bioihbjkbjnl/mcl2 huggingface dataset:
A Dose Escalation Study of BLU-5937 in Unexplained or Refractory Chronic Cough
Condition: Chronic Refractory Cough
MeSH terms: Cough
Intervention: BLU-5937; Placebo
Intervention description: Four escalating doses of BLU-5937 administered BID over the course of the study; Matching placebo for BLU-5937
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: Two-arm, Two-Period, crossover assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 68.0
Sex: All
Minimum age: 18 Years
Maximum age: 80 Years
Healthy volunteers: No
Keywords: Unexplained or refractory chronic cough, P2X3 antagonists

Summary:

      This is a multi-center, randomized, double-blind, placebo-controlled, crossover, dose
      escalation study of BLU-5937 in subjects with unexplained or refractory chronic cough
    

Eligibility criteria:

        Inclusion Criteria:

          -  Unexplained or refractory chronic cough for at least one year

          -  Chest radiograph or CT thorax within the last 60 months not demonstrating any
             abnormality considered to be significantly contributing to the chronic cough

          -  Cough count of ≥ 10 per hour (Awake Cough Count) at Screening

          -  Score of ≥ 40mm on the Cough Severity VAS at Screening

          -  Women of child-bearing potential must have a negative serum pregnancy test at
             Screening

          -  Women of child-bearing potential must use a highly effective contraception method from
             Screening through the Follow-Up Visit

          -  Male subjects and their partners of child-bearing potential must use 2 methods of
             acceptable birth control

        Exclusion Criteria:

          -  Current smoker or individuals who have given up smoking within the past 6 months, or
             those with >20 pack-year smoking history

          -  Diagnosis of COPD, bronchiectasis, idiopathic pulmonary fibrosis

          -  Treatment with an ACE-inhibitor as the potential cause of a subject's cough, or
             requiring treatment with an ACE-inhibitor during the study, or within 12 weeks prior
             to the Screening Visit

          -  FEV1/FVC < 60%

          -  History of upper respiratory tract infection or recent significant change in pulmonary
             status within 4 weeks of the Baseline Visit

          -  History of concurrent malignancy or recurrence of malignancy within 2 years prior to
             Screening

          -  History of a diagnosis of drug or alcohol dependency or abuse within the last 3 years

          -  Clinically significant abnormal electrocardiogram (ECG) or laboratory tests at
             Screening

          -  Other severe, acute, or chronic medical or psychiatric condition or laboratory
             abnormality that may increase the risk associated with trial participation or
             investigational product administration or may interfere with the interpretation of
             trial results
ACTIMMUNE in Intermediate Osteopetrosis
Condition: Osteopetrosis
MeSH terms: Osteopetrosis
Intervention: Interferon gamma-1b
Intervention description: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Intervention MeSH terms: Interferons; Interferon-gamma
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 5.0
Sex: All
Minimum age: 1 Year
Healthy volunteers: No

Summary:

      This study evaluates the effects of ACTIMMUNE (IFN-γ1b) in children and adults with
      intermediate osteoporosis. All participants will receive treatment with ACTIMMUNE for 12
      months. The investigators hypothesize that ACTIMMUNE will be tolerated by participants for
      the full 12 months and result in decreased disease severity.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of osteopetrosis; and

          -  Anemia (Hemoglobin <12 g/dL) not related to iron deficiency, or

          -  Neutropenia (Neutrophil count <1000 neutrophils/ul unsupported with cytokines), or

          -  Thrombocytopenia (Platelet count <50,000 cells x 109/L), or

          -  History of impaired bone healing, or

          -  ≥ 1 serious infection over prior year defined as requiring hospitalization and/or IV
             antibiotics, and

          -  Age > 1 year; and

          -  Ability to travel to a study center for every 3-6 month study visits; and

          -  Patient or parent/legal guardian is able and willing to provide informed consent. For
             patients 7 to 17 years of age, assent must also be provided.

