Clinical trials: Multiple Congenital Anomaly
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00304512
- Testing
- migalastat HCl
- Where
- 6 sites in United States, Australia and 4 more
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
Uterine Fibroid, Arteriovenous Malformations
An Observational Study of Radial Access Embolization Procedures Using HydroPearl Microspheres
This registry study is designed to collect data on the procedural success and complication rates in real-world patients undergoing HydroPearl embolization procedures via radial access.
- Conditions
- Uterine Fibroid, Arteriovenous Malformations, Hypervascular Tumors, Benign Prostatic Hyperplasia
- Trial name
- RAVI Registry (NCT04272216)
- Testing
- Treatment with HydroPearl via radial access
- Where
- 6 sites in United States
- Volunteers
- 99 people
- Sponsor
- Terumo Medical Corporation
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
Ataxia, Cerebellar, Cerebellar Ataxia
Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia
Neurodegenerative cerebellar ataxias represent a group of disabling disorders for which we currently lack effective therapies. Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been…
- Conditions
- Ataxia, Cerebellar, Cerebellar Ataxia, Spinocerebellar Ataxias, Ataxia, Spinocerebellar
- Trial name
- CStDCSAtaxia (NCT03120013)
- Testing
- Anodal cerebellar and cathodal spinal tDCS
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Phenylketonuria
Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain reduced blood Phe concentrations in PKU subjects.
- Trial name
- NCT00924703
- Testing
- rAvPAL-PEG
- Where
- 14 sites in United States
- Volunteers
- 68 people
- Sponsor
- BioMarin Pharmaceutical
Autosomal Dominant Polycystic Kidney Disease
The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
This is a Phase 2, open-label, parallel-group, multiple dose study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and tolerability of multiple doses of lixivaptan in Autosomal Dominant Polycystic Kidney…
- Trial name
- NCT03487913
- Testing
- Lixivaptan
- Where
- 13 sites in United States
- Volunteers
- 31 people
- Sponsor
- Palladio Biosciences
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of the cognitive dysfunction shown by children with Down syndrome, aged 11 to 17.
- Trial name
- NCT00754052
- Testing
- Aricept (donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.
Chronic Hepatitis C Infection
Open-Labeled Study of PSI-7977 and RBV With and Without PEG-IFN in Treatment-Naïve Patients With HCV GT2 or GT3
This study is to assess the safety and tolerability of sofosbuvir (SOF) 400 mg with and without ribavirin (RBV) and/or with and without pegylated interferon alfa-2a (PEG) in subjects with genotype 1, 2 or 3 hepatitis C (HCV)…
- Trial name
- NCT01260350
- Testing
- SOF, RBV, PEG
- Where
- 2 sites in New Zealand
- Volunteers
- 292 people
- Sponsor
- Gilead Sciences
Physical Disability
A Community Wellness Program for Adults Living With Long-term Physical Disability
For people living with long-term physical disabilities, such as spinal cord injury or multiple sclerosis, middle-age (45-64) is a period of great vulnerability for losses in function and participation.
- Trial name
- EW-D (NCT03653390)
- Testing
- EnhanceWellness for Disability (EW-D), Wellness Education
- Where
- United States
- Volunteers
- 516 people
- Sponsor
- University of Washington
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire
Atopic Dermatitis
A Trial to Evaluate the Efficacy and Safety of Different Doses of LEO 138559 in Adults With Moderate-to-severe Atopic Dermatitis
The purpose of this trial is to test different doses of the trial medicine (LEO 138559) and see how well they work and how safe they are at treating moderate to severe atopic dermatitis in adults.
- Trial name
- NCT05923099
- Testing
- LEO 138559
- Where
- 73 sites in United States, Canada and 9 more
- Volunteers
- 262 people
- Sponsor
- LEO Pharma
Healthy
Study to Evaluate Safety, Tolerability and Pharmacokinetics of rhNGF Eye Drops in Healthy Volunteers
The primary objective of this study is to assess the safety and tolerability of single and multiple ascending doses of rhNGF when administered as eye drops in healthy subjects.
- Trial name
- NGF0112 (NCT01744704)
- Testing
- rhNGF 0.5 µg/mL Sentinel, rhNGF 5 µg/mL Sentinel, rhNGF 20 µg/mL Sentinel
- Where
- 2 sites in Switzerland, United Kingdom
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Dompé Farmaceutici S.p.A
Autosomal Dominant Polycystic Kidney, Hypertension
Effect of Beetroot Juice on Reducing Hypertension in Autosomal Dominant Polycystic Kidney Disease
People with autosomal dominant polycystic kidney disease (ADPKD) develop high blood pressure and kidney disease. Previous studies have shown that a commonly occurring chemical, nitric oxide (NO), is reduced in ADPKD, and may…
- Conditions
- Autosomal Dominant Polycystic Kidney, Hypertension, Endothelial Dysfunction
- Trial name
- BEET-PKD (NCT05401409)
- Testing
- Beetroot juice, Nitrate-depleted beetroot juice
- Where
- 2 sites in Australia
- Volunteers
- 60 people
- Sponsor
- Western Sydney Local Health District
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Acute Gout Flare
Study Utilizing Rilonacept in Gout Exacerbations
This purpose of this clinical research was to determine the efficacy and safety of an experimental drug called Rilonacept in participants with an acute gout attack. Participants participated in this study for 30 days.
- Trial name
- SURGE (NCT00855920)
- Testing
- Rilonacept, Indomethacin
- Where
- 44 sites in United States
- Volunteers
- 225 people
- Sponsor
- Regeneron Pharmaceuticals
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.
Weight Loss
Engaging Rural Men With Mobile Technologies for Weight Loss
Overweight and obese men in rural Northeast Nebraska are an unrepresented, at-risk group exhibiting rising rates of cardiovascular disease, poor access to preventive care, and a rural milieu that contributes to their sedentary…
- Trial name
- NCT03329079
- Testing
- Mobile Technology Plus, Mobile Technology
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University of Nebraska
Epidermolysis Bullosa
Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).
- Trial name
- NCT02014376
- Testing
- SD-101 dermal cream (3%), SD-101 Dermal Cream (6%), Vehicle (SD-101 0%)
- Where
- 7 sites in United States
- Volunteers
- 48 people
- Sponsor
- Scioderm, Inc.
Duchenne Muscular Dystrophy
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases…
- Trial name
- FOR-DMD (NCT01603407)
- Testing
- Prednisone, Deflazacort
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 196 people
- Sponsor
- University of Rochester