bioihbjkbjnl/mcl2 huggingface dataset:
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
Condition: GLUT1DS1
Intervention: Triheptanoin
Intervention description: Dietary supplementation with triheptanoin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Minimum age: 30 Months
Maximum age: 35 Years
Healthy volunteers: No
Keywords: G1D, glucose, transporter

Summary:

      To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and
      seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical
             genotyping at a CLIA-certified laboratory.

          -  Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have
             taken place for 2 months). The initiation of a ketogenic diet is previous to - and
             thus is not part of this study.

          -  Males and females 30 months to 35 years and 11 months old inclusive.

        Exclusion Criteria:

          -  Subjects with evidence of independent, unrelated metabolic and/or genetic disease.

          -  Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome,
             crohn's disease, or colitis that could increase the subject's risk of developing
             diarrhea or stomach pain.

          -  Subjects with a BMI (body mass index) greater than or equal to 30.

          -  Subjects currently not on ketogenic diet.

          -  Women who are pregnant or breast feeding may not participate. Women who plan to become
             pregnant during the course of the study, or who are unwilling to use birth control to
             prevent pregnancy (including abstinence) may not participate. Females age 10 and over
             will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects
             will be asked to agree to abstinence or another form of birth control for the duration
             of the study.

          -  Allergy/sensitivity to C7

          -  Previous use of triheptanoin less than 1 month prior to study initiation.

          -  Treatment with medium chain triglycerides in the last 24 hours.

          -  Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain
             disorder (such as Alzheimer's disease) that would confound assessment of cognitive
             changes, in the opinion of the investigator.

          -  Active drug or alcohol use or dependence that, in the opinion of the investigator,
             would interfere with adherence to study requirements.

          -  Inability or unwillingness of subject or legal guardian/representative to give written
             informed consent, or assent for children age 10-17,

          -  Addition of a new antiseizure drug in the previous 3 months.
A Study to Evaluate SAGE-217 in Participants With Parkinson's Disease
Condition: Parkinson Disease
MeSH terms: Parkinson Disease
Intervention: SAGE-217; Levodopa; Antiparkinsonian Agent(s)
Intervention description: Levodopa (including carbidopa-levodopa) administered as antiparkinsonian agent(s).; Antiparkinsonian agent(s) were administered as a clinical practice as standard of care, which includes levodopa (i.e. levodopa/carbidopa) or dopamine agonists or catechol-O-methyltransferase (COMT) inhibitors or monoamine oxidase (BMAO-B) inhibitors.
Intervention MeSH terms: Levodopa; Antiparkinson Agents
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Masking: None (Open Label)
Enrollment: 29.0
Sex: All
Minimum age: 40 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Parkinson's

Summary:

      This study assesses the safety, tolerability, pharmacokinetics and efficacy of SAGE-217 in 29
      participants with moderate to severe Parkinson's Disease.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participant has a diagnosis of idiopathic Parkinson's Disease (Hoehn and Yahr Stage 2
             or Stage 3) with a duration of less than 7 years prior to screening [Part A]

          -  Participant has a diagnosis of idiopathic Parkinson's Disease (Hoehn and Yahr Stage
             1-4, assessed during the "on" period) [Part B]

          -  Participant is on a stable dose (at least 1 month prior to baseline visit) of an
             antiparkinsonian agent and is willing to remain on this dose for the duration of the
             study

        Exclusion Criteria:

          -  Participant has early (Hoehn and Yahr Stage 1) or advanced (Hoehn and Yahr Stage 4 or
             Stage 5) Parkinson's Disease [Part A]

          -  Participant has advanced (Hoehn and Yahr Stage 5) Parkinson's Disease [Part B]

          -  Participant with presence of drug-induced parkinsonism, metabolic identified
             neurogenetic disorders, encephalitis, or other atypical Parkinsonian syndromes

          -  Participant with medical history of electroconvulsive therapy or stereotaxic brain
             surgery for Parkinson's Disease

          -  Participant with medical history of suicide attempt within 2 years of screening or
             current suicidal ideation

