bioihbjkbjnl/mcl2 huggingface dataset:
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
Condition: Hemophilia A
MeSH terms: Hemophilia A
Intervention: Valoctocogene Roxaparvovec
Intervention description: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 1.0
Sex: Male
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Hemophilia A; Gene Therapy; Clotting Disorders; Blood Disorder; Blood Coagulation Disorders; Inherited Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases; Inborn; Factor VIII; Coagulants

Summary:

      This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity,
      during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270
      on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week
      5 to week 52.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as
             evidenced by medical history.

          2. Must have been on prophylactic FVIII replacement therapy for at least 12 months prior
             to study entry.

          3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure
             days.

          4. No previous documented history of a detectable FVIII inhibitor of less than 0.6
             Bethesda Units (BU).

        Exclusion Criteria:

          1. Detectable pre-existing antibodies to the AAV5 capsid.

          2. Any evidence of active infection or any immunosuppressive disorder, including HIV
             infection.

          3. Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver
             cirrhosis of any etiology or history of hepatic malignancy.

          4. Evidence of any bleeding disorder not related to hemophilia A.

          5. Active Hepatitis C.

          6. Prior treatment with any vector/gene transfer agent.
Comparison of Telavancin and Vancomycin for Complicated Skin and Skin Structure Infections With a Focus on Methicillin-resistant Staphylococcus Aureus
Condition: Staphylococcal Skin Infection
MeSH terms: Infections; Communicable Diseases; Cellulitis; Skin Diseases, Infectious; Staphylococcal Skin Infections
Intervention: Telavancin; Vancomycin
Intervention description: Telavancin 10 mg/kg/day, IV for up to 14 days.; Vancomycin 1 Gm IV q 12 hrs for up to 14 days.
Intervention MeSH terms: Vancomycin; Telavancin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 862.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: staph; MRSA; cSSSI

Summary:

      Study 0017 compares the safety and effectiveness of an investigational drug, telavancin, and
      an approved drug, vancomycin, for the treatment of complicated skin and skin structure
      infections.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients must have a diagnosis of one of the following complicated skin and skin
             structure infections with Methicillin Resistant Staphylococcus aureus (MRSA) either
             suspected or confirmed as the major cause of the infection:

               -  major abscess requiring surgical incision and drainage

               -  infected burn (see exclusion criteria for important qualifications)

               -  deep/extensive cellulitis

               -  infected ulcer (see exclusion criteria for important qualifications)

               -  wound infections

          -  Patients must be expected to require at least 7 days of intravenous antibiotic
             treatment

        Exclusion Criteria:

          -  Received more than 24 hours of potentially effective systemic (IV, IM or PO)
             antibiotic therapy prior to randomization

          -  Burns involving > 20% of body surface area or third-degree/full-thickness in nature,
             diabetic foot ulcers, ischemic ulcers/wounds, necrotizing fascitis, gas gangrene, or
             mediastinitis.
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: LUM/IVA
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 62.0
Sex: All
Minimum age: 2 Years
Maximum age: 5 Years
Healthy volunteers: No
Keywords: CF

Summary:

      This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the
      pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of
      lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic
      fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in
      Part B, if they meet the eligibility criteria.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects who weigh ≥8 kilogram (kg) without shoes and wearing light clothing at the
             Screening Visit

          -  Subjects with confirmed diagnosis of CF at the Screening Visit

          -  Subjects who are homozygous for the F508del-cystic fibrosis transmembrane conductance
             regulator (CFTR) mutation

        Exclusion Criteria:

          -  Any clinically significant laboratory abnormalities at the Screening Visit that would
             interfere with the study assessments or pose an undue risk for the subject

          -  An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
             therapy (including antibiotics) for pulmonary disease within 28 days before Day 1

          -  A standard 12-lead ECG demonstrating QTc >450 millisecond (msec) at the Screening
             Visit.

          -  History of solid organ or hematological transplantation.

          -  Ongoing or prior participation in an investigational drug study (including studies
             investigating LUM and/or IVA) within 30 days of the Screening Visit.

          -  History of cataract/lens opacity or evidence of cataract/lens opacity determined to be
             clinically significant by a licensed ophthalmologist during the ophthalmologic
             examination at the Screening Visit
A Study of Monthly Subcutaneous (SC) Mircera for Maintenance Treatment of Participants With Chronic Kidney Disease on Peritoneal Dialysis
Condition: Anemia
MeSH terms: Kidney Diseases; Renal Insufficiency, Chronic
Intervention: Methoxy polyethylene glycol-epoetin beta
Intervention description: Mircera will be administered SC every 4 weeks for a total of 48 weeks. The first dose of 120 or 200 mcg will be determined by the dose of ESA received prior to administration of study treatment, while subsequent doses will be adjusted to maintain hemoglobin within the target range.
Intervention MeSH terms: Epoetin Alfa
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 96.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This single-arm study will assess the efficacy and safety of monthly administration of SC
      Mircera for the maintenance of hemoglobin levels in participants with chronic kidney disease
      on peritoneal dialysis. Participants currently receiving maintenance treatment with SC
      erythropoietin stimulating agents (ESAs) will receive monthly SC injections of Mircera, with
      the starting dose derived from the last weekly ESA they had been receiving.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Adults greater than or equal to (≥) 18 years of age

          -  Chronic kidney disease-related anemia on peritoneal dialysis for ≥3 months

          -  Continuous SC maintenance stable ESA therapy for 4 weeks prior to study start

        Exclusion Criteria:

          -  Transfusion of red blood cells during previous 8 weeks

          -  Poorly controlled hypertension requiring interruption of ESA treatment in previous 6
             months

          -  Significant acute or chronic bleeding during previous 8 weeks
Study of Pitavastatin in Elderly Patients With Primary Hypercholesterolemia or Combined Dyslipidemia
Condition: Hypercholesterolemia; Dyslipidemias
MeSH terms: Hypercholesterolemia; Dyslipidemias
Intervention: Pitavastatin
Intervention MeSH terms: Pitavastatin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 545.0
Sex: All
Minimum age: 65 Years
Healthy volunteers: No
Keywords: pitavastatin; hypercholesterolemia; combined dyslipidemia

Summary:

      This is an open-label extension study of NK-104-306 (NCT00257686) for elderly patients with
      hypercholesterolemia or combined dyslipidemia.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Greater than or equal to 65 years of age

          -  Primary hypercholesterolemia

          -  Combined dyslipidemia

          -  Completed study NK-104-306 (NCT00257686)

        Exclusion Criteria:

          -  Failed to complete study NK-104-306(NCT00257686)
Study Comparing Pitavastatin and Atorvastatin in Patients With Type II Diabetes Mellitus and Combined Dyslipidemia
Condition: Type II Diabetes Mellitus; Dyslipidemia
MeSH terms: Diabetes Mellitus; Dyslipidemias; Diabetes Mellitus, Type 2
Intervention: Pitavastatin; Atorvastatin
Intervention description: Pitavastatin 4 mg QD; Atorvastatin 20 mg
Intervention MeSH terms: Atorvastatin; Pitavastatin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 418.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Kowa; Type II Diabetes Mellitus; Combined Dyslipidemia; pitavastatin; NK-104; Atorvastatin

Summary:

      The purpose of this study is to compare the efficacy and safety of pitavastatin with that of
      atorvastatin in patients with type II diabetes mellitus (type II DM) and combined
      dyslipidemia.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Males and females (ages 18-75 years)

          -  Type II DM treated with oral anti-diabetic medication (sulfonylurea, metformin,
             glitazones, or combination therapy)

          -  Must have been following a restrictive diet

          -  Diagnosis of combined dyslipidemia

        Exclusion Criteria:

          -  Homozygous familial hypercholesterolemia

          -  Conditions which may cause secondary dyslipidemia

          -  Uncontrolled diabetes mellitus

          -  Abnormal pancreatic, liver, or renal function

          -  Abnormal serum creatine kinase (CK) above the pre-specified level

          -  Significant heart disease
A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
Condition: Factor X Deficiency
MeSH terms: Factor X Deficiency
Intervention: FACTOR X
Phase: Phase 3
Study type: Interventional
Primary purpose: Prevention
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Maximum age: 11 Years
Healthy volunteers: No

Summary:

      The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of
      bleeding when given as routine prophylaxis over 12 months.

      The secondary objectives of the study are:

        1. To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.

        2. To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26
           weeks).
    

Eligibility criteria:

        Inclusion Criteria

          1. Children with hereditary severe or moderate FX deficiency (FX:C <5 IU/dL), based on
             their lowest reliable FX:C recorded.

          2. Children under 12 years old, whose parent/guardian has given informed consent.

          3. Children with a history of severe bleeding e.g.: intracranial haemorrhage, before
             starting prophylactic therapy, OR a mutation in the F10 gene causing a documented
             severe bleeding phenotype.

        Exclusion Criteria

          1. Children must not suffer from clinically significant liver disease, renal disease, or
             other coagulopathy or thrombophilia

          2. Children must have no history or suspicion of inhibitors to factor X.

          3. Children who have known or suspected hypersensitivity to the investigational medicinal
             product or its excipients.

          4. Children with a history of unreliability or non-cooperation.

          5. Children who are participating or have taken part in another trial within the last 30
             days.

          6. Children planning more than 4 weeks' continuous absence from the locality of the
             investigational site, between the Screening Visit and the End of Study Visit at
             approximately 6 months (26 weeks) post-Baseline.
A Study of Subcutaneous Mircera for the Treatment of Anemia in Peritoneal Dialysis Patients.
Condition: Anemia
MeSH terms: Anemia
Intervention: Epoetin alfa; methoxy polyethylene glycol-epoetin beta [Mircera]
Intervention description: As prescribed, SC; 120-360 micrograms SC monthly, starting dose
Intervention MeSH terms: Epoetin Alfa
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 80.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This 2 arm study will compare the efficacy of monthly Mircera and epoetin alfa in peritoneal
      dialysis patients who self-inject at home or receive in-centre injections. The safety of
      subcutaneous (sc) Mircera and injection site reactions and patient satisfaction will also be
      assessed. Eligible patients will be randomized either to receive monthly sc injections of
      Mircera (and will be switched from sc epoetin alfa) at a starting dose of 120-360 micrograms,
      or to remain on standard of care sc epoetin alfa. Dose adjustments will be permitted to
      reach/maintain a hemoglobin level of 10-12g/dL. The anticipated time on study treatment is
      3-12 months, and the target sample size is 380 individuals.
    

Eligibility criteria:

        Inclusion Criteria:

          -  adult patients, >=18 years of age;

          -  chronic kidney disease stage V;

          -  on peritoneal dialysis for 3 months prior to screening;

          -  on epoetin alfa sc >=3 months prior to screening.

        Exclusion Criteria:

          -  patients expecting to change dialysis modality over course of study;

          -  patients hospitalized during previous 3 months for any clinically significant
             condition;

          -  active malignancy;

          -  bleeding episode necessitating transfusion, or overt gastrointestinal bleeding within
             3 months prior to screening;

          -  transfusion of red blood cells within 3 months prior to screening.
Safety and Tolerability Study of NBI-98854 for the Treatment of Tardive Dyskinesia
Condition: Tardive Dyskinesia
MeSH terms: Dyskinesias; Tardive Dyskinesia
Intervention: NBI-98854
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 167.0
Sex: All
Minimum age: 18 Years
Maximum age: 85 Years
Healthy volunteers: No

Summary:

      Phase 3, open-label, study to evaluate the safety and tolerability of NBI-98854 administered
      once daily (qd) for a total of 48 weeks of treatment. This study will enroll approximately
      150 medically stable male and female subjects with clinical diagnoses of schizophrenia or
      schizoaffective disorder with neuroleptic-induced TD or mood disorder with
      neuroleptic-induced TD.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subjects of childbearing potential must agree to use hormonal or two forms of
             nonhormonal contraception (dual contraception) consistently during the screening,
             treatment and follow-up periods of the study.

          2. Female subjects must not be pregnant.

          3. Have one of the following clinical diagnoses for at least 3 months prior to screening:
             Schizophrenia or Schizoaffective Disorder, or Mood Disorder

          4. Have a clinical diagnosis of neuroleptic-induced TD for at least 3 months prior to
             screening.

          5. Have moderate or severe TD

          6. If using maintenance medication(s) for schizophrenia or schizoaffective disorder, or
             mood disorder, be on stable doses.

          7. Be in general good health.

          8. Have adequate hearing, vision, and language skills to perform the procedures specified
             in the protocol.

          9. Have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine,
             phencyclidine, cocaine, opiates, or cannabinoids.

        Exclusion Criteria

          1. Have an active, clinically significant unstable medical condition within 1 month prior
             to screening.

          2. Have a known history of substance dependence, substance (drug) or alcohol abuse.

          3. Have a significant risk of suicidal or violent behavior.

          4. Have a known history of neuroleptic malignant syndrome.

          5. Have a known history of long QT syndrome or cardiac tachy-arrhythmia.

          6. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed).

          7. Have received an investigational drug within 30 days before screening or plan to use
             an investigational drug (other than NBI-98854) during the study.

          8. Have a blood loss ≥550 mL or donated blood within 30 days prior to Baseline.

          9. Have an allergy, hypersensitivity, or intolerance to tetrabenazine.

         10. Are currently pregnant or breastfeeding.
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
Condition: Familial Hypercholesterolemia
MeSH terms: Hyperlipoproteinemia Type II; Homozygous Familial Hypercholesterolemia; Hypercholesterolemia
Intervention: lomitapide
Intervention description: 5-60 mg po every day
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 19.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Homozygous; Familial; Hypercholesterolemia; MTP; Apheresis; lomitapide; Homozygous Familial Hypercholesterolemia (HoFH)

Summary:

      This was a long term follow on study to assess the continued long term safety and efficacy of
      lomitapide in patients with homozygous familial hypercholesterolemia.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Completed UP1002 or 733-005.

          2. Willing and able to provide consent and comply with the requirements of the study
             protocol.

        Exclusion Criteria:

          1. Met any of the stopping rules for study discontinuation at the final visit of study
             UP1002 or 733-005.
Study of CP-690,550 Versus Placebo In Rheumatoid Arthritis Patients On Background Methotrexate With Inadequate Response To Tumor Necrosis Factor (TNF) Inhibitors
Condition: Arthritis, Rheumatoid
MeSH terms: Arthritis; Arthritis, Rheumatoid
Intervention: CP-690,550; CP-690,550; Placebo; CP-690,550; Placebo; CP-690,550
Intervention description: Oral tablets administered at 5 mg BID daily for 6 months during the double-blind, placebo-controlled period.; Oral tablets administered at 10 mg BID daily for 6 months during the double-blind, placebo-controlled period.; Oral placebo tablets administered BID daily during the first 3 months of the double-blind, study period.; Oral tablets administered at 5 mg BID daily during the second 3 months of the double-blind, study period.; Oral placebo tablets administered BID daily during the first 3 months of the double-blind, placebo-controlled period.; Oral tablets administered at 10 mg BID daily during the second 3 months of the double-blind, study period.
Intervention MeSH terms: Tofacitinib
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 399.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: randomized double-blind placebo-controlled investigational drug oral therapy safety and efficacy

Summary:

      This study will test if CP-690,550 is safe and effective in rheumatoid arthritis patients
      taking methotrexate who have an inadequate response to tumor necrosis factor inhibitor
      treatment.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Adults with moderate to severe rheumatoid arthritis on a stable dose of methotrexate
             who have inadequate response to Tumor Necrosis Factor (TNF) inhibitors.

        Exclusion Criteria:

          -  Pregnancy, severe acute or chronic medical conditions, including serious infections or
             clinically significant laboratory abnormalities.
Comparison of Safety and Efficacy of Intravenous Iron Versus Oral Iron in Chronic Renal Failure Subjects With Anemia
Condition: Anemia
MeSH terms: Renal Insufficiency; Kidney Failure, Chronic; Anemia
Intervention: Venofer; Ferrous Sulfate
Intervention description: iron sucrose injection; 500 mg intravenous (IV) infusion administered over 3.5-4 hours on Days 0 and 14, or 200 mg injections administered over 2-5 minutes on 5 different occasions from Day 0 to Day 14.; oral iron tablets; 325 mg three times a day orally for 56 days
Intervention MeSH terms: Iron; Ferric Oxide, Saccharated
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 182.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: CKD; iron; anemia

Summary:

      To assess the safety and efficacy of two forms of iron therapy for the treatment of anemia in
      non-dialysis dependent, chronic renal failure in patients receiving or not receiving
      erythropoietin.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Hemoglobin < or = 11.5 gm/dL

          -  Stable dose or not receiving EPO

          -  Renal Anemia

        Exclusion Criteria:

          -  IV iron with last 6 months

          -  Chronic infection, malignancy,major surgery within last month

          -  Blood Transfusion with last two months

          -  Significant blood loss within last 3 months

          -  Concomitant sever diseases of the liver