bioihbjkbjnl/mcl2 huggingface dataset:
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Condition: Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC); Amyloidosis, Hereditary; Amyloid Neuropathies, Familial; Amyloid Neuropathies; Amyloidosis, Hereditary, Transthyretin-Related; Familial Transthyretin Cardiac Amyloidosis
MeSH terms: Amyloid Neuropathies; Amyloid Neuropathies, Familial; Cardiomyopathies; Amyloidosis, Familial; Amyloidosis
Intervention: Revusiran (ALN-TTRSC); Sterile Normal Saline (0.9% NaCl)
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 206.0
Sex: All
Minimum age: 18 Years
Maximum age: 90 Years
Healthy volunteers: No
Keywords: Cardiomyopathy; Heart Failure; FAC; Amyloid; Transthyretin; TTR; RNAi therapeutic

Summary:

      The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in
      patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has
      been discontinued; patients are being followed-up for safety.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Documented TTR mutation

          -  Amyloid deposits in cardiac or non-cardiac tissue

          -  Medical history of heart failure

          -  Evidence of cardiac involvement by echocardiogram

        Exclusion Criteria:

          -  Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non-FAC hereditary
             cardiomyopathy, hypertensive cardiomyopathy, or cardiomyopathy due to valvular heart
             disease

          -  Has known peripheral vascular disease affecting ambulation

          -  Has a Polyneuropathy Disability score >2

          -  Has a New York Heart Association (NYHA) classification of IV
A Long-Term Study To Evaluate Safety And Efficacy Of Pregabalin For Pain Associated With Diabetic Peripheral Neuropathy
Condition: Diabetic Neuropathy, Painful
MeSH terms: Peripheral Nervous System Diseases; Diabetic Neuropathies
Intervention: pregabalin
Intervention description: Dosage: 150-600 mg/day (75-300 mg bid), oral administration, Treatment duration: 52 weeks
Intervention MeSH terms: Pregabalin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 123.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to assess the safety and efficacy of the long-term use of
      pregabalin at doses up to 600 mg/day in patients with painful diabetic peripheral neuropathy
      who have completed 13 weeks of dosing in Study A0081163
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients who completed the 13-week treatment of painful diabetic peripheral neuropathy
             in Study A0081163.

          -  Patients must be able to understand and cooperate with study procedures and have
             signed a written informed consent prior to entering the study

        Exclusion Criteria:

          -  Patients who experienced serious adverse events in the preceding study (A0081163) that
             were determined by the investigator or the study sponsor to be causally related to the
             study medication.

          -  Patients exhibiting treatment non-compliance in the preceding study (A0081163)
A Study to Evaluate Ocrelizumab Compared With Placebo in Patients With Rheumatoid Arthritis Who Have an Inadequate Response to Methotrexate Therapy
Condition: Rheumatoid Arthritis
MeSH terms: Arthritis; Arthritis, Rheumatoid
Intervention: Methotrexate; Ocrelizumab; Placebo
Intervention description: Oral or parenteral repeating dose; Ocrelizumab was administered as a slow intravenous (iv) infusion during each course as either 200 mg on Day 1 and Day 15 (OCR 200×2) or as 400 mg given on Day 1 (OCR 400×1).
Ocrelizumab was administered in combination with Methotrexate.; Intravenous repeating dose
Intervention MeSH terms: Methotrexate; Ocrelizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 314.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: RA

Summary:

      This study will evaluate the efficacy and safety of ocrelizumab, compared to placebo, in
      patients with active rheumatoid arthritis who have an inadequate response to methotrexate
      therapy. Patients will be randomized 2:2:1 to receive 1) infusions of ocrelizumab 200mg iv on
      Days 1 and 15, 2) infusions of ocrelizumab 400mg iv on Day 1 and placebo iv on Day 15, or 3)
      infusions of placebo iv on Days 1 and 15. At the end of the placebo-controlled treatment
      period at 24 weeks, patients in groups 1 and 3 will be re-randomized to receive either a
      single infusion of 400mg iv ocrelizumab or 2 infusions of 200mg iv ocrelizumab, and group 2
      will receive a second single infusion of 400mg iv ocrelizumab. All patients will receive a
      stable dose of concomitant methotrexate (7.5-25mg/week) throughout the study. The anticipated
      time on study treatment is 1-2 years. Target number of patients to be enrolled in this trial
      is 300.
    

