bioihbjkbjnl/mcl2 huggingface dataset:
Androgen Effect on Klinefelter Syndrome Motor Outcome
Condition: Klinefelter Syndrome
MeSH terms: Klinefelter Syndrome; Syndrome
Intervention: androgen oxandrolone; placebo
Intervention description: Oxandrolone ,06 >mg/kg/day, orally, for 2 years; an inactive substance
Intervention MeSH terms: Androgens; Oxandrolone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 93.0
Sex: Male
Minimum age: 4 Years
Maximum age: 12 Years
Healthy volunteers: No
Keywords: Klinefelter syndrome; androgen; androgen oxandrolone

Summary:

      The purpose of this study is to evaluate the effects of low-dose androgen on the motor and
      cognitive development of boys with Klinefelter syndrome.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Karyotype diagnosis of Klinefelter syndrome

          -  Chronological age of 4-12 years

          -  No treatment with androgen in the past year

        Exclusion Criteria:

          -  Major liver, kidney or other systemic disease

          -  Variant karyotypes including 47,XYY males

          -  Evidence of spontaneous onset of puberty, defined as testicular size > 4ml
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Condition: Inherited Metabolic Disorders (IMD)
MeSH terms: Metabolic Diseases
Intervention: MGTA-456
Intervention description: Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 8.0
Sex: All
Minimum age: 0 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: inherited metabolic disorders; cerebral adrenoleukodystrophy; Hurler syndrome; globoid cell leukodystrophy; Krabbe disease; metachromatic leukodystrophy; hematopoietic stem cells; hematopoietic stem cell transplant; umbilical cord blood; umbilical cord blood transplant; MGTA-456; myeloablative conditioning regimen; bone marrow transplant; mucopolysaccharidosis-1H

Summary:

      This study is designed to assess the safety and efficacy of using MGTA-456 in patients with
      Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Age < 2.5 years with Hurler syndrome, age 2-17 years with cerebral
             adrenoleukodystrophy (cALD), age < 16 years with metachromatic leukodystrophy (MLD)
             and age ≤ 10 years with globoid cell leukodystrophy (GLD) (also referred to as Krabbe)

          -  Cord blood grafts require genetic testing and/or demonstration of enzyme activity for
             patients with Hurler syndrome, MLD or GLD and are tested for very long chain fatty
             acids (VLCFA) to confirm there is no evidence of VLCFA consistent with ALD

          -  Adequate organ function

          -  Availability of eligible donor material

        Exclusion Criteria:

          -  Availability of a matched-related donor who is not a carrier of the same genetic
             defect

          -  Active infection at screening

          -  Prior myeloablative conditioning

          -  History of human immunodeficiency virus (HIV) infection
Study to Assess the Efficacy and Safety of a PARP Inhibitor for the Treatment of BRCA-positive Advanced Ovarian Cancer
Condition: Ovarian Neoplasm
MeSH terms: Ovarian Neoplasms; Carcinoma, Ovarian Epithelial
Intervention: KU-0059436 (AZD2281)(PARP inhibitor); KU-0059436 (AZD2281)(PARP inhibitor)
Intervention description: oral; oral
Intervention MeSH terms: Olaparib; Poly(ADP-ribose) Polymerase Inhibitors
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 58.0
Sex: Female
Minimum age: 18 Years
Maximum age: 130 Years
Healthy volunteers: No
Keywords: Advanced ovarian cancer; Poly(ADP ribose) polymerases; KU-0059436; AZD2281; BRCA1 protein; BRCA2 protein

Summary:

      The purpose of the study is to see if the drug KU 0059436 is effective and well tolerated in
      treating patients with measurable BRCA1- or BRCA2-positive advanced ovarian cancer and for
      whom no curative therapeutic option exists.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Advanced ovarian cancer with positive BRCA1 or BRCA2 status

          -  Failed at least one prior chemotherapy

          -  In investigators opinion, no curative standard therapy exists

          -  Measurable disease

        Exclusion Criteria:

          -  Brain metastases

          -  Less than 28 days since last treatment used to treat the disease

          -  Considered a poor medical risk due to a serious uncontrolled disorder
A Multi-Center, Double-Masked Evaluation of the Efficacy and Safety of CSF-1 in the Treatment of Presbyopia
Condition: Presbyopia
MeSH terms: Presbyopia
Intervention: CSF-1; CSF-1 Component #1; CSF-1 Component #2
Intervention description: This treatment arm consists of 2 different concentrations of CSF-1. Subjects randomized to the CSF-1 treatment arm will receive their first dose of CSF-1 in-office at Visit 2. All subjects will dose twice a day in both eyes with a single drop for approximately 1 week. At Visit 3, subjects randomized to the CSF-1 arm will now receive a different concentration of CSF-1. Subjects will continue dosing twice a day in both eyes for approximately 1 week.; This treatment arm consists of 2 different concentrations of CSF-1 Component #1.
Subjects randomized to the CSF-1 Component #1 treatment arm will receive their first dose of CSF-1 Component #1 in-office at Visit 2. All subjects will dose twice a day in both eyes with a single drop for approximately 1 week. At Visit 3, subjects randomized to the CSF-1 Component #1 arm will now receive a different concentration of CSF-1 Component #1. Subjects will continue dosing twice a day in both eyes for approximately 1 week.; This treatment arm consists of a single concentration of CSF-1 Component #2. Subjects randomized to the CSF-1 Component #2 treatment arm will receive their first dose of CSF-1 Component #2 in-office at Visit 2. All subjects will dose twice a day in both eyes with a single drop for approximately 1 week. At Visit 3, subjects randomized to the CSF-1 Component #2 arm will continue dosing with the same concentration of CSF-1 Component #2. Subjects will continue dosing twice a day in both eyes for approximately 1 week.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 166.0
Sex: All
Minimum age: 45 Years
Maximum age: 64 Years
Healthy volunteers: No

Summary:

      This is a 4-visit, multi-center, randomized, double-masked, parallel group study evaluating
      the safety and efficacy of CSF-1 in the treatment of presbyopia.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects must:

               1. Have presbyopia

        Exclusion Criteria:

          -  Subjects must not:

               1. Have any contraindications to the study medications or diagnoses that would
                  confound the study data
Use of Entresto Sacubitril/Valsartan for the Treatment of Peripheral Arterial Disease
Condition: Peripheral Arterial Disease
MeSH terms: Peripheral Arterial Disease; Peripheral Vascular Diseases
Intervention: Entresto; Placebo group
Intervention description: Oral pills; The placebo pills
Intervention MeSH terms: Sacubitril and valsartan sodium hydrate drug combination
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 6.0
Sex: All
Minimum age: 18 Years
Maximum age: 80 Years
Healthy volunteers: No

Summary:

      This study proposes the use of Entresto (sacubitril/valsartan) to test the effects on pain
      free walking duration on patients with peripheral arterial disease, a condition caused by
      decreased blood flow to the muscles in the legs.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subject with symptoms of intermittent claudication, such as exercise-induced pain,
             cramps, fatigue, or other equivalent discomfort, involving large muscle groups of the
             leg(s) (calf, thigh, buttocks), relieved by rest.

          2. Ankle-brachial index ≤ 0.90 acquired according to the American Heart Association
             guidelines.

          3. Highest ankle pressure reduced by at least 25 mm Hg after exercise compared to resting
             pressure (or loss of previously present Doppler signal for both the posterior tibial
             and anterior tibial arteries immediately after exercise if arteries were
             incompressible).

          4. Patients on medical treatment for PAD without significant improvement in intermittent
             claudication within the last 6 months.

        Exclusion Criteria:

          1. Age < 18 and > 80 years.

          2. Patients with physician diagnosed chronic kidney disease or heart failure stage II or
             IV or unstable angina.

          3. Echocardiographic evidence of cardiomyopathies and pulmonary hypertension.

          4. Patients that have received cancer treatment within the last year (except skin
             cancer).

          5. Severe limitations in mobility due to osteomuscular disorders present at time of
             interview.

          6. Dementia or other mental disorders that prevent patients from following a research
             protocol present at time of interview

          7. Patients engaged in an exercise rehabilitation program within the past 6 months.

          8. Patients schedule to undergo an arterial revascularization procedure during the study
             or have undergone one within the past 6 months.

          9. Inconsistent maximal walking distance on the treadmill test.
A Phase 2 Study of BGJ398 in Patients With Recurrent GBM
Condition: Recurrent Glioblastoma or Other Glioma Subtypes
MeSH terms: Glioblastoma; Recurrence
Intervention: BGJ398
Intervention description: Capsule for oral use.
Intervention MeSH terms: Infigratinib
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 26.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: BGJ398,; recurrent glioblastoma'; recurrent GBM,; FGFR,

Summary:

      This is an open-label non-randomized, multicenter, phase II study of BGJ398 administered to
      adult patients with histologically confirmed GBM and/or other glioma subtypes with
      FGFR1-TACC1, FGFR3-TACC3 fusion and/or activating mutation in FGFR1, 2 or 3.
    

Eligibility criteria:

        Inclusion criteria:

          1. Patients with histologically confirmed GBM and/or other glioma subtypes at the time of
             diagnosis or prior relapse.

          2. Written documentation of local or central laboratory determination of amplification or
             translocation to FGFR1-TACC1, FGFR3-TACC-3 fusion and/or activating mutation in FGFR1,
             FGFR2,or FGFR3

          3. RANO defined tumor progression by MRI in comparison to a prior scan

          4. Patients must have received prior external beam radiotherapy and temozolomide.

        Exclusion criteria:

          1. History of another primary malignancy

          2. Prior or current treatment with a FGFR inhibitor

          3. Neurological symptoms related to underlying disease requiring increasing doses of
             corticosteroids

          4. Patients must not be taking Enzyme Inducing Anti-Epileptic Drug (EIAED). If previously
             on an EIAED, the patient must be off of it for at least two weeks prior to study
             treatment.
A Study of Topical Steroids as Preemptive Therapy for EGFR Inhibitor-Induced Papulopustular Eruption
Condition: Acneiform Rash; Papulopustular Eruption
MeSH terms: Exanthema
Intervention: Triamcinolone
Intervention description: 6 weeks of twice daily application of 0.1% triamcinolone cream to the face, chest, and back.
Intervention MeSH terms: Triamcinolone
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 14.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This randomized, controlled, phase II clinical study is designed to assess the efficacy of
      preemptive treatment with topical steroids in preventing the papulopustular eruption induced
      by epidermal growth factor receptor inhibitor (EGFRI) treatment in cancer patients.
      Participants will be followed up for 6 weeks of twice daily application of triamcinolone
      cream to the face, chest, and back.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients must have a histologically or cytologically confirmed cancer diagnosis for
             which EGFRI treatment is indicated

          -  Initiation of topical steroids or control treatment within 3 days of initiation of
             cetuximab, erlotinib, panitumumab, or afatinib

          -  Patients must be age ≥ 18 years.

          -  Life expectancy of greater than 6 weeks

          -  Patient able to use topical medications reliably and complete questionnaires with
             assistance if needed

          -  Patients must have the ability to understand and the willingness to sign a written
             informed consent prior to registration on study.

        Exclusion Criteria:

          -  Patients who have used systemic or topical steroids within 7 days of trial
             registration, or start systemic or topical steroids for reasons unrelated to trial
             during the 6-week follow up period

          -  Patients who have used antibiotics within 7 days of trial registration, or start
             antibiotics for other conditions during the 6-week follow up period

          -  History of allergic reactions to topical steroids

          -  Patients with any rash at the time of study registration

          -  Uncontrolled intercurrent illness including, but not limited to, ongoing or active
             infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
             arrhythmia, or psychiatric illness/social situations that would limit compliance with
             study requirements.

