Clinical trials: Metabolic Evaluation
Myelofibrosis, Gaucher Disease
Clinical and Pathophysiological Investigations Into Erdheim Chester Disease
Background: Erdheim Chester Disease (ECD) is a very rare disease in which abnormal white blood cells start growing and affect the bones, kidneys, skin, and brain.
- Conditions
- Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS)
- Trial name
- NCT01417520
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Malnutrition, Child
Study of Malnutrition in Primary School Children
Study of malnutrition in primary school age children aim to asses the prevelance of malnutrition amoung the children in this age group and also ldintified the factors that affect in malnutrition between the children and the study…
- Trial name
- NCT04810468
- Testing
- Anthropometric measurment
- Volunteers
- 440 people, healthy volunteers welcome
- Sponsor
- Sohag University
Child Obesity
Childhood Obesity Risk Assessment Longitudinal Study
Childhood obesity is a major public health problem worldwide. Europe has a high prevalence of obesity, which is accentuated in Mediterranean countries.
- Trial name
- CORALS (NCT06317883)
- Where
- 8 sites in Spain
- Volunteers
- 1,508 people, healthy volunteers welcome
- Sponsor
- Consorcio Centro de Investigación Biomédica en Red (CIBER)
Thalassemia, Brain Function (Measured by ERP Level)
A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.
β-thalassemia syndromes are a group of hereditary disorders characterized by a genetic deficiency in the synthesis of beta-globin chains.
- Trial name
- NCT01935661
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Trial name
- NCT03153319
- Testing
- Adalimumab Injection [Humira], Saline Solution for Injection
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
An Observational Study in Children With CLN2 Batten Disease
This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
- Trial name
- NCT04462692
- Sponsor
- REGENXBIO Inc.
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
HIV Infections, HIV Wasting Syndrome
A Study to Evaluate the Effect of Nandrolone Decanoate in Women With HIV-Associated Weight Loss
The purpose of this study is to see if giving nandrolone decanoate (a hormonal drug) will cause weight gain in HIV-positive women who have HIV-associated weight loss (wasting). Wasting has become an AIDS-defining condition.
- Trial name
- NCT00000854
- Testing
- Nandrolone decanoate
- Where
- 22 sites in United States, Puerto Rico
- Volunteers
- 38 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Malnutrition
Validation of the STAMP Screening Tool For Pediatric Nutritional Risk
Background: Insufficient nutritional intake, with or without concomitant morbidity, leads to weight loss or insufficient weight gain, is related to an increase in morbidity and mortality and exposes the child to medical…
- Trial name
- STAMP2 (NCT01718769)
- Testing
- questionnaire STAMP
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- Meir Medical Center
Undernutrition
Nutributter Programming to Prevent Undernutrition: an Evaluation
This randomized, controlled effectiveness study examines Nutributter programming within an integrated package of maternal and child health services offered by Haiti's Ministry of Health (MSPP) to prevent undernutrition among…
- Trial name
- NPPU (NCT01552512)
- Testing
- Nutributter Program, Integrated Package
- Where
- Haiti
- Volunteers
- 1,228 people
- Sponsor
- Washington University School of Medicine
Hypercholesterolemia
Efficacy of Lapaquistat Acetate Co-Administered With Current Lipid-Lowering Treatment on Blood Cholesterol Levels in Subjects With Homozygous Familial Hypercholesterolemia
The purpose of the study is to determine the efficacy of lapaquistat acetate, once daily (QD), to lower cholesterol in subjects with homozygous familial hypercholesterolemia undergoing lipid-lowering treatment.
- Trial name
- NCT00263081
- Testing
- Lapaquistat acetate and current lipid-lowering treatment, Current lipid-lowering treatment
- Where
- 10 sites in United States, Canada and 4 more
- Volunteers
- 44 people
- Sponsor
- Takeda
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Nephropathic Cystinosis
CYSTEA-BONE Clinical Study
Nephropathic Cystinosis (NC) is an orphan inherited autosomal recessive disease characterised as a generalized lysosomal storage disease due to a deficiency of the cystine lysosomal transport protein, cystinosin.
- Trial name
- CYSTEA-BONE (NCT03919981)
- Testing
- Blood sampling
- Where
- 13 sites in France, Germany and 2 more
- Volunteers
- 42 people
- Sponsor
- Hospices Civils de Lyon
Dyslipidemias
Phase 3 Trial to Evaluate the Efficacy and Safety of YYC506.
Phase 3 study to evaluate the effiacay and safety of YYC506
- Trial name
- Atorf-YOOrct (NCT04858308)
- Testing
- YYC506, Control
- Where
- 3 sites in South Korea
- Volunteers
- 554 people
- Sponsor
- Yooyoung Pharmaceutical Co., Ltd.
Type 1 Diabetes Mellitus
Group Education Curriculum for Older Teens With Type 1 Diabetes
SMART T1D is a research study that offers interactive diabetes education for teens with type 1 diabetes in peer groups led by diabetes nurse educators.
- Trial name
- SMART T1D (NCT03147274)
- Testing
- Intervention
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Boston Children's Hospital
Childhood Obesity
Evaluation of a Commercial Gaming Impact in a Childhood Obesity Intervention
Recently, researchers have been interested in the use of game play, mechanics and psychology for health-related engagement and behavior change.
- Trial name
- NCT01757925
- Testing
- weight managment program, Active Gaming
- Where
- 11 sites in United States
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- UnitedHealth Group
Severe Combined Immunodeficiency, Atypical
Generalized Neonatal Screening of Severe Combined Immunodeficiencies
Severe combined Immunodeficiencies ( SCID ) are a group of inherited diseases of the immune system by characterised profound abnormalities of T cell development .
- Trial name
- DEPISTREC (NCT02244450)
- Testing
- SCID screening
- Where
- 51 sites in France
- Volunteers
- 190,539 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Anderson-Fabry Disease
Functional Capacity in Anderson-Fabry Disease Patients
The goal of this observational study is to observe the relation between excercise parameters - assessed by CPET - and rest/stress hemodynamic parameters - assessed by echocardiogram and CMR - in patients with a genetic diagnosis…
- Trial name
- OPTIMA-FD (NCT06884358)
- Where
- 5 sites in Italy
- Volunteers
- 100 people
- Sponsor
- IRCCS Policlinico S. Donato
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
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