Clinical trials: Multiple Anomalies
Healthy, Sickle Cell Anemia
Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease
This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in…
- Trial name
- NCT04255875
- Testing
- PF-07209326
- Where
- 16 sites in United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Pfizer
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants
The purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of VX-407 in healthy participants.
- Trial name
- NCT07022119
- Testing
- VX-407
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Type 2 Diabetic
The Correlation Between Testosterone and Cortisol Level and Inflammatory Markers Levels in Type 2 Diabetic Men.
Many type 2 diabetic men have low serum testosterone levels. Our aim in this trial is to check if there is a correlation between serum or hair testosteron levels and serum or hair cortisol levels in type 2 diabetic men.
- Trial name
- BZMC (NCT02529306)
- Where
- Israel
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Bnai Zion Medical Center
Mucopolysaccharidosis Type II, Metabolic Diseases
A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers
It is a phase I open-label single-dose, dose-escalation cohort study to evaluate of the tolerance, safety, and pharmacokinetics of GNR-055 in healthy volunteers
- Trial name
- NCT06475404
- Testing
- verenafusp alfa 6 mg/kg, verenafusp alfa 9 mg/kg, verenafusp alfa 12 mg/kg
- Where
- Russia
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM
Hyperuricemia or Gout
D-0120 Safety and PK/PD Study in China
It is a randomized, double-blind, placebo-controlled, multiple-administration, multiple-dose, dose-escalating, phase Ib/IIa clinical study to evaluate the safety, tolerability, PK and PD of D-0120 in healthy subjects and…
- Trial name
- NCT03923868
- Testing
- D-0120
- Where
- 6 sites in China
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- InventisBio Co., Ltd
Duchenne Muscular Dystrophy
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT06565208
- Testing
- SAT-3247
- Where
- 2 sites in Australia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Satellos Bioscience, Inc.
Hereditary Angioedema
Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations
This is a 3 part, phase 1 crossover study in healthy subjects to evaluate the pharmacokinetic profile of KVD824 following single and multiple doses of novel KVD824 modified-release formulations compared with a reference KVD824…
- Trial name
- NCT05118958
- Testing
- KVD824 Prototype 1 modified-release tablet, KVD824 Prototype 2 modified-release tablet, KVD824 Immediate-Release Capsule
- Where
- United Kingdom
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Diabetic Retinopathy, Macular Degeneration
Advancing Understanding of Transportation Options
This Stage II randomized, controlled, longitudinal trial seeks to assess the acceptability, feasibility, and effects of a driving decision aid use among geriatric patients and providers.
- Conditions
- Diabetic Retinopathy, Macular Degeneration, Glaucoma, Retinitis Pigmentosa
- Trial name
- AUTO (NCT04141891)
- Testing
- Driving Decision Aid, Older Drivers Website
- Where
- 3 sites in United States
- Volunteers
- 529 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Healthy
A Comparative Bioavailability/Food Effect Study of Immediate-Release and Extended-Release Tramadol HCl/Acetaminophen Combination Products Following Single-Dose and Multiple-Dose Administration in Healthy Adult Volunteers
The objectives of this two-part study were to compare the single-dose and multiple-dose pharmacokinetics of extended release and immediate-release fixed combination formulations containing tramadol-HCl/acetaminophen and to…
- Trial name
- NCT00973232
- Testing
- DDS-06C (650 mg acetaminophen and 75 mg tramadol HCl), DDS-06C (650 mg acetaminophen and 75 mg tramadol), Zaldiar®
- Where
- United Kingdom
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Labopharm Inc.
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of QBW251 in Healthy Subjects and Cystic Fibrosis Patients
This study is designed to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and cystic fibrosis patients following single and multiple doses.
- Trial name
- NCT02190604
- Testing
- QBW251
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 153 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Healthy Volunteer, Atopic Dermatitis
A Single and Multiple Ascending Dose Trial of KT-474 in Healthy Adult Volunteers and Patients With Atopic Dermatitis (AD) or Hidradenitis Suppurativa (HS)
KT-474 is an oral heterobifunctional small molecule IRAK4 degrader being developed for the treatment of interleukin-1 receptor (IL-1R)/toll-like receptor (TLR)-driven immune-inflammatory diseases.
- Conditions
- Healthy Volunteer, Atopic Dermatitis, Hidradenitis Suppurativa
- Trial name
- NCT04772885
- Testing
- KT-474
- Where
- 14 sites in United States
- Volunteers
- 154 people, healthy volunteers welcome
- Sponsor
- Kymera Therapeutics, Inc.
Down Syndrome
Symbolic Representation in Young Children With Down Syndrome
The aim of this study is to characterize the process of constitution of symbolic representation in infants with Down syndrome, as well as to investigate the relationship between gestures and the emergence of oral language.
- Trial name
- NCT00668980
- Where
- 3 sites in Brazil
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Sotos Syndrome, Beckwith-Wiedemann Syndrome
Body Perception and Representation in Overgrowth Syndromes, Behavioral Assessment and Neuropsychological Development
Sotos Syndrome (SS) and Beckwith-Wiedemann Syndrome (BWS) are known as overgrowth syndromes as they involve an excessive growth of the whole body or of specific body parts.
- Conditions
- Sotos Syndrome, Beckwith-Wiedemann Syndrome, Overgrowth Syndrome, Body Image
- Trial name
- Body-PROBAND (NCT04993235)
- Testing
- NEPSY-II, CBCL and AQ questionnaires, BUT questionnaire and experimental tasks
- Where
- Italy
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Long QT Syndrome
Phase 1 Study of LQT-1213 in Healthy Adults
This is a single-center, randomized, double-blind, placebo-controlled study to be conducted in 2 parts: single ascending dose (SAD) incorporating a food effect arm and multiple ascending dose (MAD).
- Trial name
- NCT05759962
- Testing
- LQT-1213
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Thryv Therapeutics, Inc.
X-Linked Hypohidrotic Ectodermal Dysplasia, Exercise-induced Rise of Body Temperature
Short Term Effects and Risks of Physical Exercise in Subjects With Hypohidrotic Ectodermal Dysplasia
Because of their lack of sweat glands individuals with hypohidrotic ectodermal dysplasia (HED) are at particular risk of life-threatening hyperthermia during exercise in a warm environment.
- Trial name
- NCT01135888
- Testing
- Skin cooling devices
- Where
- Germany
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- University Hospital Erlangen
Skin Laxity
The INFORM Study: Rotational Fractional Resection for Submental Contouring
Efficacy and safety of Rotational Fractional Resection (skin resection) in patients with moderate to severe submental skin laxity.
- Trial name
- NCT03853980
- Testing
- Focal Contouring System
- Where
- 14 sites in United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Recros Medica, Inc.
Neoplastic Syndrome, Non-melanomatous Skin Cancer
Topical Sirolimus in Patients With Basal Cell Nevus Syndrome and in Healthy Participants
RATIONALE: Studying samples of blood and tissue from patients with basal cell nevus syndrome and from healthy participants in the laboratory may help doctors learn more about changes that may occur in DNA and identify biomarkers…
- Trial name
- NCT00433485
- Testing
- sirolimus, comparative genomic hybridization, gene expression analysis
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Yale University
Congenital Ichthyosis
Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
This is an experimental non-randomized clinical study aimed at expanding the indications for the use of biological drugs with the aim of using them for the pathogenetic therapy of children with congenital ichthyosis.
- Trial name
- NCT04996485
- Testing
- Secukinumab Injection, Ustekinumab Injection, Dupilumab Injection
- Where
- Russia
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- National Medical Research Center for Children's Health, Russian Federation
Autosomal Dominant Polycystic Kidney Disease
Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy
Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation. This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic…
- Trial name
- NCT00920309
- Testing
- Rapamycin
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Yale University
Irritable Bowel Syndrome
A Phase 1, Randomized, Placebo-Controlled, Ascending Cohort, Dose Escalation Study in Normal Healthy Volunteers
GSK3179106 is a potent and relatively selective inhibitor of RET kinase which has been designed to be a safe and effective therapy for irritable bowel syndrome (IBS) patients .
- Trial name
- NCT02727283
- Testing
- GSK3179106
- Where
- Australia
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline