Clinical trials: Newborn Diseases
Muscular Dystrophies
First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect
The FTIH study with GSK3439171A will evaluate the safety of GSK3439171A in healthy subjects in order to avoid confounding factors due to the disease or concomitant drugs in patients.
- Trial name
- NCT03627494
- Testing
- GSK3439171A
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Healthy Volunteers
A Study of Marstacimab to Compare Prefilled Pen (PFP) Device to Prefilled Syringe (PFS) Device
The goal in this study is to show that there are not significant differences in biologic activity of the study drug when administered using either the prefilled pen and prefilled syringe.
- Trial name
- NCT04832139
- Testing
- Marstacimab PFP, Marstacimab PFS
- Where
- Belgium
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Pfizer
Haemophilia A, Haemophilia B
Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B
The puropse of this non-interventional register and survey study is to identify the patterns of prescribed pain, anti-depressive and anti-anxiety medication and management of pain, depression and anxiety for people with…
- Trial name
- MIND (NCT03276130)
- Where
- 5 sites in Denmark, Finland and 1 more
- Volunteers
- 2,300 people
- Sponsor
- Swedish Orphan Biovitrum
Atrial Septal Defect
Safety and Efficacy of the AbsnowTM II Absorbable Atrial Septal Defect Closure System for Atrial Septal Defect Closure Therapy
A prospective, multicenter, randomized controlled, non-inferiority clinical trial to evaluate the safety and efficacy of the AbsnowTM II absorbable atrial septal defect occluder system for atrial septal defect closure therapy.
- Trial name
- NCT07529028
- Testing
- Absnow II Absorbable Atrial Septal Defect Occluder System, Cera Atrial Septal Defect Occluder
- Volunteers
- 236 people
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.
Sickle Cell Disease
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs).
- Trial name
- NCT05098028
- Testing
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bausch Health Americas, Inc.
HIV Infections
A Study of Delavirdine Mesylate in Combination With Other Anti-HIV Drugs in HIV-Infected Children and Babies
The purpose of this study is to see if it is safe and effective to give delavirdine mesylate (Rescriptor) plus two nucleoside reverse transcriptase inhibitors (NRTIs) to HIV-infected children and babies.
- Trial name
- NCT00002210
- Testing
- Delavirdine mesylate
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Pharmacia and Upjohn
Hypoxia Neonatal
Owlet Smart Sock 3 Accuracy on Infants
The purpose of this research study is to evaluate the Owlet Smart Sock 3 wireless pulse oximeter performance in assessing the heart rate and oxygen saturation in neonatal population, while measuring the same parameter with a…
- Trial name
- NCT05635513
- Testing
- Owlet OSS V3
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Owlet Baby Care, Inc.
Prader-Willi Syndrome
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS).
- Trial name
- NCT07266324
- Testing
- BMB-101
- Where
- 2 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Bright Minds Biosciences Pty Ltd
Gout
Evaluate the Efficacy and Safety of Arhalofenate for Preventing Flares and Reducing Serum Uric Acid in Gout Patients
The purpose of this study is to determine whether arhalofenate is effective in preventing flares and reducing serum uric acid in gout patients.
- Trial name
- NCT02063997
- Testing
- Arhalofenate 600 mg, Allopurinol 300 mg, Colchicine 0.6 mg
- Where
- 56 sites in United States, Canada and 1 more
- Volunteers
- 248 people
- Sponsor
- Gilead Sciences
Atopic Dermatitis
ICP-332 in Subjects With Moderate to Severe Atopic Dermatitis
A randomized, double-blind, placebo-controlled phase III study evaluating the efficacy and safety of oral ICP-332 in subjects with moderate to severe atopic dermatitis
- Trial name
- NCT06775860
- Testing
- ICP-332 Tablets
- Where
- 62 sites in China
- Volunteers
- 552 people
- Sponsor
- Beijing InnoCare Pharma Tech Co., Ltd.
Retinal Degeneration, Retinitis Pigmentosa
Safety & Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients
Patients who are legally blind, caused by retinal degeneration of photoreceptor rods \& cones (e.g. Retinitis pigmentosa), receive a subretinal implant to restore vision partially.
- Trial name
- NCT01497379
- Testing
- surgical implantation of subretinal device
- Where
- Hong Kong
- Volunteers
- 2 people
- Sponsor
- Retina Implant AG
Nevus, Dysplasia; Epithelial
Skin Fluorescent Imaging (SFI) System in Patients With Nevi
The Sponsor has developed the Skin Fluorescent Imaging (SFI) system, an in vivo imaging modality, for the purpose of providing physicians with insight into the biological changes occurring during tissue remodeling in a nevus.
- Trial name
- NCT04368247
- Testing
- Skin Fluorescent Imaging
- Where
- 4 sites in United States
- Volunteers
- 300 people
- Sponsor
- Orlucent, Inc
Hereditary Angioedema (HAE)
A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants
The objective of this study is to evaluate the efficacy, pharmacokinetics (PK), and safety of icatibant for the treatment of acute attacks in Japanese participants with type I or type II hereditary angioedema (HAE).
- Trial name
- NCT03888755
- Testing
- Icatibant
- Where
- 8 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
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.
Pediatric Obstructive Sleep Apnea, Down Syndrome (DS)
Pediatric Down Syndrome Post-Approval Study
The purpose of this Post Approval research study is to assess ongoing safety and effectiveness of the Inspire therapy in adolescents and young adults (age 13-18) with Down syndrome and severe sleep apnea.
- Trial name
- NCT06851338
- Testing
- Hypoglossal Nerve Stimulation
- Where
- 6 sites in United States
- Volunteers
- 60 people
- Sponsor
- Inspire Medical Systems, Inc.
Severe Hemophilia A
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
- Trial name
- NCT01712438
- Testing
- Human cl rhFVIII
- Where
- 37 sites in United States, Belarus and 15 more
- Volunteers
- 110 people
- Sponsor
- Octapharma
Hereditary Angioedema (HAE)
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.
- Trial name
- NCT05460325
- Testing
- Lanadelumab
- Where
- 4 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00759876
- Testing
- Ataluren
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- PTC Therapeutics
Advanced Nonhaematologic Malignancies
Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors
This study will be conducted to assess the safety and tolerability of BOS172738 when administered to patients with advanced solid tumors with rearranged during transfection (RET) gene alterations and also to establish the maximum…
- Trial name
- NCT03780517
- Testing
- BOS172738
- Where
- 21 sites in United States, Belgium and 5 more
- Volunteers
- 117 people
- Sponsor
- Boston Pharmaceuticals
Primary Immunodeficiency Diseases (PID)
Phase 2/3 Study of IGSC, 20% in PIDD
The purpose of this study is to develop a 20% subcutaneous (SC) immunoglobulin preparation for the treatment of patients with primary immunodeficiency diseases (PIDD).
- Trial name
- NCT01218438
- Testing
- Immune Globulin Intravenous (Human), 10% Solution, Immune Globulin Subcutaneous (Human), 20% Solution
- Where
- 18 sites in United States, Canada
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
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