bioihbjkbjnl/mcl2 huggingface dataset:
Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
Condition: Waldenstrom's Macroglobulinemia
MeSH terms: Waldenstrom Macroglobulinemia
Intervention: Perifosine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 37.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: relapsed Waldenstrom's Macroglobulinemia; refractory Waldenstrom's Macroglobulinemia; WM; perifosine

Summary:

      Waldenström's Macroglobulinemia (lymphoplasmacytic lymphoma, WM) remains incurable with
      limited therapeutic options and notably absent FDA approved therapy with any WM indication.
      Therefore, there is a need to identify new therapeutic agents for WM patients both in the
      upfront and relapsed/refractory setting. The purpose of this research study is to assess the
      efficacy of perifosine in patients with relapsed or refractory WM.
    

Eligibility criteria:

        Inclusion Criteria:

          -  18 years of age or older

          -  Must have received prior therapy for their WM and have relapsed or refractory WM. Any
             number of prior therapies is acceptable

          -  Measurable disease, defined as presence of immunoglobulin M paraprotein with a minimum
             IgM level of equal to or greater than 2 times the ULN and over 10% of
             lymphoplasmacytic cells in bone marrow

          -  ECOG Performance Status 0,1, or 2

          -  Laboratory values as described in the protocol

          -  Life expectancy of greater than 12 weeks

        Exclusion Criteria:

          -  Uncontrolled infection

          -  Other active malignancies

          -  CNS involvement

          -  Cytotoxic chemotherapy less than 3 weeks, or biologic therapy less than 2 weeks, or
             corticosteroids less than 2 weeks prior to registration.

          -  Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy
             considered investigational

          -  Pregnant or nursing women

          -  Known to be HIV positive

          -  Radiation therapy less than 2 weeks prior to registration
Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Condition: Fragile X Tremor/Ataxia Syndrome
MeSH terms: Ataxia; Cerebellar Ataxia; Tremor; Syndrome
Intervention: citocoline
Intervention description: Ten subjects will receive 1,000mg once daily of citicoline. Each subject will stay on the study drug for 12 months. The outcome measures will be assessed at baseline and then again at the end of month 3, month 6, and month 12. The subjects will continue taking the same study drug at the same dose throughout the entire trial.
Intervention MeSH terms: Choline
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Minimum age: 18 Years
Maximum age: 90 Years
Healthy volunteers: No

Summary:

      The objective of this study is to determine if citocoline is effective for balance
      abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor
      ataxia syndrome. The study will test 1000mg twice daily of citocoline for 12 months in an
      open label pilot study, with study visits at baseline, 3, 6, and 12 months.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Clinical diagnosis of FXTAS

               -  Serum creatine kinase, complete metabolic panel, complete blood count, liver
                  function tests, renal function tests, platelets and EKG are within normal limits

        Exclusion Criteria:

          -  Legal incapacity or limited legal capacity

          -  Presence of severe renal disease (BUN 50% greater than normal or creatinine clearance
             <60 mL/min) or hepatic disease.

          -  Abnormal creatine kinase and/or platelet count in the past 6 months (as determined by
             lab reports obtained from primary care physicians or conducted at baseline).

          -  Women of childbearing potential who are pregnant at the time of screening or who will
             not use adequate protection during participation of the study.

          -  Allergy/sensitivity to the drug of its formulations.

          -  Concurrent participation in another clinical study.

          -  Active substance use or dependence.

          -  Serious illness (requiring systematic treatment/or hospitalization) until the subject
             either completes therapy or is clinically stable on therapy, in the opinion of the
             site investigator, for at least 60 days prior to study entry.

