Clinical trials: Multiple Anomalies
Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
- Trial name
- FLY (NCT06328608)
- Testing
- PRX-102 1 mg/kg every two weeks
- Where
- 12 sites in United States, Austria and 4 more
- Volunteers
- 22 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Sepsis Newborn, Sepsis
A Multi-center Quasi-experimental Study in Northern Italy to Evaluate the Impact of Sepsis Bundle in Obstetric Settings: the SOS Study
Despite advances in the diagnosis and treatment of sepsis, the studies available in the literature report very high maternal and fetal/neonatal mortality and morbidity in cases of sepsis occurring during pregnancy or the…
- Trial name
- NCT07375108
- Testing
- microbiological results from blood cultures or other materials.
- Where
- Italy
- Volunteers
- 52 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Gastroschisis
Multi-Centre Gastroschisis Interventional Study Across Sub-Saharan Africa
This study is a multi-centre interventional study at seven tertiary paediatric surgery centres in Ghana, Zambia, Malawi and Tanzania aimed at reducing mortality from gastroschisis.
- Trial name
- NCT03724214
- Testing
- Pre-hospital interventional care bundle, In-hospital interventional care bundle
- Where
- 9 sites in Canada, Ghana and 4 more
- Volunteers
- 235 people
- Sponsor
- King's College London
Primary Immunodeficiency Disease
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study.
- Trial name
- NCT04842643
- Testing
- IGSC 20% infusion
- Where
- 8 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Takeda
Huntington Disease
IMarkHD: in Vivo Longitudinal Imaging of HD Pathology
iMarkHD is an adaptive, longitudinal positron emission tomography (PET) and magnetic resonance (MR) imaging study in Huntington's disease (HD) that aims to assess abnormal molecular, functional, and structural changes in…
- Trial name
- NCT03434548
- Testing
- PET imaging, Multi-modal MRI imaging
- Where
- United Kingdom
- Volunteers
- 113 people, healthy volunteers welcome
- Sponsor
- King's College London
Retinal Diseases, Stargardt's Disease
Microcurrent Stimulation to Treat Macular Degeneration
Microcurrent stimulation thereapy improves retinal efficiency and may restore and/or improve retinal function.
- Conditions
- Retinal Diseases, Stargardt's Disease, Retinitis Pigmentosa
- Trial name
- NCT01790958
- Where
- 4 sites in United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Retina Institute of Hawaii
Turner Syndrome, Bicuspid Aortic Valve
Aortopathy in Persons With Bicuspid Aortic Valve, Turner and Marfan Syndrome
The study aim is: 1. To examine aortic tissue by light microscopy 2. To examine aortic tissue by electron microscopy 3. To study changes in the epigenome and transcriptome of the X chromosome specific to aortic tissue. 4.
- Conditions
- Turner Syndrome, Bicuspid Aortic Valve, Marfan Syndrome
- Trial name
- NCT01760668
- Where
- Denmark
- Volunteers
- 5 people
- Sponsor
- University of Aarhus
Stargardt's Macular Dystrophy
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
- Trial name
- NCT02941991
- Testing
- hESC-RPE
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Atopic Dermatitis
A Phase 2 Study of CIM331 for Atopic Dermatitis Patients
To assess the safety, tolerability and efficacy of CIM331, compared to placebo, in atopic dermatitis patients who are inadequately controlled by or intolerant to topical therapy
- Trial name
- NCT01986933
- Testing
- nemolizumab (CIM331)
- Where
- 18 sites in United States
- Volunteers
- 264 people
- Sponsor
- Chugai Pharmaceutical
Pulmonary Hypertension, Familial Persistent, of the Newborn
A Study To Evaluate Safety And Efficacy Of IV Sildenafil In The Treatment Of Neonates With Persistent Pulmonary Hypertension Of The Newborn
This study will evaluate whether IV sildenafil can reduce the time on inhaled nitric oxide treatment and reduce the failure rate of available treatments for persistent pulmonary hypertension of the newborn.
- Trial name
- NCT01720524
- Testing
- iv sildenafil
- Where
- 42 sites in United States, Belgium and 10 more
- Volunteers
- 59 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Dermatitis, Atopic
A Phase 1 Study of KHK4083 in Subjects With Atopic Dermatitis
The objective of this study is to evaluate the safety and tolerability of multiple intravenous (IV) infusions of KHK4083 in subjects with moderate or severe atopic dermatitis.
- Trial name
- NCT03096223
- Testing
- KHK4083
- Where
- Japan
- Volunteers
- 26 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Pompe Disease
Drug-drug Interaction Study
This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe Disease.
- Trial name
- NCT01380743
- Testing
- duvoglustat, rhGAA
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- Amicus Therapeutics
Basal Cell Carcinoma, Keratosis Palmaris et Plantaris
The Evaluation of Oral Acitretin in the Treatment of Psoriasis, Cutaneous Disorders of Keratinization, Multiple Basal Cell Carcinomas and Other Retinoid Responsive Diseases
This is a continuing study which evaluates the long-term safety and efficacy of oral acitretin in an open manner in the treatment of psoriasis, cutaneous disorders of keratinization, multiple basal cell carcinomas and other…
- Conditions
- Basal Cell Carcinoma, Keratosis Palmaris et Plantaris, Psoriasis
- Trial name
- NCT00005660
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Cancer Institute (NCI)
Medullary Thyroid Carcinoma, Multiple Endocrine Neoplasia Type 2
Study of Molecular Pathways in Medullary Thyroid Carcinoma and Correlation of Molecular Data With Clinical Behavior of the MTC in Individuals Patients
Background: Medullary thyroid carcinoma (MTC) is a rare malignancy, occurring either as a sporadic disease (75% of cases), or in a hereditary pattern as multiple endocrine neoplasia (MEN) type 2 (MEN2A or MEN2B) or familial…
- Trial name
- NCT01424878
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Cancer Institute (NCI)
Atopic Dermatitis
Bioequivalence of Two Tacrolimus 0.1% Topical Ointment Formulations in Patients With Atopic Dermatitis
The primary objectives are to establish the therapeutic equivalence of tacrolimus ointment 0.1%, manufactured by Taro Pharmaceuticals Inc.
- Trial name
- NCT00833079
- Testing
- Tacrolimus 0.1% manufactured by Taro, Protopic - Tacrolimus 0.1%, Tacrolimus Vehicle manufactured by Taro
- Volunteers
- 500 people
- Sponsor
- Sun Pharmaceutical Industries, Inc.
Healthy Volunteer, Urea Cycle Disorder
Safety and Tolerability of SYNB1020-CP-001
A Phase 1, First-in-human, Oral Single and Multiple Dose-Escalation, Randomized, Double-blinded, Placebo-controlled Study of SYNB1020 in Healthy Adult Volunteers to Evaluate Safety, Tolerability, Dosing, and Pharmacodynamics
- Trial name
- SYNB1020CP001 (NCT03179878)
- Testing
- SYNB1020
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Synlogic
Polycystic Kidney Disease
Early PKD Observational Cohort Study
This observational study will collect blood and urine and clinical information from individuals with early-stages of polycystic kidney disease (PKD), their unaffected siblings and normal volunteers to create a biobank, also…
- Trial name
- EPOC (NCT02936791)
- Where
- 3 sites in United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Retinitis Pigmentosa, Usher Syndromes
Prospective Open Clinical and Genetic Study of Patients With Retinitis Pigmentosa
This study is aimed to characterize Russian population of Retinitis Pigmentosa
- Trial name
- RU-RP (NCT03901391)
- Testing
- Whole Exome Sequencing
- Where
- 2 sites in Russia
- Volunteers
- 130 people
- Sponsor
- Sensor Technology for Deafblind
Respiratory Tract Neoplasms, Thymic Neoplasms
High Dose Somatostatin Analogues in Neuroendocrine Tumors
Octreotide (OCT) is a somatostatin analogue (SSA) available in a long-acting formulation, conventionally administered every 28 days at the maximum dose of 30 mg.
- Conditions
- Respiratory Tract Neoplasms, Thymic Neoplasms, Pancreatic Neoplasms, Gastrointestinal Neoplasms
- Trial name
- HIDONET (NCT00990535)
- Testing
- Octreotide-LAR
- Where
- Italy
- Volunteers
- 28 people
- Sponsor
- Federico II University