Clinical trials: Genetic Disorder
- Condition Genetic Disorder
- Funder type Industry (pharma, biotech, devices)
- Accepts healthy volunteers yes
Growth Hormone Deficiency
Safety Study of Syntropin (Human Growth Hormone) for the Treatment of Growth Hormone Deficiency
The purpose of this study is to evaluate the pharmacokinetics of Syntropin (a human growth hormone) and to determine the serum concentration of IGF-1 after Syntropin injection.
- Trial name
- NCT00489294
- Testing
- Syntropin
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Phage Pharmaceuticals, Inc.
Atopic Dermatitis, Atopic Dermatitis Eczema
Bioavailability and Tolerability of Fexofenadine Hydrochloride Topical Lotion 1%
The investigational lotion is envisaged as an short- and long term "Ease \& Prevent" monotherapy for adults and children with mild to moderate eczema.
- Conditions
- Atopic Dermatitis, Atopic Dermatitis Eczema, Atopic Rash, Rash
- Trial name
- ATOFEX-1 (NCT05461456)
- Testing
- Fexofenadine Hydrochloride Topical Lotion 1% (OP2101)
- Where
- Bangladesh
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- OrchestratePharma GmbH
Asthma, COPD
An Evaluation of 9MW1911 Injection in Healthy Subjects
This study is a randomized, double-blind, dose-escalating phase I clinical study to evaluate the safety, tolerability and pharmacokinetics of 9MW1911 injection in healthy subjects.
- Conditions
- Asthma, COPD, Atopic Dermatitis
- Trial name
- NCT05803902
- Testing
- Experimental drug 9MW1911
- Where
- China
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Dermatitis, Atopic
Phase IIa Study of WBI-1001 Cream for Atopic Dermatitis
WBI-1001 is a synthetic,new, non-steroid, small molecule being developed as a candidate drug for the topical, cream treatment of inflammatory skin diseases.
- Trial name
- WBI-1001-201 (NCT00837551)
- Testing
- WBI-1001
- Where
- Canada
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Welichem Biotech Inc.
Hereditary Tyrosinemia, Type I
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product 1) and Nitisinone 10 mg Tablets 'Baked' for 6 months @ 40°C/75% RH (Test Product 2) are bioequivalent to the reference product Orfadin 10 mg…
- Trial name
- NCT02750345
- Testing
- Nitisinone, Nitisinone Baked Tablet, Orfadin
- Where
- South Africa
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Liver Diseases, Primary Biliary Cirrhosis
Repeatability and Reproducibility of Multiparametric MRI
This study aims to prospectively assess the repeatability and reproducibility of iron-corrected T1 (cT1), T2*, and hepatic proton density fat fraction (PDFF) quantification with multiparametric MRI using the LiverMultiScan™ (LMS…
- Conditions
- Liver Diseases, Primary Biliary Cirrhosis, Non-Alcoholic Fatty Liver Disease, Non-Alcoholic Steatohepatitis
- Trial name
- NCT03743272
- Testing
- LiverMultiScan
- Where
- 4 sites in Netherlands, United Kingdom
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Perspectum
Healthy Volunteers
A Study to Determine the Relative Bioavailability of Capsule and Tablet Formulations of GDC-0941 and the Effect of Food and Proton Pump Inhibition on GDC-0941 Tablet Pharmacokinetics in Healthy Volunteers
This is a Phase I, randomized, two-part, single-center pharmacokinetics (PK) study to assess the relative bioavailability of capsule and tablet formulations of GDC-0941 in the fasted state (Part 1) and to determine the effect of…
- Trial name
- NCT00999128
- Testing
- GDC-0941, rabeprazole
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Genentech, Inc.
Familial Amyloid Polyneuropathy (FAP)
Study of SOM0226 in Familial Amyloid Polyneuropathy
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
- Trial name
- NCT02191826
- Testing
- SOM0226
- Where
- Spain
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- SOM Innovation Biotech SA
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn What Happens to Elinzanetant in the Body When Taken With or Without Carbamazepine, and How Safe it is in Healthy Men and Women Aged 18 to 60 Years.