          -  Inability or unwillingness of the subject or legal guardian/representative to give
             written informed consent.
A Study to Evaluate the Efficacy and Safety of CT-P27 in Acute Uncomplicated Influenza A Infection
Condition: Influenza A
MeSH terms: Influenza, Human
Intervention: CT-P27 90 mg/kg; CT-P27 45 mg/kg; Placebo
Intervention description: Influenza A treatment drug.; Influenza A treatment drug.; Placebo.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 228.0
Sex: All
Minimum age: 19 Years
Maximum age: 64 Years
Healthy volunteers: No
Keywords: efficacy; safety

Summary:

      This study is a Phase IIb, Randomized, Double-blind, Multicenter, Placebo-controlled study
      evaluating the efficacy and safety of CT-P27 in subjects with acute uncomplicated influenza A
      infection.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or Female subjects between the ages of 19 and 64 years, both inclusive

          -  Diagnosed with influenza A at screening using rapid influenza diagnostic test

          -  Onset of influenza symptoms and fever is within 48 hours at screening

          -  At least 2 of symptoms (moderate to severe in intensity)

          -  ≥38.0'C body temperature at screening

        Exclusion Criteria:

          -  Taking antiviral treatment for influenza or has a history of using these antivirals
             within 14 days prior to the administration of study drug

          -  Positive influenza B or A+B infection
Ambrisentan in Single Ventricle
Condition: Hypoplastic Left Heart Syndrome; Hypoplastic Right-sided Heart Complex
MeSH terms: Univentricular Heart; Hypoplastic Left Heart Syndrome; Dextrocardia
Intervention: Ambrisentan; Placebo
Intervention description: Once daily oral dosing
Intervention MeSH terms: Ambrisentan
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 16.0
Sex: All
Minimum age: 24 Months
Maximum age: 120 Months
Healthy volunteers: No
Keywords: Single ventricle

Summary:

      Purpose: To evaluate the pharmacokinetics, bioavailability and hemodynamic efficacy of
      ambrisentan after Fontan surgical palliation of single ventricle heart defects.

      Study activities and population group: Children undergoing Fontan surgical palliation for
      single ventricle defects will be eligible for the study. Up to 20 subjects will be enrolled
      (16 ambrisentan, 4 placebo) and will receive 3 days (3 doses) of ambrisentan starting on
      post-operative day #1 upon returning from the operating room. Ambrisentan plasma levels will
      be obtained at specified time points during treatment. Post-operative monitoring lines will
      be used to measure effects of ambrisentan on hemodynamics and pulmonary / systemic
      endothelial function.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Age ≥ 24 months; ≤120 months.

          2. History of congenital heart disease with severe hypoplasia of a right or left
             ventricle.

          3. Undergoing Fontan surgery as part of standard clinical care.

          4. Availability and willingness of the parent/legally authorized representative to
             provide written informed consent and, as appropriate, assent from the child.

        Exclusion Criteria:

          1. History of serious adverse event related to ambrisentan administration.

          2. History of ambrisentan exposure within 48 hours of the study.

          3. Presence of pulmonary venous obstruction.

          4. Treatment with cyclosporin.

          5. Any of the following - as determined by the attending physician

               -  Significant hemodynamic instability

               -  Sepsis.

               -  Need for ECMO support.

          6. Renal failure defined as serum creatinine > 2 times higher than the upper limit of
             normal.

          7. Liver dysfunction defined as alanine aminotransferase or aspartate aminotransferase >
             3 times higher than the upper limit of normal.

          8. Thrombocytopenia defined as a platelet count < 50 000 cells/µL.

          9. Leukopenia defined as white blood cells < 2500 cells/µL.

         10. Anemia defined as hemoglobin < 8mg/dL.

         11. Atrial hypertension (mean LA pressure > 12mm Hg).
Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
Condition: Myotonic Dystrophy Type 1
MeSH terms: Myotonic Dystrophy
Intervention: rhIGF-I/rhIGFBP-3; placebo
Intervention description: 1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.; 1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.
Phase: Phase 2
Study type: Interventional
Primary purpose: Supportive Care
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 69.0
Sex: All
Minimum age: 21 Years
Maximum age: 65 Years
Healthy volunteers: No

Summary:

      To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance,
      ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and
      strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients
    

Eligibility criteria:

        Inclusion Criteria (list is not inclusive):

          -  A diagnosis of DM1, confirmed by DM1 genetic mutation

          -  Ability to walk 30 feet - assistance with cane and/or leg bracing permitted

          -  Able to self-administer study medication by subcutaneous injection or caregiver is
             available to administer study medication

        Exclusion Criteria (list is not inclusive):

          -  Congenital DM1

          -  Weight greater than 100 kg or body mass index greater than 30 kg/m2

          -  Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone,
             investigational agent within 60 days of screening

          -  Current diagnosis or history of malignancy expect for surgically cured skin cancer or
             pilomatricoma

          -  Changes in lipid lowering medications during the 3 months prior to screening

          -  Diaphragmatic weakness such that patients are unable to tolerate the supine position,
             or swallowing impairment such that patients are unable to maintain nutrition without
             use of gastrostomy.

