Clinical trials: Hereditary Disease
Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Evaluation of Long-Term Efficacy of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia at High Cardiovascular Risk
The purpose of this study is to see if bemedoic acid (ETC-1002) is effective versus placebo in patients with high cardiovascular risk and elevated LDL cholesterol not adequately controlled by their current therapy.
- Trial name
- CLEAR Wisdom (NCT02991118)
- Testing
- bempedoic acid
- Where
- 2 sites in United States
- Volunteers
- 779 people
- Sponsor
- Esperion Therapeutics, Inc.
Thyroid Associated Ophthalmopathies, Thyroid-Associated Ophthalmopathy
Teprotumumab (RV 001) Treatment in Patients With Active Thyroid Eye Disease
The primary objective of this study is to investigate the efficacy, safety, and tolerability of RV 001 (teprotumumab), a fully human anti-IGF1R antibody, administered q3W for 6 months, in comparison to placebo, in the treatment…
- Trial name
- NCT01868997
- Testing
- teprotumumab, normal saline
- Where
- 15 sites in United States, Germany and 2 more
- Volunteers
- 88 people
- Sponsor
- Amgen
Atopic Dermatitis / Eczema
To Evaluate the in Use Tolerance of E45 Eczema Repair Emollient in Babies and Children With (Very(Dry/Atopic Skin
The investigation will evaluate, under dermatological control, the cutaneous in-use tolerance of E45 Eczema Repair Emollient with two or three times daily application, over a period of two weeks on children and babies with…
- Trial name
- NCT02857062
- Testing
- E45 Eczema Repair Emollient
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Reckitt Benckiser Healthcare (UK) Limited
Alzheimer's Disease, Huntington's Disease
A Study Evaluating The Absorption Of Dimebon Into The Body From A Dimebon Solution Applied To The Skin
To estimate the absorption, safety, and tolerability of a dimebon transdermal solution relative to the dimebon immediate release oral formulation.
- Trial name
- NCT00990613
- Testing
- Dimebon IR, Dimebon Transdermal
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Pfizer
Hemophilia A
Efficacy and Safety of Recombinant Factor VIII (GreenGene) in Patients With Hemophilia A
The purpose of this study is to study the evaluation of efficacy and safety of GreenGene (Recombinant Factor VIII).
- Trial name
- NCT01568580
- Testing
- GreenGene
- Volunteers
- 71 people
- Sponsor
- Green Cross Corporation
Chorea, Huntington
Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in Canada
This study will evaluate long-term safety and tolerability of valbenazine in participants with chorea associated with Huntington Disease (HD) who participated in Study NBI-98854-HD3006 (NCT04400331) in Canada.
- Trial name
- NCT06312189
- Testing
- Valbenazine
- Where
- 3 sites in Canada
- Volunteers
- 7 people
- Sponsor
- Neurocrine Biosciences
Hemophilia B
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
- Trial name
- NCT03855280
- Testing
- APVO101
- Where
- 11 sites in Brazil, Georgia and 4 more
- Volunteers
- 21 people
- Sponsor
- Medexus Pharma, Inc.
Atopic Dermatitis
Phase 2a Study of the Safety, Tolerability, and Pharmacokinetics of Topically Administered PRN473 (SAR444727) in Patients With Mild to Moderate Atopic Dermatitis
This was a Ph2a study that consists of a double-blind, intra-patient placebo-controlled treatment period and an open-label uncontrolled treatment period with objective to evaluate the safety, tolerability, PK and preliminary…
- Trial name
- NCT04992546
- Testing
- PRN473 (SAR444727)
- Where
- 12 sites in United States, Canada
- Volunteers
- 39 people
- Sponsor
- Principia Biopharma, a Sanofi Company
Atopic Dermatitis, Psoriasis
Analytical Validation of Wrist-Based Assessment of Nocturnal Scratch in Atopic Dermatitis and Psoriasis
Pruritis is a common symptom in patients with atopic dermatitis and psoriasis. Patients with atopic dermatitis and psoriasis often report pruritis-associated sleep impairments and scratching behaviors during nighttime and the…
- Trial name
- DECODE-Scratch (NCT06448689)
- Where
- France
- Volunteers
- 90 people
- Sponsor
- ActiGraph L.L.C
Healthy
Controlled Randomized Study With Athena Base Plate Versus SenSura Base Plate on Perspiring Skin
Aim The aim of the Athena project is to develop an ostomy product that is softer and more flexible in order to improve the comfort for people with a stoma.