          -  Patients using any other topical medications in the treatment areas (face, chest, or
             back).
Sirolimus as Secondary Therapy in Chronic Graft-Versus-Host Disease Not Responding To Prior Treatment
Condition: Graft Versus Host Disease
MeSH terms: Bronchiolitis Obliterans Syndrome; Graft vs Host Disease
Intervention: sirolimus
Intervention description: Given PO
Intervention MeSH terms: Sirolimus
Phase: Phase 2
Study type: Interventional
Primary purpose: Supportive Care
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 44.0
Sex: All
Healthy volunteers: No

Summary:

      This phase II trial studies the side effects and how well sirolimus works as secondary
      therapy in treating patients with chronic graft-versus-host disease (GVHD) that did not
      respond to prior treatment. Sirolimus may be an effective treatment for chronic GVHD
    

Eligibility criteria:

        Inclusion Criteria:

          -  Biopsy-confirmed diagnosis of clinical extensive chronic GVHD with inadequate response
             to previous treatment and where secondary systemic therapy is indicated because of

               -  Clinical progression of signs and symptoms of chronic GVHD in a previously
                  involved organ, or

               -  Development of signs and symptoms of chronic GVHD in a previously uninvolved
                  organ, or

               -  Absence of improvement after 3 months of primary treatment, or

               -  Continued need for treatment with prednisone at doses >= 1.0 mg/kg/day for more
                  than 2 months, without qualification for type of donor, graft or conditioning
                  regimen

          -  Patient or guardian able and willing to provide informed consent

          -  Stated willingness to use contraception in women of child-bearing potential (Food and
             Drug Administration [FDA] requirement)

          -  Stated willingness of the patient to comply with study procedures and reporting
             requirements

          -  Stated willingness of the physician most involved in management of chronic GVHD (the
             "managing physician,") to comply with study procedures and reporting requirements

        Exclusion Criteria:

          -  Fungal or viral infection with no radiographic evidence of improvement during
             continued appropriate antimicrobial therapy

          -  Cytomegalovirus (CMV) antigenemia unresponsive to antiviral therapy

          -  Active disseminated varicella zoster virus (VZV) infection with persistent non-crusted
             lesions

          -  Inability to tolerate oral medications

          -  Absolute neutrophil count (ANC) < 1500/uL

          -  Platelet count < 50,000/uL

          -  Persistent or recurrent malignancy, including histopathologic evidence of myeloma or
             lymphoma; patients with breakpoint cluster region-abelson (bcr/abl) detected by
             polymerase chain reaction (PCR) assay as the only evidence of persistent chronic
             myeloid leukemia may be enrolled

          -  Pregnancy

          -  Known history of hypersensitivity to sirolimus
A Safety, Efficacy and Pharmacokinetic Study of AGN-199201 and AGN-190584 in Patients With Presbyopia
Condition: Presbyopia
MeSH terms: Presbyopia
Intervention: AGN-199201 ophthalmic solution; AGN-190584 ophthalmic solution; AGN-199201 Vehicle; AGN-190584 Vehicle
Intervention description: 1 drop of AGN-199201 ophthalmic solution Doses A, B, C in the eye.; 1 drop of AGN-190584 ophthalmic solution Doses A, B, C in the eye.; Vehicle to AGN-199201; Vehicle to AGN-190584
Intervention MeSH terms: Pharmaceutical Solutions; Ophthalmic Solutions
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 151.0
Sex: All
Minimum age: 40 Years
Maximum age: 55 Years
Healthy volunteers: No

Summary:

      This is a safety, efficacy and pharmacokinetics study of the fixed combination of AGN-199201
      and AGN-190584 in participants with presbyopia (inability to focus on items close-up).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Normal vision at distance, either natural or post corneal laser refractive surgery,
             with presbyopia in each eye and complaints of poor near vision that impacts activities
             of daily living

        Exclusion Criteria:

          -  Use of any topical ophthalmic medications, including artificial tears other than the
             study medications during the study

          -  Corneal abnormalities in either eye that interfere with visual acuity

          -  History of cataract surgery, phakic intraocular lens surgery, corneal inlay surgery or
             any intraocular surgery

          -  Diagnosis of glaucoma or ocular hypertension.
A Study to Evaluate the Efficacy and Safety of PF-06700841 in Subjects With Active Psoriatic Arthritis
Condition: Psoriatic Arthritis
MeSH terms: Arthritis; Arthritis, Psoriatic
Intervention: PF-06700841; Placebo
Intervention description: Starting after the Week 16 visit, subjects receiving PF-06700841 10 mg once daily will start to randomly receive either the 60 mg QD dose or 30 mg QD dose until Week 52, as predetermined at randomization. All subjects will receive blinded dosing throughout the 52 weeks study treatment period in order to maintain the study blind.; Starting after the Week 16 visit, subjects receiving placebo will start to randomly receive either the 60 mg QD dose or 30 mg QD dose until Week 52, as predetermined at randomization. All subjects will receive blinded dosing throughout the 52 weeks study treatment period in order to maintain the study blind.
Intervention MeSH terms: PF-06700841
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 219.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Spondyloarthritis

Summary:

      This is a 52 week Phase 2b study designed to evaluate the efficacy at 16 weeks and to
      evaluate the safety and efficacy up to 1 year in subjects with active psoriatic arthritis.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Active arthritis at screening/baseline as indicated by >/= 3 tender/painful and 3
             swollen joints.

          -  Active plaque psoriasis at screening and baseline.

        Exclusion Criteria:

          -  Non-plaque forms of psoriasis (with exception of nail psoriasis).

          -  History of autoimmune rheumatic disease other than PsA; also prior history of or
             current, rheumatic inflammatory disease other than PsA.
Study of Bevacizumab Followed by Bevacizumab Consolidation for Ovarian Cancer
Condition: Advanced Ovarian Carcinoma; Primary Peritoneal Carcinoma; Ovarian Carcinosarcoma
MeSH terms: Carcinoma; Ovarian Neoplasms; Carcinoma, Ovarian Epithelial; Carcinosarcoma; Mixed Tumor, Mullerian
Intervention: Avastin; Paclitaxel; Cisplatin
Intervention description: Initial Treatment Bevacizumab 15mg/kg Day 1 IV every 21 days x 5 cycles (beginning with cycle 2)
Consolidation Treatment:
Avastin 15mg/kg IV every 21 days x 12 cycles; Paclitaxel 135mg/m2 IV Day 1 every 21 days x 6 cycles; 75mg/m2 IP day 2 every 21 days x 6 cycles
Intervention MeSH terms: Paclitaxel; Bevacizumab; Cisplatin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: Female
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Ovarian Cancer; Gynecologic Cancer; Ovarian Carcinoma; Peritoneal Carcinoma; IP chemotherapy; Intraperitoneal; Avastin; Bevacizumab

Summary:

      The purpose of this study is to evaluate the tolerability of intraperitoneal cisplatin with
      intravenous paclitaxel and Avastin as defined by the proportion of patients able to complete
      6 cycles of treatment.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients with stage II and III epithelial ovarian carcinoma, primary peritoneal
             carcinoma, or ovarian carcinosarcoma.

          -  Adequate bone marrow, renal, and hepatic function

          -  Patients must be entered no more than twelve weeks postoperatively

        Exclusion Criteria:

          -  Patients with epithelial ovarian carcinoma of low malignant potential (borderline
             carcinomas).

          -  Stage IV or suboptimally debulked disease following primary cytoreductive surgery

          -  Patients who have received prior radiotherapy or chemotherapy.