bioihbjkbjnl/mcl2 huggingface dataset:
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Condition: Gaucher Disease, Type 1; Type 1 Gaucher Disease; Gaucher Disease
MeSH terms: Gaucher Disease
Intervention: Afegostat tartrate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 30.0
Sex: All
Minimum age: 18 Years
Maximum age: 74 Years
Healthy volunteers: No
Keywords: afegostat tartrate; isofagomine tartrate; AT2101; Amicus Therapeutics

Summary:

      This study was conducted to test the safety and tolerability of afegostat tartrate in
      participants with type 1 Gaucher disease already receiving enzyme replacement therapy.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Had a confirmed diagnosis of type 1 Gaucher disease with a known documented missense
             gene mutation in at least 1 of the 2 gene-encoding β-glucosidase alleles

          -  Clinically stable

          -  Male or female participants, 18 to 74 years old inclusive

          -  All participants of childbearing potential used adequate birth control

          -  Provided written informed consent to participate in the study

        Exclusion Criteria:

          -  Clinically significant disease, severe complications from Gaucher disease, or serious
             illness that precluded participation in the study in the opinion of the Investigator
             that compromised the safety of the participant or precluded the participant from
             completing the study

          -  During the screening period, any clinically significant findings, as deemed by the
             Investigator

          -  Partial or total splenectomy (removal of spleen) within the 2 years prior to study
             entry

          -  History of pulmonary hypertension or Gaucher related lung disease

          -  History of allergy or sensitivity to the study drug or any excipients, including any
             prior serious adverse reaction to iminosugars (for example, N-butyldeoxynojirimycin or
             miglustat)

          -  Pregnant or breast-feeding

          -  Current/recent drug or alcohol abuse

          -  Treatment with any investigational product in the 90 days before study entry

          -  Treatment in the previous 90 days with any drug known to have a well-defined potential
             for toxicity to a major organ

          -  Presence or symptoms of gastrointestinal, liver or kidney disease, or other conditions
             known to interfere with the absorption, distribution, metabolism, or excretion of
             drugs
Efficacy and Safety Study of MP-435 in Combination With Methotrexate (MTX) in Patients With Rheumatoid Arthritis
Condition: Rheumatoid Arthritis
MeSH terms: Arthritis; Arthritis, Rheumatoid
Intervention: MP-435(dose1) + Methotrexate; Placebo + Methotrexate
Intervention description: MP-435 dose1 + stable weekly dose of Methotrexate; Placebo + stable weekly dose of Methotrexate
Intervention MeSH terms: Methotrexate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 112.0
Sex: All
Minimum age: 20 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Rheumatoid Arthritis

Summary:

      The purpose of this study is to determine the Efficacy, Safety, and Pharmacokinetics of
      MP-435 administered for 12 weeks in subjects with rheumatoid arthritis (RA) on stable doses
      of Methotrexate.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects with a diagnosis of RA according to the diagnostic criteria of the American
             College of Rheumatology (ACR) (revised in 1987) for at least 6 months.

          -  Subjects who inadequately response for stable dose of MTX.

        Exclusion Criteria:

          -  Patients with Class IV functional activity by the Steinbrocker's scale.

          -  Patients who have received a biological agent in the past.

          -  Patients who have other rheumatic diseases, or who have other diseases with joint
             symptoms.

          -  Patients with severe or uncontrolled endocrine, psychiatric, cardiac, hematological,
             pulmonary, hepatic, kidney, gastrointestinal, or thyroid disease.
In Vivo Preoperative Skin Preparation Persistence Evaluation
Condition: Bacterial Recovery of Skin Flora Post-product Application
Intervention: 3M CHG/IPA Prep; ChloraPrep
Intervention description: Apply topically to intact dry skin (abdominal site) for 30 seconds or moist skin (inguinal site) for 2 minutes.; Apply topically to intact dry skin (abdominal site) for 30 seconds or moist skin (inguinal site) for 2 minutes.
Intervention MeSH terms: Ethanol; Chlorhexidine; Chlorhexidine gluconate
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Enrollment: 69.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: Accepts Healthy Volunteers

Summary:

      Evaluate persistent antimicrobial effectiveness of chlorhexidine gluconate containing
      products on abdominal and inguinal sites following a saline and wipe challenge.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects of any race

