Clinical trials: Multiple Congenital Anomaly
Drug-resistant Focal Epilepsies in Pediatric Population
Genetic and Electrophysiologic Study in Focal Drug-resistant Epilepsies
Brain somatic mutations are increasingly recognized as a major cause of focal epilepsies. These include mTOR pathway mutations underlying cortical malformations such as focal cortical dysplasia and hemimegalencephaly, and SLC35A2…
- Trial name
- GENEPHY (NCT02890641)
- Testing
- Sampling of blood, frozen resected tissues, and cerebrospinal fluid (CSF)
- Where
- France
- Volunteers
- 450 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Sphingomyelin Lipidosis
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.
- Trial name
- ASCEND-Peds (NCT02292654)
- Testing
- Olipudase alfa
- Where
- 6 sites in United States, Brazil and 4 more
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Alagille Syndrome
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US.
- Trial name
- NCT04530994
- Testing
- maralixibat
- Where
- 14 sites in United States
- Sponsor
- Mirum Pharmaceuticals, Inc.
Breast Cancer, Chronic Myeloproliferative Disorders
Study of the Emotional Needs of Caregivers of Stem Cell Transplantation Patients
RATIONALE: Understanding the emotional needs of spouses or others who are living with and caring for patients who have undergone stem cell transplantation may help improve the quality of life of both the caregivers and the…
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00082654
- Testing
- psychosocial assessment and care
- Where
- United States
- Sponsor
- Fred Hutchinson Cancer Center
Wilson Disease
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation…
- Trial name
- NCT03403205
- Testing
- ALXN1840, SoC Therapy
- Where
- 56 sites in United States, Australia and 20 more
- Volunteers
- 214 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Congenital Heart Disease
Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on exercise capacity through cardiopulmonary exercise testing.
- Trial name
- RUBATO (NCT03153137)
- Testing
- Macitentan 10 mg
- Where
- 32 sites in United States, Australia and 10 more
- Volunteers
- 142 people
- Sponsor
- Actelion
Adult Spinal Deformity
Neurologic Complications in Spinal Deformity Surgery - Extension
272 subjects with "high risk" adult spinal deformity requiring surgical correction were enrolled in the previous prospective multi-center international Scoli-RISK-1 study.
- Trial name
- NCT02949245
- Testing
- Interventions
- Where
- 11 sites in United States, Canada and 4 more
- Volunteers
- 77 people
- Sponsor
- AO Foundation, AO Spine
Advanced Carcinoid Tumor, Digestive System Neuroendocrine Neoplasm
Lenvatinib and Everolimus in Treating Patients With Advanced, Unresectable Carcinoid Tumors
This phase II trial studies how well lenvatinib and everolimus work in treating patients with carcinoid tumors that have spread to other places in the body (advanced) and cannot be removed by surgery (unresectable).
- Conditions
- Advanced Carcinoid Tumor, Digestive System Neuroendocrine Neoplasm, Multiple Endocrine Neoplasia Type 1, Neuroendocrine Neoplasm
- Trial name
- NCT03950609
- Testing
- Everolimus, Lenvatinib
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- M.D. Anderson Cancer Center
Twin Dichorionic Diamniotic Placenta, Twin Monochorionic Monoamniotic Placenta
Austrian Registry on the Outcome of Monochorionic Pregnancies
About one third of twin pregnancies are resulting from a single fertilized oocyte. Two third of these monozygotic twins share a common placenta and are therefore called monochorionic.
- Conditions
- Twin Dichorionic Diamniotic Placenta, Twin Monochorionic Monoamniotic Placenta, Twin-To-Twin Transfusion Syndrome, Twin Pregnancy With Antenatal Problem
- Trial name
- MonoReg (NCT03171909)
- Where
- 13 sites in Austria
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003
This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1…
- Trial name
- NCT06714006
- Testing
- PYC-003
- Where
- 12 sites in Australia, Hong Kong and 1 more
- Volunteers
- 166 people, healthy volunteers welcome
- Sponsor
- PYC Therapeutics
Neonatal Alloimmune Thrombocytopenia
Study on the Anti-HPA Antibodies Caused Neonatal Alloimmune Thrombocytopenia in Chinese Pregnant Women
This clinical study aims to investigate the anti-HPA antibodies caused neonatal alloimmune thrombocytopenia in Chinese pregnant women. The survey and data collect will help form an appropriate clinical screening procedure
- Trial name
- NCT02934906
- Testing
- Standard clinical treatment, including intravenous immunoglobulin and/or platelet transfusion
- Volunteers
- 55,497 people, healthy volunteers welcome
- Sponsor
- Xinqiao Hospital of Chongqing
Healthy
A Pharmacokinetics and Safety of Methylphenidate HCl in Healthy Japanese Adult Male Volunteers
The purpose of the study is to evaluate the pharmacokinetics and safety of 3 single doses of Methylphenidate HCl in healthy Japanese adult male volunteers.
- Trial name
- NCT01202734
- Testing
- Methylphenidate HCl
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Primary Immunodeficiency
A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency
The purpose of the study is to determine whether biweekly (every 2 weeks) administration of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) produces a steady-state area under the…
- Trial name
- NCT04566692
- Testing
- IGSC 20%
- Where
- 12 sites in United States
- Volunteers
- 33 people
- Sponsor
- Grifols Therapeutics LLC
Atopic Dermatitis
An Evaluation of LEO 138559 in Adults With Moderate to Severe Atopic Dermatitis.
This is a clinical study in adult participants with moderate to severe atopic dermatitis (AD). The purpose of the study is to test a new medicine (LEO 138559) given by injection to see if it works to treat AD and what the side…
- Trial name
- NCT04922021
- Testing
- LEO 138559
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 58 people
- Sponsor
- LEO Pharma
Thalassemia
Colla Corii Asini Treating Anemia in Pregnant Women With Thalassemia(Presenting the Syndrome of Blood Deficiency)
480 pregnant patients diagnosed of silent or standard α-thalassemia, HbH disease, minor or intermediate β-thalassemia with mild anemia will be randomly assigned to treatment group and control group.
- Trial name
- NCT03374111
- Testing
- Colla corii asini, a Simulate Agent of Colla corii asini granule
- Where
- 12 sites in China
- Volunteers
- 480 people
- Sponsor
- The First Affiliated Hospital, Guangzhou University of Traditional Chinese Medicine
Exostoses, Multiple Hereditary
The Health-Related Quality of Life in Patients With Hereditary Multiple Exostoses
The purpose of this study is to assess the health-related quality of life of subjects who have Hereditary Multiple Exostoses and to develop a disease specific quality of life survey.
- Trial name
- NCT00474448
- Where
- Canada
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
Congenital Heart Disease
Study on the Relationship Between Maternal Exposure to Environmental Factors and Fetal Congenital Heart Disease
The objective of this study is to research the relationship between maternal exposure to environmental factors and fetal congenital heart disease in a community-based case-control study.
- Trial name
- NCT02914392
- Where
- 2 sites in China
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Androgenic Alopecia
Effect of Pumpkin Seed Oil on Hair Growth in Men With Androgenetic Alopecia
Androgenic alopecia is the most common cause of hair loss in men. Finasteride and minoxidil are available in treatment of androgenetic alopecia, although the former causes decreased libido or ejaculate volume, erectile…
- Trial name
- NCT01852487
- Testing
- 400mg /day during 6 months
- Where
- South Korea
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Dream Plus
Primary Ciliary Dyskinesia
Early Detection and Characterization of Primary Ciliary Dyskinesia
Primary Ciliary Dyskinesia (PCD) is a severe genetic disorder caused by various mutations in genes affecting ciliary motility. Various new and complementary diagnostic techniques, including measurements of nasal nitric oxide…
- Trial name
- NCT01070914
- Where
- Israel
- Volunteers
- 130 people
- Sponsor
- Ziv Hospital
Acquired Factor X Deficiency
A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis
The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants with acquired factor X deficiency associated with AL amyloidosis.
- Trial name
- NCT06963216
- Testing
- Coagadex(R)
- Sponsor
- Kedrion S.p.A.