Clinical trials: Newborn Diseases
Hemophilia A
Study Evaluating The Safety Of Xyntha In Usual Care Settings
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year…
- Trial name
- NCT00765726
- Testing
- Moroctocog alfa(AF-CC), Blood draw for laboratory testing
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 12 people
- Sponsor
- Pfizer
Dermatitis, Atopic
Tacrolimus Ointment Long Term Safety in Young Children With Atopic Dermatitis
The objective of this study is to assess the safety and efficacy of 0.03% tacrolimus ointment as long-term treatment in paediatric patients with atopic dermatitis.
- Trial name
- NCT00560326
- Testing
- Tacrolimus Ointment 0.03%
- Where
- 8 sites in Canada, Finland and 3 more
- Volunteers
- 50 people
- Sponsor
- Astellas Pharma Inc
Thyroid Eye Disease
A Study of TEPEZZA® Treatment in Participants With Thyroid Eye Disease
The main goal of this study is to learn how teprotumumab will be processed in the body (Pharmacokinetics) subcutaneously and whether it is safe and tolerable after administration into adult patients with thyroid eye disease.
- Trial name
- NCT06389578
- Testing
- Teprotumumab
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Amgen
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
Staphylococcus Aureus
Aurexis® in Cystic Fibrosis Subjects Chronically Colonized With Staphylococcus Aureus in Their Lungs
Patients who are at least 7 years old with stable Cystic Fibrosis who have Staphylococcus aureus in their Lungs will be enrolled into the study and receive one dose of Aurexis® intravenously on Study Day 1, and will be followed…
- Trial name
- NCT00198289
- Testing
- Aurexis® (tefibazumab)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Bristol-Myers Squibb
Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants…
- Trial name
- NCT03959189
- Testing
- ERX-963
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Expansion Therapeutics, Inc.
Atopic Dermatitis
A Study of Lebrikizumab (LY3650150) With/Without Topical Corticosteroid Treatment in Participants With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to evaluate the efficacy and safety of lebrikizumab with/without Topical Corticosteroid Treatment in Participants with Moderate-to-Severe Atopic Dermatitis.
- Trial name
- NCT06280716
- Testing
- Lebrikizumab, Topical Corticosteroid
- Where
- 46 sites in China, South Korea
- Volunteers
- 301 people
- Sponsor
- Eli Lilly and Company
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
Familial Hypercholesterolemia
Atorvastatin Three Year Pediatric Study
The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial…
- Trial name
- NCT00827606
- Testing
- atorvastatin
- Where
- 40 sites in United States, Belgium and 13 more
- Volunteers
- 272 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Hereditary Tyrosinemia, Type I
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
The purpose of this study is to look at the long term safety profile of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1 (HT-1).
- Trial name
- OPAL (NCT02320084)
- Testing
- Nitisinone
- Where
- 77 sites in Austria, Belgium and 15 more
- Volunteers
- 315 people
- Sponsor
- Swedish Orphan Biovitrum
Fragile X Syndrome, Behavior
An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome
Diverse symptomatology makes Fragile X Syndrome (FXS) difficult to treat, and currently there are no approved prevention or treatment methods for FXS.
- Conditions
- Fragile X Syndrome, Behavior, Cognitive Dysfunction
- Trial name
- NCT05832255
- Testing
- Psilocybin, 1.5 mg
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Nova Mentis Life Science Corp
Hemophilia A
PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for…
- Trial name
- NCT02492984
- Testing
- Intravenous infusions of Xyntha
- Where
- 15 sites in China
- Volunteers
- 85 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data.
- Trial name
- NCT01239758
- Testing
- ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03), ACE-031 (Extension of cohort 3 from core study, A031-03)
- Where
- 4 sites in Canada
- Volunteers
- 11 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Ichthyosis, Pruritus
Study Determining Safety and Efficacy of Avena Sativa (Oat) Skincare Products for Treating Skin Dryness and Itching in Cancer Patients
This study will test the safety and efficacy of three topical agents containing oat kernel flour to determine how well they relieve skin dryness and itching related to cancer therapies.
- Trial name
- NCT03796052
- Testing
- Avena Sativa Skincare Regimen
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Johnson & Johnson Consumer Inc. (J&JCI)
Huntington Disease
Mass Balance Study of [14C] LPM3770164 in Healthy Participants
This is a phase 1, single-center, single-dose, open-label mass-balance study to evaluate radioactive recovery rate, radioactive PK characteristics, metabolite identification, and to observe the safety in healthy male subjects of…
- Trial name
- NCT07516899
- Testing
- [14C] LPM3770164
- Where
- China
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Luye Pharma Group Ltd.
Primary Immunodeficiency (PID)
Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or Pumps
The purpose of this study is to compare satisfaction (LQI questionnaire, factor I: treatment interference) in PID patients receiving subcutaneous injections of Gammanorm® 165 mg/mL according to the delivery device.
- Trial name
- NCT02180763
- Testing
- Gammanorm
- Where
- 8 sites in France
- Volunteers
- 31 people
- Sponsor
- Octapharma
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.
Soft Tissue Injuries, Gouty Arthritis
AMPED Outcomes Registry of Post-ED Pain Management
Study aims to assess patient-recorded outcomes of pain control medications prescribed in the ER after visits for specific painful injuries/illnesses.
- Conditions
- Soft Tissue Injuries, Gouty Arthritis, Fractures, Ureteral Colic
- Trial name
- AMPED (NCT01626235)
- Where
- 11 sites in United States
- Volunteers
- 843 people
- Sponsor
- Radnor Registry Research, Inc
Carcinoid, Neuroendocrine
Safety Study of Seneca Valley Virus in Patients With Solid Tumors With Neuroendocrine Features
The primary purpose of the study is to determine if Seneca Valley Virus may be administered safely to patients with certain types of advanced cancer.
- Trial name
- NCT00314925
- Testing
- Seneca Valley Virus (biological agent)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Neotropix
Short Stature Born Small for Gestational Age (SGA)
Long Term Study of Genotropin (Somatropin) for Short Children Born Small for Gestational Age (SGA)
To assess the long-term safety of Genotropin(somatropin) on Small for Gestational Age (SGA) without epiphyseal closing.
- Trial name
- NCT01859949
- Testing
- Genotropin (somatropin)
- Where
- 19 sites in Japan
- Volunteers
- 62 people
- Sponsor
- Pfizer
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