Clinical trials: Storage Disease
Adult Immunoblastic Lymphoma, Central Nervous System Non-Hodgkin Lymphoma
Collecting and Storing Tissue Samples From Patients With Rare or Cutaneous Non-Hodgkin Lymphoma
This research study is collecting and storing tissue samples from patients with rare or cutaneous non-Hodgkin lymphoma. Collecting and storing samples of tissue from patients with cancer to test in the laboratory may help the…
- Conditions
- Adult Immunoblastic Lymphoma, Central Nervous System Non-Hodgkin Lymphoma, Childhood Immunoblastic Lymphoma, Grade 1 Follicular Lymphoma
- Trial name
- NCT01000753
- Testing
- Cytology Specimen Collection Procedure
- Where
- 130 sites in United States, Australia and 2 more
- Volunteers
- 174 people
- Sponsor
- Children's Oncology Group
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant.
- Trial name
- NCT05634512
- Testing
- Laronidase therapy and a stem cell transplant
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Gaucher Disease, Fabry Disease
Therapeutic Exercise to Treat Neuropathic Pain
A quasi-experimental, prospective clinical trial with pre and post intervention measurements, whose porpuose is assess the efficacy of a therapeutic exercise protocol to treat neuropathic pain in Fabry Disease.
- Trial name
- Aps (NCT04050137)
- Testing
- Therapeutic Exercise
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Universidad de Zaragoza
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo…
- Trial name
- NCT02053064
- Testing
- SAF-301
- Where
- France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
Fabry Disease
FASHION Fabry Disease Hypertrophic Cardiomyopathy and Infammation
In Fabry disease (FD) and hypertrophic cardiomyopathy (HCM) systemic inflammation recently gained attention as a possible key pathophysiologic process involved in the development of cardiac hypertrophy and progression of the…
- Trial name
- FASHION (NCT05761834)
- Testing
- Sample blood and 2D-echocardiography with Doppler
- Where
- Italy
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
IBS (Irritable Bowel Syndrome), Bloating
A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating
This study is a randomized, double-blind, placebo-controlled, within-individual crossover trial designed to assess the impact of regular use of a consumer-grade FODMAP-targeting digestive enzyme blend (FODZYME®) on…
- Conditions
- IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
- Trial name
- NCT07591584
- Testing
- FODMAP-targeting digestive enzyme blend
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Kiwi Health Inc
Melanoma
A Storage Facility for Tissues Obtained From Patients With Malignant Melanoma
This study collects and stores blood and tumor samples from patients with malignant melanoma and healthy individuals. The purpose of this study is to gain a better understanding of the causes of melanoma and how melanoma tumors…
- Trial name
- NCT04567706
- Testing
- Biospecimen Collection
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Ohio State University Comprehensive Cancer Center
Gaucher Disease, Acid SphingoMyelinase Deficiency
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and oncohematology clinics, and adult internal medicine and hematology.
- Trial name
- NCT05992532
- Where
- 23 sites in Spain
- Volunteers
- 210 people
- Sponsor
- Fundación Española de Hematología y Hemoterapía
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all…
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Trial name
- NIFAMA (NCT05284006)
- Testing
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Nemours Children's Clinic
Mucopolysaccharidosis Type VI
Gene Therapy in Patients With Mucopolysaccharidosis Disease
This study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B (ARSB) enzyme.
- Trial name
- NCT03173521
- Testing
- AAV2/8.TBG.hARSB
- Where
- 2 sites in Italy, Turkey (Türkiye)
- Volunteers
- 9 people
- Sponsor
- Fondazione Telethon
Parkinson Disease, Gaucher Disease
Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism
The purpose of this study is to determine if participants have changes in dopamine cells in their brain using DaTSCAN™ brain imaging.
- Conditions
- Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
- Trial name
- NCT01358474
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Sanfilippo Syndrome, MPS3
The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
The natural history study of patients with Sanfilippo disease(s) (MPS3)
- Trial name
- NCT05705674
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Gaucher Disease, Cerebroside Lipidosis Syndrome
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status.
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00358943
- Where
- 317 sites in United States, Argentina and 50 more
- Volunteers
- 12,000 people
- Sponsor
- Genzyme, a Sanofi Company
Hurler Syndrome, Cerebral Palsy
Effects of Growth Hormone in Chronically Ill Children
The specific aims for this study are - 1. To determine the effect of GH on height, height velocity, body weight and lean body mass.
- Conditions
- Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease
- Trial name
- NCT00286689
- Testing
- Growth Hormone, Whole body Protein turnover, DEXA scan
- Where
- United States
- Sponsor
- University of Texas Southwestern Medical Center
Kidney Transplant; Complications, Kidney Failure
Normothermic Machine Perfusion (NMP) Versus Static Cold Storage (SCS) in Human Kidney Transplantation
Due to organ shortage in kidney transplantation (KT) several strategies have been implemented in an attempt to increase donor pool utilization, including transplantation of extended criteria donor (ECD) allografts.
- Conditions
- Kidney Transplant; Complications, Kidney Failure, Graft Dysfunction
- Trial name
- NMP-DBD (NCT05031052)
- Testing
- End-ischemic normothermic oxygenated machine perfusion, Static cold storage
- Where
- 4 sites in Germany
- Volunteers
- 194 people
- Sponsor
- Charite University, Berlin, Germany
Iron Deficiency Anemia of Pregnancy, Iron Storage Disease
Analysis of the Effect of Iron Supplements on Iron Deficiency Anemia in Pregnancy
Subjects were tested for hemoglobin, ferritin, serum iron, transferrin saturation and reticulocyte count during routine prenatal examination at 24-26 weeks of gestation, and blood samples were taken for serum hepcidin detection…
- Trial name
- NCT05804071
- Testing
- Niferex
- Where
- China
- Volunteers
- 452 people, healthy volunteers welcome
- Sponsor
- Qianfoshan Hospital
Gaucher Disease, Type 1
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework…
- Trial name
- NCT04120506
- Testing
- VPRIV
- Volunteers
- 15 people
- Sponsor
- Shaare Zedek Medical Center
Fabry Disease
Safety Study of Replagal® Therapy in Children With Fabry Disease
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
- Trial name
- NCT01363492
- Testing
- Replagal (agalsidase alfa)
- Where
- 5 sites in United States
- Volunteers
- 15 people
- Sponsor
- Shire
Gaucher Disease Type 3
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
- Trial name
- NCT07715084
- Testing
- AZ-3102
- Where
- 7 sites in India, Pakistan and 1 more
- Volunteers
- 20 people
- Sponsor
- Azafaros B.V.
Mucopolysaccharidosis (MPS), Hunter Syndrome
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Hunter syndrome (Mucopolysaccharidosis II, [MPS II]) is a rare, genetically linked lysosomal storage disease (LSD) caused by deficiency of the enzyme, iduronate-2-sulfatase (I2S).
- Trial name
- NCT01822184
- Where
- 7 sites in United States, Argentina and 3 more
- Volunteers
- 100 people
- Sponsor
- Shire
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