        Exclusion Criteria:

          -  12 months or fewer following HCT;

          -  Pregnancy or breastfeeding;

          -  Known or suspected allergy to interferon gamma-1b or related products;

          -  Participation in simultaneous therapeutic study that involves an investigational study
             drug or agent within 4 weeks of study enrollment;

          -  ALT greater than 3 fold higher than normal; or

          -  Any other social or medical condition that the Investigator believes would pose a
             significant hazard to the subject if the investigational therapy were initiated or be
             detrimental to the study.
Auranofin in Decreasing Pain in Patients With Paclitaxel-Induced Pain Syndrome
Condition: Pain
Intervention: auranofin; placebo; questionnaire administration
Intervention description: Given PO; Given PO; Ancillary studies
Intervention MeSH terms: Auranofin
Phase: Phase 2
Study type: Interventional
Primary purpose: Supportive Care
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 30.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This randomized pilot clinical trial studies whether auranofin will relieve pain following
      paclitaxel in patients who have previously experienced paclitaxel-induced pain. Auranofin is
      a drug given by mouth to treat other diseases such as rheumatoid arthritis, and is being
      studied to see if it will decrease pain following paclitaxel.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Absolute neutrophil count (ANC) >= 1500/mm^3

          -  Platelet count (PLT) >= 100,000/mm^3

          -  Creatinine =< 2 x upper limit of normal (ULN)

          -  Either serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT])
             or serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST])
             =< 1.5 x ULN

          -  Total/direct bilirubin =< 1.5 x ULN

          -  Alkaline phosphatase =< 1.5 x ULN

          -  Hemoglobin >= 9 mg/dL

          -  Negative urine or serum pregnancy test performed =< 7 days prior to registration, for
             women of childbearing potential only

          -  Previously experienced paclitaxel induced pain during a current or past paclitaxel
             treatment that the treating healthcare provider thinks is consistent with the
             paclitaxel-induced acute pain syndrome; note: formal documentation of prior pain is
             not required

          -  Scheduled to receive paclitaxel at a dose >= 70 mg/m^2 =< 14 days from randomization

          -  Ability to complete the questionnaires or to do so with assistance

        Exclusion Criteria:

          -  Pregnant women

          -  Nursing women

          -  Any woman of childbearing potential or male partner of a woman of childbearing
             potential unwilling to employ acceptable contraception throughout the study and for at
             least 30 days after the last dose of the study drug

          -  History of gold-induced disorders, including but not limited to, necrotizing
             enterocolitis, pulmonary fibrosis, exfoliative dermatitis, bone marrow aplasias or
             other severe hematologic disorders; history of severe allergic or anaphylactic
             reactions or hypersensitivity to auranofin or other gold compounds

          -  Currently receiving Dilantin (phenytoin) or auranofin or another gold-containing
             compound

          -  Anticipated use of filgrastim (G-CSF) or sargramostim (GM-CSF) within 30 days after
             receiving auranofin

          -  Currently receiving immune-modulating therapies
Safety, Pharmacokinetics, and Preliminary Efficacy Study of CDZ173 in Patients With Primary Sjögren's Syndrome
Condition: Primary Sjögren's Syndrome
MeSH terms: Sjogren's Syndrome; Syndrome
Intervention: CDZ173; Placebo
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 30.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No

Summary:

      This Study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary
      therapeutic efficacy of oral administrations of CDZ173 in patients with primary Sjögren's
      syndrome.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of primary Sjögren's syndrome (pSS)

          -  ESSDAI score ≥ 6 at screening visit

        Exclusion Criteria:

          -  Secondary Sjögren's syndrome

        Other protocol-defined inclusion/exclusion criteria may apply.
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Intravenous Brivaracetam in Subjects >= 1 Month to < 16 Years of Age With Epilepsy
Condition: Epilepsy
MeSH terms: Epilepsy
Intervention: Brivaracetam
Intervention description: Pharmaceutical form: Solution for iv injection
Route of administration: intravenous use
Concentration: 10 mg/ml
Intervention MeSH terms: Brivaracetam
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 50.0
Sex: All
Minimum age: 1 Month
Maximum age: 16 Years
Healthy volunteers: No
Keywords: Epilepsy; Brivaracetam; Briviact; Open-label; pediatric; intravenous

Summary:

      The purpose of the study is to evaluate the pharmacokinetics (PK), safety, and tolerability
      of brivaracetam (BRV) administered intravenously (iv) in subjects >= 1 month to < 16 years of
      age with epilepsy.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female from >= 1 month to < 16 years of age. For subjects who are < 1 year
             from birth and who were preterm infants, the corrected gestational age should be used
             for this entry requirement

          -  Weight >= 3 kg (6.6 lbs)

          -  Diagnosis of epilepsy

          -  Acceptable candidate for venipuncture and intravenous (iv) infusion

          -  Treatment with >=1 anti epileptic drug (AED; including BRV) without a change of dose
             regimen for at least 7 days prior to Screening

          -  No treatment with vagus nerve stimulation (VNS), OR the subject is being treated with
             VNS and the settings have been constant for >=7 days prior to Screening

          -  For female subjects: not of childbearing potential, OR of childbearing potential and
             not sexually active/negative pregnancy test, OR of childbearing potential and sexually
             active/negative pregnancy test/uses medically acceptable contraceptive methods

        Exclusion Criteria:

          -  Subject has previously received iv Brivaracetam (BRV) in this study

          -  Subject is being treated with BRV at a dose >5mg/kg/day (rounded) or >200mg/day for
             subjects with body weights >40kg

          -  Subject requires or is likely to require a change in concomitant antiepileptic drug(s)
             (AED[s]), dose of concomitant AED(s), or formulation of AED(s) during the 7 days prior
             to the intravenous (iv) pharmacokinetic (PK) Period

          -  Subject is likely, in the opinion of the Investigator, to require rescue medication
             during the Initiating Oral BRV (IOB) Treatment or iv PK Periods

          -  Subject has experienced generalized convulsive status epilepticus in the 28 days prior
             to Screening or during the Screening Period
A 6 Month Safety Extension Study of MBGS205
Condition: Hypogonadotropic Hypogonadism
MeSH terms: Hypogonadism
Intervention: BGS649
Intervention description: Capsules will be taken weekly for a maximum of 24 weeks
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 143.0
Sex: Male
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Hypogonadotropic; hypogonadism; testosterone

Summary:

      Phase IIb, multicentre, double-blind, randomised, placebo-controlled parallel-group 36-week
      study evaluating safety and efficacy of BGS649 in male obese subjects with hypogonadotropic
      hypogonadism (HH)
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participating in Study MBGS205 and completion of the 24 week treatment period without
             meeting any discontinuation criteria of Study MBGS205

          -  In opinion of the investigator has been compliant with the requirements of the Study
             MBGS205 protocol.

        Exclusion Criteria:

        -Meeting any of the discontinuation criteria of initial Study MBGS205
Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms
Condition: Spasms, Infantile
MeSH terms: Spasm; Spasms, Infantile
Intervention: Cannabidiol Oral Solution
Intervention description: 20 or 40 mg/kg/day BID
Intervention MeSH terms: Cannabidiol
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Minimum age: 6 Months
Maximum age: 36 Months
Healthy volunteers: No

Summary:

      Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy
      that usually strikes children in the first year of life. There is a great need for safe and
      effective therapies in the treatment of IS. This need is even more important for infants and
      toddlers still sick after being treated with medicine that is already available.

      This is a multi-center study to evaluate the efficacy and safety of Cannabidiol Oral Solution
      (CBD) in the treatment of children aged 6 months through 36 months with a diagnosis of
      infantile spasms who have not responded to first line therapies.

      The overall study duration is expected to be 64 weeks for those subjects who respond to CBD
      treatment. The maximum possible study duration for each patient is approximately 64 weeks,
      however a subject will be deemed to have completed the study after 58 weeks.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Meets protocol-specified criteria for qualification, including infantile spasms

          -  Parent(s)/caregiver(s) fully comprehend and sign the informed consent form, understand
             all study procedures, and can communicate satisfactorily with the Investigator and
             study coordinator.

        Exclusion Criteria:

          -  History or current use of over-the-counter medications, dietary supplements, or drugs
             outside protocol-specified parameters

          -  Signs, symptoms or history of any condition that, per protocol or in the opinion of
             the investigator, might compromise:

               1. the safety or well-being of the participant or study staff

               2. the analysis of results

          -  During the Safety Treatment and Follow-up Periods, subjects are not to receive the
             following:

               1. any cannabinoids (CBD, Δ9-tetrahydrocannabinol (THC), hemp oil, Realm Oil or
                  marijuana)

               2. any other investigational drug or investigational device
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: TAS-205; Placebo
Intervention description: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal; 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 36.0
Sex: Male
Minimum age: 5 Years
Healthy volunteers: No

Summary:

      The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of
      TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Able to give an informed consent. If applicable, able to give an informed assent.

          -  Phenotypic evidence of DMD.

          -  Male and ≧5 years of age.

          -  Bodyweight ≧7.5 kg and <60 kg.

          -  Able to complete the 6MWD test with a distance of at least 75 m.

          -  Able to take tablets.

          -  If taking oral glucocorticoids no significant change in the total daily or dosing 6
             months before enrollment.

        Exclusion Criteria:

          -  Any serious drug allergy.

          -  A forced vital capacity (FVC) of <50% of predicted value.

          -  Wearing a respirator continuously (except for the use during sleep).

          -  A left ventricular ejection fraction (EF) of <40% or fractional shortening (FS) of
             <25% on echocardiogram.

          -  Clinically significant cardiac failure and respiratory failure.

          -  Ongoing immunosuppressive therapy (other than corticosteroids) .

          -  Surgical history or plan for surgery that may affect muscular strength or motor
             function.

          -  Any injury that may affect muscular strength or motor function.

          -  With any systemic allergic disease or any chronic inflammatory disease.

          -  Previous gene therapy (exon skipping, or stop codon read through therapy), cell-based
             therapy, or any other investigational agents.
A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Condition: Haemostasis; Haemophilia A
MeSH terms: Hemophilia A
Intervention: Concizumab; Turoctocog alfa
Intervention description: 0.15 mg/kg (with potential stepwise dose administration to 0.25 mg/kg) administered daily s.c (subcutaneously, under the skin). Treatment duration is 24 weeks in the main phase, and 52 weeks in the extension phase; Breakthrough bleeding episodes will be treated by the patients at home with turoctocog alfa at the discretion of the study doctor, who will also choose dose levels
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 36.0
Sex: Male
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This trial is conducted in Asia, Europe and the United States of America (USA). The aim of
      the trial is to assess the efficacy of concizumab administered s.c. (subcutaneously, under
      the skin) once daily in preventing bleeding episodes in patients with severe haemophilia A
      without inhibitors.
    

Eligibility criteria:

        Inclusion Criteria: - Informed consent obtained before any trial-related activities.
        Trial-related activities are any procedures that are carried out as part of the trial,
        including activities to determine the suitability for the trial - Male patients aged 18
        years or older at the time of signing informed consent, diagnosed with severe haemophilia A
        (FVIII activity below 1%), based on medical records or results at screening Exclusion
        Criteria: - Known or suspected hypersensitivity to trial product(s) or related products -
        Known inherited or acquired bleeding disorder other than haemophilia A - Presence of
        inhibitors (neutralising antibodies) to Factor VIII (equal to or above 0.6 Bethesda Units)
        at screening measured by the Nijmegen method
Antihistamines in Eosinophilic Esophagitis
Condition: Eosinophilic Esophagitis
MeSH terms: Esophagitis; Eosinophilic Esophagitis
Intervention: Famotidine; Loratadine; Placebo
Intervention description: 40 mg tab twice daily by mouth for 12 week duration; 10 mg tab once daily by mouth for 12 week duration; Contains no active ingredient
Intervention MeSH terms: Loratadine; Famotidine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Participant)
Enrollment: 1.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      Researchers are assessing the safety and effectiveness of antihistamines in the treatment of
      eosinophilic esophagitis.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients over the age of 18, male and female.

          -  Patients who carry the diagnosis of Eosinophilic Esophagitis (EoE) based on esophageal
             biopsies obtained within 6 months prior to enrollment (>15 eosinophils per hpf on at
             least 2 esophageal levels)

          -  Subjects must be able to give appropriate informed consent

        Exclusion Criteria:

          -  Not willing or able to sign consent.

          -  Patients who have used topical or systemic corticosteroid therapy for any reason in
             the previous 6 weeks.

          -  Patients who have been treated with acid-suppressing medications (PPI or H2 receptor
             antagonists) in the previous 6 weeks.

          -  Patients on immunosuppressive or immunomodulating medications in the previous 6 weeks.

          -  Patients with known allergies or hypersensitivity to anti-histamines.

          -  Patients who have contraindications to the procurement of esophageal biopsies
             including patients who have known bleeding disorders, a history of bleeding diathesis,
             or who are currently using warfarin or clopidogrel.

          -  Patients who have a contraindication to the performance of an
             esophagogastroduodenoscopy (EGD) including previous cardiopulmonary arrest during an
             endoscopic procedure.

          -  Patients who are pregnant.