          -  Participant with medical history of impulse control disorder
Intravenous Nitrite Infusion for Reversal of Cerebral Vasospasm After Subarachnoid Hemorrhage
Condition: Subarachnoid Hemorrhage
MeSH terms: Subarachnoid Hemorrhage; Vasospasm, Intracranial; Hemorrhage
Intervention: Sodium Nitrite
Intervention description: Subjects will receive continuous intravenous infusion of study drug for 7 days at a dose of 64 nmol/min/kg. The infusion will begin shortly after angiographic demonstration of cerebral vasospasm and will be for 7 days unless side effects of the drug dictate stopping the infusion.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 6.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: subarachnoid hemorrhage; vasospasm; nitrite; sodium nitrite

Summary:

      The hypothesis is that intravenous infusion of sodium nitrite is safe and effective for the
      reversal of cerebral vasospasm after subarachnoid hemorrhage in patients with a cerebral
      aneurysm.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Informed consent obtained from a patient or legal representative before enrollment;

          -  Admission to hospital following subarachnoid hemorrhage;

          -  Aneurysm confirmed by digital subtraction angiography or CT-angiography after
             admission;

          -  Development of the clinical symptoms and /or signs of cerebral vasospasm and
             CT-angiography results warranting cerebral angiography.

        Exclusion Criteria:

          -  Rupture of a fusiform, traumatic, or mycotic aneurysm;

          -  Pregnancy (confirmed by a serum human chorionic gonadotropin pregnancy test) or breast
             feeding;

          -  Methemoglobin > 2%

          -  History of sickle cell disease, thalassemia, or other hemoglobinopathy;

          -  Anemia with hemoglobin level less than 6 g/dL;

          -  Significant acute or chronic concomitant diseases (including renal, hepatic,
             cardiovascular, pulmonary, or oncologic disease, sepsis, pulmonary edema, pulmonary
             embolism) that would be inconsistent with survival for at least 6 months;

          -  History of allergy to nitrites or allergy to other substances that could interfere
             with nitrite metabolism, within 30 days before screening;

          -  History of red blood cell glucose-6-phosphate dehydrogenase (G6PD)deficiency;

          -  Treatment with allopurinol, a medication that could interfere with nitrite metabolism,
             within 30 days before screening;

          -  Other investigational drug within the past 30 days;

          -  other cerebral injury within the past 30 days including previous subarachnoid
             hemorrhage, stroke, transient ischemic attacks (TIA), concussion, head trauma, cranial
             surgery, radiation therapy to the head.
FET-PET for Evaluation of Response of Recurrent GBM to Avastin
Condition: Glioblastoma Multiforme; GBM
MeSH terms: Glioblastoma
Intervention: 18F-FET
Intervention description: Radiotracer, surrogate marker for protein synthesis
Phase: Phase 2
Study type: Interventional
Primary purpose: Diagnostic
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 13.0
Sex: All
Minimum age: 18 Years
Maximum age: 85 Years
Healthy volunteers: No
Keywords: FET; Fluoroethyl tyrosine; GBM; Response to treatment; Avastin; Brain Tumor

Summary:

      Hypothesis: The central hypothesis underlying the proposed research study is that FET-PET
      will predict durable benefit in patients receiving anti-angiogenic benefit for presumed
      recurrent GBM (i.e. progression-free survival and overall survival). We have defined one
      primary specific aim, for which we expect to obtain definitive results, and two secondary
      aims, under which we plan to generate preliminary data to support a future, larger project.
    

Eligibility criteria:

        Inclusion Criteria:

          -  1. GBM patients with changes on MRI suggestive of recurrence who have not yet
             initiated antiangiogenic therapy. 2. Age ≥ 18 3. Anticipated survival >3 months 4.
             Able to give informed consent 5. Capable of undergoing scan without the need for
             sedation or general anesthesia.

        Exclusion Criteria:

          1. Active intracranial infection or nonglial brain mass.

          2. Recent large intracranial hemorrhage (<1 month; size to be determined by principal
             investigator)

          3. Pregnant or nursing. Quantitative serum hCG testing will be performed prior to the
             initial and each -subsequent FET- PET scan on all females of childbearing potential.
             Our BWH Radiation Safety Committee and Partners IRB requires stat serum ß-hcG
             pregnancy tests.

