bioihbjkbjnl/mcl2 huggingface dataset:
Safety and Efficacy of LCI699 in Cushing's Disease Patients
Condition: Cushings Disease; Cushing Disease
MeSH terms: ACTH-Secreting Pituitary Adenoma; Pituitary ACTH Hypersecretion
Intervention: LCI699
Intervention description: Osilodrostat 1 mg and 5 mg capsules, was prepared by Novartis and supplied to the Investigator. The capsule formulation of osilodrostat was later changed to tablets and this change was implemented in the study with Protocol amendment 6. Osilodrostat was open labeled 1 mg, 5 mg, 10 mg and 20 mg tablets.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 31.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Cushing Disease; osilodrostat; LCI699; Pituitary Gland; Adrenocorticotropic Hormone

Summary:

      This exploratory study is a proof of concept study to determine whether LCI699 can safely
      reduce the level of urinary free cortisol in patients with Cushing's disease.

      In addition, this study evaluated the long term efficacy and safety of LCI699 including an
      additional 12 week of treatment followed by a 12 month long term optional extension.

      A second extension provided patients who were clinically benefitting from LCI699 an
      opportunity to continue to have access to the drug until LCI699 was commercially available
      and reimbursed or through the availability of a local access program.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients with a confirmed diagnosis of Cushing's Disease (persistent or recurrent) as
             evidenced by increased 24-hour urine free cortisol (UFC), normal or increased morning
             plasma Adrenocorticotropic Hormone (ACTH), and pituitary origin of excess ACTH.

          -  Patients with de novo Cushing's disease can be included only if they are not
             considered candidate for surgery

        Exclusion Criteria:

          -  Patients treated with mitotane 6 months prior to Visit 1

          -  Patients with compression of the optic chiasm

          -  Patients with a known inherited syndrome as the cause for hormone over secretion

          -  Patients with Cushing's syndrome due to ectopic ACTH secretion or adrenal Cushing's
             syndrome

          -  Patients with pseudo-Cushing's syndrome

          -  Patients who are not biochemically euthyroid

          -  Diabetic patients with poorly controlled diabetes (HbA1c >9%)

          -  Women of child-bearing potential, defined as all women physiologically capable of
             becoming pregnant, unless they are using highly effective methods of contraception
             during dosing and for 1 week after completion of dosing.

          -  Patients who have received pituitary irradiation within five years prior to Visit 1.

          -  Patients with risk factors for QTc prolongation or Torsade de Pointes.
A Dose-Finding Study of Vedolizumab for Treatment of Steroid-Refractory Acute Intestinal Graft-Versus-Host Disease (GvHD) in Participants Who Have Undergone Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
Condition: Allogeneic Hematopoietic Stem Cell Transplantation
MeSH terms: Graft vs Host Disease
Intervention: Vedolizumab
Intervention description: Vedolizumab IV infusion
Intervention MeSH terms: Vedolizumab
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 17.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Drug Therapy

Summary:

      The purpose of this study is to assess the initial activity, tolerability, safety and to
      identify a recommended dose and regimen of vedolizumab intravenous (IV) administered for
      treatment of steroid-refractory acute intestinal GvHD in participants who have undergone
      allo-HSCT.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Recipient of 1 allogeneic hematopoietic stem cell transplantation (allo-HSCT) but not
             more than 1 allo-HSCT.

          2. Has primary steroid-refractory graft-versus-host disease (GvHD). Steroid-refractory
             disease is defined as worsening or no improvement in 5 to 7 days of treatment with
             methylprednisolone 2 milligram per kilogram (mg/kg) or equivalent or lack of a CR
             after 14 days of primary treatment with methylprednisolone 2 mg/kg or equivalent. Note
             that participants who develop intestinal GvHD while receiving systemic therapy for
             other GvHD are still eligible after 5 to 7 days, even if the intestinal GvHD has not
             been present for the entire duration. Participants who may have received an increase
             in their steroid dose treatment (example, increased methylprednisolone from 1 mg/kg to
             2 mg/kg) before enrollment will be eligible, provided the participant has met the
             definition of steroid refractory above. Participants who develop toxicity on
             corticosteroids or who are otherwise medically unable to be dosed to this level, will
             also be eligible.

          3. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 3.

          4. Evidence of myeloid engraftment defined by absolute neutrophil count greater than or
             equal to (>=) 0.5*109/liter (L) on 3 consecutive days.

        Exclusion Criteria:

          1. Presence of chronic GvHD at Screening (including acute-chronic overlap syndrome).

          2. Relapse of underlying malignant disease after allo-HSCT.

          3. Hyperacute GvHD defined as onset of GvHD within the first 15 days following
             hematopoietic stem cell infusion.

          4. Received systemic agents other than corticosteroids for treatment of acute GvHD. GvHD
             prophylaxis agents (example, calcineurin inhibitors) may be continued.

          5. Life expectancy of <3 weeks.
Gallium-68 PSMA-11 PET in Patients With Biochemical Recurrence
Condition: Prostate Cancer; Prostate Adenocarcinoma; Prostate Cancer Metastatic; Prostate Cancer Recurrent
MeSH terms: Prostatic Neoplasms; Recurrence
Intervention: 68Ga-PSMA
Intervention description: PET/CT imaging with 68Ga-PSMA:
68Ga-PSMA is an investigational radioactive drug that binds to receptors on prostate cancer cells. The intravenously administered drug dose will be about 5 mCi (range 3-7 mCi).
Intervention MeSH terms: Gallium 68 PSMA-11
Phase: Phase 2
Study type: Interventional
Primary purpose: Diagnostic
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 1435.0
Sex: Male
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This study evaluates the value of Gallium-68 labeled PSMA (68Ga-PSMA) for PET/CT imaging of
      prostate cancer recurrence. 68Ga-PSMA is a radioactive molecule, which binds to prostate
      cancer cells. Together with a PET/CT scanner, the distribution of 68Ga-PSMA can be determined
      in the body.

      To test this new drug, participants will receive an intravenous injection of Ga-68-PSMA and
      then have a PET/CT scan. The scan results will be made available to study participants and
      treating physicians.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Histopathological proven prostate adenocarcinoma.

          2. Rising prostate specific antigen (PSA) after definitive therapy with prostatectomy or
             radiation therapy (external beam or brachytherapy).

               1. Post radical prostatectomy (RP) - according to American Urological Association
                  (AUA) recommendation criteria: PSA greater than 0.2 ng/mL measured more than 6
                  weeks after RP and confirmatory persistent PSA greater than 0.2 ng/mL.

               2. Post-radiation therapy - according to ASTRO-Phoenix consensus definition: Nadir +
                  greater than or equal to 2 ng/mL rise in PSA.

          3. Karnofsky performance status of ≥ 50.

          4. Age ≥ 18.

          5. Ability to understand a written informed consent document, and the willingness to sign
             it.

        Exclusion Criteria:

          1. Current investigational therapy for prostate cancer.

          2. Unable to lie flat, still or tolerate a PET/CT scan.

          3. Prior history of a malignancy within the last 2 years, except skin basal cell or
             cutaneous superficial squamous cell carcinoma that has not metastasized, and except
             superficial bladder cancer.

          4. Prisoner.
Gentamicin Intravesical Efficacy for Infection of Urinary Tract
Condition: Postoperative Urinary Tract Infection; Pelvic Organ Prolapse; Stress Urinary Incontinence
MeSH terms: Infections; Communicable Diseases; Urinary Tract Infections; Urinary Incontinence; Urinary Incontinence, Stress; Prolapse; Pelvic Organ Prolapse
Intervention: gentamicin sulfate; Catheter clamping only
Intervention description: Subjects in the gentamicin arm will receive an bladder instillation of 80mg of gentamicin sulfate in 50 mL of normal saline through a standard-of-care Foley catheter.; Subjects in the control arm will have their standard-of-care transurethral catheter clamped for 1 hour post-placement to maintain subject masking to study arm.
Intervention MeSH terms: Gentamicins
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Participant)
Masking description: The subject will be masked as to whether she has been assigned to the gentamicin arm or the control arm. The subject will be unmasked at the completion of the 6 week follow up period.
Enrollment: 370.0
Sex: Female
Minimum age: 18 Years
Healthy volunteers: No
Keywords: gentamicin sulfate; pelvic organ prolapse surgery; midurethral sling; bladder instillation

Summary:

      Urinary tract infection (UTI) is a common problem after surgery for pelvic organ prolapse and
      stress urinary incontinence. This prospective, randomized, single-masked (subject),
      two-parallel armed study aims to determine the effect of a single postoperative intravesical
      instillation of 80 mg of gentamicin sulfate in 50 mL of saline versus usual care on the
      proportion of women treated for UTI within 6 weeks following surgery for pelvic organ
      prolapse (POP) or stress urinary incontinence (SUI).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Adult women undergoing pelvic organ prolapse surgery and/or stress urinary
             incontinence surgery with surgical plan for at least 1 cystoscopy and will leave the
             operating room with a standard-of-care transurethral catheter.

          -  Negative urine culture within 4 weeks or completion of UTI treatment ≥48 hours prior
             to surgery

        Exclusion Criteria:

          -  History of allergic reaction or anaphylaxis to gentamicin sulfate or to sodium
             metabisulfite (one of the preservatives in the gentamicin sulfate product)

          -  Abnormal intraoperative urinary tract finding (e.g. bladder mass, stone, or fistula)

          -  Intraoperative urinary tract injury

          -  Suppressive recurrent UTI treatment

          -  Chronic indwelling catheter/self-catheterization

          -  Unable to provide informed consent

          -  Severe renal impairment - glomerular filtration rate of less than 30 mL / minute

          -  Current pregnancy

          -  Currently incarcerated
Effect of mTOR Inhibition and Other Metabolism Modulating Interventions on the Elderly
Condition: Aging
Intervention: Rapamycin; Placebo; Rapamycin
Intervention description: treatment; control; No placebo control in this substudy group
Intervention MeSH terms: Sirolimus; Temsirolimus; MTOR Inhibitors
Phase: Phase 2
Study type: Interventional
Primary purpose: Basic Science
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 34.0
Sex: All
Minimum age: 70 Years
Maximum age: 95 Years
Healthy volunteers: Accepts Healthy Volunteers
Keywords: Geriatrics

Summary:

      The ability to mount an effective immune response declines with age, leaving the elderly
      increasingly susceptible to infectious diseases and cancer. Rapamycin, an FDA approved drug
      to prevent transplant rejection, increases the lifespan and healthspan of mice and
      ameliorates age-related declines in immune responsiveness, cancer survival, and cognition in
      laboratory animals. Investigators are conducting a translational trial to test whether
      rapamycin also improves life functions in humans focusing on elderly persons (aged 70-95).
    

Eligibility criteria:

        Inclusion Criteria: age 70-95

          -  participants will be in good health with all chronic diseases (hypertension, coronary
             artery disease, etc.) clinically stable.

          -  participants must have adequate cognitive function to be able to give informed
             consent. This will be established by enrolling participants with CLOX 1 scores of ≥10.

        Exclusion Criteria:

          -  unstable ischemic heart disease

          -  clinically significant pulmonary disease

          -  history of immunodeficiency or receiving immunosuppressive therapy

          -  history of a coagulopathy or receiving a medical condition requiring anticoagulation

          -  an estimated glomerular filtration rate of <30ml/min

          -  uncontrolled hypercholesteremia >350mg/dl;

          -  uncontrolled hypertriglyceridemia >500mg/dl

          -  diabetes

          -  history of skin ulcers or poor wound healing

          -  smoking

          -  liver disease

          -  treatment with drugs known to affect cytochrome P450 3A (diltiazem, erythromycin)
Preschool Wheeze: Inflammation/Infection Guided Management
Condition: Asthma
Intervention: Beclometasone; Co-amoxiclav; Azithromycin
Intervention description: Beclometasone dipropionate 200mcg bd for 4 months; Co-amoxiclav 0.3ml/kg bd for 4 weeks; Azithromycin 10mg/kg od for 4 weeks
Intervention MeSH terms: Azithromycin; Amoxicillin-Potassium Clavulanate Combination; Beclomethasone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Participant)
Enrollment: 60.0
Sex: All
Minimum age: 1 Year
Maximum age: 5 Years
Healthy volunteers: No
Keywords: Preschool; Wheeze; Moderate; Severe

Summary:

      This is a single-centre, randomised-controlled trial, comparing management of preschool
      wheeze. It specifically aims to compare management of preschool wheeze using current clinical
      guidelines to management determined by eosinophilic inflammation and infection. Participants
      will be children aged 1-5 years who have recurrent wheezing and will be allocated to one of
      two treatment groups, either current clinical care or pathological phenotype based
      management. They will be asked to make 2 study visits to the Royal Brompton Hospital over the
      course of 4 months.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Reported recurrent wheeze, needing at least 2 courses of oral steroids in the last 12
             months, at least one course of oral steroids in the last 6 months

          -  Past wheeze confirmed by a clinician

        Exclusion Criteria:

          -  Any known cardiac disease

          -  Any chronic respiratory condition (other than preschool wheeze) diagnosed by a
             physician

          -  Any chronic condition that increases susceptibility to respiratory tract infections
             such as severe developmental delay and feeding difficulty/unsafe swallow Prematurity
             <34 weeks, or requirement of ventilation in the newborn period History of neonatal
             chronic lung disease Family not contactable by telephone
Nitrate Effect on Exercise Capacitance
Condition: Fontan
Intervention: Isosorbide Dinitrate
Intervention description: All patients will be given a 6 week titration regimen of study medication. Patients will begin at a 5mg dosage and titrate up to 30mg three times per day if each subsequent dose is tolerated.
Intervention MeSH terms: Isosorbide; Isosorbide Dinitrate; Isosorbide-5-mononitrate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 9 Years
Healthy volunteers: No

Summary:

      The investigators will investigate the safety and preliminary efficacy of ISDN therapy to
      reduce venous congestion and improve exercise tolerance in children and adults after the
      Fontan operation. This will be accomplished by recruiting 15 Fontan physiology patients from
      the Cincinnati Children's Fontan clinic and University of Kentucky Pediatric Cardiology
      clinic for the investigation. The investigators will non-invasively measure both central
      venous pressures at rest and during graded cardiopulmonary exercise testing. In addition the
      study team will obtain a measurement of liver stiffness before and after a 4-week regimen of
      ISDN therapy. Patients will be seen twice in clinic, once before and after ISDN therapy, and
      phone calls will be made to ensure safety, compliance, and make appropriate alterations to
      medications throughout the study period. Pre- and post-intervention central venous pressure
      and exercise tolerance will be compared.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Underlying Fontan physiology

          -  On a stable medication regimen for the past 3 months

          -  Nine years of age or older

        Exclusion Criteria:

          -  Pregnant or nursing

          -  Prior hospitalization for heart failure in past year

          -  Presence of uncontrolled arrhythmias within the past 6 months

          -  Non-cardiac conditions which significantly limited exercise

          -  Moderate or severe ventricular dysfunction by echocardiogram or cardiac MRI

          -  Currently treated with a phosphodiesterase-5 inhibitor or organic nitrates

          -  Concurrent enrollment in other investigational drug trial

          -  End stage Liver Disease (ESLD)
Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
Condition: Hemophilia B
MeSH terms: Hemophilia A; Hemophilia B
Intervention: Coagulation Factor IX variant; Coagulation Factor IX variant
Intervention description: Single intravenous injection of CB2679d/Dalcinonacog alfa; Daily subcutaneous injections of CB2679d/Dalcinonacog alfa for 28 days
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Masking: None (Open Label)
Enrollment: 6.0
Sex: Male
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      Phase 2b, single-center, open-label study designed to evaluate the pharmacokinetics,
      pharmacodynamics, efficacy and safety of subcutaneous (SC) prophylaxis treatment regimens
      with CB2679d in 6 adult, male subjects with severe congenital hemophilia B.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Confirmed diagnosis of severe (<2%) congenital hemophilia B.

          -  Male, age 18 or older.

          -  Agreement to use highly effective birth control throughout the study.

          -  Affirmation of informed consent with signature confirmation before any trial-related
             activities.

          -  Stated willingness to comply with all study procedures and availability for the
             duration of the study.

        Exclusion Criteria:

          -  History or a family history of FIX inhibitors.

          -  Positive antibody to FIX detected by central laboratory at screening.

          -  Previous participation in and subsequent treatment in a clinical trial within the
             previous 30 days or 3-half-lives, whichever is longer, or absence of clinical effect.

          -  Have a coagulation disorder other than congenital hemophilia B.

          -  Factor IX gene mutation 128G>A.

          -  Significant contraindication to participation.
A Study to Assess the Long-term Safety and Efficacy of Erenumab (AMG 334) in Chronic Migraine Prevention.
Condition: Treatment for Prevention of Chronic Migraine
MeSH terms: Migraine Disorders
Intervention: Erenumab
Intervention description: Administered by subcutaneous injection once a month
Intervention MeSH terms: Erenumab
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 609.0
Sex: All
Minimum age: 18 Years
Maximum age: 66 Years
Healthy volunteers: No
Keywords: Chronic Migraine

Summary:

      To assess the long-term safety and efficacy of erenumab.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subject has provided informed consent prior to initiation of any study-specific
             activities/procedures

          2. Completed the 12-week study visit and did not end IP early during the double-blind
             treatment period of the AMG 334 20120295 (NCT02066415) parent study, and is
             appropriate for continued treatment.

        Exclusion Criteria:

          1. Development of any unstable or clinically significant medical condition, laboratory or
             electrocardiogram (ECG) abnormality following randomization into the parent study,
             that in the opinion of the investigator, would pose a risk to subject safety or
             interfere with the study evaluation, procedures or completion.

          2. Systolic blood pressure (BP) 160 mm Hg and/or diastolic BP 100 mm Hg or greater at
             screening/Day 1.

          3. Subject who used excluded concomitant medications between week 8 and week 12 of the
             parent study
Phase 2 Study of OPA-15406 Ointment in Pediatric Patients With Atopic Dermatitis
Condition: Atopic Dermatitis
MeSH terms: Dermatitis, Atopic; Dermatitis; Eczema
Intervention: OPA-15406; Placebos
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 73.0
Sex: All
Minimum age: 2 Years
Maximum age: 14 Years
Healthy volunteers: No

Summary:

      To evaluate the safety, efficacy (dose response) and pharmacokinetics of 0.3% and 1%
      OPA-15406 when applied twice daily for 4 weeks in pediatric patients with atopic dermatitis.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of atopic dermatitis based on the criteria of Hanifin and Rajka

        Exclusion Criteria:

          -  Subjects who have an atopic dermatitis or contact dermatitis flare-up defined as a
             sudden intensification of atopic dermatitis.

          -  Subjects who have an active viral skin infection.

          -  Subjects with a current or history of malignancy.