Clinical trials: Congenital Deformity
Fabry Disease
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
- Trial name
- NCT01124643
- Testing
- Replagal
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Shire
Sickle Cell Disease
Crizanlizumab Pregnancy Outcomes Intensive Monitoring
This was a non-interventional study to evaluate pre-specified pregnancy and infant outcomes in women treated with crizanlizumab during pregnancy or within 105 days before the last menstrual period (LMP).
- Trial name
- PRIM (NCT07554547)
- Where
- Switzerland
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia, Sickle ß0-Thalassemia
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT01179217
- Testing
- L-glutamine
- Where
- 31 sites in United States
- Volunteers
- 230 people
- Sponsor
- Emmaus Medical, Inc.
Safety, Tolerability
A Study of GenSci098 in Subjects With Active Thyroid Eye Disease
To evaluate the safety and tolerability of single and multiple ascending subcutaneous doses of GenSci098 in patients with thyroid eye disease (TED)
- Conditions
- Safety, Tolerability, GenSci098, Thyroid Eye Disease (TED)
- Trial name
- NCT06569758
- Testing
- GenSci098
- Where
- China
- Volunteers
- 76 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of PG-011 Gel in Adolescents and Adults With Atopic Dermatitis
This is a Multicenter, Randomized, Double-blind, Placebo-controlled seamless and adaptive-designed phase IIb/III study to Assess the Efficacy and Safety of topical use of PG-011 Gel in adolescents and Adults With Atopic…
- Trial name
- NCT06587685
- Testing
- PG-011Gel, Vehicle
- Where
- China
- Volunteers
- 472 people
- Sponsor
- Prime Gene Therapeutics Co., Ltd.
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.
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