          4. Patient lives too far from BWH and/or is unwilling/ unable to return for scheduled
             imaging visits.

             -
LHA510 Proof-of-Concept Study as a Maintenance Therapy for Patients With Wet Age-Related Macular Degeneration
Condition: Exudative Age-Related Macular Degeneration
MeSH terms: Macular Degeneration
Intervention: LHA510 ophthalmic suspension; LHA510 vehicle; Ranibizumab ophthalmic solution
Intervention description: Inactive ingredients used as a placebo comparator; For intravitreal (IVT) injection
Intervention MeSH terms: Ranibizumab; Ophthalmic Solutions; Acrizanib
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 136.0
Sex: All
Minimum age: 50 Years
Maximum age: 90 Years
Healthy volunteers: No
Keywords: LHA510; PoC; Age-related macular degeneration

Summary:

      The purpose of this study is to evaluate the efficacy of 84 successive days of topically
      administered LHA510 compared to vehicle in reducing the number of patients requiring
      intravitreal (IVT) anti-vascular endothelial growth factor (VEGF) therapy (Lucentis®) for
      recurrence of active choroidal neovascularization (CNV).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Sign written informed consent form;

          -  Wet AMD;

          -  IVT anti-VEGF therapy for at least 6 months and a maximum of 7 years since the 3rd
             loading dose;

          -  BCVA 50 letters (approximate Snellen equivalent 20/100) or better in the study eye;

          -  Demonstrate ability to administer eye drops (subject or care-giver);

          -  CNV recently demonstrated high need for frequent anti-VEGF therapy and a sustained
             functional and clear anatomical response to the therapy in the study eye;

          -  Other protocol-specified inclusion criteria may apply.

        Exclusion Criteria:

          -  Any active ocular or periocular infection or intraocular inflammation;

          -  Current or history of macular or retinal disease (if visually significant) other than
             wet AMD in the study eye;

          -  Current clinically significant vitreous hemorrhage or history of rhegmatogenous
             retinal detachment affecting the macula in the study eye;

          -  History of hypersensitivity to any of the study drugs or clinically relevant
             sensitivity to fluorescein dye or povidone iodine;

          -  Women of child-bearing potential;

          -  History of a medical condition that, in the opinion of the Investigator, would
             preclude scheduled study visits, completion of the study or a safe administration of
             investigational product;

          -  Other protocol-specified exclusion criteria may apply.
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
Condition: Sly Syndrome; MPS VII; Mucopolysaccharidosis; Mucopolysaccharidosis VII
MeSH terms: Mucopolysaccharidoses; Mucopolysaccharidosis VII; Syndrome
Intervention: UX003
Intervention description: solution for intravenous infusion
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 8.0
Sex: All
Minimum age: 1 Day
Maximum age: 5 Years
Healthy volunteers: No
Keywords: MPS 7; Sly Syndrome; MPS VII; Enzyme Replacement Therapy; Rare Disease; Mucopolysaccharidosis type 7; Lysosomal Storage Disease; Metabolic Disorder

Summary:

      The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII
      participants less than 5 years of age on safety, tolerability, and efficacy as determined by
      the reduction of urinary glycosaminoglycans (uGAG) excretion.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme
             assay, or genetic testing.

          2. Under 5 years of age at the time of informed consent.

          3. Written informed consent of Legally Authorized Representative after the nature of the
             study has been explained, and prior to any research-related procedures.

        Exclusion Criteria:

          1. Undergone a successful bone marrow or stem cell transplant or has evidence of any
             degree of detectable chimaerism with donor cells.

          2. Any known hypersensitivity to rhGUS or its excipients that, in the judgment of the
             Investigator, places the subject at increased risk for adverse effects.

          3. Use of any investigational product (drug or device or combination) other than UX003
             within 30 days prior to Screening, or requirement for any investigational agent prior
             to completion of all scheduled study assessments at any time during the study.

          4. Has a condition of such severity and acuity, in the opinion of the Investigator, which
             may not allow safe study participation. For patients with hydrops fetalis, the ongoing
             interventions to manage fluid balance can be continued; if the addition of enzyme
             replacement therapy (ERT) is considered a fluid-overload risk, the individual should
             be excluded.

