Clinical trials: Multiple Congenital Anomaly
- Condition Multiple Congenital Anomaly
- Funder type Industry (pharma, biotech, devices)
- Accepts healthy volunteers yes
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
Mass Balance and Absolute Bioavailability Study of JMKX003142 In Healthy Volunteers
A phase 1 healthy volunteer study to assess the excretion and metabolism as well as the absolute bioavailability of oral JMKX003142.
- Trial name
- NCT07463053
- Testing
- [14C]JMKX003142, JMKX003142 tablets, [14C]JMKX003142 solution for infusion
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Jemincare
Chromosome 13 Aneuploidy, Chromosome 18 Aneuploidy
Non-invasive Prenatal Diagnostic Validation Study
The primary purpose of this study is to collect maternal blood samples from pregnant women to develop a non-invasive prenatal diagnostic test based on fetal DNA isolated from maternal blood.
- Conditions
- Chromosome 13 Aneuploidy, Chromosome 18 Aneuploidy, Chromosome 21 Aneuploidy, Sex Chromosome Aberrations
- Trial name
- NIPD (NCT01574781)
- Testing
- Blood draw, Cheek swab/Saliva Sampling
- Where
- United States
- Volunteers
- 1,781 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
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
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn More About How Much of the Study Treatment Elinzanetant (or BAY3427080) Gets Absorbed, How Safe it is and How it Affects the Body in Healthy Female and Male Participants
Researchers are looking for a better way to treat men and women with vasomotor symptoms, a condition of having hot flashes caused by hormonal changes.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05351892
- Testing
- Elinzanetant (BAY3427080)
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Bayer
Dermatitis, Atopic, Dermatitis Eczema
A Study To Evaluate ASN002 In Subjects With Atopic Dermatitis
This is a dose escalation study to test the safety, tolerability and preliminary efficacy of ASN002 in people with moderate to severe atopic dermatitis (AD).
- Conditions
- Dermatitis, Atopic, Dermatitis Eczema, Dermatitis, Eczematous
- Trial name
- NCT03139981
- Testing
- ASN002
- Where
- 10 sites in United States, Canada
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Asana BioSciences
Healthy Volunteer, Down Syndrome
A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662
This single-center, single-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down…
- Trial name
- NCT01667367
- Testing
- RG1662
- Where
- United Kingdom
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Trisomy 13, Trisomy 18
High Risk Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our proprietary algorithm method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278874
- Where
- 4 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Cystic Fibrosis
Drug-Drug Interaction Study of VX-809 and VX-770 in Healthy Subjects
The purpose of this study is to assess the pharmacokinetics and safety of co-administration of VX-770 and VX-809 in healthy adults.
- Trial name
- NCT00966602
- Testing
- VX-809, VX-770, VX-809 & VX-770
- Where
- Netherlands
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Von Willebrand Diseases
Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)
The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other…
- Trial name
- NCT05776069
- Testing
- VGA039
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 116 people, healthy volunteers welcome
- Sponsor
- Vega Therapeutics, Inc
Atopic Dermatitis (AD)
Study to Investigate the Safety, Tolerability, and Pharmacokinetics of GB-7624 in Healthy Adult Participants
This is a randomised, double-blinded, placebo-controlled first in human (FIH) trial to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity of GB-7624.
- Trial name
- NCT06920693
- Testing
- GB-7624
- Where
- Australia
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Generate Biomedicines
Hereditary Hemorrhagic Telangiectasia
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers evaluate the efficacy, safety, tolerability and PD of…
- Trial name
- InsigHHT (NCT06659640)
- Testing
- ALN-6400
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Healthy Volunteers, Atopic Dermatitis
A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome
The study is divided in 3 parts, starting with the safety assessment of BPR277 ointment in Healthy volunteers (Part 1). If found to be well tolerated in Part 1, BPR277 ointment will be assessed in two different patients groups to…
- Conditions
- Healthy Volunteers, Atopic Dermatitis, Netherton Syndrome
- Trial name
- 3 (NCT01428297)
- Testing
- BPR277 ointment (controlled application), BPR277 ointment, BPR277
- Where
- 4 sites in United States, Netherlands
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult Subjects
This study evaluated the safety and pharmacokinetics of multiple ascending doses of VX-440 in combination with tezacaftor/ivacaftor (TEZ/IVA) (triple combination [TC]) administered for 13 days to healthy male and female subjects
- Trial name
- NCT03486236
- Testing
- VX-440, TEZ, IVA
- Where
- United Kingdom
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Healthy Volunteers, Sickle Cell Disease
A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients
FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias.
- Trial name
- NCT03815695
- Where
- 18 sites in United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Forma Therapeutics, Inc.
Amyloid Cardiomyopathy, Transthyretin-Related
Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
- Trial name
- NCT03294707
- Testing
- AG10 oral tablet
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Idiopathic Pulmonary Fibrosis
Assess the Safety, Tolerability and Pharmacokinetics of AZD5055 Following Single and Multiple Ascending Doses in Healthy Participants
This is a phase I, First-in-Human study in healthy participants, performed at a single study center, consisting of 2 parts: Part 1 is a single ascending dose (SAD) study and Part 2 is a multiple ascending dose (MAD) study.
- Trial name
- NCT05134727
- Testing
- AZD5055
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Healthy Volunteers, Polycystic Kidney, Autosomal Dominant
Effect of Venglustat in Patients With Renal Impairment
Primary Objective: To study the effect of mild, moderate and severe renal impairment on the pharmacokinetics (PK) of Venglustat following a single dose.
- Trial name
- NCT03687554
- Testing
- Venglustat GZ/SAR402671
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Primary Hyperoxaluria
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
This is a double-blind, placebo-controlled, dose escalation trial of DCR-PHXC in Healthy Volunteers (HVs) and patients with Primary Hyperoxaluria (PH).
- Trial name
- NCT03392896
- Testing
- DCR-PHXC
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Hereditary Angioedema
First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX7353 in healthy subjects, and single and…
- Trial name
- NCT02448264
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC