates born at 35 weeks' gestation or above, and to assess the influence of pre-defined…
- Conditions
- Diastolic Dysfunction, Neonatal Encephalopathy, Small for Gestational Age at Delivery, Transient Tachypnea of the Newborn
- Trial name
- DiFuSE (NCT06200519)
- Where
- Ireland
- Volunteers
- 216 people, healthy volunteers welcome
- Sponsor
- University College Cork
Lipedema, Dercum's Disease
Insight Into Subcutaneous Adipose Tissue Disorders
The INSIGHT study aims to phenotype individuals with subcutaneous adipose tissue (SAT) disorders specifically lipedema and Dercum's disease with an aim to find a cure for the SAT in these disorders that is resistant to diet and…
- Conditions
- Lipedema, Dercum's Disease, Familial Multiple Lipomatosis, Madelung's Disease
- Trial name
- INSIGHT (NCT02838277)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Arizona
Language Development Disorders, Down Syndrome
Strategies for Teaching Verbs
This study is designed to evaluate whether children with Down syndrome and children with typical development exhibit different levels of accuracy demonstrating novel verbs taught under three conditions: semantic cues (perform…
- Conditions
- Language Development Disorders, Down Syndrome, Child Language
- Trial name
- NCT03441685
- Testing
- Semantic cues (perform action), syntactic cues (sentence frame), or combined semantic and syntactic cues
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Asthma, COPD
Acoustic Waveform Respiratory Evaluation
The study will evaluate the feasibility of using smartphone speakers and microphones to evaluate the caliber of the airways, detect airway obstruction, aid in airway disease diagnosis, and identify disease exacerbations.
- Conditions
- Asthma, COPD, Cystic Fibrosis, Ciliary Motility Disorders
- Trial name
- AWARE (NCT06512064)
- Testing
- AWARE
- Where
- 2 sites in United States
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- Indiana University
ADPKD (autosomal Dominant Polycystic Kidney Disease)
Study to Evaluate the Drug-drug Interaction of JMKX003142 in Healthy Subjects
To evaluate the effects of oral administration rifampicin or itraconazole capsules on the pharmacokinetics of JMKX003142 in healthy subjects.
- Trial name
- NCT06658964
- Testing
- Cohort 1: JMKX003142 and itraconazole, Cohort 2: JMKX003142 and rifampicin
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Jemincare
Cystic Fibrosis
Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People with Cystic Fibrosis
This is a 3-part, single-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy volunteers (Part A) and multiple-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy…
- Trial name
- ABCI (NCT05802264)
- Testing
- ABCI
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Cystetic Medicines, Inc.
Thalassemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers
- Trial name
- NCT03165864
- Testing
- IONIS TMPRSS6-Lrx
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Myopathy
Maintenance of Muscle Mass in Older People: the Negative Impact of Statin Therapy
A major contributor to frailty and immobility in the elderly is the age related loss of muscle mass (sarcopenia). Cardiovascular disease (CVD) is the leading cause of mortality in the elderly, with high blood cholesterol and…
- Trial name
- NCT01047163
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Autosomal Dominant Polycystic Kidney Disease, Kidney Disease
New Quantitive MRI Parameters in Assessing Kidneys of Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to establish normal Magnetic Resonance quantitative values (tissues stiffness, Apparent Diffusion Coefficient values and Blood Oxygen Level Determination values for both renal cortex and medullary…
- Trial name
- MRI Pilot (NCT02250287)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Drunk Driving, Alcohol Drinking
Design and Evaluation of an In-Vehicle Real-Time Drunk Driving Detection System
To analyze driving behavior of individuals under the influence of alcohol while driving in a real car. Based on the in-vehicle variables, the investigators aim at establishing algorithms capable of discriminating sober and drunk…
- Conditions
- Drunk Driving, Alcohol Drinking, Impaired Driving
- Trial name
- DRIVE (NCT05796609)
- Testing
- Driving under the influence of alcohol
- Where
- Switzerland
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- University of Bern