Clinical trials: Newborn Diseases
Von Willebrand Disease
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (recombinant von Willebrand factor [rVWF]), with or without ADVATE, in the treatment and control of nonsurgical bleeding events…
- Trial name
- NCT02932618
- Testing
- Vonicog alfa, Antihemophilic Factor (Recombinant)
- Where
- 46 sites in United States, Austria and 11 more
- Volunteers
- 47 people
- Sponsor
- Baxalta now part of Shire
Uncontrolled Gout
Pegloticase and Methotrexate Co-administered in Participants With Uncontrolled Gout Who Previously Failed Pegloticase Monotherapy
This is a Phase 4, multicenter, open-label trial of pegloticase with methotrexate (MTX) in adult participants with uncontrolled gout who were previously treated with pegloticase without a concomitant immunomodulator and stopped…
- Trial name
- NCT04772313
- Testing
- Pegloticase, Methotrexate (MTX)
- Where
- 15 sites in United States
- Volunteers
- 11 people
- Sponsor
- Amgen
Perinatal Asphyxia
A Study of 2-Iminobiotin in Neonates With Perinatal Asphyxia
In case of insufficient oxygen supply to the brain of a newborn child (perinatal asphyxia), toxic compounds will be formed. These toxic compounds will damage the cells of the brain.
- Trial name
- NCT01626924
- Testing
- 2-Iminobiotin
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Neurophyxia B.V.
Polycystic Kidney, Autosomal Dominant
A Safety, Pharmacokinetic & Dose-Escalation Study of KD019 in Subjects With Autosomal Dominant Polycystic Kidney Disease
The primary objective of this study Phase 1b was to determine the safety, plasma pharmacokinetics, and maximum tolerated dose (MTD) of tesevatinib when administered to participants with autosomal dominant polycystic kidney…
- Trial name
- NCT01559363
- Testing
- Tesevatinib
- Where
- 11 sites in United States
- Volunteers
- 69 people
- Sponsor
- Kadmon, a Sanofi Company
Atopic Dermatitis
A Phase II Clinical Study Evaluating of ZL-82 Tablets in Atopic Dermatitis
This clinical trial aims to explore whether the drug ZL-82 tablets can be used to treat moderate to severe atopic dermatitis in adults, and to understand the safety and tolerability of the drug.
- Trial name
- NCT07193589
- Testing
- The high-dose group of ZL-82 tablets, The low-dose group of ZL-82 tablets
- Where
- China
- Volunteers
- 111 people
- Sponsor
- Chengdu Zenitar Biomedical Technology Co., Ltd
Homozygous Familial Hypercholesterolemia (HoFH)
A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
The purpose of the study is to establish the efficacy, safety and tolerability of rosuvastatin in children and adolescents with homozygous familial hypercholesterolemia.
- Trial name
- HYDRA (NCT02226198)
- Testing
- Rosuvastatin 20mg
- Where
- 9 sites in Belgium, Canada and 5 more
- Volunteers
- 20 people
- Sponsor
- AstraZeneca
Primary Immunodeficiency
Study of ProMetic BioTherapeutics Immune Globulin Intravenous (Human) 10%
Phase 3 multicenter, open-label study of safety, tolerability, efficacy, and pharmacokinetics (PK) of ProMetic's Immune Globulin Intravenous (Human) 10%, the investigational medicinal product [IMP]), in Adults and Children with…
- Trial name
- NCT02269163
- Testing
- Immune Globulin Intravenous, Prometic's Immune Globulin Intravenous 10%
- Where
- 13 sites in United States
- Volunteers
- 82 people
- Sponsor
- Prometic Biotherapeutics, Inc.
Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy
A Phase 1/ Phase 2 Study of TTHX1114(NM141)
Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study
- Conditions
- Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy, Endothelial Dysfunction
- Trial name
- INTREPID (NCT04520321)
- Testing
- TTHX1114(NM141)
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Trefoil Therapeutics, Inc.
Mucopolysaccharidosis I
Long Term Follow-Up for RGX-111
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase…
- Trial name
- NCT06103487
- Testing
- No Intervention
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
Hyperuricemia, Gout
Study of Levotofisopam 50 mg Three Times a Day (TID) Administered for 7 Days on Hyperuricemia and Gout
The purpose of this study is to determine whether levotofisopam is safe and effective in the treatment of hyperuricemia and gout.
- Trial name
- NCT01519687
- Testing
- levotofisopam
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Pharmos
Low Back Pain
A Study of Tanezumab in Adults With Chronic Low Back Pain
The purpose of this study is to evaluate the efficacy and safety of multiple doses of tanezumab administered every 8 weeks in treating chronic low back pain.
- Trial name
- NCT00876187
- Testing
- Tanezumab 20 mg IV, Tanezumab 10 mg IV, Tanezumab 5 mg IV
- Where
- 135 sites in United States
- Volunteers
- 1,359 people
- Sponsor
- Pfizer
Gout
A Phase Ib Clinical Trial of Peguricase for Injection With Methotrexate in Patients With Uncontrolled Gout.
To evaluate the safety and tolerability of peguricase for injection with methotrexate in patients with gout who remain uncontrolled after standardized treatment with conventional uric acid-lowering drugs, to determine the…
- Trial name
- NCT06298071
- Testing
- SIBP-R002, Methotrexate
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Shanghai Institute Of Biological Products
Squamous Cell Carcinoma of the Oral Cavity, Leukoplakia Oral
Infectious Aetiology of Potentially Malignant Disorders and Squamous Cell Carcinomas of the Oral Cavity
Recent studies estimated that 15 to 20% of all cancers in humans are associated with viruses. Among oral cancer about 90% are oral squamous cell carcinomas (OSCC).
- Trial name
- INECOC (NCT01636544)
- Testing
- contralateral healthy tissue biopsy
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Institut Pasteur
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn How Much Rosuvastatin Gets Into the Blood When Taken With or Without Elinzanetant and to Learn About the Safety of Elinzanetant in Healthy Participants
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, like those that happen in women during menopause. These symptoms can include vasomotor symptoms.
- Trial name
- NCT04889287
- Testing
- Elinzanetant (BAY3427080), Rosuvastatin
- Where
- Germany
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Bayer
Hemophilia B, Allergic Reactions
Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B
Retrospective, multicenter study in patients with hemophilia B. This study will be placed at 20 to 50 hemophilia treatment centers in North America and the EU, and is designed to estimate the frequency of Class II and III…
- Trial name
- NCT00195221
- Where
- 38 sites in United States, Austria and 7 more
- Volunteers
- 166 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A
Quality of Life Study of Helixate NexGen
The aim of this study is to describe Health Related Quality of Life (HRQoL) in adolescents and adults with Hemophilia A treated prophylactically or on-demand with Helixate NexGen.
- Trial name
- NCT01053715
- Where
- 31 sites in Austria, Belgium and 5 more
- Volunteers
- 78 people
- Sponsor
- CSL Behring
Cholangiocarcinoma
A Study of E7090 in Participants With Unresectable Advanced or Metastatic Cholangiocarcinoma With Fibroblast Growth Factor Receptor (FGFR) 2 Gene Fusion
The primary purpose of the study is to assess the objective response rate (ORR) of E7090 by Response Evaluation Criteria In Solid Tumors (RECIST) 1.1 based on independent imaging review (IIR) in participants with unresectable…
- Trial name
- NCT04238715
- Testing
- E7090
- Where
- 56 sites in China, Japan
- Volunteers
- 63 people
- Sponsor
- Eisai Co., Ltd.
Atopic Dermatitis
Oral DS107 in Moderate to Severe Atopic Dermatitis
This study will compare the safety and efficacy of Oral DS107 (2 g) to placebo in patients with moderate to severe atopic dermatitis. Oral DS107 (2 g) will be orally administered for 8 weeks, and will be compared against placebo.
- Trial name
- NCT02211417
- Testing
- Oral DS107
- Where
- 14 sites in United States, Canada
- Volunteers
- 102 people
- Sponsor
- Dignity Sciences Ltd.
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Ilaris NIS in Korea
This is a study to evaluate safety and effectiveness of Ilaris in adult and pediatric patients receiving the drug in a clinical setting for any of the following indications, Hereditary Periodic Fever Syndromes…
- Conditions
- Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS), Colchicine Resistance Familial Mediterranean Fever (crFMF), TNF Receptor Associated Periodic Syndrome (TRAPS)
- Trial name
- REASSURE (NCT06838143)
- Testing
- Ilaris
- Where
- South Korea
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Hyalinosis, Hyaline Membrane Disease
Biomarker for Hyaline Fibromatosis Syndrome (BioHFS)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Hyaline fibromatosis syndrome from the blood
- Conditions
- Hyalinosis, Hyaline Membrane Disease, Juvenile Hyaline Fibromatosis, Fibromatosis Hyalinica Multiplex Juvenilis
- Trial name
- BioHFS (NCT03196115)
- Where
- 3 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
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