          -  Major psychiatric illness (major depression, bipolar disorder or schizophrenia) within
             twelve months of screening

          -  History of non-compliance with other therapies
Trial of IW-1973, A Stimulator of Soluble Guanylate Cyclase (sGC) in Patients With Stable Type 2 Diabetes and Hypertension
Condition: Diabetes Mellitus, Type 2; Hypertension
MeSH terms: Hypertension; Diabetes Mellitus; Diabetes Mellitus, Type 2
Intervention: IW-1973; Placebo
Intervention description: Oral Tablet; Oral Tablet
Intervention MeSH terms: Praliciguat
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking description: Double-Blind
Enrollment: 26.0
Sex: All
Minimum age: 30 Years
Maximum age: 75 Years
Healthy volunteers: No

Summary:

      To compare the safety, tolerability, pharmacokinetic (PK) profile, and pharmacodynamic (PD)
      effects of 2 treatment regimens of IW-1973 tablet (40 mg per day) administered orally for 2
      weeks to patients with stable type 2 diabetes mellitus and hypertension.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patient is ambulatory male or female

          -  Patient's body mass index score is > 20 and < 40 kg/m^2 at the Screening Visit

          -  Women of childbearing potential must have a negative pregnancy test at the time of
             screening and check-in and must agree to use protocol-specified contraception
             throughout the duration of the study

          -  Patient's health is stable with no clinically significant findings on physical
             examination

          -  Patient has type 2 (ie, adult onset) diabetes mellitus diagnosed by a physician or
             nurse practitioner > 6 months before the Screening Visit, is on a stable glycemic
             control medication, and protocol specified hemoglobin (Hb)A1c values at the Screening
             Visit

          -  Patient has hypertension diagnosed by a physician or nurse practitioner > 6 months
             before the Screening Visit, and blood pressure (BP) within the protocol's acceptable
             range

          -  Patients must be on a stable regimen for hypertension control that includes an
             angiotensin converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB),
             stable for 28 days

          -  Other inclusion criteria per protocol

        Exclusion Criteria:

          -  Patient has a clinically significant active or unstable medical condition that, in the
             opinion of the Investigator, would preclude trial participation

          -  Patient is on medication(s) that, when co-administered with a sGC stimulator, could
             increase the risk of hypotension

          -  Patient has evidence of severe or active end-organ damage

          -  Patient is an active smoker or has used any nicotine-containing products (cigarettes,
             e-cigarettes, vape pens, cigars, chewing tobacco, gum, patches) during the 6 months
             before Check-in. Use of nicotine is excluded during the study until after the End of
             Trial Visit.

          -  Other exclusion criteria per protocol
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Condition: Familial Partial Lipodystrophy
MeSH terms: Lipodystrophy, Familial Partial; Lipodystrophy
Intervention: AKCEA-ANGPTL3-LRx
Intervention description: AKCEA-ANGPTL3-LRx solution for SC injection.
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: 18 Years
Healthy volunteers: No
Keywords: Lipodystrophy; Lipodystrophy, Familial Partial; Lipid Metabolism Disorders; Dyslipidemias; Kobberling-Dunnigan syndrome (type 1 and 2); Lipoatrophic Diabetes

Summary:

      This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for
      reduction of fasting triglycerides in participants with familial partial lipodystrophy.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Must give written informed consent to participate in the study.

          -  Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes
             mellitus and hypertriglyceridemia.

          -  Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with
             hemoglobin A1c (HbA1c) ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as
             defined in study protocol.

