Clinical trials: Inherited Genetic Conditions (Diagnosis)
Hyperalgesia, Pain Insensitivity, Congenital
The Relationship Between Saliva β-endorphins Levels, Cold Pressor Test and Perception of Pain in Oral Surgery Procedures
The purpose of this study is to evaluate the correlation between β-endorphin levels in blood plasma and saliva in healthy participants with different pain sensitivity and in those with acute pain in oral and maxillofacial region.
- Trial name
- NCT03164161
- Testing
- Cold pressor test, β-endorphins evaluation in saliva, β-endorphins evaluation in blood plasma
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Lithuanian University of Health Sciences
Fibrodysplasia Ossificans Progressiva (FOP)
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Primary Objective(s): To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.) To describe the prevalence of clinical characteristics of…
- Trial name
- NCT06064656
- Testing
- Non-Interventional
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- Regeneron Pharmaceuticals
To Determine Bioequivalence Under Fed Conditions
A Comparative Bioavailability Study of Lisinopril Tablets, 40 mg - Effect of Food Study
The purpose of this study is to compare the bioavailability of Par Lisinopril 40 mg Tablets and IPR Pharmaceuticals, Inc. Zestril (R).
- Trial name
- NCT01380431
- Testing
- Lisinopril
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Rare Diseases, Fabry Disease
MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.
- Conditions
- Rare Diseases, Fabry Disease, Adherence, Medication, Quality of Life
- Trial name
- MALTA-FABRY (NCT03683966)
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Wuerzburg University Hospital
Williams Syndrome, Supravalvular Aortic Stenosis
Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank
Williams syndrome is a rare genetic disorder occurring in 1:8000-12,000 individuals. It is caused by the deletion of 25-27 coding genes, including elastin (ELN) on the 7th human chromosome.
- Conditions
- Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
- Trial name
- NCT02706639
- Where
- 3 sites in United States
- Volunteers
- 305 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Stage IV Prostate Cancer, Adenocarcinoma of the Prostate
Docetaxel With or Without Thalidomide in Treating Patients With Metastatic Prostate Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Thalidomide may stop the growth of prostate cancer by stopping blood flow to the tumor.
- Conditions
- Stage IV Prostate Cancer, Adenocarcinoma of the Prostate, Recurrent Prostate Cancer
- Trial name
- NCT00020046
- Testing
- docetaxel, thalidomide
- Where
- United States
- Sponsor
- National Institutes of Health Clinical Center (CC)
Psoriasis (PsO), Vitiligo Vulgaris
Evaluation of Stigma Toward Patients With Alopecia Areata, Atopic Dermatitis, Vitiligo, and Psoriasis
Primary objective: The primary objective is to evaluate in the general population the difference between emotional reactions associated with facial involvement by four major dermatologic diseases: psoriasis, atopic dermatitis…
- Conditions
- Psoriasis (PsO), Vitiligo Vulgaris, Atopic Dermatitis, Areata Alopecia
- Trial name
- STIGMADERM (NCT06801821)
- Where
- Italy
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sickle Cell Disease
Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)
An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia.
- Trial name
- NCT01077921
- Testing
- Propranolol
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Laura M. De Castro, MD
Rheumatoid Arthritis, Ankylosing Spondylitis
Induction of Regulatory t Cells by Low Dose il2 in Autoimmune and Inflammatory Diseases
TRANSREG will assess the safety and biological efficacy of low-dose IL2 as a Treg inducer in a set of 14 autoimmune and auto-inflammatory diseases, with the aim to select diseases in which further therapeutic development will be…
- Conditions
- Rheumatoid Arthritis, Ankylosing Spondylitis, Systemic Lupus Erythematosus, Psoriasis
- Trial name
- TRANSREG (NCT01988506)
- Testing
- Interleukin 2
- Where
- 11 sites in France
- Volunteers
- 81 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How Safe the Study Treatment Elinzanetant is and How it Moves Into, Through and Out of the Body When Given as Single Increasing Doses That Are Higher Than Normally Used Compared to Placebo and Moxifloxacin in Healthy Participants
Researchers are looking for a better way to treat vasomotor symptoms, a condition of having hot flashes. The condition is caused by hormonal changes and occurs primarily in women, but can also affect men.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05481528
- Testing
- Moxifloxacin, Elinzanetant (BAY3427080)
- Where
- Germany
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Bayer
Hyperuricemia, Gout
Phase IIa Study of ALLN-346 (Engineered Urate Oxidase) in Subjects With Hyperuricemia, Gout and Chronic Kidney Disease
The purpose of this Phase IIa study is to evaluate the safety, tolerability and pharmacodynamics of ALLN-346 in subjects with hyperuricemia and gout, and with mild to moderate chronic kidney disease.
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Trial name
- NCT04987294
- Testing
- ALLN-346
- Where
- 17 sites in United States
- Volunteers
- 19 people
- Sponsor
- Allena Pharmaceuticals
Alpha-1 Antitrypsin Deficiency (AATD)
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
This study is the first study in the RestorAATion clinical program. The purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess…
- Trial name
- RestorAATion-1 (NCT06186492)
- Testing
- WVE-006
- Where
- United Kingdom
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Wave Life Sciences USA, Inc.
Homozygous Familial Hypercholesterolemia (HoFH)
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia…
- Trial name
- NCT02651675
- Testing
- AAV directed hLDLR gene therapy
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 9 people
- Sponsor
- REGENXBIO Inc.
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Rare Diseases, Marfan Syndrome
Psychological Support in Adult Patients With Marfan Syndrome
The goal of this observational study is to identify key factors leading to psychotherapy or psychological support in adult subjects with Marfan syndrome (MSF).
- Conditions
- Rare Diseases, Marfan Syndrome, Psychology, Social
- Trial name
- MARFANPSY (NCT05712577)
- Testing
- Questionnaire
- Where
- Italy
- Volunteers
- 125 people
- Sponsor
- IRCCS Policlinico S. Donato
Choreatic Disorder
Heart and Skeletal Muscle Problems in Neuroacanthocytosis
The purpose of this study is to learn about heart and skeletal muscle problems related to neuroacanthocytosis (also called Mcleod's syndrome and Levine-Critchley disease).
- Trial name
- NCT00007228
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Intracerebral Hemorrhage, CAA - Cerebral Amyloid Angiopathy
MRI-markers to Monitor Small Vessel Disease Dynamics in the Prognosis of Small Vessel Disease-associated, Cerebrovascular Events
This is a nested cohort study in the PRO-SVD cohort. Small vessel disease is a chronic disease and is thought to progress over time.
- Conditions
- Intracerebral Hemorrhage, CAA - Cerebral Amyloid Angiopathy, Cerebral Small Vessel Diseases
- Trial name
- MRI-PRO-SVD (NCT05773235)
- Testing
- Combined 3- and 7 Tesla-MRI
- Where
- Switzerland
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Down Syndrome
tDCS for Catatonic Depression in Down Syndrome: A Pilot Study
This study evaluates the efficacy of transcranial direct current stimulation (tDCS) for depression with catatonia in individuals with Down syndrome (DS).
- Trial name
- TOTORO (NCT06866925)
- Testing
- Transcranial stimulation
- Where
- France
- Volunteers
- 62 people
- Sponsor
- Hôpital le Vinatier
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