Eligibility criteria:

        Inclusion criteria:

          -  Adult patients, ≥ 18 years of age

          -  Active rheumatoid arthritis

          -  Inadequate treatment with any DMARD other than methotrexate

        Exclusion criteria:

          -  Rheumatic autoimmune disease or inflammatory joint disease other than rheumatoid
             arthritis

          -  Concurrent treatment with any DMARD other than methotrexate

          -  Previous treatment with any cell-depleting therapies

          -  Any surgical procedure in past 12 weeks, or planned within 48 weeks after baseline
EZN-2279 in Patients With ADA-SCID
Condition: ADA-SCID; Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency
MeSH terms: Severe Combined Immunodeficiency; Immunologic Deficiency Syndromes
Intervention: EZN-2279; Adagen
Intervention description: Weekly administration of EZN-2279 via IM injection
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 7.0
Sex: All
Healthy volunteers: No

Summary:

      The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of
      EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated
      with Adagen.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Diagnosis of ADA-deficient combined immunodeficiency

          2. Stable clinical status while receiving therapy with Adagen®. Patients previously
             receiving gene therapy or undergoing hematopoietic stem cell transplantation who still
             require Adagen® treatment are eligible. The dose of Adagen® must be stable for at
             least 6 months prior to study entry.

          3. Have both of the following during the Adagen® Lead-in phase of the study prior to
             EZN-2279 transition:

               1. Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen® and

               2. Total erythrocyte dAXP ≤0.02 μmol/mL from a trough blood sample

          4. Patients or parent/guardian must be capable of understanding the protocol requirements
             and risks and providing written informed assent/consent

        Exclusion Criteria:

          1. Autoimmunity requiring immunosuppressive treatment

          2. Patients with detectable neutralizing anti-Adagen® antibodies at screening evaluation

          3. Severe thrombocytopenia (platelet count <50 x 10^9/L)

          4. Current participation in other therapeutic protocols for ADA-deficient combined
             immunodeficiency

          5. Current or prior participation in another clinical study with an investigational agent
             and/or use of an investigational drug in the 30 days before study entry

          6. Known planned participation in a gene-therapy study for the planned duration of this
             study

          7. Any condition that, in the opinion of the PI, makes the patient unsuitable for the
             study

          8. Inability or unwillingness to administer Adagen® or EZN-2279 on a one-time-per-week
             regimen

          9. Inability to comply with the study protocol

         10. Female patients who are pregnant or lactating

         11. Female patients who are breast-feeding

         12. Female subjects of childbearing potential who are not using an FDA approved birth
             control method
Fixed-dose Safety and Efficacy Study of Asenapine for the Treatment of Acute Manic or Mixed Episode in Bipolar 1 Disorder (P05691)
Condition: Bipolar 1 Disorder
Intervention: Asenapine; Asenapine; Placebo
Intervention description: asenapine tablet, 5 mg sublingually BID for 21 days; asenapine tablet, 10 mg sublingually BID for 21 days; placebo sublingual tablet, administered BID for 21 days
Intervention MeSH terms: Asenapine
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 367.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This trial will study the efficacy and safety of a fixed dose of asenapine in participants
      diagnosed with Bipolar 1 Disorder. Participants who qualify for the study will be randomly
      assigned to receive a fixed dose of asenapine (either 5 mg or 10 mg twice daily [BID]) or
      placebo (BID) for 3 weeks. Throughout the trial, observations will be made on each
      participant at various times to assess the safety and effectiveness of the study treatment.
      The primary hypothesis is that there is at least one dose of asenapine that is superior to
      placebo in the change from baseline in manic symptoms (as measured by Young Mania Rating
      Scale [YMRS]) at Day 21 of the trial.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Each participant must be at least 18 years of age

          -  Male, or a female who is not of child-bearing potential or who is non-pregnant,
             non-lactating, and is using a medically accepted method of contraception

          -  Each participant must have a current diagnosis of Bipolar I Disorder, current episode
             manic or mixed

          -  Each participant must be confirmed to be experiencing an acute manic or mixed bipolar
             1 episode

          -  Each participant must have discontinued the use of all prohibited psychotropic
             medications

        Exclusion Criteria:

          -  A participant must not have a primary Axis I disorder other than Bipolar 1 Disorder
             (i.e., an Axis 1 disorder other than Bipolar 1 Disorder that is primarily responsible
             for current symptoms and functional impairment)

          -  A participant must not currently (within the past 6 months) meet the Diagnostic and
             Statistical Manual of Mental Disorders Fourth Edition, Text Revision (DSM-IV-TR™)
             criteria for substance abuse or dependence (excluding nicotine)

          -  A participant must not be at imminent risk of self-harm or harm to others, in the
             investigator's opinion based on clinical interview and responses provided on the
             Columbia Suicide Severity Rating Scale (CSSRS).
Comparison of Sofosbuvir/Velpatasvir Fixed Dose Combination for 12 Weeks With Sofosbuvir and Ribavirin for 24 Weeks in Adults With Chronic Genotype 3 HCV Infection
Condition: Hepatitis C Virus Infection
MeSH terms: Infections; Communicable Diseases; Hepatitis C; Hepatitis
Intervention: SOF/VEL; SOF; RBV
Intervention description: 400/100 mg FDC tablet administered orally once daily; 400 mg tablet administered orally once daily; Tablets administered orally in a divided daily dose according to package insert weight-based dosing recommendations (< 75 kg = 1000 mg and ≥ 75 kg = 1200 mg)
Intervention MeSH terms: Sofosbuvir
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 558.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Sofosbuvir; Velpatasvir; SOF/VEL; GS-5816; Hepatitis C; HCV; Cirrhosis; Genotype 3

Summary:

      The primary objectives of this study are to compare the efficacy of treatment with
      sofosbuvir/velpatasvir (SOF/VEL) fixed-dose combination (FDC) for 12 weeks with that of
      sofosbuvir (SOF) + ribavirin (RBV) for 24 weeks and to evaluate the safety and tolerability
      of each treatment regimen in participants with chronic genotype 3 hepatitis C virus (HCV)
      infection.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Willing and able to provide written informed consent

          -  HCV RNA ≥ 10^4 IU/mL

          -  HCV genotype 3

          -  Chronic HCV infection (≥ 6 months)

          -  Females of childbearing potential must have a negative serum pregnancy test

          -  Males and females of childbearing potential who engage in heterosexual intercourse
             must agree to use protocol specified method(s) of contraception

        Exclusion Criteria:

          -  Current or prior history of clinically-significant illness (other than HCV) that may
             interfere with subject treatment, assessment or compliance with the protocol

          -  Screening ECG with clinically significant abnormalities

          -  Laboratory results outside of acceptable ranges at Screening

          -  Pregnant or nursing female or male with pregnant female partner

          -  Chronic liver disease of a non-HCV etiology (e.g., hemochromatosis, Wilson's disease,
             alfa-1 antitrypsin deficiency, cholangitis)

          -  Infection with hepatitis B virus (HBV) or human immunodeficiency virus (HIV)
A Comparison of Midazolam and Zolpidem as Oral Premedication in Children
Condition: Parental/Caregiver Anxiety; Child's Anxiety
MeSH terms: Anxiety Disorders
Intervention: zolpidem; Midazolam
Intervention description: Zolpidem will be given one time at 0.25 mg/kg pre-operatively
Intervention MeSH terms: Midazolam; Zolpidem
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 86.0
Sex: All
Minimum age: 2 Years
Maximum age: 9 Years
Healthy volunteers: No
Keywords: Pre-medication; Pre-operative anxiety

Summary:

      The purpose of this investigator-initiated study is to compare the efficacy of oral midazolam
      and zolpidem for preoperative sedation, anxiety of patient, and caregiver anxiety at the time
      of separation, and ease of mask acceptance at induction in children. Subjects will be
      randomized to receive oral medication midazolam or zolpidem approximately 30 minutes prior to
      surgery. No placebo will be administered in this study. Subjects will be male and female
      children between 2 and 9 years of age. In total, subject participation will last
      approximately the duration of their preoperative, perioperative, and immediate postoperative
      period. A member of the research team will recruit subjects preoperatively in the operating
      room holding area prior to surgery. Consent will take place at the time of recruitment in the
      preoperative holding area following a detailed explanation of the study and medications
      involved in the study. Participants will be of ASA (American Society of Anesthesiologists)
      class I-II, undergoing surgical procedures of at least 2 hours duration, and expected to
      remain inpatient for at least 23 hours postoperatively.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Pediatric patients ages 2-9 years

          -  ASA class I-II

          -  inpatient surgeries of at least 2 hours duration

          -  requiring postoperative admission of at least 23 hours Exclusion Criteria:

        Exclusion Criteria:

          -  contraindication to preoperative sedation, known allergy or sensitivity to the study
             medications,

          -  those who lack legal representative consent

          -  Patients with weights lying below the 5th percentile or above the 95th percentile
             according to the current published CDC growth chart will also be excluded regardless
             of existing specific contraindication.
Safety and Efficacy of Pomalidomide, Bortezomib and Low-dose Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma
Condition: Multiple Myeloma
MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell
Intervention: Pomalidomide; Bortezomib; Dexamethasone
Intervention description: Pomalidomide 4 mg will be taken orally on Days 1-14 of a 21-day cycle.; Bortezomib 1.3 mg/m2 will be administered subcutaneously on Days 1, 4, 8 and 11 of 21 days for cycles 1 -8 and on Days 1, 8 of 21 days for cycle 9 and onward until disease progression.; Dexamethasone 20 mg/day [≤ 75 years old] or 10 mg/day [>75 years old] will be taken orally on Days 1, 2, 4, 5, 8, 9, 11, 12 of 21 days for cycles 1-8 and on Days 1, 2, 8, 9 of 21 days for cycles 9 and onward until disease progression.
Intervention MeSH terms: Thalidomide; Dexamethasone; Bortezomib; Pomalidomide
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 559.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Multiple Myeloma; Pomalidomide