          -  Hypertriglyceridemia as defined by fasting triglycerides (TG) levels ≥ 500 milligrams
             per deciliter (mg/dL) at both Screening and Qualification visits. Participants with
             the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 millimoles per
             liter [mmol/L]) to < 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or family history
             criteria for study inclusion may be further screened and enrolled in the study.

          -  Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic
             resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.

        Key Exclusion Criteria:

          -  Diagnosis of generalized lipodystrophy.

          -  Diagnosis of acquired partial lipodystrophy (APL).

          -  Acute pancreatitis within 4 weeks of Screening.

          -  Acute coronary syndrome within 6 months of Screening.

          -  Major surgery within 3 months of Screening.

          -  Have any other conditions in the opinion of the investigator which could interfere
             with the participant participating in or completing the study.
Efficacy and Safety of Increasing Doses of DaxibotulinumtoxinA for Injection (DAXI for Injection) in the Treatment of Moderate or Severe Lateral Canthal Lines
Condition: Lateral Canthal Lines
Intervention: DaxibotulinumtoxinA for injection
Intervention description: Intramuscular injection
Intervention MeSH terms: Botulinum Toxins, Type A; abobotulinumtoxinA
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Masking: None (Open Label)
Enrollment: 63.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Lateral canthal wrinkle; Crow's feet lines; LCL

Summary:

      A Phase 2a, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Treatment of
      Moderate or Severe Lateral Canthal Lines (LCL).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Provide written informed consent consistent with International Conference on
             Harmonisation (ICH)-Good Clinical Practice (GCP) guidelines and local laws, including
             authorization to release health information, signed prior to any study procedures
             being performed

          -  Be outpatient, male or female subjects, in good general health, 18-65 years old

          -  Have a score of moderate (2) or severe (3) LCL at maximum smile effort as assessed by
             the IGA-LCWS

          -  Able to understand the requirements of the study and be willing and able to follow all
             study procedures, attend all scheduled visits, and successfully complete the study.

        Exclusion Criteria:

          -  Active skin disease, infections, or inflammation at the injection sites

          -  History of clinically significant bleeding disorders

          -  Clinically significant laboratory values at screening that may interfere with a
             subject's ability to complete the study, as determined by the investigator

          -  Planned or anticipated need for surgery or hospitalization through the end of the
             study

          -  Pregnant, nursing, or planning a pregnancy during the study; or is Women of Child
             Bearing Potential (WOCBP) but is not willing to use an effective method of
             contraception

          -  Current enrollment in an investigational drug or device study or participation in such
             a study within the last 30 days prior to screening through the end of the study
In Vivo Patient Preoperative Skin Prep Persistence
Condition: Bacterial Recovery of Skin Flora Post-Product Application
Intervention: 3M CHG/IPA Surgical Skin Preparation - Abdominal Region; Normal Saline - Abdominal Region; 3M CHG/IPA Surgical Skin Preparation - Inguinal Region; Normal Saline - Inguinal Region
Intervention description: Chlorhexidine gluconate 2% / Isopropyl alcohol 70%; 0.9% sodium chloride applied with foam applicator; Chlorhexidine gluconate 2% / Isopropyl alcohol 70%; 0.9% sodium chloride applied with foam applicator
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Enrollment: 27.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: Accepts Healthy Volunteers

Summary:

      The objective of the study is to demonstrate persistence of the CHG/IPA Prep on skin flora of
      the abdominal and inguinal regions of human subjects.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects of any race

          -  Subjects in good general 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 areas

          -  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
Fragmin for the Treatment of Acute VTE in Pediatric Cancer Patients
Condition: Venous Thromboembolism
MeSH terms: Thromboembolism; Venous Thromboembolism
Intervention: dalteparin
Intervention description: dalteparin subcutaneous injection
Intervention MeSH terms: Dalteparin; Tinzaparin; Heparin, Low-Molecular-Weight
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 38.0
Sex: All
Maximum age: 18 Years
Healthy volunteers: No
Keywords: VTE

Summary:

      Three month treatment of acute VTE with Fragmin in pediatric cancer patients
    

Eligibility criteria:

        Inclusion Criteria:

        -

        Exclusion Criteria:

        -