Clinical trials: Genetic Conditions
Unverricht-Lundborg Disease
Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
- Trial name
- NCT00357669
- Testing
- Brivaracetam 25 mg, Brivaracetam 50 mg
- Where
- 13 sites in Finland, France and 5 more
- Volunteers
- 50 people
- Sponsor
- UCB Pharma SA
Acute Ischemic Stroke
Research Into the Effect of a Clot-dissolving Agent and Its Inhibitor
Single-chain urokinase-type plasminogen activator (pro-urokinase) is a highly effective thrombolytic drug. At pharmacologic concentrations however, pro-urokinase is converted to urokinase - a non specific thrombolytic, limiting…
- Trial name
- NCT01694381
- Testing
- M5, C1-inhibitor
- Where
- Netherlands
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- TSI, LLC
Osteoarthritis
Tanezumab In Osteoarthritis Of The Hip Or Knee
The purpose of the study is to test the efficacy and safety of 2 doses of tanezumab compared to oxycodone CR and placebo in patients with osteoarthritis
- Trial name
- NCT00985621
- Testing
- tanezumab 10 mg, tanezumab 5 mg, oxycodone
- Where
- 112 sites in United States, Austria and 5 more
- Volunteers
- 614 people
- Sponsor
- Pfizer
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cancer, Colorectal Cancer
FOG-001 in Locally Advanced or Metastatic Solid Tumors
The goal of this clinical trial is to determine if FOG-001 is safe and effective in participants with locally advanced or metastatic solid tumors or in participants with familial adenomatous polyposis (FAP).
- Conditions
- Cancer, Colorectal Cancer, Solid Tumor, Locally Advanced Solid Tumor
- Trial name
- NCT05919264
- Testing
- FOG-001, mFOLFOX-6, Nivolumab
- Where
- 33 sites in United States, Australia
- Volunteers
- 619 people
- Sponsor
- Parabilis Medicines, Inc.
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
Retinitis Pigmentosa (RP), Usher Syndrome
Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance
Retinitis pigmentosa and similar degenerative diseases of the retina lead to progressive loss of vision. TES therapy with the CE-marked OkuStim® System is a treatment approved in the EU for slowing the progression of the disease.
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome, Cone Rod Dystrophy
- Trial name
- TES-STEP (NCT07548944)
- Testing
- Transcorneal Electrical Stimulation using the OkuStim System
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Okuvision GmbH
Short Stature, Idiopathic, Infant, Small for Gestational Age
Evaluation of the Ease of Use, Preference, and Safety of EutropinPen Inj.
The purpose of this study is to assess the ease of use, preference, and safety after 8 weeks subcutaneous administration of EutropinPen Inj. in patients pretreated with recombinant human growth hormone by reusable device.
- Conditions
- Short Stature, Idiopathic, Infant, Small for Gestational Age, Growth Hormone Deficiency, Chronic Renal Failure
- Trial name
- NCT03015909
- Testing
- Somatropin
- Where
- South Korea
- Volunteers
- 116 people
- Sponsor
- LG Life Sciences
Atopic Dermatitis (Eczema)
The Effects of a Botanical Moisturizer, an Oral Supplement, and an Unscented Soap on Eczema and Itch
The purpose of this study is to examine the effects of a botanical moisturizer, an oral supplement, and an unscented soap on eczema severity, itch, mood, and various skin measures (such as skin hydration) in those with mild to…
- Trial name
- NCT06819709
- Testing
- Botanical Moisturizer + Oral Supplement + Unscented Soap
- Where
- Poland
- Volunteers
- 35 people
- Sponsor
- Codex Labs Corporation
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Familial Adenomatous Polyposis Coli, FAP
Chemoprevention Trial in Familial Adenomatous Polyposis (FAP) Coli Using EPA
This purpose of this study is to investigate whether the number and size of rectal polyps can be reduced in patients with Familial Adenomatous Polyposis (FAP) by using a highly-purified form of a naturally occurring substance…
- Trial name
- NCT00510692
- Testing
- Eicosapentanoic Acid (EPA), Endoscopy, Biopsies taken
- Where
- United Kingdom
- Volunteers
- 58 people
- Sponsor
- S.L.A. Pharma AG
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.
Stargardt Disease, Stargardt Macular Dystrophy
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- DEVOTE (NCT07734064)
- Testing
- ASP2020
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Onychomycosis of Toenails
Efficacy and Safety Evaluation of AN2690 Topical Solution to Treat Onychomycosis of the Toenail
The purpose of this study is to determine whether AN2690 topical solution is a safe and effective treatment for onychomycosis of the toenail.
- Trial name
- NCT01270971
- Testing
- AN2690 Topical Solution, 5%, Solution Vehicle
- Where
- 27 sites in United States
- Volunteers
- 594 people
- Sponsor
- Pfizer
Chronic Kidney Diseases, Diabetes
KidneYou - Innovative Digital Therapy
The aim of the study is to assess the improvement of health in patients affected by CKD when they are exposed to non-pharmacological treatment strategies as nutritional program (NP), physical activity program (PA) and mindfulness…
- Conditions
- Chronic Kidney Diseases, Diabetes, Arterial Hypertension, Chronic Glomerulonephritis
- Trial name
- NCT05286632
- Testing
- KidneYou APP
- Where
- Italy
- Volunteers
- 210 people
- Sponsor
- Advice Pharma Group srl
Familial Adenomatous Polyposis (FAP)
Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
The main goal of this clinical trial is to learn if the drug eRapa works to slow down the progression of disease in patients diagnosed with Familial Adenomatous Polyposis (FAP). Researchers will compare eRapa to Placebo.
- Trial name
- SERENTA (NCT06950385)
- Testing
- eRapa (encapsulated rapamycin)
- Where
- 29 sites in United States, Denmark and 5 more
- Volunteers
- 168 people
- Sponsor
- Rapamycin Holdings Inc.
Lennox Gastaut Syndrome, Dravet Syndrome
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- NCT05044819
- Testing
- Cannabidiol
- Where
- 26 sites in United States
- Volunteers
- 154 people
- Sponsor
- Jazz Pharmaceuticals
Lax Skin
Treatment to Lift Facial Lax Skin and Improve Facial Wrinkles Using Precise and Lift Applicators
Open-label, non-randomized single-arm prospective, multi-center, self-controlled clinical study with masked evaluation.
- Trial name
- NCT06333067
- Testing
- Sofwave
- Where
- 2 sites in United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD