bioihbjkbjnl/mcl2 huggingface dataset:
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Condition: Muscular Dystrophy, Duchenne
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: Edasalonexent; Placebo
Intervention description: 100 mg/kg/day; Placebo
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 131.0
Sex: Male
Minimum age: 4 Years
Maximum age: 7 Years
Healthy volunteers: No
Keywords: Muscular Dystrophies; Musculoskeletal Diseases; Neuromuscular Diseases; Duchenne muscular dystrophy; DMD; dystrophin; dystrophy; Duchenne

Summary:

      The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of
      edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male
      patients from 4-7 years of age (up to 8th birthday) will be enrolled.

      Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the key
      link between loss of dystrophin and disease pathology and plays a fundamental role in the
      initiation and progression of skeletal and cardiac muscle disease in DMD.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Written consent/assent by patient and/or legal guardian as per regional and/or
             Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements

          -  Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase
             (CK) and documentation of mutation(s) in the dystrophin gene known to be associated
             with a DMD phenotype

          -  Able to perform stand from supine without assistance in ≤ 10 seconds

          -  Able to perform the 10MWT and 4-stair climb

          -  Followed by a doctor or medical professional who coordinates Duchenne care on a
             regular basis and willingness to disclose patient's study participation with medical
             professionals

        Exclusion Criteria:

          -  Use of corticosteroids within 24 weeks prior to Day 1; use of inhaled, intranasal, and
             topical corticosteroids is permitted

          -  Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4
             weeks. Exception: Patients who have received at least 24 weeks of a stable dose of
             eteplirsen prior to Day 1, and expected to continue treatment, will be eligible

          -  Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy,
             warfarin, phenytoin, S mephenytoin, cyclosporine, dihydroergotamine, ergotamine,
             fentanyl, alfentanil, pimozide, quinidine, sirolimus, tacrolimus, or paclitaxel

          -  Use of human growth hormone within 3 months prior to Day 1

          -  Other prior or ongoing significant medical conditions
A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: ELX/TEZ/IVA; IVA
Intervention description: Fixed dose combination (FDC) tablets for oral administration.; Tablets for oral administration.
Intervention MeSH terms: Ivacaftor; Elexacaftor
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 69.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      This study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on
      glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the
      F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose
      metabolism.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Heterozygous for F508del and an MF mutation (F/MF genotypes)

          -  Forced expiratory volume in 1 second (FEV1) value ≥ 30% of predicted mean for age,
             sex, and height

          -  Abnormal glucose tolerance determined by an OGTT as either:

               -  Impaired glucose tolerance (IGT) defined as 2 hour post OGTT blood glucose level
                  ≥140 to <200 mg/dL (≥7.77 to <11.10 mmol/L) and fasting blood glucose level <126
                  mg/dL (<7.00 mmol/L)

               -  CF-related diabetes (CFRD) defined as either fasting hyperglycemia (blood glucose
                  level ≥126 mg/dL [≥7.00 mmol/L] after an 8-hour fast) or 2-hour post OGTT blood
                  glucose level ≥200 mg/dL (≥11.10 mmol/L)

        Key Exclusion Criteria:

          -  Clinically significant liver cirrhosis with or without portal hypertension

          -  Solid organ or hematological transplantation

          -  Lung infection with organisms associated with a more rapid decline in pulmonary status

          -  Type 1 or Type 2 diabetes

          -  Duration of CFRD ≥5 years

        Other protocol defined Inclusion/Exclusion criteria may apply.
Hydroxychloroquine for the First Thrombosis Prevention in Antiphospholipid Antibody Positive Patients
Condition: Antiphospholipid Syndrome
MeSH terms: Thrombosis; Antiphospholipid Syndrome
Intervention: Hydroxychloroquine
Intervention MeSH terms: Hydroxychloroquine
Phase: Phase 3
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 11.0
Sex: All
Minimum age: 18 Years
Maximum age: 60 Years
Healthy volunteers: No
Keywords: antiphospholipid autoantibodies; antiphospholipid positive

Summary:

      In this multi-center international study, our aim is to determine the effectiveness of HCQ
      for primary thrombosis prophylaxis in persistently aPL-positive but thrombosis-free patients
      without systemic autoimmune diseases.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Persistent(at least 12 weeks apart)aPL-positivity within 12 months prior to the
             screening defined as:

               -  aCL IgG/M (>40U,medium-to-high titer,and/or greater than the 99th
                  percentile)and/or

               -  aβ2GPI IgG/M(>40U, medium-to-high titer, and/or greater than the 99th
                  percentile)and/or

               -  Positive LA test based on the International Society of Thrombosis & Haematosis
                  Recommendations

        Selected Exclusion Criteria:

          -  History of thrombosis (arterial, venous, and/or biopsy proven microthrombosis

          -  History of Transient Ischemic Attack Confirmed by a Neurologist

          -  SLE Diagnosis based on the ACR Classification Criteria > 4/11

          -  Other Systemic Autoimmune Diseases diagnosed based on ACR Classification Criteria

          -  Current Hydroxychloroquine or another antimalarial treatment (-3 months)

          -  Current warfarin treatment (-3 months)

          -  Current heparin therapy( -3 months)

          -  Current pregnancy

          -  History of Hydroxychloroquine eye toxicity

          -  History of Hydroxychloroquine allergy

          -  Known glucose-6-phosphate dehydrogenase deficiency
Clinical Safety Study of the Progesterone Vaginal Ring in Women Undergoing Assisted Reproductive Technology Procedures
Condition: Progesterone Supplementation in Women Undergoing ART
Intervention: Progesterone vaginal ring
Intervention description: A flexible, non-degradable PVR containing progesterone in micronized formulation and dispersed evenly throughout the ring.
Intervention MeSH terms: Progesterone
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Masking description: Open-label trial
Enrollment: 352.0
Sex: Female
Minimum age: 18 Years
Maximum age: 34 Years
Healthy volunteers: No
Keywords: Progesterone vaginal ring (PVR); Vaginal progesterone ring; Assisted reproductive technology (ART); Infertility

Summary:

      The purpose of the study is to assess the safety of Progesterone Vaginal Ring (PVR) in women
      undergoing fresh embryo transfer (ART).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Pre-menopausal women aged 18-34 at the time of consent.

          -  Documentation of a normal uterine cavity by hysteroscopy, hydrosonogram, or
             hysterosalpingogram within one year of screening.

          -  Normal Pap smear test within 24 months of screening.

          -  At least one cycle without reproductive hormone medication prior to screening
             follicle-stimulating hormone (FSH) and estradiol blood draw.

          -  Tubal, idiopathic, male factor, ovulatory dysfunction, or endometriosis-linked
             infertility.

        Exclusion Criteria:

          -  Body mass index greater than 38 kg/m^2.

          -  FSH greater than 15 IU/L during the early follicular phase (Day 2-4). For those
             participants with polycystic ovarian syndrome, a Day 2-4 FSH level can be obtained
             following a progestogen withdrawal or spontaneous menses.

          -  Clinically significant gynecologic pathology, such as submucosal fibroids, intramural
             fibroids >5 cm, communicating hydrosalpinx, uncorrected uterine septum, endometrial
             cancer or endometrial atypia, scar tissue inside the cavity or poorly developed
             uterine lining from prior uterine surgery, pelvic tuberculosis, or any other
             conditions that could adversely affect pregnancy success.

          -  Uncontrolled elevation of prolactin or too little thyroid hormone in the blood.

          -  History of more than one failed fresh in vitro fertilization cycle. A failed cycle is
             defined as having started a cycle and not becoming pregnant or pregnancy loss prior to
             the 20th week of pregnancy.
A Trial of CM-AT in Children With Autism With All Levels of FCT (The Blum Study)
Condition: Autism
MeSH terms: Autistic Disorder
Intervention: CM-AT; PLACEBO
Intervention description: Single unit dose powder of active substance (CM-AT) administered 3 times per day for 90 days; Single unit dose powder of non-active substance administered 3 times per 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: 190.0
Sex: All
Minimum age: 3 Years
Maximum age: 8 Years
Healthy volunteers: Accepts Healthy Volunteers
Keywords: Autism

Summary:

      The purpose of this study is to determine whether CM-AT is safe and effective in treating the
      core symptoms of autism in children with all levels of fecal chymotrypsin.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Meets the current Diagnostic and Statistical Manual with Mental Disorders (DSM-IV-TR)
             for Autism (Autistic Disorder), screened by SCQ and confirmed by ADI-R;

        Exclusion Criteria:

          -  Patient weighing < 13kg (28.6 lbs)

          -  Previous allergy to porcine (pork) products

          -  Previous history of severe head trauma or stroke, loss of consciousness, seizure (or
             need for seizure medication either present or past) within one year of entering study
             or uncontrolled systemic disease

          -  Diagnosis of: HIV, cerebral palsy, endocrine disorder, pancreatic disease, muscular
             dystrophy, known genetic disorder, blood dyscrasia, ongoing GI disease

          -  Evidence of severe, moderate or uncontrolled systemic disease; and/or any co-morbid
             condition which in the Investigator's or Medical Director's opinion makes it
             undesirable for the subject to participate in the study or jeopardizes compliance with
             the protocol;

          -  Within 30 days of starting the study, certain supplementation, chelation or dietary
             restriction (a 30 day washout period would be required for inclusion);

          -  Ongoing dietary restriction for allergy or other reasons except nut allergies
             (lactose-free allowable);

          -  Use of of any stimulant medication must be discontinued 5 days prior to entering the
             study.

          -  Subject must have a stable dose of SSRI's for at least 30 days.

          -  Inability to ingest study drug and/or follow prescribed dosing schedule
A Study of Duloxetine (LY248686) in Japanese Children and Adolescents With Depressive Disorder
Condition: Depressive Disorder
MeSH terms: Depressive Disorder; Depression
Intervention: Duloxetine Hydrochloride; Placebo
Intervention description: Administered orally; Administered orally
Intervention MeSH terms: Duloxetine Hydrochloride
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 149.0
Sex: All
Minimum age: 9 Years
Maximum age: 17 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to determine the efficacy of duloxetine hydrochloride versus
      placebo in the treatment of Japanese children and adolescents with depressive disorder.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participants diagnosed with Major Depressive Disorder or persistent depressive
             disorder and completely meet the criteria of major depressive episode as defined by
             the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) with
             the Mini International Neuropsychiatric Interview for Children and Adolescents
             (MINI-KID) ver.7.0.2.

          -  Participants whose incipient age of depression was ≥7 years old.

          -  Total score of CDRS-R is ≥40 and CGI-S score is ≥4 at both screening and baseline.

        Exclusion Criteria:

          -  Have remarkable response to psycho-education (defined as >30% decrease in the total
             score of CDRS-R between screening and baseline).

          -  Have a current or previous diagnosis (DSM-5) of the following as judged by the
             investigator:

               -  Neurodevelopmental disorders

               -  Schizophrenia spectrum and other psychotic disorders

               -  Bipolar and related disorders

               -  Trauma and stressor-related disorders

               -  Disruptive · Impulse Control · and Conduct disorders

          -  Have a current diagnosis (DSM-5) of the following as judged by the investigator:

               -  Obsessive-compulsive and related disorders

               -  Anorexia nervosa, Bulimia nervosa, Binge-eating disorder

               -  Sleep-wake disorders

               -  Neurocognitive disorders

               -  Disruptive mood dysregulation disorder

          -  Have personality disorders, in the judgement of the investigator.
Follow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES)
Condition: Acquired Thrombotic Thrombocytopenic Purpura
MeSH terms: Purpura; Purpura, Thrombocytopenic; Purpura, Thrombotic Thrombocytopenic
Intervention: Caplacizumab; Standard of Care
Intervention description: • PE with plasma (e.g., fresh frozen plasma, solvent detergent/viral-inactivated plasma, cryosupernatant), • Corticosteroid treatment and • Use of other immunosuppressive agents (e.g., rituximab).
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 104.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      The objectives of this study were to evaluate long-term safety and efficacy of caplacizumab,
      to evaluate safety and efficacy of repeated use of caplacizumab and to characterize long-term
      impact of acquired thrombotic thrombocytopenic purpura (aTTP).
    

Eligibility criteria:

        Inclusion Criteria:

          1. Completed the Final (28 day) follow-up visit in Study ALX0681-C301.

          2. >= 18 years of age at the time of signing the informed consent form.

          3. Provided informed consent prior to initiation of any study specific
             activity/procedure.

        Exclusion Criteria:

          1. Not being able/willing to comply with the study protocol procedures.

          2. Currently enrolled in a clinical study with another investigational drug or device.
Efficacy and Safety Study of a Test Naproxen Sodium 220mg Tablet in Postoperative Dental Pain
Condition: Post-operative Dental Pain
MeSH terms: Toothache
Intervention: Test naproxen sodium tablet; Commercial naproxen sodium tablet; Commercial naproxen sodium liquid gels capsule; Placebo tablet
Intervention description: Single dose of 2 test naproxen sodium 220 mg tablets; Single dose of 2 naproxen sodium 220 mg tablets; Single dose of 2 naproxen sodium 220 mg liquid gel capsules; 2 placebo tablets
Intervention MeSH terms: Naproxen
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Post-operative dental pain following third molar extraction.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 501.0
Sex: All
Minimum age: 17 Years
Maximum age: 50 Years
Healthy volunteers: No

Summary:

      To evaluate analgesic onset, efficacy, and safety of a single dose of 440 mg of naproxen
      sodium administered as two Test Naproxen Sodium 220 mg tablets compared with two commercial
      naproxen sodium products (two naproxen sodium 220 mg tablets and two naproxen sodium 220 mg
      liquid gels capsules) and placebo in the dental pain model following third-molar extractions.
    

