Clinical trials: Hereditary Disease
Atopic Dermatitis
Correlation Study Between PEST and SCORAD in Management of Atopic Dermatitis With Ceradan® Regimen
This is a prospective, open label, single arm, and observational and multicenter study to assess the correlation between PEST and SCORAD scores in the management of AD with the Ceradan® regimen.
- Trial name
- COPES (NCT02073591)
- Testing
- Ceradan Cream, Ceradan Wash
- Where
- 2 sites in Singapore
- Volunteers
- 58 people
- Sponsor
- Hyphens Pharma Pte Ltd
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).
- Trial name
- NCT03912233
- Testing
- VX-121, TEZ, VX-561
- Where
- 26 sites in United States, Germany and 3 more
- Volunteers
- 87 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Asthma, COPD
A Clinical Study in Healthy Subjects to Evaluate 9MW1911 Injection
This is a Phase 1, randomized, placebo-controlled study, enrolling approximately 38 healthy adult subjects (18-65 yrs). The purpose of this study is to evaluate the safety, tolerability and PK of single ascending dose of 9MW1911…
- Conditions
- Asthma, COPD, Atopic Dermatitis
- Trial name
- NCT05170243
- Testing
- 9MW1911 Injection
- Where
- China
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Dermatitis, Atopic, Eczema
A Study to Evaluate Safety and Effectiveness of Cendakimab (CC-93538) in Participants With Moderate to Severe Atopic Dermatitis
The purpose of this study is to evaluate the effectiveness and safety of 3 dose regimen of CC-93538 in adult participants with moderate to severe Atopic Dermatitis (AD).
- Trial name
- NCT04800315
- Testing
- CC-93538
- Where
- 157 sites in United States, Canada and 4 more
- Volunteers
- 221 people
- Sponsor
- Celgene
Skin Laxity
Radiofrequency and Ultrasound for Improvement of Skin Laxity and Wrinkles: Efficacy and Safety Evaluation
This study will evaluate the clinical efficacy, safety and the performance of radiofrequency heating and ultrasound delivered by the BTL-585-2 applicator of the BTL-585F system for non-invasive treatment of facial wrinkles and…
- Trial name
- NCT05929625
- Testing
- BTL-585-2 RF+US, BTL-585-2 RF
- Where
- 2 sites in United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- BTL Industries Ltd.
Dermatitis, Atopic
Pharmacokinetic Study of Topical GSK2894512 Cream
This study will assess the systemic exposure and pharmacokinetic parameters of GSK2894512 following twice daily topical administration of 1% and 2% cream in adult subjects with AD, and will provide information about the systemic…
- Trial name
- NCT02466152
- Testing
- GSK2894512 Topical Cream
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- GlaxoSmithKline
Atopic Dermatitis
Multiple-Dose Pharmacokinetics and Potential for Adrenal Suppression Following Treatment With Halobetasol Spray in Patients With Atopic Dermatitis
This is an open-label, safety study designed to assess the multiple-dose pharmacokinetics and potential for adrenal suppression following topical treatment with halobetasol propionate 0.05% spray applied twice-daily in adult…
- Trial name
- NCT03245385
- Testing
- Treatment with Halobetasol propionate 0.05% topical spray
- Where
- 13 sites in United States
- Volunteers
- 20 people
- Sponsor
- Sun Pharmaceutical Industries Limited
Retinitis Pigmentosa
Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
The purpose of this study is to evaluate the safety of intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa by a prospective, single-center, randomized, parallel, double-blind, phase I…
- Trial name
- TC/RP (NCT02280135)
- Testing
- Intravitreal injection of Autologous bone marrow Stem Cell, Subconjunctival injection of saline
- Where
- Spain
- Volunteers
- 8 people
- Sponsor
- Red de Terapia Celular
Atrial Septal Defect
Cera™ ASD Occluder Post-Market Clinical Follow-Up Study
The objective of this post-market study is to: collect real-word clinical data and confirm the long-time safety and performance of the Lifetech Cera™ ASD Occluder identify previously unknown side-effects
- Trial name
- NCT06849635
- Where
- 6 sites in Poland
- Volunteers
- 139 people
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.
Beta-Thalassemia
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06024876
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Primary Immune Deficiency
Retrospective Chart Review of Subcutaneous IgG Use in Infants
This study is a retrospective chart review of the use of subcutaneous IgG (SCIG) in infants less than two years old.
- Trial name
- NCT01166074
- Testing
- Subcutaneous IgG (SCIG)
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- CSL Behring
Gout
Review of Safety Using Rilonacept in Preventing Gout Exacerbations (RE-SURGE)
The purpose of this clinical research study is to determine the safety and effectiveness of an experimental drug called rilonacept in subjects with gout who are on urate-lowering therapy.
- Trial name
- NCT00856206
- Testing
- Rilonacept
- Where
- 124 sites in United States, Germany and 4 more
- Volunteers
- 1,315 people
- Sponsor
- Regeneron Pharmaceuticals
Sickle Cell Disease
Managed Access Programs for SEG101, Crizanlizumab
The purpose of this registration is to list Managed Access Programs (MAPs) related to SEG101, Crizanlizumab
- Trial name
- NCT03720626
- Testing
- crizanlizumab
- Sponsor
- Novartis Pharmaceuticals
Epidermolysis Bullosa
Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa
This was a Phase III, Efficacy and Safety Study of Oleogel-S10 in Participants with Inherited Epidermolysis Bullosa (EB). EB is a rare group of genetic skin fragility disorders characterised by blistering of the skin in response…
- Trial name
- EASE (NCT03068780)
- Testing
- Oleogel-S10, Control gel
- Where
- 51 sites in United States, Argentina and 24 more
- Volunteers
- 223 people
- Sponsor
- Amryt Research Limited
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Dermatitis, Atopic
Tacrolimus Ointment Pharmacokinetics in Infants With Atopic Dermatitis
The purpose of this study is to estimate the systemic exposure to tacrolimus in infants with atopic dermatitis after repeated application of tacrolimus ointment.
- Trial name
- NCT00535691
- Testing
- Tacrolimus Ointment 0.03%
- Where
- 8 sites in Canada, Finland and 3 more
- Volunteers
- 53 people
- Sponsor
- Astellas Pharma Inc
Atopic Dermatitis
Phase 2a Study of Efficacy and Safety of OpSCF in Moderate to Severe AD (Atopic Dermatitis)
The purpose of this study is to determine the efficacy and safety of a monoclonal antibody, OpSCF, in the treatment of adults with moderate to severe Atopic Dermatitis (Eczema). OpSCF will be compared to a placebo.
- Trial name
- NCT06101823
- Testing
- OpSCF
- Where
- 22 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Insmed Incorporated
Von Willebrand Disease, Type 3
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants…
- Trial name
- WILL-EMI NIS (NCT06883240)
- Testing
- Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates
- Where
- 26 sites in United States, Belgium and 12 more
- Volunteers
- 44 people
- Sponsor
- Hoffmann-La Roche
Protein C Deficiency
A Study of Freeze-dried Human Protein C Concentrate (TAK-662) in Participants With Congenital Protein C Deficiency
This study is conducted in Japan of Freeze-dried Human Protein C Concentrate (TAK-662) used to treat participants with congenital protein C deficiency.
- Trial name
- NCT06590974
- Testing
- Freeze-dried Human Protein C Concentrate
- Where
- Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
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