Clinical trials: Congenital Diseases
Genetic Disorder, Noonan Syndrome
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as…
- Trial name
- NCT03435627
- Testing
- Somatropin
- Where
- 22 sites in Japan
- Volunteers
- 71 people
- Sponsor
- Novo Nordisk A/S
Healthy
Bioequivalence Study of Colchicine Tablets
This randomized, single dose, three-way crossover study will evaluate the bioequivalence of two formulations of colchicine, the test product (colchicine 0.6mg Mutual) and a marketed combination product (colchicine 0.5 mg with…
- Trial name
- NCT01021020
- Testing
- Colchicine, Colchicine/Probenecid
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Mutual Pharmaceutical Company, Inc.
Cystic Fibrosis
Study of Interferon Gamma-1b by Injection for the Treatment of Patients With Cystic Fibrosis
The purpose of this research study is to evaluate the safety, tolerability, and efficacy of Interferon gamma-1b (IFN-gamma 1b) when administered by subcutaneous injection over a period of 4 weeks to patients with mild-to-moderate…
- Trial name
- NCT00043342
- Testing
- interferon gamma-1b
- Volunteers
- 51 people
- Sponsor
- InterMune
Atopic Dermatitis
POC Study to Evaluate BSI-045B in Moderate-to-severe Atopic Dermatitis
The study is a multicenter clinical trial and is designed as a proof-of-concept study to evaluate the efficacy, safety, tolerability, PK, immunogenicity, and PD of BSI-045B following SC injections.
- Trial name
- ADAMANT (NCT05932654)
- Testing
- BSI-045B
- Where
- 17 sites in United States
- Volunteers
- 22 people
- Sponsor
- Biosion, Inc.
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants…
- Trial name
- AFFINE (NCT04370054)
- Testing
- PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Where
- 45 sites in United States, Australia and 14 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
Cardiomyopathies, Heart Diseases
PKP2-ACM Natural History Study
The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2 Variants (PKP2-ACM) managed with standard of care.
- Conditions
- Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Genetic Diseases
- Trial name
- NCT06644742
- Where
- 3 sites in United States, Netherlands
- Volunteers
- 36 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Neonatal Abstinence Syndrome
Study to Collect Data for Neonatal Abstinence Syndrome (NAS) and Evaluate the Automated Data Collection Process
Substance abuse during pregnancy is on the rise through both prescribed and illicit use of controlled substances, which has increased neonatal abstinence syndrome (NAS).
- Trial name
- NCT06303986
- Testing
- NeoMonki
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Rekovar Inc.
Atopic Dermatitis, Atopic Dermatitis, Unspecified
A Study to Learn About the Study Medicine Etrasimod in Adults With Moderate to Severe Atopic Dermatitis (AD) Who Have Already Tried Treatments Taken by Mouth or by Injection
The purpose of this study is to learn about the safety and effects of the study medicine called etrasimod for the possible treatment of atopic dermatitis (AD), also called eczema, in adults who have already tried AD treatments…
- Conditions
- Atopic Dermatitis, Atopic Dermatitis, Unspecified, Eczema, Eczema, Atopic
- Trial name
- NCT05732454
- Testing
- etrasimod
- Where
- 74 sites in United States, Canada and 2 more
- Volunteers
- 58 people
- Sponsor
- Pfizer
Atopic Dermatitis
Topical YR001 Ointment in Adult With Mild to Moderate Atopic Dermatitis
This study is to evaluate the safety, tolerability, and pharmacokinetics of YR001 topical ointment in adult patients with mild to moderate atopic dermatitis
- Trial name
- NCT06309355
- Testing
- YR001 ointment
- Where
- 4 sites in United States
- Volunteers
- 35 people
- Sponsor
- Hangzhou Yirui Pharmaceutical Technology Co., Ltd
Atrial Septal Defect, Atrial Fibrillation
Feasibility Study on the VERAFEYE System
The objective of the study is to evaluate the performance of the VERAFEYE System in subjects entitled to undergo a standard of care ablation or closure procedure.
- Conditions
- Atrial Septal Defect, Atrial Fibrillation, Atrial Arrhythmia, Left Atrial Appendage Closure
- Trial name
- LUMINIzE (NCT05931835)
- Testing
- VERAFEYE System
- Where
- Ireland
- Volunteers
- 18 people
- Sponsor
- LUMA Vision Ltd.
Cystic Fibrosis
A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
Evaluate the safety and tolerability of VX-659 in healthy subjects
- Trial name
- NCT03029455
- Testing
- VX-659, Tezacaftor, Ivacaftor
- Where
- 10 sites in United Kingdom
- Volunteers
- 163 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hereditary Angioedema (HAE)
Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).
- Trial name
- NCT01059526
- Testing
- ecallantide
- Where
- 40 sites in United States
- Volunteers
- 81 people
- Sponsor
- Shire
Sickle Cell Disease
ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed.
- Trial name
- NCT04707235
- Testing
- Hydroxycarbamide
- Where
- 87 sites in France, French Guiana and 6 more
- Volunteers
- 2,093 people
- Sponsor
- Theravia
Wilson Disease
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy…
- Trial name
- NCT04884815
- Testing
- UX701, Standard of Care (SOC)
- Where
- 16 sites in United States, Portugal and 2 more
- Volunteers
- 82 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
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
Hemophilia A, Congenital
Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
The primary objective of this study is to investigate weekly prophylaxis dosing regimens used in standard clinical practice. In addition the study will capture reported bleed rate, pattern of change in KOVALTRY prophylaxis dose…
- Trial name
- TAURUS (NCT02830477)
- Testing
- Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973
- Where
- 25 sites in United States, Belgium and 11 more
- Volunteers
- 313 people
- Sponsor
- Bayer
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Phenylketonuria
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated…
- Trial name
- NCT01732471
- Testing
- Kuvan®
- Where
- Germany
- Volunteers
- 90 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.