Eligibility criteria:

        Inclusion Criteria:

          1. 17 - 50 years old

          2. Weigh 100 pounds or greater and have a body mass index (BMI) of 17.5 to 30.4
             (inclusive) at screening

          3. Dental extraction of three or four third molars

          4. Meets post-surgical pain

          5. Females of childbearing potential and males agree to contraceptive requirements of
             study

          6. Have a negative urine drug screen at screening, and on day of surgical procedure

        Exclusion Criteria:

          1. Pregnant female, breastfeeding, trying to be pregnant or male with pregnant partner or
             partner currently trying to become pregnant

          2. Have a known allergy or hypersensitivity to naproxen or other NSAIDs, including
             aspirin, or to acetaminophen, hydrocodone or other opioids

          3. Not able to swallow large tablets or capsules

          4. History of any condition (s) in investigator's opinion, may jeopardize subject safety,
             well-being and integrity of study

          5. Use analgesics 5 or more times per week

          6. History of chronic tranquilizer use, heavy drinking, substance abuse as judged by
             investigator site staff within last 5 years

          7. Use of immunosuppressive drugs within 2 weeks of screening

          8. History of endoscopically documented peptic ulcer disease or bleeding disorder in last
             2 years
A Study of Galcanezumab in Participants With Chronic Cluster Headache
Condition: Chronic Cluster Headache
MeSH terms: Cluster Headache; Headache
Intervention: Galcanezumab 300 mg; Placebo
Intervention description: Administered SC; Administered SC
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 240.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No

Summary:

      The main purpose of this study is to evaluate the efficacy of the study drug known as
      galcanezumab in participants with chronic cluster headache.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participants with a history of chronic cluster headache occurring without a remission
             period, or with remissions lasting <1 month, for at least 1 year.

          -  Participants are able to distinguish cluster headache attacks from other headaches.

        Exclusion Criteria:

          -  Current enrollment in or discontinuation within the last 30 days from, a clinical
             trial involving any investigational drug or device.

          -  Current use or any prior exposure to any calcitonin-gene-related peptide (CGRP)
             antibody, any antibody to the CGRP receptor, or antibody to nerve growth factor (NGF).

          -  Are taking indomethacin and/or are suspected of having another distinct trigeminal
             autonomic cephalalgia.

          -  A history of migraine variants that could implicate or could be confused with
             ischemia.

          -  Known hypersensitivity to multiple drugs, monoclonal antibodies or other therapeutic
             proteins.

          -  A history or presence of other medical illness that indicates a medical problem that
             would preclude study participation.

          -  Evidence of significant active or unstable psychiatric disease, in the opinion of the
             investigator.

          -  Women who are pregnant or nursing.
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
Condition: Polycythemia Vera
MeSH terms: Polycythemia Vera; Polycythemia
Intervention: Best Available Therapy; Ruxolitinib
Intervention description: Best Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib.; Ruxolitinib was administered at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 149.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Polycythemia Vera; Hematologic Diseases; Myeloproliferative Disorders; Bone Marrow Diseases; Hydroxyurea; INC424; Ruxolitinib

Summary:

      This study compared the efficacy and safety of ruxolitinib to Best Available Therapy (BAT) in
      patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and
      did not have a palpable spleen.
    

Eligibility criteria:

        Inclusion Criteria:

        Confirmed diagnosis of PV according to the 2008 World Health Organization criteria,
        Non-palpable spleen, Phlebotomy dependent, Resistant to or intolerant of hydroxyurea, ECOG
        performance status of 0, 1 or 2.

        Exclusion Criteria:

        Inadequate liver or renal function, Significant bacterial, fungal, parasitic, or viral
        infection requiring treatment, Active malignancy within the past 5 years, excluding
        specific skin cancers, Previously received treatment with a JAK inhibitor, Being treated
        with any investigational agent, Women who are pregnant or nursing.

        Other inclusion/exclusion criteria apply.