Clinical trials: Multiple Anomalies
Atopic Dermatitis, Hidradenitis Suppurativa (HS)
A Phase I Study of QLS12010 Capsules: Safety, Tolerability, PK, PD, and Food Effects in Healthy Adults and Moderate to Severe Atopic Dermatitis Patients
The goal of this clinical trial is to evaluate the safety, tolerability, PK, and PD of single and multiple ascending doses of QLS12010 Capsules and the effect of food on their PK profiles in healthy adult participants and…
- Conditions
- Atopic Dermatitis, Hidradenitis Suppurativa (HS), Rheumatoid Arthritis (RA)
- Trial name
- NCT06946641
- Testing
- QLS12010
- Where
- China
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Shanghai Qilu Pharmaceutical Research and Development Center LTD
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Lymphangioleiomyomatosis, a Study on Cathepsin K
This is a physiopathological case-control, non-interventional, monocentric study of adult patients with lymphangioleiomyomatosis.
- Trial name
- LAM-CAK (NCT05323370)
- Testing
- 24h urine, urine sample
- Where
- France
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- University Hospital, Tours
Parkinson Disease, Parkinson's Disease and Parkinsonism
Complex Eye Movements in Parkinson's Disease and Related Movement Disorders
Diagnosing Parkinson's disease (PD) depends on the clinical history of the patient and the patient's response to specific treatments such as levodopa.
- Conditions
- Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, Corticobasal Degeneration
- Trial name
- NCT04925622
- Testing
- Complex eye movement exam
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Saccadous, Inc.
Atopic Dermatitis
A Phase 1b Study Assessing the PK, PD, Safety & Tolerability of SB414 in Atopic Dermatitis
This is a Phase 1b, 2-week study assessing the pharmacokinetics, pharmacodynamics, safety and tolerability of SB414 in subjects with mild to moderate AD.
- Trial name
- NCT03431610
- Testing
- SB414 2%, SB414 6%, Vehicle
- Where
- 9 sites in United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Novan, Inc.
Healthy Volunteers, Atopic Diathesis and Atopic Dermatitis
Single Dose and Multiple Dose Study to Assess Safety and Tolerability of LOU064
This is a 6-part first-in-human study in up to approximately 184 participants. Parts 1 to 5 is in health volunteers and part 6 is in subjects with atopic dermatitis.
- Trial name
- NCT03918980
- Testing
- LOU064
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Pregnancy
Evaluation of a Decision Assisting Tool for Prenatal Testing
This is a randomized study of an interactive, computerized descision-assisting tool for prenatal testing decision making (PT Tool) versus standard educational booklets distributed to all pregnant women by the State of California…
- Trial name
- NCT00686062
- Testing
- Control - computerized age-appropriate prenatal testing educational brochure, PT Tool - interactive, computerized, prenatal testing decision tool
- Where
- United States
- Volunteers
- 494 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Down Syndrome, ADHD
Evaluating Treatment of ADHD in Children With Down Syndrome
Children with Down syndrome (DS) have a 3-5 time greater prevalence of Attention Deficit Hyperactivity Disorder (ADHD) than typically developing (TD) children.
- Trial name
- TEAM-DS (NCT04219280)
- Testing
- Quillivant XR
- Where
- 4 sites in United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Healthy, Men
Study of Daily Application of Nestorone® (NES) and Testosterone (T) Combination Gel for Male Contraception
The long term objective is to develop a gel to be used as a male contraceptive.
- Conditions
- Healthy, Men, Male Contraception
- Trial name
- NCT03452111
- Testing
- Nestorone + Testosterone Combination Gel
- Where
- 17 sites in United States, Chile and 5 more
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Premier Research
Down Syndrome
Blood Flow and Blood Pressure Investigation in Down Syndrome
Down syndrome (DS) is a chromosomal condition that occurs in approximately 1 in 800 births worldwide, and causes impairments in physical function, including a reduced work capacity (as measured by VO2peak or aerobic capacity).
- Trial name
- FBI21 (NCT05528549)
- Testing
- Exercise
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- University of Illinois Chicago
Down Syndrome, Amyloid Beta-protein
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
To determine the sensitivity of the visual assessment of BAY94-9172 PET images in detecting cerebral β-amyloid in individuals with Down Syndrome (DS) and specificity in individuals without DS.
