Clinical trials: Storage Disease
Diabetic Foot, Leg Ulcer
Obtaining and Storing Standard of Care Wound Biopsies for Immediate or Future Wound-Related Scientific Study
The objective of this study protocol is to use wound biopsies that have been obtained during standard of care management for further scientific evaluation after standard evaluation by a pathologist.
- Conditions
- Diabetic Foot, Leg Ulcer, Decubitus Ulcer
- Trial name
- NCT00759889
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Southwest Regional Wound Care Center
Pompe Disease
Alglucosidase Alfa Pompe Safety Sub-Registry
To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment…
- Trial name
- NCT01710813
- Testing
- alglucosidase alfa
- Where
- 18 sites in United States, Belgium and 4 more
- Volunteers
- 110 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis I, Hurler's Syndrome
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Trial name
- NCT00144768
- Testing
- laronidase
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Microbiota
Effects of Resistant Potato Starch on the Gut Microbiota
This was a randomized, double-blind, three-arm, parallel group clinical trial, which assessed the shifts that occur in microbial populations after four weeks of supplementation with resistant potato starch (SolnulTM) at two…
- Trial name
- NCT05242913
- Testing
- Resistant Potato Starch
- Where
- Canada
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- MSP Starch Products Inc.
Hermansky Pudlak Syndrome
A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis
Hermansky-Pudlak Syndrome (HPS) is a rare genetic disease that is associated with oculocutaneous albinism, bleeding, granulomatous colitis, and pulmonary fibrosis in some subtypes, including HPS-1, HPS-2, and HPS-4.
- Trial name
- NCT02368340
- Testing
- Pulmonary function test, Chest CT, Sample collection
- Where
- 5 sites in United States
- Volunteers
- 55 people
- Sponsor
- Vanderbilt University
Thrombocytopenia
Efficacy and Safety Study of Platelets Treated for Pathogen Inactivation and Stored for Up to Seven Days
Objective: To determine if platelets treated for pathogen inactivation and stored for 6 to 7 days are safe and effective compared to platelets collected by the same method, stored for the same amount of time and not treated for…
- Trial name
- TESSI (NCT00261924)
- Testing
- Transfusion of Pathogen Inactivated Platelets stored for 6-7 days
- Where
- United Kingdom
- Volunteers
- 211 people
- Sponsor
- Cerus Corporation
Neutral Lipid Storage Disease
The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)
Neutral Lipid Storage Disease With Myopath (NLSDM) is a disease caused by a defect in the PNPLA2 gene encoding ATGL. Patients with NLSDM accumulate triglycerides and exhibit muscle weakness, cardiac failure and hepatosteatosis.
- Trial name
- NLSDM (NCT01527318)
- Testing
- Fibrate treatment
- Where
- Netherlands
- Volunteers
- 6 people
- Sponsor
- Maastricht University Medical Center
McArdle Disease
A Study of the Safety of REN001 in Patients With McArdle Disease
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease
- Trial name
- NCT04226274
- Testing
- REN001
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 19 people
- Sponsor
- Reneo Pharma Ltd
Maroteaux-Lamy Syndrome
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic…
- Trial name
- NCT02156674
- Testing
- Naglazyme®
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Anxiety
Uncertainty and Firearms: Obtaining Secure Storage
The goal of this clinical trial is to test whether inclusion of cognitive behavioral therapy (CBT) for Uncertainty-Enhanced (CUE) with Lethal Means Counseling (LMC) is acceptable, feasible, and efficacious in addressing secure…
- Trial name
- UFOS (NCT05818696)
- Testing
- Lethal Means Counseling (LMC), CBT for Uncertainty - Enhanced (CUE)
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Gaucher's Disease, Osteopenia
Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
OBJECTIVES: I. Determine the efficacy of alendronate sodium in treating osteopenia (generalized bone density and focal bone lesions) in patients with Gaucher's disease.
- Trial name
- NCT00004488
- Testing
- alendronate sodium, calcium carbonate, cholecalciferol
- Where
- 3 sites in United States, Israel
- Volunteers
- 82 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Chronic Disease
Using "Store and Forward" Tele-health as an Adjunct to Traditional Outpatient or Home Health Physical Therapy
This study will take about 3-16 months. Participants will be inpatients in the Hennepin Health System that are transitioning into outpatient or home health Physical Therapy or current outpatient patients.
- Trial name
- TELE (NCT02659280)
- Testing
- Adjunct Therapy, Standard of Care
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Hennepin Healthcare Research Institute
Niemann-Pick Disease, Type C
Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with…
- Trial name
- NCT03687476
- Testing
- VTS-270
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Glycogen Storage Disease Type II
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to ERT with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769…
- Trial name
- NCT05164055
- Testing
- Avalglucosidase alfa (GZ402666)
- Where
- 11 sites in France
- Volunteers
- 17 people
- Sponsor
- Genzyme, a Sanofi Company
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
This is a prospective longitudinal natural history study with a retrospective cross-sectional arm aimed at determining the natural history of sphingosine phosphate lyase insufficiency syndrome (SPLIS), a recently recognized…
- Trial name
- SPLIS-HIS (NCT06669949)
- Testing
- no intervention
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of California, San Francisco
Parkinson Disease
Dopaminergic restauratIon by intraVEntriculaire Administration
Prospective monocentric randomized controlled open-label proof-of-concept study in cross-over of two 1-month periods and a long-term follow-up period not to exceed September 30, 2023, with 2 groups: Intracerebroventricular…
- Trial name
- DIVE (NCT04332276)
- Testing
- A-dopamine, optimized oral treatment
- Where
- France
- Volunteers
- 12 people
- Sponsor
- University Hospital, Lille
Alpha-Mannosidosis
Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
- Trial name
- NCT01681940
- Testing
- Lamazym
- Where
- 4 sites in Belgium, Denmark and 2 more
- Volunteers
- 10 people
- Sponsor
- Zymenex A/S
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Type II Gaucher Disease
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Trial name
- NCT06272149
- Testing
- VGN-R08b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Thrombocytopenia
Mirasol-Treated Platelets - (Pathogen Reduction Extended Storage Study)
The PRESS trial is a single-center, two-part, randomized cross-over pilot study in Copenhagen that will evaluate 1) the safety and performance of Mirasol-treated and untreated platelet products stored for 2-4 days and for 7-days…
- Trial name
- PRESS (NCT01368211)
- Testing
- 2-4-day-old Mirasol-treated Platelets Transfusion, 2-4-day-old Untreated Platelets Transfusion
- Where
- Denmark
- Volunteers
- 16 people
- Sponsor
- Terumo BCTbio
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