          -  Subjects in good health

          -  Minimum skin flora baseline requirements on abdomen and groin

        Exclusion Criteria:

          -  Any tattoos, scars, breaks in the skin, or any form of dermatitis, or other skin
             disorders (including acne) on the applicable test area

          -  Topical antimicrobial exposure within 14 days prior to screening and treatment days

          -  Use of systemic or topical antibiotics, steroid medications, or any other products
             known to affect the normal microbial flora of the skin within 14 days prior to
             screening and treatment days
AIN457 Regimen Finding Extension Study in Participants With Moderate to Severe Psoriasis
Condition: Plaque-type Psoriasis
MeSH terms: Psoriasis
Intervention: AIN457; Placebo
Intervention description: AIN457A 150 mg powder for solution; Placebo to AIN457A 150 mg powder for solution
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 275.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Chronic plaque-type psoriasis; AIN457; dermatology; Moderate to severe chronic plaque-type psoriasis

Summary:

      The purpose of this study was to provide long term clinical data for the compound for the
      treatment of the indication of moderate to severe chronic plaque-type psoriasis.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Patients who completed the core study CAIN457A2211. A patient is defined as having
             completed the core study if he/she completed the study up to and including visit 13
             (F4) of the core study

          -  Patients must be able to understand and communicate with the investigator and comply
             with the requirement of the study and must given written, signed and dated informed
             consent before any study assessment is performed.

          -  Patients must be expected to benefit from the ongoing treatment with AIN457, as
             assessed by the patient and investigator

          -  Male patients must consent to practice reliable contraception during the study and for
             16 weeks after the last dose of study drug administration Note: Due to new data
             available from the toxicology studies, the need for male contraception was removed.

        Key Exclusion Criteria:

          -  Patients who experience a second consecutive full relapse at visit 13 ( week F4) of
             the core study CAIN457A2211

          -  Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a
             female after conception and until termination of gestation, confirmed by a positive
             hCG laboratory test (> 5mlU/mL)

          -  Women of child-bearing potential, defined as all women physiologically capable of
             becoming pregnant, unwilling to use effective contraception during the study and for
             16 weeks after stopping treatment.
Cardiac Surgery Neuroprotection Study in Elders
Condition: Post-operative Cognitive Decline; Post-operative Delirium
MeSH terms: Emergence Delirium; Cognitive Dysfunction
Intervention: Aspart insulin; Normal saline; Intranasal mucosal atomizer device
Intervention description: 40 IU of aspart insulin (200 microliters per nostril) will be administered intranasally using an intranasal mucosal atomizer device within 2 hours prior to surgery, and then four times a day for 7 days or until hospital discharge (whichever occurs first); 200 microliters of normal saline will be administered per nostril using an intranasal mucosal atomizer device within 2 hours prior to surgery, and then four times a day for 7 days or until hospital discharge (whichever occurs first); Insulin and placebo will be drawn into identical syringes. Nurses will administer the insulin or placebo by connecting the mucosal atomizer device (MAD) to the syringe, placing the MAD tip in the nostril and compressing the syringe plunger to spray atomized solution into the nasal cavity.
Intervention MeSH terms: Insulin Aspart
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 22.0
Sex: All
Minimum age: 65 Years
Healthy volunteers: No

Summary:

      The goal of this study is to determine the potential ability of intranasal insulin to prevent
      post-operative cognitive decline and post-operative delirium in an elderly cardiac surgery
      population.
    

Eligibility criteria:

        Inclusion Criteria:

          1. elderly patients (>=65 years old)

          2. undergoing elective coronary artery bypass graft and/or valve surgery requiring
             cardiopulmonary bypass

          3. English or Spanish-speaking

        Exclusion Criteria:

          1. severe dementia, neurodegenerative, or psychiatric disease that prevents patient from
             living independently at baseline

          2. emergent surgery

          3. inability to perform cognitive testing (i.e. difficulty hearing or inability to speak)

          4. contraindications to intranasal administration of medication
Safety and Efficacy Study of a New Antiviral Drug to Prevent Cytomegalovirus Reactivation in Bone Marrow Transplanted Patients
Condition: HCMV Reactivation or HCMV End-Organ Disease
Intervention: 60 mg AIC246; 120 mg AIC246; 240 mg AIC246; Placebo
Intervention description: Oral administration; Oral administration; Oral administration; Oral administration
Intervention MeSH terms: Letermovir
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 133.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: HCMV

