bioihbjkbjnl/mcl2 huggingface dataset:
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
Condition: Hereditary Angioedema, HAE
MeSH terms: Angioedema; Angioedemas, Hereditary
Intervention: BCX7353 capsules; BCX7353 capsules; Placebo oral capsule
Intervention description: BCX7353 capsules administered orally once daily; BCX7353 capsules administered orally once daily; Matching placebo capsules administered orally once daily
Intervention MeSH terms: Berotralstat
Phase: Phase 3
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 19.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No
Keywords: BCX7353

Summary:

      This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to
      evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in
      patients with Type I and Type II HAE who live in Japan.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  A clinical diagnosis of hereditary angioedema (HAE) Type 1 or Type 2, defined as
             having a C1-INH functional level and a C4 level below the lower limit of the normal
             (LLN) reference range, as assessed during the Screening period.

          -  Access to and ability to use one or more acute medications approved by the relevant
             competent authority for the treatment of acute attacks of HAE

          -  Subjects must be medically appropriate for on-demand treatment as the sole medicinal
             management for their HAE during the study.

          -  Subjects must have a specified number of expert-confirmed attacks during the run-in
             period of 56 days from the Screening visit.

          -  Acceptable effective contraception

          -  Written informed consent

        Key Exclusion Criteria:

          -  Pregnancy or breast-feeding

          -  Any clinically significant medical condition or medical history that, in the opinion
             of the Investigator or Sponsor, would interfere with the subject's safety or ability
             to participate in the study

          -  Any laboratory parameter abnormality that, in the opinion of the Investigator, is
             clinically significant and relevant for this study

          -  Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/
             anaphylaxis with unclear etiology

          -  Use of C1-INH within 14 days or use of androgens or tranexamic acid within 28 days
             prior to the Screening visit for prophylaxis of HAE attacks, or initiation of these
             drugs during the study

          -  Current participation in any other investigational drug study or received another
             investigational drug within 30 days of the Screening visit

          -  Prior enrollment in a BCX7353 study
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: IVA
Intervention description: Granules in sachet for oral administration.
Intervention MeSH terms: Ivacaftor
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 57.0
Sex: All
Minimum age: 1 Month
Maximum age: 24 Months
Healthy volunteers: No

Summary:

      The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of
      ivacaftor and metabolites in participants with cystic fibrosis (CF) who are <24 months of age
      at treatment initiation and have an ivacaftor-responsive CF transmembrane conductance
      regulator (CFTR) gene mutation.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Confirmed diagnosis of CF by sweat chloride value or CF mutation criteria.

          -  Have 1 of the following 10 CFTR mutations on at least 1 allele: G551D, G178R, S549N,
             S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H (eligible in regions where
             ivacaftor is approved for use). Part A/B group may also have other
             ivacaftor-responsive mutations.

          -  Hematology, serum chemistry, and vital signs results at screening with no clinically
             significant abnormalities that would interfere with the study assessments, as judged
             by the investigator.

        Exclusion Criteria:

          -  History of any illness or condition that, in the opinion of the investigator, might
             confound the results of the study or pose an additional risk in administering study
             drug to the participant

          -  Colonization with organisms associated with a more rapid decline in pulmonary status
             at screening (Only for Parts A and B)

          -  History of abnormal liver function or abnormal liver function at screening

          -  History of solid organ or hematological transplantation

          -  Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A
             within 2 weeks before Day 1

          -  Participation in a clinical study involving administration of either an
             investigational or a marketed drug within 30 days or 5 terminal half-lives before
             screening

          -  Hemoglobin (Hgb) <9.5 g/dL at screening

          -  Chronic kidney disease of Stage 3 or above

          -  Presence of a non-congenital or progressive lens opacity or cataract at Screening

        Other protocol defined Inclusion/Exclusion Criteria may apply.
Study of Cingal for the Relief of Knee Osteoarthritis Compared to Triamcinolone Hexacetonide at 39 Weeks Follow up
Condition: Knee Osteoarthritis
MeSH terms: Osteoarthritis; Osteoarthritis, Knee
Intervention: Cingal; Monovisc; Triamcinolone Hexacetonide
Intervention description: Hyaluronic Acid with Triamcinolone Hexacetonide; Hyaluronic Acid; Triamcinolone Hexacetonide
Intervention MeSH terms: Triamcinolone; Triamcinolone Acetonide; Triamcinolone hexacetonide; Triamcinolone diacetate
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: In the 16-02 study, subjects were randomized in a 4:4:1 ratio into the Cingal, Monovisc (hyaluronic acid) or TH (triamcinolone hexacetonide) arm. The 17-02 study is a 39-week follow-up to the 16-02 study.
Masking: Double (Participant, Outcomes Assessor)
Masking description: Study subjects and the Outcomes Assessor are blinded to the study treatment.
Enrollment: 526.0
Sex: All
Minimum age: 40 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Osteoarthritis; Intra-articular Injection; Hyaluronic Acid; Triamcinolone Hexacetonide

Summary:

      Extension study to Cingal 16-02 to obtain 39-week safety and efficacy follow-up data
    

Eligibility criteria:

        Inclusion Criteria:

          1. Only subjects that were enrolled and met the inclusion criteria for the Cingal 16-02
             trial and signed the informed consent are eligible for Cingal 17-02 trial.

          2. Subject is able to understand and comply with the requirements of Cingal 17-02 and
             voluntarily provides consent.

        Exclusion Criteria:

        Patients will not be rescreened at enrollment to Cingal 17-02 as these patients met the
        inclusion / exclusion criteria for the Cingal 16-02 clinical trial.
OTX-14-007: A Phase 3 Study Evaluating the Safety and Efficacy of OTX-DP for the Treatment of Chronic Allergic Conjunctivitis
Condition: Chronic Allergic Conjunctivitis
MeSH terms: Conjunctivitis; Conjunctivitis, Allergic
Intervention: Dexamethasone; Placebo Vehicle
Intervention MeSH terms: Dexamethasone
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 73.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: Accepts Healthy Volunteers

Summary:

      The objective of the study was to evaluate the efficacy and safety of OTX-DP (dexamethasone
      insert) 0.4 mg for intracanalicular use when placed in the canaliculus of the eyelid for the
      treatment of the signs and symptoms of chronic allergic conjunctivitis
    

Eligibility criteria:

        Inclusion Criteria:

          -  Has a positive history of ocular allergies and a positive skin test reaction to a
             perennial allergen and a seasonal allergen

          -  Has a positive bilateral CAC reaction to a perennial allergen within minutes of
             instillation

        Exclusion Criteria:

          -  History of refractive surgery (including LASIK procedures)

          -  History of retinal detachment, diabetic retinopathy, or active retinal disease

          -  Presence of an active ocular infection or positive history of an ocular herpetic
             infection at any visit

          -  Use any of the disallowed medications during the period indicated

          -  History of IOP increase as a result of steroid treatment
Once-Daily Investigational Nasal Spray In Adults And Adolescents With Vasomotor Rhinitis
Condition: Vasomotor Rhinitis; Rhinitis, Vasomotor
MeSH terms: Rhinitis; Rhinitis, Vasomotor
Intervention: GW685698X
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 350.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No
Keywords: nonallergic rhinitis; GW685698X; VMR; vasomotor rhinitis

Summary:

      The primary objective of this study is to compare the efficacy and safety of GW685698X 100mcg
      once daily (QD) aqueous nasal spray with vehicle placebo nasal spray in adult and adolescent
      subjects (12 years of age and older) with vasomotor rhinitis (VMR).
    

Eligibility criteria:

        Inclusion criteria:

          -  Must be outpatients.

          -  Diagnosis of VMR (vasomotor rhinitis).

          -  Literate in English or native language.

        Exclusion criteria:

          -  Significant concomitant medical condition.

          -  Use corticosteroids or other allergy medications during the study.

          -  Used tobacco products within the past year.
Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis
Condition: Refractory Generalized Myasthenia Gravis
MeSH terms: Myasthenia Gravis; Muscle Weakness
Intervention: Eculizumab; Placebo
Intervention description: Intravenous administration of eculizumab.; Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
Intervention MeSH terms: Eculizumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Intervention model description: Following the Blind Induction Phase, all participants received open-label eculizumab.
Masking: None (Open Label)
Masking description: The study consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, followed by an Open-Label Maintenance Phase.
Enrollment: 117.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: gMG; Myasthenia Gravis; safety; efficacy

Summary:

      To evaluate the safety and efficacy of eculizumab in the treatment of refractory generalized
      myasthenia gravis (gMG) as an extension study for the participants who previously completed
      Study ECU-MG-301(NCT01997229).
    

Eligibility criteria:

        Inclusion Criteria:

          1. Participant has completed Study ECU-MG-301.

          2. Participant has given written informed consent.

          3. Participant was willing and able to comply with the protocol requirements for the
             duration of the study.

          4. Female participant of childbearing potential must have had a negative pregnancy test
             (serum human chorionic gonadotropin). All participants were required to practice an
             effective, reliable, and medically approved contraceptive regimen during the study and
             for up to 5 months following discontinuation of treatment.

        Exclusion Criteria:

          1. Participants who withdrew from Study ECU-MG-301 as a result of an adverse event
             related to study drug.

          2. Female participants who were pregnant, breastfeeding, or intended to conceive during
             the course of the study.

          3. Unresolved meningococcal infection

          4. Hypersensitivity to murine proteins or to one of the excipients of eculizumab

          5. Any medical condition or circumstances that, in the opinion of the investigator, might
             have interfered with the participant's participation in the study, posed any added
             risk for the participant, or confounded the assessment of the participants.
Study to Assess the Efficacy and Safety of ACT-541468 (Daridorexant) in Adult and Elderly Subjects Suffering From Difficulties to Sleep
Condition: Insomnia Disorder
MeSH terms: Sleep Initiation and Maintenance Disorders
Intervention: Daridorexant; Placebo
Intervention description: Daridorexant will be administered as tablets, orally, once daily in the evening.; Matching placebo will be administered as tablets, orally, once daily in the evening.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 924.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      The main purpose of this study is to assess efficacy and safety of ACT-541468 (daridorexant)
      in adult and elderly subjects with insomnia disorder. Efficacy will be evaluated on objective
      and subjective sleep parameters.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Signed informed consent prior to any study-mandated procedure;

          -  Male or female aged ≥ 18 years;

          -  Insomnia disorder according to DSM-5 criteria;

          -  Insomnia Severity Index score ≥ 15;

          -  Insufficient sleep quantity as collected subjectively in the sleep diary and validated
             objectively by polysomnography;

          -  Women of childbearing potential must have a negative and urine pregnancy test and use
             the contraception scheme up to at least 30 days after last study treatment intake.

        Exclusion Criteria:

          -  Body mass index below 18.5 or above 40.0 kg/m2;

          -  Any lifetime history of of related breathing disorder, periodic limb movement
             disorder, restless legs syndrome, circadian rhythm disorder, rapid eye movement (REM)
             behavior disorder, narcolepsy, or apnea/hypopnea;

          -  Cognitive behavioral therapy (CBT) only allowed if, the treatment started at least 1
             month prior to Visit 3 and the subject agrees to continue this CBT throughout the
             study;

          -  Self-reported usual daytime napping ≥ 1 hour per day and ≥ 3 days per week;

          -  Acute or unstable psychiatric conditions diagnosed by the Mini International
             Neuropsychiatric Interview;

          -  Mini Mental State Examination (MMSE) score < 25 in subjects ≥ 50 years;

          -  For female subjects: pregnant, lactating or planning to become pregnant during
             projected duration of the study;

          -  History or clinical evidence of any disease or medical condition or treatment, which
             may put the subject at risk of participation in the study or may interfere with the
             study assessments.

          -  Any circumstances or conditions, which, in the opinion of the investigator, may affect
             the subject's full participation in the study or compliance with the protocol.
Durvalumab ± Tremelimumab in Combination With Platinum Based Chemotherapy in Untreated Extensive-Stage Small Cell Lung Cancer (CASPIAN)
Condition: Small Cell Lung Carcinoma Extensive Disease
MeSH terms: Carcinoma; Lung Neoplasms; Small Cell Lung Carcinoma
Intervention: Durvalumab; Tremelimumab; Carboplatin; Cisplatin; Etoposide
Intervention description: IV infusions every 3 weeks for 12 weeks (4 cycles) and every 4 weeks thereafter until PD or other discontinuation criteria.; IV infusions every 3 weeks for 12 weeks(4 cycles). An additional dose of tremelimumab will be administered in the week 16.; up to 4 cycles every 3 weeks in Arm 1 and 2, up to 6 cycles every 3 weeks in Arm 3; up to 4 cycles every 3 weeks in Arm 1 and 2, up to 6 cycles every 3 weeks in Arm 3; up to 4 cycles every 3 weeks in Arm 1 and 2, up to 6 cycles every 3 weeks in Arm 3
Intervention MeSH terms: Carboplatin; Etoposide; Durvalumab; Tremelimumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 987.0
Sex: All
Minimum age: 18 Years
Maximum age: 130 Years
Healthy volunteers: No
Keywords: Carcinoma, Small Cell Lung; Oat Cell Carcinoma of Lung; Oat Cell Lung Cancer; Small Cell Cancer Of The Lung; Small Cell Lung Cancer

Summary:

      This is a phase III, randomized, open-label, multicenter, global study to determine the
      efficacy and safety of combining durvalumab ± tremelimumab with platinum based chemotherapy
      (EP) followed by durvalumab ± tremelimumab maintenance therapy versus EP alone as first-line
      treatment in patients with extensive-stage small-cell lung cancer
    

Eligibility criteria:

        Inclusion criteria:

          1. Histologically or cytologically documented extensive disease. Brain metastases; must
             be asymptomatic or treated and stable off steroids and anti-convulsants for at least 1
             month prior to study treatment.

          2. Suitable to receive a platinum-based chemotherapy regimen as 1st line treatment.

          3. Life expectancy ≥12 weeks at Day 1.

          4. ECOG 0 or 1 at enrolment.

          5. No prior exposure to immune-mediated therapy excluding therapeutic anticancer
             vaccines.

        Exclusion criteria:

          1. Any history of radiotherapy to the chest prior to systemic therapy or planned
             consolidation chest radiation therapy (except paliative care outside of the chest).

          2. Paraneoplastic syndrome of autoimmune nature, requiring systemic treatment or clinical
             symptomatology suggesting worsening of PNS

          3. Active infection including tuberculosis, HIV, hepatitis B anc C

          4. Active or prior documented autoimmune or inflammatory disorders

          5. Uncontrolled intercurrent illness, including but not limited to interstitial lung
             disease.
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Condition: Epidermolysis Bullosa
MeSH terms: Epidermolysis Bullosa
Intervention: SD-101-6.0 cream; Placebo (SD-101-0.0) cream
Intervention description: applied topically once a day for 90 days; applied topically once a day for 90 days
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 169.0
Sex: All
Minimum age: 1 Month
Healthy volunteers: No
Keywords: Amicus Therapeutics; Scioderm, Inc.; Simplex; Recessive Dystrophic; Junctional non-Herlitz; Epidermolysis Bullosa; SD-101 6%; SD-101-6.0; Allantoin 6%; Zorblisa

Summary:

      The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0)
      cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding
      Source - United States Food and Drug Administration (FDA) Office of Orphan Products
      Development (OOPD).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Informed Consent form signed by the participant or participant's legal representative;
             if the participant was under the age of 18 but capable of providing assent, signed
             assent from the participant.

          -  Participant (or caretaker) must have been willing to comply with all protocol
             requirements.

          -  Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

          -  Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.

          -  Participants 1 month and older.

          -  Target wound must have been present for at least 21 days.

        Exclusion Criteria:

          -  Participants who did not meet the entry criteria outlined above.

          -  Selected target wound did not have clinical evidence of local infection.

          -  Use of any investigational drug within the 30 days before enrollment.

          -  Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.

          -  Use of systemic or topical steroidal therapy within the 30 days before enrollment.
             (Inhaled steroids and ophthalmic drops containing steroids were allowed).

          -  Use of systemic antibiotics within the 7 days before enrollment.

          -  Current or former malignancy.

          -  Arterial or venous disorder resulting in ulcerated lesions.

          -  Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at
             screening and every 30 days until the final visit for female participants of
             childbearing potential).

          -  Females of childbearing potential who were not abstinent and not practicing a
             medically acceptable method of contraception.
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Condition: Primary Hyperoxaluria Type 1; Primary Hyperoxaluria
MeSH terms: Hyperoxaluria, Primary
Intervention: Lumasiran
Intervention description: Lumasiran will be administered by subcutaneous (SC) injection.
Intervention MeSH terms: Lumasiran
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 21.0
Sex: All
Healthy volunteers: No
Keywords: PH1; Primary Hyperoxaluria; Hyperoxaluria; RNAi Therapeutic; siRNA; AGT; Systemic Oxalosis

Summary:

      The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and
      pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1
      (PH1).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Has documented diagnosis of primary hyperoxaluria type 1 (PH1)

          -  Estimated glomerular filtration rate (eGFR) ≤45 mL/min/1.73 m^2 for patients ≥12
             months of age (<12 months of age, must have serum creatinine considered elevated for
             age)

          -  Meets plasma oxalate level requirements

          -  If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90
             days

          -  If on dialysis, may be on hemodialysis therapy only and must have been on a stable
             regimen for at least 4 weeks

        Exclusion Criteria:

          -  Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone

          -  Diagnosis of conditions other than PH1 contributing to renal insufficiency

          -  History of liver transplant

          -  History of kidney transplant and currently receiving immunosuppressants