Summary:

      The purpose of this study is to compare the efficacy of the combination of pomalidomide,
      bortezomib and low dose dexamethasone to the combination of bortezomib and low dose
      dexamethasone in participants with relapsed/refractory multiple myeloma. This study will also
      assess how safe the combination of pomalidomide, bortezomib and low dose dexamethasone is
      compared to the combination of bortezomib and low dose dexamethasone.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Must be ≥ 18 years at the time of signing informed consent.

          -  Must have documented diagnosis of multiple myeloma and have measureable disease by
             serum and urine protein electrophoresis.

          -  Must have had at least 1 but no greater than 3 prior anti-myeloma regimens.

          -  Must have documented disease progression during or after their last anti-myeloma
             therapy.

          -  All subjects must have received prior treatment with a lenalidomide containing regimen
             for at least 2 consecutive cycles.

        Exclusion Criteria:

          -  Documented progressive disease during therapy or within 60 days of the last dose of a
             bortezomib-containing therapy under the 1.3 mg/m^2 dose twice weekly dosing schedule.

          -  Peripheral neuropathy Grade 3, Grade 4 or Grade 2 with pain within 14 days prior to
             randomization.

          -  Non-secretory multiple myeloma.

          -  Subjects with severe renal impairment requiring dialysis.

          -  Previous therapy with pomalidomide.
A Study of Rapastinel as Adjunctive Therapy in Major Depressive Disorder (RAP-MD-02)
Condition: Depressive Disorder, Major
MeSH terms: Depressive Disorder; Depression; Depressive Disorder, Major
Intervention: Rapastinel; Placebo
Intervention description: Rapastinel pre-filled syringes for weekly IV injections.; Placebo-matching rapastinel pre-filled syringes for weekly IV injections.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 658.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No

Summary:

      This study will evaluate the efficacy, safety, and tolerability of two doses of rapastinel,
      225 milligrams (mg) and 450 mg, compared to placebo adjunctive to antidepressant therapy
      (ADT) in patients with major depressive disorder (MDD) who have a partial response to ADT.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Meet Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5)
             criteria for MDD

          -  Current major depressive episode of at least 8 weeks and not exceeding 18 months in
             duration at Visit 1

          -  Have no more than partial response (< 50% improvement) to ongoing treatment with a
             protocol-allowed antidepressant

          -  If female of childbearing potential, have a negative serum β-human chorionic
             gonadotropin (β-hCG) pregnancy test.

        Exclusion Criteria:

          -  DSM-5-based diagnosis of any disorder other than MDD that was the primary focus of
             treatment within 6 months before Visit 1

          -  Lifetime history of meeting DSM-5 criteria for:

               1. Schizophrenia spectrum or other psychotic disorder

               2. Bipolar or related disorder

               3. Major neurocognitive disorder

               4. Neurodevelopmental disorder of greater than mild severity or of a severity that
                  impacts the participant's ability to consent, follow study directions, or
                  otherwise safely participate in the study

               5. Dissociative disorder

               6. Posttraumatic stress disorder

               7. MDD with psychotic features

          -  Significant suicide risk, as judged by the Investigator.
Study to Evaluate Potential Decrease in Hospitalization Events, Time Between Events, and Increasing Longevity in Patients With Symptomatic Heart Failure (0954-948)
Condition: Heart Failure
MeSH terms: Heart Failure
Intervention: Losartan 50 mg; Losartan 150 mg
Intervention description: 50-mg losartan oral tablet; 100-mg losartan oral tablet + 50-mg losartan oral tablet
Intervention MeSH terms: Losartan
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 3834.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This is a multicenter study to evaluate potential decrease in hospitalization events and time
      between events and increasing longevity in patients with symptomatic congestive heart failure
      and intolerant of first-line medication for heart failure. This study will evaluate if higher
      doses of the investigational drug given daily will be superior to the lower dose of the same
      investigational drug given daily.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Symptomatic heart failure with known or recently diagnosed intolerance of treatment
             with first-line medication for heart failure

          -  May or may not be receiving an alternative and/or additional drug treatment