Summary:

      The aim of the study is to find out whether AIC246 is safe and efficacious in lowering the
      chances of the cytomegalovirus becoming active again and causing illness after an HBPC
      transplant (allogeneic stem cell transplant).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Seropositive for HCMV IgG antibodies before transplantation

          -  First allogeneic Human blood precursor cell (HBPC) transplantation performed for 1 of
             the following diagnoses: leukaemia, lymphoma, Hodgkin's lymphoma, non-Hodgkin's
             lymphoma, multiple myeloma, myelodysplastic and myeloproliferative disorder

          -  Evidence of post transplantation engraftment

          -  Able to swallow tablets.

        Exclusion Criteria:

          -  Previous anti-HCMV therapy after this allogeneic HBPC transplantation

          -  Mismatched or cord blood transplant recipients

          -  Current or history of end-organ HCMV disease

          -  Graft versus host disease (GVHD)

          -  Impaired liver function

          -  Reduced renal function
Pharmacokinetic Response to BPI in Burns
Condition: Burns
MeSH terms: Burns
Intervention: BPI
Intervention description: BPI will be give IV with an initial bolus given over 30 minutes and then a continuous infusion for the next 47.5 hours
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 4.0
Sex: All
Minimum age: 13 Years
Maximum age: 60 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to evaluate the safety of a protein called BPI that is naturally
      made by the body's white blood cells to fight infection.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Ages 13 years to 60 years old

          -  Sustained partial-thickness or full-thickness burn greater than 20% and less than 41%
             TBSA

          -  Parental consent (if subject is less than 18 years old) or individual (if subject is
             equal to or older than 18) signed, informed consent.

          -  Able to start BPI in infusion within 8 hours of burn injury.

        Exclusion Criteria:

          -  Age > 61.

          -  Inhalational injury requiring mechanical ventilation.

          -  Partial and full thickness burns totaling > 41% total body surface area

          -  Cardiac dysfunction, defined as the presence of any of the following:

               1. New York Heart Class 3 or 4 heart failure.

               2. Unstable angina or acute myocardial infarction.

               3. Left ventricular ejection fraction known to be < 35%.

          -  Electrical or lightening induced burn injury or any injury that required
             cardio-pulmonary resuscitation.

          -  Concomitant non-burn trauma with an ISS > 9.

          -  Non-survivable injury secondary to burn size - patients made DNR or comfort care due
             to burn size.

          -  Patients with known causes of immunosuppression:

               1. Known history of HIV/AIDS

               2. Chronic steroid use for underlying medical conditions (equivalent to 15 mg
                  prednisone/day).

               3. Active oncolytic therapy for known malignancy

          -  Known or suspected pregnancy

          -  Known allergy to rBPI21
An Investigational Drug, Palbociclib (PD-0332991), Is Being Studied In Combination With Velcade And Dexamethasone In Patients With Multiple Myeloma. Patients Must Have Received Prior Treatment For Multiple Myeloma.
Condition: Multiple Myeloma
MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell
Intervention: Bortezomib; Dexamethasone; PD 0332991
Intervention description: Escalating doses of bortezomib will be administered intravenously on Days 8, 11, 15 and 18 of a 28-day cycle (Schedule A) or of a 21-day cycle (Schedule B). The planned doses to be evaluated are 0.7, 1 and 1.3 mg/m2 in combination with PD 0332991 and dexamethasone.; 20 mg, orally on Days 8, 11, 15 and 18 of a 28 day cycle (Schedule A) or of a 21-day cycle (Schedule B) in combination with PD 0332991 and bortezomib.; Escalating doses of PD 0332991 will be administered orally on Days 1-21 of a 28-day cycle for Schedule A and on Days 1-12 of a 21-day cycle for Schedule B. The planned doses to be evaluated are 50, 75, 100 mg and 125 mg once daily in combination with bortezomib and dexamethasone.
Intervention MeSH terms: Dexamethasone; Bortezomib; Palbociclib
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 53.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This is a Phase 1/2 study evaluating the safety and anti-tumor activity of PD 0332991 in
      combination with Velcade® [bortezomib] and dexamethasone in patients who have received at
      least one previous treatment for multiple myeloma.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of symptomatic multiple myeloma as defined by International Myeloma Working
             Group (IMWGURC).

