Clinical trials: ALD
Sickle Cell Disease, Thalassemia
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who…
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03367546
- Testing
- Blood and Marrow Transplant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Adrenoleukodystrophy
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II.
- Trial name
- NCT00004418
- Testing
- glyceryl trierucate/glyceryl trioleate
- Where
- 2 sites in United States
- Volunteers
- 126 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Infantile Refsum's Disease, Zellweger Syndrome
Study of Bile Acids in Patients With Peroxisomal Disorders
OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis. II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Bifunctional Enzyme Deficiency, Adrenoleukodystrophy
- Trial name
- NCT00004442
- Testing
- chenodeoxycholic acid, cholic acid, ursodiol
- Volunteers
- 25 people
- Sponsor
- University of Cincinnati
Steatotic Liver Disease, MASLD/MASH (Metabolic Dysfunction-Associated Steatotic Liver Disease / Metabolic Dysfunction-Associated Steatohepatitis)
Long-term Follow-up to Determine Outcome in Liver Disease (LOVE Study)
The rational to conduct the LOVE study builds on the lack of available data on outcomes in steatotic liver disease in well characterized patients over a time frame of several years.
- Conditions
- Steatotic Liver Disease, MASLD/MASH (Metabolic Dysfunction-Associated Steatotic Liver Disease / Metabolic Dysfunction-Associated Steatohepatitis), Steatotic Liver Disease of Mixed Origin (MetALD), ALD - Alcoholic Liver Disease
- Trial name
- LOVE (NCT06907563)
- Where
- 4 sites in France, Germany and 2 more
- Volunteers
- 30,000 people
- Sponsor
- Universität des Saarlandes
Liver Cirrhosis
A PROspective Faecal MIcrobiota tranSplantation Trial to Improve outcomEs in Patients With Cirrhosis
A feasibility trial called PROFIT has previously shown that FMT administered endoscopically into the jejunum in patients with cirrhosis is safe and feasible and have identified some potential mechanisms of action that warrant…
- Trial name
- PROMISE (NCT06461208)
- Testing
- Encapsulated FMT
- Where
- 23 sites in United Kingdom
- Volunteers
- 300 people
- Sponsor
- King's College London
Alcoholic Liver Disease
Quantitative MRI for Non-invasive Assessment of Severity of Alcoholic Liver Disease (ALD)
The current goal in the treatment of Alcoholic Liver Disease (ALD) is to manage ALD-associated complications as there are no disease-specific therapies.
- Trial name
- NCT03113929
- Testing
- MRI
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- The Cleveland Clinic
Adrenoleukodystrophy
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the adrenomyeloneuropathy (AMN) phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the…
- Trial name
- Advance (NCT03231878)
- Testing
- Leriglitazone
- Where
- 10 sites in United States, France and 6 more
- Volunteers
- 116 people
- Sponsor
- Minoryx Therapeutics, S.L.
Pulmonary Disease, Chronic Obstructive, Lung Diseases, Interstitial
BREATHE ALD: A Shared Decision-Making Intervention for Adults With Advanced Lung Disease
This study aims to: 1. Develop the BREATHE-ALD intervention for adults with Advanced Lung Disease, multiple chronic conditions, and palliative care needs and their caregivers using interviews with 10 advanced lung disease (ALD)…
- Trial name
- BREATHE-ALD (NCT04930666)
- Testing
- BRief intervention to Enhance Adherence to Treatment and HEalth advice in Advanced Lung Disease
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Columbia University
Self-Efficacy, Health Literacy
Health Literacy and Self-efficacy Expectations in Patients With Alcohol-related Liver Cirrhosis
The goal of this observational study is to quantify the health literacy and self-efficacy in people with alcohol-related liver cirrhosis.
