Clinical trials: Fat Disorder
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Parenteral Nutrition-Associated Liver Disease
Low Dose Parenteral Fat for Prevention of Parenteral Nutrition Associated Cholestasis in Preterm Neonates
The goal of the study is to determine if parenteral nutrition-associated cholestasis (PNAC) is related to the amount of parenteral (intravenous) fat administered to premature babies until full enteral nutrition is achieved.
- Trial name
- NCT01050660
- Testing
- Intravenous fat emulsion, Restriction of intravenous fat emulsion to 1 gm/kg/d
- Where
- United States
- Volunteers
- 136 people
- Sponsor
- Yale University
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Hypertriglyceridemia, Pediatric Obesity
Effect of Omega-3 Fatty Acids Supplementation on Hypertriglyceridemia in Pediatric Patients With Obesity.
The primary objective was to evaluate the safety and efficacy the 3 grams per day of omega-3 in adolescents with obesity and hypertriglyceridemia ( ≥ 150 mg/dl and ≤ 1000 mg/dl) for 12 weeks, compared with placebo.
- Trial name
- NCT03216057
- Testing
- Omega-3 fatty acid 3 grams per day
- Volunteers
- 168 people
- Sponsor
- Hospital Infantil de Mexico Federico Gomez
Gestational Diabetes, Hyperlipidemia
Lipid Metabolism in Gestational Diabetes
The purpose of this pilot study is to determine if, during pregnancy, women with GDM have an exaggerated lipid response compared to non-gestational diabetics.
- Trial name
- NCT00534105
- Testing
- Total Cholesterol, Triglyceride, HDL
- Where
- United States
- Volunteers
- 106 people
- Sponsor
- Baystate Medical Center
Cardiovascular Disease (CVD), Hypertension
Veteran Peer Coaches Optimizing and Advancing Cardiac Health
The purpose of this study is to test if having a Veteran peer health coach will improve blood pressure control among Veterans with high blood pressure and at least one other Cardiovascular disease (CVD) risk factor.
- Conditions
- Cardiovascular Disease (CVD), Hypertension, Hyperlipemia, Obesity
- Trial name
- Vet-COACH (NCT02697422)
- Testing
- Community-based peer health coach intervention
- Where
- United States
- Volunteers
- 264 people
- Sponsor
- VA Office of Research and Development
Niemann-Pick Disease, Type C
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
- Trial name
- NCT03759639
- Testing
- IB1001
- Where
- 9 sites in United States, Germany and 3 more
- Volunteers
- 33 people
- Sponsor
- IntraBio Inc
Cholestasis
Compassionate Use of Omegaven to Reverse Parenteral Nutrition Induced Cholestasis
The purpose of this research study is to see if giving Omegaven (an intravenous fat emulsion containing fish oil) instead of the current lipid emulsion, which contains fat derived from soybeans, as part of your child's…
- Trial name
- NCT01173159
- Testing
- Omegaven
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Lipomatosis, Multiple Symmetrical
Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
Participants (homozygous MFN2 [gene that provides instructions to produce the Mitofusin 2 protein] R707W) will be treated with Metreleptin, and effects on body composition, metabolic parameters and safety will be assessed over a…
- Trial name
- NCT05351164
- Testing
- Metreleptin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Michigan
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Obesity, Impaired Glucose Tolerance
Effects of Rosiglitazone on the Metabolic Phenotype of Impaired Glucose Tolerance in Youth
The purpose of the study is to determine whether treatment of children and adolescents with Impaired Glucose Tolerance (IGT) with rosiglitazone will lead to improvements in insulin sensitivity and glucose tolerance.
- Conditions
- Obesity, Impaired Glucose Tolerance, Type 2 Diabetes Mellitus
- Trial name
- NCT00413335
- Testing
- Rosiglitazone
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Yale University
Cerebrotendinous Xanthomatosis (CTX)
Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Iiopathic bilateral cataracts occur in many children with Cerebrotendinous Xanthomatosis (CTX) so the opportunity to use contact with health care providers in connection with pediatric cataract evaluation and treatment is a…
- Trial name
- NCT02638220
- Where
- 44 sites in United States
- Volunteers
- 442 people
- Sponsor
- Travere Therapeutics, Inc.
Smith-Lemli-Opitz Syndrome
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
The purpose of this study is to determine whether supplementation with an oil-based cholesterol suspension will correct the biochemical abnormalities in cholesterol and its precursors in individuals with the Smith-Lemli-Opitz…
- Trial name
- NCT00272844
- Testing
- crystalline cholesterol oil-based suspension
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Boston Children's Hospital
Hypercholesterolemia
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Primary Objective: To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment…
- Trial name
- NCT03510715
- Testing
- Alirocumab SAR236553 (REGN727), Atorvastatin, Simvastatin
- Where
- 10 sites in Brazil, Canada and 8 more
- Volunteers
- 18 people
- Sponsor
- Sanofi
Sarcoma
Safety and Efficacy Study of Torisel and Liposomal Doxorubicin for Patients With Recurrent Sarcoma
The purpose of this study is to identify a safe dosing regimen for the combination of Torisel and liposomal doxorubicin in patients with recurrent sarcoma.
- Trial name
- NCT00949325
- Testing
- temsirolimus plus liposomal doxorubicin
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Non-Alcoholic Fatty Liver Disease, Non-Alcoholic Steatohepatitis
Pediatric Liver Fat Quantification (LFQ) Phase 2 Pilot Study
This clinical study will involve performing a series of medical imaging procedures of the abdomen using both ultrasound and MRI modalities in subjects at risk for or already diagnosed with Nonalcoholic Fatty Liver Disease…
- Trial name
- NCT04800094
- Testing
- Investigational Liver Fat Quantification Software
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Philips Clinical & Medical Affairs Global
Metastatic Myxoid Liposarcoma, Metastatic Round Cell Liposarcoma
Testing the Combination of Two Experimental Drugs MK-3475 (Pembrolizumab) and Interferon-gamma for the Treatment of Mycosis Fungoides and Sézary Syndrome and Advanced Synovial Sarcoma
This phase II trial studies how well pembrolizumab and interferon gamma-1b work in treating patients with stage IB-IVB mycosis fungoides and Sezary syndrome that has come back (relapsed) or has not responded to previous treatment…
- Conditions
- Metastatic Myxoid Liposarcoma, Metastatic Round Cell Liposarcoma, Metastatic Synovial Sarcoma, Recurrent Mycosis Fungoides and Sezary Syndrome
- Trial name
- NCT03063632
- Testing
- Interferon Gamma-1b, Laboratory Biomarker Analysis, Pembrolizumab
- Where
- 7 sites in United States
- Volunteers
- 28 people
- Sponsor
- National Cancer Institute (NCI)
HIV Infections, Lipodystrophy
Therapeutic Approaches to HAART-Induced Lipodystrophy
To determine the efficacy and safety of 4 therapeutic interventions on HAART-Induced lipodystrophy. The interventions are: 1) Dietary - the effect of a high carbohydrate vs.a high cis-monounsaturated fatty acid diet.
- Trial name
- NCT00461552
- Testing
- Leptin
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- University of Texas Southwestern Medical Center