- Trial name
- NCT00928304
- Testing
- Florbetaben (BAY94-9172)
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Lantheus Biosciences Ltd.
Healthy Adult Subjects, Sickle Cell Disease
Safety, Tolerability and Pharmacokinetics of FTX-6058
This is a study to evaluate the safety, tolerability and pharmacokinetics of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease (SCD).
- Trial name
- NCT04586985
- Testing
- FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Fulcrum Therapeutics
Retinopathy of Prematurity, ROP
Validation of i-ROP DL to Detect More Than Mild ROP
The purpose of the pivotal reader study is to assess the readers' accuracy in diagnosing plus disease versus no plus or pre-plus disease with or without the aid of the i-ROP DL.
- Trial name
- NCT06109285
- Testing
- iROP DL
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Siloam Vision
Cystic Fibrosis, Bronchiectasis
Dose Escalation Study of ALX-009 in Healthy Men and Cystic Fibrosis (CF) and Non-CF Bronchiectasis Patients
This is a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and pharmacokinetics of a single ascending doses (SAD) and multiple ascending doses (MAD) of Hypothiocyanite (OSCN-)…
- Trial name
- NCT02598999
- Testing
- ALX-009, OSCN-, bLF
- Where
- France
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Alaxia SAS
Down Syndrome (Trisomy 21), Edward's Syndrome (Trisomy 18)
Non-Invasive Determination of Fetal Chromosome Abnormalities
The overall significance of this study is to develop a laboratory developed test (LDT) to use a new marker in the maternal blood to better identify pregnancies that have a child with a chromosome abnormality such as Down syndrome…
- Conditions
- Down Syndrome (Trisomy 21), Edward's Syndrome (Trisomy 18), Patau Syndrome (Trisomy 13), Klinefelter Syndrome (47, XXY)
- Trial name
- NCT00891852
- Where
- 7 sites in United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Lenetix Medical Screening Laboratory
Cystic Fibrosis
A Study to Investigate the Safety and Tolerability of Single and Multiple Ascending Doses of KIT2014 in Healthy Subjects
This is the first-in-human study with KIT2014 designed to provide safety, tolerability and pharmacokinetic data in healthy volunteers.
- Trial name
- NCT06659757
- Testing
- KIT2014
- Where
- Australia
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Kither Biotech Srl
Down Syndrome, Balance
The Effects of Kinesiotaping on Balance in Children With Down Syndrome.
This study aimed to explore the effects of Kinesio tape applied to plantar soles on balance in children with Down Syndrome (DS). Two groups including children with DS and a group with their typically developing peers evaluated.
- Trial name
- NCT04156607
- Testing
- Kinesio taping
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Gazi University
Healthy Subjects, Cystic Fibrosis
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
This Phase 1/2a study is a double-blinded (subject and Investigator), randomized, placebo-controlled, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple…
- Trial name
- NCT03647228
- Testing
- IONIS-ENaCRx
- Where
- 8 sites in Germany, United Kingdom
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
22q11 Deletion Syndrome, DiGeorge Syndrome
SNP-based Microdeletion and Aneuploidy RegisTry (SMART)
This multi-center prospective observational study is designed to track birth outcomes and perinatal correlates to the Panorama prenatal screening test in the general population among ten thousand women who present clinically and…
- Conditions
- 22q11 Deletion Syndrome, DiGeorge Syndrome, Trisomy 21, Trisomy 18
- Trial name
- SMART (NCT02381457)
- Where
- 21 sites in United States, Australia and 4 more
- Volunteers
- 20,960 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Sickle Cell Disease
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
- Trial name
- NCT05036512
- Testing
- GBT021601
- Where
- 4 sites in United States, Australia
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- Pfizer
Sleep Disorder
Characterizing Sleep Disorders in Children and Adults With Tuberous Sclerosis Complex (TSC)
The proposed research project is aimed at further characterization of sleep problems and evaluation of their impact in children and adults with TSC, excluding epilepsy as contributing factor.
- Trial name
- NCT03326765
- Testing
- Questionnaires, electroencephalogram (EEG), polysomnography
- Where
- Belgium
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Universitair Ziekenhuis Brussel