Clinical trials: Brain Diseases, Metabolic
Type 1 Gaucher Disease
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth.
- Trial name
- NCT00875160
- Testing
- AT2101
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 1 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Care Pathway for Patients With Fabry's Disease (Fabry-PATH)
Fabry's disease is an X-linked disorder that manifests differently in men and women, leading to differences in healthcare pathways.
- Trial name
- Fabry-PATH (NCT07485660)
- Testing
- Self-questionnaire
- Where
- France
- Volunteers
- 200 people
- Sponsor
- University Hospital, Toulouse
Gaucher Disease
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from…
- Trial name
- NCT03625882
- Where
- 35 sites in Japan
- Volunteers
- 63 people
- Sponsor
- Takeda
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Adrenomyeloneuropathy, Adrenoleukodystrophy
A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of Lorenzo's oil.
- Trial name
- NCT00545597
- Testing
- Lorenzo's oil
- Where
- United States
- Volunteers
- 240 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Fabry Disease, Anderson Fabry Disease
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
In the shared decision-making process, patients should express their expectations and preferences regarding treatment to the physician.
- Trial name
- NCT04043273
- Testing
- Noninterventional characterization of patients expectations and preferences regarding their treatment
- Where
- 15 sites in France
- Volunteers
- 69 people
- Sponsor
- Amicus Therapeutics France SAS
Phenylketonuria
The Effectiveness of Kuvan in Amish PKU Patients
The purpose of this study is to determine if Amish patients with PKU show responsiveness after a high dose, prolonged Saproterin trial.
- Trial name
- NCT02677870
- Testing
- saproterin dihydrochloride, Diet treatment
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University Hospitals Cleveland Medical Center
Ischemic Stroke, Atherosclerosis of Artery
Efficacy of Probucol Combined with Statins Treatment for Ischemic Stroke
The goal of this clinical trial is to evaluate whether the combination of Probucol with statin therapy can reduce the risk of vascular events and improve atherosclerosis outcomes in adults with ischemic stroke and confirmed…
- Conditions
- Ischemic Stroke, Atherosclerosis of Artery, Lipid Disorder, Statin
- Trial name
- EPCIS (NCT06604117)
- Testing
- Statin, Probucol
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Xuanwu Hospital, Beijing
Insulin Resistance, Transcranial Direct Current Stimulation
Improving Insulin Sensitivity by Non-invasive Brain Stimulation
Efforts in curing and preventing obesity and type 2 diabetes (T2D) have been elusive thus far. One reason for that is the lack of understanding of the role of the brain in the development and treatment of the disease.
- Conditions
- Insulin Resistance, Transcranial Direct Current Stimulation, Obesity, Diabetes
- Trial name
- NCT04420650
- Testing
- transcranial direct current stimulation
- Where
- Germany
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen
Cancers
Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)
The purpose of this study is to safely remove ovarian tissue in pre-pubertal pediatric patients, who are at risk for infertility from their medical treatment, for freezing for future restoration of fertility and hormone function.
- Trial name
- NCT05299710
- Testing
- Laparoscopic surgery
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- Erin Rowell
Respiratory Insufficiency, Pulmonary Disease, Chronic Obstructive
The Effect of Aromatherapy Massage on Pain, Anxiety and Comfort
Patients in the intensive care unit (ICU) experience physical and psychological discomfort, including pain. Anxiety is a condition frequently encountered in the ICU.
- Conditions
- Respiratory Insufficiency, Pulmonary Disease, Chronic Obstructive, Diabetes Mellitus, Type 2, High Blood Pressure
- Trial name
- NCT06295874
- Testing
- Effect of aromatherapy massage on vital signs, pain, anxiety and comfort level
- Where
- Turkey (Türkiye)
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Nilgün Erdoğan
Glycogen Storage Disease Type II
High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease
The research protocol will be submitted for approval to the institutional review board of Columbia University Medical Center. An attempt will be made to recruit at least 6 juvenile patients between the ages of 8 and 17…
- Trial name
- NCT01656590
- Testing
- High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
- Where
- United States
- Sponsor
- Columbia University
Phenylketonuria (PKU)
Prevalence and Determinants of Obesity in PKU Patients
This observational cross-sectional multicenter study aims to assess the prevalence of overweight and obesity in adult patients with phenylketonuria (PKU) and to identify associated risk factors.
- Trial name
- NCT07703020
- Where
- Switzerland
- Volunteers
- 100 people
- Sponsor
- Christel Tran
Acromegaly
Effects of Sandostatin LAR® in Acromegaly
This study aims primarily to determine the effect Insulin-like Growth Factor 1 (IGF-1) normalization into current IGF-I normal ranges with Sandostatin LAR® therapy on biochemical metabolic, cardiovascular and body composition…
- Trial name
- NCT01424241
- Testing
- Sandostatin LAR
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Columbia University
HIV Infections, Hypertension
ENHANCE-EvideNce Led Co-created HeAlth Systems interventioNs for MLTCs CarE
The goal of this study is to determine the effect of the ENHANCE intervention in improving clinical outcomes and evaluating the effects of the intervention on implementation processes and outcomes.
- Conditions
- HIV Infections, Hypertension, Diabetes Mellitus, Asthma
- Trial name
- ENHANCE (NCT06248190)
- Testing
- ENHANCE intervention (health systems)
- Where
- 32 sites in South Africa
- Volunteers
- 1,837 people
- Sponsor
- University of KwaZulu
Fabry Disease
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients.
- Trial name
- LDN6702 (NCT01581424)
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- University of Minnesota
Epilepsy; Seizure, Neuromuscular Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
The "North Carolina Clinical Genomic Evaluation by Next-gen Exome Sequencing, 2 (NCGENES 2)" study is part of a larger consortium project investigating the clinical utility, or net benefit of an intervention on patient and family…
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability
- Trial name
- NCGENES2 (NCT03548779)
- Testing
- Pre-visit prep, usual care + exome seq
- Where
- 3 sites in United States
- Volunteers
- 548 people
- Sponsor
- University of North Carolina, Chapel Hill
Krabbe Disease
Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.
- Trial name
- NCT06308718
- Testing
- FBX-101
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Duke University
Cerebral Amyloid Angiopathy
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
The purpose of the study is to evaluate the effect of ALN-APP on measures of CAA disease progression and to characterize the safety, tolerability, and pharmacodynamics (PD) of ALN-APP in adult patients with sporadic CAA (sCAA)…
- Trial name
- cAPPricorn-1 (NCT06393712)
- Testing
- ALN-APP
- Where
- 57 sites in United States, Australia and 4 more
- Volunteers
- 210 people
- Sponsor
- Alnylam Pharmaceuticals
Friedreich's Ataxia
Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept
Friedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extremely severe disease.
- Trial name
- ACTFRIE (NCT00811681)
- Testing
- pioglitazone
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.