OPEN ENROLLMENT
Biobank for Potential or Known Genetic Diseases
This study is for patients of all ages with either known or suspected genetic diseases. There is no cost associated with participation in this research study. We would like to obtain a…
- Investigator
- Estrella Lizbeth Mellin Sanche, Pamela Trapane
- IRB Number
- IRB202002626
- Ages
- 0 Years – 100 Years
- Sexes
- All
- Location
- Jacksonville
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Rare Disease Registry
This is a registry (data bank) for patients of all ages with Gaucher, Fabry, Pompe, or MPS1 disease. The sponsors of this registry are Genzyme Corporation (Genzyme) (United States) and Sanofi…
- Investigator
- Pamela Trapane
- IRB Number
- IRB202101353
- Ages
- 0 Years – 100 Years
- Sexes
- All
- Location
- Jacksonville
A Multicenter, Interventional, Retrospective and Prospective study of Enzyme Replacement Therapy (VPRIV) Clinical Outcomes and Safety in Gaucher Disease Type 1 Patients Previously Treated with Substrate Reduction Therapy
Adult patients with Gaucher Disease Type 1 who have previously received SRT (substrate replacement therapy) and are now receiving ERT (enzyme replacement therapy) with velaglucerase alfa. For group…
- Investigator
- Estrella Lizbeth Mellin Sanche
- IRB Number
- WCG20210806
- Ages
- 18 Years – 100 Years
- Sexes
- All
- Location
- Jacksonville
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Sildi-Safe: Safety of Sildenafil in Premature Infants with Severe Bronchopulmonary Dysplasia (BPD)
This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants with severe bronchopulmonary dysplasia (BPD)….
- Investigator
- Mark Hudak, Josef Cortez, William Driscoll, Ma Ingyinn, James Kirk, Padma Nandula, Sfurti Nath, Sanket Shah, Renu Sharma, Winston Sheen, Shalinkumar Patel, Orlyn Claire Lavilla, John Logan, Grace Twitty, Dawn McDaniel
- IRB Number
- CED000000503
- Sexes
- All
- Location
- Jacksonville
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STOMP study
Phase 3, randomized, placebo-controlled, double-blind trial of tecovirimat for the treatment of human monkeypox virus (HMPXV) disease (Arm A and B). The study will also include a cohort of people…
- Investigator
- Mobeen Rathore
- IRB Number
- CED000000716
- Ages
- 7 Years – 60 Years
- Sexes
- All
- Location
- Jacksonville
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BabyCOVE study
This is a Phase 2, two-part, open-label in Part 1 and observer-blind, randomized, placebo controlled in Part 2 study to evaluate the safety, tolerability, reactogenicity, and effectiveness of…
- Investigator
- Mobeen Rathore
- IRB Number
- CED000000720
- Ages
- 3 Years – 6 Years
- Sexes
- All
- Location
- Jacksonville
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The Connection study (20191002)
A randomized, double blind, parallel-group, placebo controlled study to evaluate the efficacy and safety of IBP-9414 in premature infants 500-1500g birth weight in the prevention of necrotizing…
- Investigator
- Josef Cortez, William Driscoll, Mark Hudak, Ma Ingyinn, Orlyn Claire Lavilla, John Logan, Dawn McDaniel, Padma Nandula, Sfurti Nath, Shalinkumar Patel, Sanket Shah, Renu Sharma, Winston Sheen
- IRB Number
- 1262352
- Sexes
- All
- Location
- Jacksonville
OPEN ENROLLMENT
Safety of Sildenafil in Premature Infants at Risk of Bronchopulmonary Dysplasia (20180529)
You are being asked to consider permitting your baby to take part in this research study, because your baby was born prematurely and is at risk for developing a lung disease called bronchopulmonary…
- Investigator
- Mark Hudak, William Driscoll, Ma Ingyinn, James Kirk, Padma Nandula, Sfurti Nath, Sanket Shah, Renu Sharma, Winston Sheen, Josef Cortez, Shalinkumar Patel, Orlyn Claire Lavilla, John Logan, Grace Twitty, Dawn McDaniel
- IRB Number
- 1304032
- Sexes
- All
- Location
- Jacksonville
OPEN ENROLLMENT
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care / (POPS) (20193297)
This study is interested in learning more about how drugs, that are given to children by their health care providers, act in the bodies of children and young adults in hopes to find the most safe and…
- Investigator
- Mobeen Rathore
- IRB Number
- 1283493
- Ages
- 0 Years – 20 Years
- Sexes
- All
- Location
- Jacksonville
OPEN ENROLLMENT
Central Nervous System Derived Exosomes: A Novel Source of Biomarkers for Neonatal Hypoxic Ischemic Encephalopathy
The purpose of this research study is to look at biomarkers that come from the baby’s blood and see if these biomarkers are associated with brain injury or if they can predict brain…
- Investigator
- Mark Hudak
- IRB Number
- IRB202102111
- Ages
- 0 Years – 1 Years
- Sexes
- All
- Location
- Jacksonville
OPEN ENROLLMENT
Open-Label, Phase 2, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Patiromer in Children under 12 Years of Age with Hyperkalaemia
A Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Patiromer in Children under 12 Years of Age with Hyperkalaemia. Clinical data shown the benefits of patiromer in…
- Investigator
- Mohammad Ilyas
- IRB Number
- 20226442
- Ages
- 0 Years – 12 Years
- Sexes
- All
- Location
- Jacksonville
OPEN ENROLLMENT
A Global Prospective Observational Registry of Patients with Pompe Disease
This is an international Registry that would like to collect and evaluate outcomes on patients with Pompe. The Sponsor of the study will look at long-term safety of treatments, effectiveness of…
- Investigator
- Pamela Trapane
- IRB Number
- POM-005
- Ages
- 0 Years – 100 Years
- Sexes
- All
- Location
- Jacksonville