          5. Has a concurrent disease or condition that, in the view of the Investigator, places
             the subject at high risk of poor treatment compliance or of not completing the study,
             or would interfere with study participation or affect safety. Since hydropic patients
             have a high rate of mortality, the risk of death prior to 1 year of age should not be
             considered sufficient to exclude the patient from the study for compliance.
Detection of High Grade Prostate Cancer With Subharmonic Ultrasound Imaging, A Pilot Study
Condition: Prostatic Neoplasm
MeSH terms: Prostatic Neoplasms
Intervention: Perflutren Lipid Microsphere Intravenous Suspension
Intervention description: The intravenous administration of Perflutren Lipid Microsphere will provide enhancement of vascular tissue when performing subharmonic prostate ultrasound imaging. This enhancement will be used to guide targeted biopsy of the prostate to vascular areas which are more likely to contain aggressive prostate cancer.
Phase: Phase 2
Study type: Interventional
Primary purpose: Diagnostic
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 55.0
Sex: Male
Minimum age: 18 Years
Healthy volunteers: No
Keywords: prostate cancer; prostate ultrasound; subharmonic imaging; prostate biopsy

Summary:

      This is a phase II single center study using contrast-enhanced ultrasound to identify
      aggressive forms of prostate cancer with subharmonic imaging.

      The following are the study objectives:

        1. To implement subharmonic imaging technology on a transrectal probe suitable for prostate
           imaging and biopsy

        2. To demonstrate visualization of prostatic vascularity using subharmonic
           contrast-enhanced imaging.

        3. To provide a preliminary estimate of the diagnostic accuracy of contrast-enhanced
           subharmonic imaging for detection of clinically significant PCa.

      The study will include 50 subjects who are scheduled for prostate biopsy. Each subject will
      receive an intravenous infusion of microbubble contrast material immediately prior to a
      prostate biopsy procedure. The study will demonstrate whether subharmonic imaging with a
      microbubble contrast agent allows for detection of clinically significant prostate cancer.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subject must be scheduled for a clinically indicated needle biopsy of the prostate
             based upon an elevated PSA, abnormal digital rectal examination, or based upon active
             surveillance of prostate cancer.

          2. Subject must be able and willing to give written informed consent for a contrast
             enhanced ultrasound study of the prostate.

          3. Subject must be a male at least 18 years of age when informed consent is obtained.

          4. Subject must have a life expectancy that exceeds the duration of the clinical trial.

        Exclusion Criteria:

          1. Participant in a clinical trial involving an investigational drug within the past 30
             days.

          2. Prior allergic reaction to the ultrasound contrast agent Definity™

          3. Previous treatment for PCa.

          4. Clinically unstable, severely ill, or moribund.
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of ABBV-3373 in Participants With Moderate to Severe Rheumatoid Arthritis (RA)
Condition: Rheumatoid Arthritis (RA)
MeSH terms: Arthritis; Arthritis, Rheumatoid
Intervention: ABBV-3373; Placebo for ABBV-3373; Adalimumab; Placebo for adalimumab
Intervention description: ABBV-3373 is administered as intravenous (IV) infusion; Placebo for ABBV-3373 is administered as IV infusion; Adalimumab is administered as subcutaneous (SC) injection; Placebo for adalimumab is administered as subcutaneous (SC) injection
Intervention MeSH terms: Adalimumab
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 48.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Rheumatoid Arthritis (RA); ABBV-3373; Adalimumab

Summary:

      This study will assess the safety, tolerability, and efficacy of ABBV-3373 in participants
      with moderately to severely active rheumatoid arthritis (RA) on background methotrexate (MTX)
      compared with adalimumab.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participant has the clinical diagnosis of RA for > 3 months based on the 1987 American
             College of Rheumatology (ACR) classification criteria or 2010 ACR/European League
             against Rheumatism (EULAR) criteria.

          -  Participant meets the following disease activity criteria: >= 4 swollen joints (based
             on 28 joint count) and >= 4 tender joints (based on 28 joint count) at Screening and
             Baseline visits and disease activity score (28 joints) (DAS28) C-reactive protein
             (CRP) >= 3.2 at Screening.

          -  Participant has an incomplete response to methotrexate. Participants must have been on
             oral or parental MTX therapy >= 3 months and on a stable prescription of 15 to 25
             mg/week (or >= 10 mg/week in participants intolerant of MTX at doses >= 15 mg/week)
             for >= 4 weeks prior to the first dose of study drug. Participant must be expected to
             be able to continue on stable dose of MTX for the duration of study participation.

        Exclusion Criteria:

          -  Participants previously exposed to adalimumab or other anti-tumor necrosis factor
             (TNF) biologics.

          -  Participants previously exposed to non-anti-TNF biologics or targeted synthetic
             disease-modifying antirheumatic drugs (DMARDs) for RA, with exception of participants
             exposed for less than 3 months and terminated not due to lack of efficacy or
             intolerability.