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, such as hot flashes. Before a treatment can be approved for patients to take, researchers do clinical studies to better…
- Trial name
- NCT05028608
- Testing
- Elinzanetant (BAY3427080), Midazolam, Carbamazepine
- Where
- Germany
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Bayer
Gout
A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout
The purpose of this study is to: Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in…
- Trial name
- NCT05256810
- Testing
- ALN-XDH
- Where
- United Kingdom
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Gout, Hyperuricemia
Relative Bioavailability Study to Assess an LC350189 Tablet Compared to an LC350189 Capsule in Healthy Adults
This is an open-label, randomized, crossover, single dose study. Two single doses of LC350189(tablet or capsule formulations) will be administered with a washout period of at least 4-day between the doses to investigate the…
- Trial name
- NCT04886050
- Testing
- LC350189 Tablet, LC350189 Capsule
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- LG Chem
Hereditary Cancer-Predisposing Syndrome
Inherited Risk Evaluation Tool (InheRET): Identifying Patients at Increased Risk for Hereditary Disease
This study will evaluate the impact InheRET™, an online family history gathering and risk assessment reporting tool, has on facilitating National Comprehensive Cancer Network(NCCN) guideline compliant referrals for cancer genetic…
- Trial name
- NCT04510896
- Testing
- InheRET, Patient Acceptance, Physician Acceptance Survey
- Where
- 4 sites in United States
- Volunteers
- 2,232 people, healthy volunteers welcome
- Sponsor
- InheRET, Inc
Hyperuricemia, Gout
Safety, Tolerability and Pharmacokinetic/Pharmacodynamic Characteristics of LC350189
This will be a dose block-randomized, double-blind, active and placebo controlled, single and multiple dosing, dose-escalation study to evaluate tolerability, safety and pharmacokinetic / pharmacodynamic characteristics.
- Trial name
- NCT01361646
- Testing
- LC350189
- Where
- South Korea
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- LG Life Sciences
Healthy Volunteer, Muscular Dystrophies
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
This is a Phase 1 2-part, single-center, open-label study in healthy male volunteers. Part A will assess the absorption, metabolism, excretion, and pharmacokinetics of one oral dose of radiolabeled EDG-5506.
- Conditions
- Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT05730842
- Testing
- EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Atopic Dermatitis
A Phase I Study of HY209 Gel in Healthy Male Volunteers for Atopic Dermatitis
A randomized, double-blind, placebo-controlled single and multiple dosing, dose escalation phase I clinical trial to investigate the safety/tolerability and pharmacokinetics of HY209 gel after transdermal administration in…
- Trial name
- Shaperon (NCT03492398)
- Testing
- HY209
- Where
- South Korea
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Shaperon
Cystic Fibrosis
A Phase 1 Relative Bioavailability Study of a VX-581 Tablet Formulation
The purpose of this study is to evaluate the relative bioavailability of VX-581 tablet formulation compared to a suspension formulation along with its safety and tolerability.
- Trial name
- NCT07813039
- Testing
- VX-581
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Healthy, Chronic Rhinosinusitis With Nasal Polyps
Study Of PF-06817024 In Healthy Subjects, In Patients With Chronic Rhinosinusitis With Nasal Polyps And in Patients With Atopic Dermatitis
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics of PF-06817024 in healthy volunteers, in participants with chronic rhinosinusitis, with nasal polyps and in participants with…
- Conditions
- Healthy, Chronic Rhinosinusitis With Nasal Polyps, Atopic Dermatitis
- Trial name
- NCT02743871
- Testing
- PF-06817024
- Where
- 20 sites in United States
- Volunteers
- 97 people, healthy volunteers welcome
- Sponsor
- Pfizer
Retinitis Pigmentosa, Retinal Dystrophy
Observational Study to Assess the Reliability and Validity of the MLYMT and MLSDT
This is a prospective Multi-Center Observational Study to assess the reliability and validity of the Multi-Luminance Y-Mobility Test (MLYMT) and Multi-Luminance Shape Discrimination Study (MLSDT) Main Outcome Measures: (i)…
- Trial name
- NCT05820100
- Testing
- Observational
- Where
- 8 sites in United States, Puerto Rico
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Nanoscope Therapeutics Inc.
Obesity, Behavior, Health
Evaluation of Brain Activity Changes After a Behavior Change Weight Loss Intervention
The aim of this study is to compare brain changes in individuals with overweight or obesity enrolled in either the Noom Healthy Weight program or a matched control.
- Conditions
- Obesity, Behavior, Health, Weight Loss
- Trial name
- NCT04522245
- Testing
- Noom Healthy Weight; mobile behavior change program for weight loss., Matched Control
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Noom Inc.
Lactose Intolerance
Effects Comparison of A1 and A2 Milk on Gastrointestinal Physiology, Symptoms and Cognitive Behavior
This is a single-site, double-blinded, randomized, controlled, 2X2 cross-over study aiming to compare effects of milk containing only A2 type beta casein versus milk containing both A1 and A2 beta casein proteins on the…
- Trial name
- NCT02406469
- Testing
- Oral consumption milk Tetra Pak
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- a2 Milk Company Ltd.