Genetics Disease: Clinical Studies Open for Enrollment
Eligibility
- Male or female < 18 years of age
- No other significant co-morbidity (independent neurological disease, seizures)
- Molecular confirmation of IOPD or LOPD
- Age at enrollment: 5 years or more for IOPD patients and 5-40 years for LOPD patients
- Not claustrophobic
- A negative urine drug screening test
- No current use of recreational drugs
- No significant alcohol history (> 7 glasses of alcohol per week)
- No participation in investigational drug study within the last 30 days
- No contraindication to MRI
- Inclusion/Exclusion Criteria: for matched healthy controls no history of drug or alcohol abuse or dependence, no history of head injury, no significant medical history of such as seizure disorder, diabetes, alcoholism, cardiac disease including coronary artery disease, drug addiction, respiratory problems, liver disease, renal disease, etc.
Enrollment Status: Actively enrolling
Principal Investigator
Davis Kronn, MD
Contact for Study Screening
Hsakho@nymc.edu
Eligibility
- Male or female younger than 18 years of age
- Patients will be eligible for inclusion if they meet the following criteria for PARDS: Acute onset of respiratory failure defined as requiring mechanical ventilation via an endotracheal tube in the past 72 hours, Oxygenation index greater than 4 or oxygen saturation index greater than 5 if an arterial gas or PaO2 is not obtained, Chest imaging consistent with a new infiltrate or acute parenchymal disease and Respiratory failure not fully explained by cardiac failure or fluid overload
- Corrected gestational age ≥ 42 weeks or ˂18 years
- At least 12 hours of hemodynamic stability defined as a mean blood pressure or systolic blood pressure above the 5th percentile for age, which may be achieved with the support of vasoactive medication, specifically milrinone at any dose, dopamine of ≤ 5mcg/kg/min, epinephrine of ≤ 0.05mcg/kg/min and norepinephrine of ≤ 0.05mcg/kg/min.
- Exclusion Criteria: for matched healthy controls; Greater than 72 hours since mechanical ventilation was initiated or since the onset of PARDS, Allogeneic bone marrow transplant within the last five years, Malignancy or other irreversible disease or condition for which six-month mortality is estimated to be greater than 50 percent, Severe chronic respiratory disease requiring home invasive or non-invasive ventilator support greater than CPAP 5cmH2O, History of a lung transplantation at any time and Neuromuscular disease that impairs ability to ventilate without assistance
Enrollment Status: Actively enrolling
Principal Investigator
Erin Rescoe, MD
Contact for Study Screening
Sere.Politano@wmchealth.org
Eligibility
- Male or female, 22-85 years of age.
- Documented history of symptomatic or asymptomatic paroxysmal or persistent AF. The duration of AF must have been > 30 seconds as documented by an external monitor or present on 12-lead ECG.
- CHA2DS2-VASC score of 1-4 without prior stroke or TIA**
- The participant is on a DOAC at the time of screening.
- Inclusion/Exclusion Criteria: for matched healthy controls; Valvular or permanent atrial fibrillation, Current treatment with warfarin and unwilling or unable to take a DOAC.
Enrollment Status: Actively enrolling
Study Information
Grant # UG3HL15065
Principal Investigator
Jason Jacobson, MD
Contact for Study Screening
FNU.Namrata@wmchealth.org
Eligibility
- Male or female at least18 years of age
- Diagnosed with HIV and under active care by licensed USA healthcare provider
- Receiving CAB+RPV LA injectable therapy monthly or every 2 months for at least 6 months but no longer than 18 months at study entry.
- PWH will be eligible if documented pattern of sub-optimal adherence on prior oral ART in the 12 months prior to CAB+RPV LA injectable therapy initiation.
- PWH who are virologically suppressed OR viremic (defined as viral load of HIV-1 RNA >200 copies/mL) at study entry.
- Inclusion/Exclusion Criteria: for matched healthy controls; PWH who have maintained virological suppression and had no adherence challenges with prior oral ART and/or PWH who have previously received CAB+RPV LA therapy as part of a clinical trial.
Enrollment Status: Actively enrolling
Study Information
ClinicalTrials.gov | NCT03635788
Principal Investigator
Rebecca Glassman, MD
Contact for Study Screening
Macy.Baca@wmchealth.org
Eligibility
- Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease.
- Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated.
- Left ventricular hypertrophy.
- Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant.
- Exclusion Criteria: for matched healthy controls; History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation, History of seizures currently requiring treatment and Underlying medical condition that may cause or contribute to left ventricular hypertrophy.
Enrollment Status: Actively enrolling
Study Information
ClinicalTrials.gov | NCT05280548
Principal Investigator
Maryam Banikazemi, MD
Contact for Study Screening
jstarace2@nymc.edu
Eligibility
- Male or female, Aged at least 50 years old at time of initiation of regimen of interest (index date).
- HIV-1 diagnosis
- Currently or previously prescribed either DTG/3TC or BIC/FTC/TAF alone to manage HIV-1 infection, initiated after market approval of DTG/3TC in the participant´s respective country
- Has at least 24 weeks of clinical data available after index date.
- Upon initiation participants must have been on a stable ART regimen for ≥3 months and have <200 copies/mL HIV-1 RNA up to 180 days prior to their index date (if more than one viral load (VL) assessment is available within the assessment window, the last available VL should be utilized)
- Exclusion Criteria
- HIV-2 diagnosis
- Previous treatment:
- For BIC/FTC/TAF cohort participants, previous treatment with DTG+3TC or DTG/3TC
- For DTG/3TC participants, previous treatment with BIC/FTC/TAF
Enrollment Status: Actively enrolling
Principal Investigator
Rebecca Glassman, MD
Contact for Study Screening
Macy.Baca@wmchealth.org