- Trial name
- NCT01245413
- Testing
- SenSura, Athena
- Where
- Denmark
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Coloplast A/S
Niemann-Pick Disease, Type C
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC).
- Trial name
- NCT05163288
- Testing
- N-Acetyl-L-Leucine
- Where
- 14 sites in United States, Australia and 6 more
- Volunteers
- 53 people
- Sponsor
- IntraBio Inc
DM1, Myotonic Dystrophy
Global Study of Del-desiran for the Treatment of DM1
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia
- Trial name
- HARBOR (NCT06411288)
- Testing
- AOC 1001 (del-desiran)
- Where
- 34 sites in United States, Canada and 8 more
- Volunteers
- 159 people
- Sponsor
- Avidity Biosciences, Inc.
Gaucher Disease Type 1
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy…
- Trial name
- NCT00319046
- Testing
- Miglustat
- Volunteers
- 42 people
- Sponsor
- Actelion
Primary Immunodeficiency Diseases (PID)
Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatment regimens and treatment administration…
- Trial name
- NCT02593188
- Testing
- HYQVIA
- Where
- 30 sites in United States
- Volunteers
- 264 people
- Sponsor
- Baxalta now part of Shire
Autosomal Dominant Polycystic Kidney Disease
The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
This is a Phase 2, open-label, parallel-group, multiple dose study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and tolerability of multiple doses of lixivaptan in Autosomal Dominant Polycystic Kidney…
- Trial name
- NCT03487913
- Testing
- Lixivaptan
- Where
- 13 sites in United States
- Volunteers
- 31 people
- Sponsor
- Palladio Biosciences
Retinitis Pigmentosa (RP), Age Related Macular Degeneration
Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies
Summary of the Study This clinical trial evaluates a novel peptide-based therapy for treating retinal dystrophies, age-related macular degeneration (AMD), and diabetic retinopathy (DR).
- Conditions
- Retinitis Pigmentosa (RP), Age Related Macular Degeneration, Diabetic Retinopathy
- Trial name
- Retino (NCT06787482)
- Testing
- ACE Retino
- Where
- 2 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Ace Cells Lab Limited
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.
Atopic Dermatitis
A Study of the Long-Term Safety of Crisaborole Ointment, 2% in Japanese Pediatric and Adult Participants With Mild to Moderate Atopic Dermatitis
This study is a Phase 3, multicenter, open-label, long-term safety extension study of Studies C3291032 and C3291031 in Japanese pediatric and adult participants with mild to moderate Atopic Dermatitis (AD).
- Trial name
- NCT04498403
- Testing
- Crisaborole 2%
- Where
- 11 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Pfizer
Wrinkle, Lax Skin
A Prospective Controlled Study to Evaluate the Safety and Effectiveness of Thermage® FLX Radiofrequency Treatment
A prospective, 180-day, randomized, multicenter, independent blinded evaluators, controlled study of treatment with the Thermage FLX System
- Conditions
- Wrinkle, Lax Skin, Lines Skin
- Trial name
- NCT07187297
- Testing
- Thermage FLX
- Where
- 7 sites in United States
- Volunteers
- 164 people, healthy volunteers welcome
- Sponsor
- Bausch Health Americas, Inc.
Skin Manifestations, Skin Abnormalities
Efficacy of Vinh Wellness Collagen on Skin Health
The most visible signs of aging are on the skin. As skin gets older it shows a decrease in epidermal thickness, with a flattening of the dermal-epidermal junction. At the same time, loss of elasticity and wrinkle formation begin.
- Trial name
- NCT04449159
- Testing
- Vinh Wellness Collagen
- Where
- Canada
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Vinh Hoan Corporation
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