- Conditions
- Self-Efficacy, Health Literacy, Liver Cirrhosis, ALD - Alcoholic Liver Disease
- Trial name
- GESA (NCT07169526)
- Where
- Germany
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Hannover Medical School
Leukemia, Myeloid, Acute, Leukemia, B-cell
Precision Exercise in Children With Malignant Hemopathies
In the early years of life and during adolescence, physical activity is crucial for good development of motor skills. It is even more so for those children and young people who are forced to undergo anti-cancer therapies and…
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin
- Trial name
- SportTherapy (NCT04090268)
- Testing
- Sport Therapy
- Where
- Italy
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Alcohol-related Liver Disease, Liver Diseases
Enhancing Alcohol Treatment Engagement in Associated Liver Disease (ALD) Patients
The purpose of this trial is to see if providing patients with alcohol-related liver disease with tailored alcohol use treatment options will increase engagement with treatment and correct possible misconceptions.
- Trial name
- ENGAGE-ALD (NCT06269510)
- Testing
- ENGAGE-ALD Application (APP), Treatment Facilitation bundle, Enhanced Usual Care
- Where
- United States
- Volunteers
- 268 people
- Sponsor
- Henry Ford Health System
Adrenoleukodystrophy
Early Diagnosis Of Childhood Cerebral ALD
The goal of this single institution study is to evaluate boys with adrenoleukodystrophy (ALD) diagnosed early in life, and to prospectively monitor them to determine parameters that will facilitate earlier detection of the…
- Trial name
- NCT02948062
- Where
- United States
- Sponsor
- Masonic Cancer Center, University of Minnesota
ALD - Alcoholic Liver Disease
Comparison of ALD, NASH, and Healthy Control Patients
The availability of biological samples from individuals with alcoholic liver disease (ALD), as well as samples from appropriate heavy drinking, yet healthy controls and non-drinking healthy controls, is an essential first step in…
- Trial name
- NCT03224949
- Testing
- Blood draw
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- The Cleveland Clinic
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Peroxisome Biogenesis Disorders
Betaine and Peroxisome Biogenesis Disorders
The PBD are a rare group of inherited disorders due to the failure to form functional cellular peroxisomes. Most patients have progressive hearing and visual loss, leading to deafness and blindness, as well as neurological…
- Trial name
- NCT01838941
- Testing
- Betaine
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Alcohol Use Disorder, Alcohol-associated Liver Disease
Pomegranate Dietary Supplements in AUD and ALD
The goal of this project is to determine an individual's ability to generate active gut microbial metabolites called urolithins upon consumption of pomegranate dietary supplements.
- Conditions
- Alcohol Use Disorder, Alcohol-associated Liver Disease, Alcoholic Cirrhosis, Healthy
- Trial name
- NCT07678567
- Testing
- Pomegranate Dietary Ssupplement
- Where
- United States
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- University of Louisville
Healthy
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers
A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers.
- Trial name
- NCT03500627
- Testing
- OP-101
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Orpheris, Inc.
Non-alcoholic Fatty Liver Disease (NAFLD), Alcoholic Liver Disease (ALD)
Screening in Primary Care of Advanced Liver Fibrosis in NAFLD and/or Alcoholic Patients
The primary objective of the SOPRANO study is to compare two blood fibrosis tests, the eLIFT and the FibroMeter, for the screening of advanced liver fibrosis in patients with NAFLD and/or ALD from primary care centers.
- Conditions
- Non-alcoholic Fatty Liver Disease (NAFLD), Alcoholic Liver Disease (ALD), Liver Fibrosis
- Trial name
- SOPRANO (NCT05699018)
- Testing
- eLIFT
- Where
- 13 sites in France
- Volunteers
- 1,788 people
- Sponsor
- University Hospital, Angers
Vitrectomy, Cataract
Objective Measurements of the Opacification of the Lens After Vitrectomy
Cataract is the opacification of the lens. Usually cataract occurs slowly, causing progressive vision loss over several months or years.
- Trial name
- MOCVIT (NCT04566237)
- Testing
- Objective Scatter Index (OSI), Average Lens Density (ALD)
- Where
- France
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Metachromatic Leukodystrophy, Adrenoleukodystrophy
Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Evaluating the safety and efficacy of Lentiviral Hematopoietic Stem Cell Gene Therapy for advanced stage of Metachromatic Leukodystrophy and adrenoleukodystrophy.
- Trial name
- NCT02559830
- Testing
- transduced CD34+ hematopoietic stem cell
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Shenzhen Second People's Hospital