          -  Phase 1: Relapsed or relapsed/refractory myeloma after at least 1 previous treatments
             and with a life expectancy of more than 3 months.

          -  Phase 2: Measurable (as defined by IMWGURC) disease after at least 1 previous
             treatment.

        Exclusion Criteria:

          -  History of allogeneic stem cell transplant.

          -  Phase 2 only: Prior bortezomib therapy will only be allowed if there was a
             demonstrated positive response, and disease progression occurred off therapy.

          -  Must have not experienced significant blood level changes, e.g. very low platelets,
             while on previous bortezomib therapy

          -  Prior radiation therapy to > 25% of the bone marrow (whole pelvis is 25%).
The Utility of 18F-DOPA-PET in the Treatment of Recurrent High-grade Glioma
Condition: Glioma
MeSH terms: Glioma
Intervention: Fluorine F 18 Fluorodopa; Intensity-Modulated Radiation Therapy; Positron Emission Tomography
Intervention description: Contrast used in PET; Radiation of different intensities are aimed at the tumor from many angles. This type of radiation therapy reduces the damage to healthy tissue near the tumor; imaging test
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 21.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This proposal is for a pilot study comparing volumes of 18F-DOPA-PET avidity with contrast
      enhancement and T2 FLAIR on MRI. Investigators then plan to compare patterns of failure with
      target volumes, pre-treatment MRI changes and pre-treatment 18F-DOPA-PET.
    

Eligibility criteria:

        Inclusion Criteria

          -  Age > or equal to 18 years

          -  ECOG PS < or equal to 3

          -  Histologically confirmed or radiographic evidence of recurrent/progressive glioma.

          -  History of radiation therapy to the brain for prior diagnosis of glioma

          -  Planned radiation treatments at Mayo Clinic Rochester

          -  Provide informed written consent

          -  Willing to sign consent onto the Mayo Clinic Radiotherapy Patient Outcomes Registry
             and Biobanking study, IRB number 15-000136 (blood draw optional)

          -  Willing to return to enrolling institution for follow-up during Active Monitoring
             Phase of the study.

        Exclusion Criteria

          -  More than one prior course of radiotherapy or prior prescription doses exceeding 60 Gy
             to re-irradiation target volumes

          -  Unable to undergo MRI scans with contrast

          -  Unable to undergo an 18F-DOPA-PET scan (e.g., Parkinson's Disease, taking anti
             dopaminergic, or dopamine agonist medication or less than 6 half-lives from
             discontinuance of dopamine agonists.)

        Note: Other potentially interfering drugs: amoxapine, amphetamine, benztropine, buproprion,
        buspirone, cocaine, mazindol, methamphetamine, methylphenidate, norephedrine, phentermine,
        phenylpropanolamine, selegiline, paroxetine, citalopram, and sertraline). If a patient is
        on any of these drugs, list which ones on the On-Study form.

        -Any of the following:

          -  Pregnant women

          -  Nursing women

          -  Men or women of childbearing potential who are unwilling to employ adequate
             contraception
Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Condition: Porphyria Cutanea Tarda
MeSH terms: Porphyria Cutanea Tarda; Porphyrias, Hepatic; Porphyria, Erythropoietic; Porphyrias
Intervention: Hydroxychloroquine; Phlebotomy
Intervention description: 100 mg by mouth twice weekly; 450 mL every 2 weeks
Intervention MeSH terms: Hydroxychloroquine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 48.0
Sex: All
Minimum age: 18 Years
Maximum age: 100 Years
Healthy volunteers: No
Keywords: Porphyria, rare disease, orphan disease, iron metabolism

Summary:

      Porphyria cutanea tarda (PCT) is an iron-related disorder that responds to treatment by
      phlebotomy or low-dose hydroxychloroquine, but comparative data on these treatments are
      limited. The hypothesis is that hydroxychloroquine is noninferior to phlebotomy in terms of
      time to remission. Patients with well documented PCT are assigned to treatment by
      randomization if specific criteria are met. All patients are followed until remission -
      defined as achieving a normal plasma porphyrin concentration.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Documented porphyria cutanea tarda (PCT)

          -  Willing to give informed consent

          -  Age 18 or greater

        Exclusion Criteria:

          -  Blistering skin lesions due to another condition