Clinical trials: Effect Neonatal
Atopic Dermatitis
A Study of Orally Administered IPG7236 in Healthy Adult Participants
The study is a phase 1, randomized, double-blind, placebo-controlled, single and multiple dose escalation study to evaluate the safety, tolerability, pharmacokinetic and food effect of orally administered IPG7236 in healthy adult…
- Trial name
- NCT05288543
- Testing
- IPG7236- Single ascending dose, IPG7236- Multiple ascending dose
- Where
- Australia
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Nanjing Immunophage Biotech Co., Ltd
Stargardt Disease
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
- Trial name
- STARLIGHT (NCT05417126)
- Testing
- Gene Therapy-vMCO-010
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Nanoscope Therapeutics Inc.
Becker Muscular Dystrophy
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 175 adult participants in a cohort…
- Trial name
- NCT05291091
- Testing
- Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg
- Where
- 51 sites in United States, Australia and 10 more
- Volunteers
- 244 people
- Sponsor
- Edgewise Therapeutics, Inc.
Alpha1-antitrypsin Deficiency
GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study
The purpose of the study is 2-fold: (1) to evaluate the safety and potential immunogenicity of GLASSIA following intravenous (IV) administration via in-line filtration; and (2) to assess the effects of GLASSIA augmentation…
- Trial name
- NCT02525861
- Testing
- GLASSIA
- Where
- 18 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
- Trial name
- NCT00743483
- Testing
- rhBSSL
- Where
- 4 sites in Netherlands, Poland
- Volunteers
- 15 people
- Sponsor
- Swedish Orphan Biovitrum
Epidermolysis Bullosa
A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa
The purpose of this study is to investigate a treatment to enhance the healing of acute and chronic nonhealing cutaneous wounds, such as the erosions experienced by patients with Epidermolysis Bullosa (EB), by the known activity…
- Trial name
- NCT00311766
- Testing
- Thymosin Beta 4
- Volunteers
- 30 people
- Sponsor
- RegeneRx Biopharmaceuticals, Inc.
Hypertension
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
The purpose of this study is to determine whether 6R-BH4 (sapropterin dihydrochloride) is safe and effective in the treatment of poorly controlled hypertension in the presence or absence of type 2 diabetes.
- Trial name
- NCT00325962
- Testing
- 6R-BH4 (sapropterin dihydrochloride)
- Where
- 24 sites in United States
- Volunteers
- 84 people
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell Disease
A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease
- Trial name
- Hestia2 (NCT02482298)
- Testing
- Ticagrelor
- Where
- 20 sites in United States, Egypt and 6 more
- Volunteers
- 87 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With Ivacaftor
To evaluate the effect of ciprofloxacin on the pharmacokinetics (PK) of ivacaftor and on the pharmacokinetics of VX-661 when administered in combination with ivacaftor
- Trial name
- NCT02015507
- Testing
- ivacaftor, VX-661, ciprofloxacin
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Alpha-1 Antitrypsin Deficiency
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
- Trial name
- NCT02900183
- Testing
- ARC-AAT Injection
- Where
- 4 sites in Canada, Ireland and 2 more
- Sponsor
- Arrowhead Pharmaceuticals
Dermatitis Atopic
Dupilumab-PEdiatric Skin Barrier Function and LIpidomics STudy in Patients With Atopic Dermatitis
Primary Objective: Evaluate changes in skin barrier function with transepidermal water loss (TEWL) assessed after skin tape stripping (STS) in predefined lesional skin in pediatric participants with moderate to severe atopic…
- Trial name
- PELISTAD (NCT04718870)
- Testing
- Dupilumab SAR231893
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Sanofi
Lactose Intolerance
Effect of Probiotic Supplementation on Lactose Maldigestion Induced by Lactose Solution
Study objectives: Primary: Difference in breath hydrogen concentration (BHC, ppm) in lactase and probiotic groups compared to placebo, measured by the incremental area under curve (iAUC) analysis Non-inferiority in breath…
- Trial name
- NCT03814668
- Testing
- Probiotic, Lactase
- Where
- France
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Danisco
Atopic Eczema
A Phase 2a Study to Investigate the Effects of Repeated Administration of AeroDerm in Subjects With Atopic Eczema
The primary objective of this study is to investigate the effect of AERODERM (also known as pitrakinra, AER 001, BAY 16-9996) on Eczema Area and Severity Index (EASI) score in subjects with moderate to severe atopic eczema
- Trial name
- NCT00676884
- Testing
- Aeroderm (also known as pitrakinra, AER 001, BAY 16-9996)
- Where
- United Kingdom
- Volunteers
- 25 people
- Sponsor
- Aerovance, Inc.
Hemophilia A
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital…
- Trial name
- ZEBRHA 2 (NCT07416604)
- Testing
- NXT007, Emicizumab
- Where
- 63 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Hoffmann-La Roche
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX…
- Trial name
- NCT03943290
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 62 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
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
Gout and Hyperuricemia
PK Effects of SHR4640 on Repaglinide and Midazolam, and the Impact of SHR4640 on QT Interval
The purpose of this study is to evaluate the PK effects of SHR4640 tablets on repaglinide and midazolam, as well as the effects of SHR4640 tablets on the QT interval in healthy subjects.
- Trial name
- NCT06196580
- Testing
- repaglinide; midazolam; SHR4640
- Where
- China
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Hypochondroplasia
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
- Trial name
- CANOPY-HCH-3 (NCT06455059)
- Testing
- Vosoritide
- Where
- 23 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- BioMarin Pharmaceutical
Healthy Volunteers, Atopic Diathesis and Atopic Dermatitis
Single Dose and Multiple Dose Study to Assess Safety and Tolerability of LOU064
This is a 6-part first-in-human study in up to approximately 184 participants. Parts 1 to 5 is in health volunteers and part 6 is in subjects with atopic dermatitis.
- Trial name
- NCT03918980
- Testing
- LOU064
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Healthy Subjects, Sickle Cell Disease
A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).
- Trial name
- NCT02285088
- Testing
- GBT440
- Where
- United Kingdom
- Volunteers
- 133 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics