bioihbjkbjnl/mcl2 huggingface dataset:
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
MeSH terms: Wolman Disease
Intervention: Sebelipase Alfa
Intervention description: Sebelipase alfa is a recombinant human lysosomal acid lipase. The investigational medicinal product is an enzyme replacement therapy intended for treatment of participants with LAL-D. Dosing occurred once weekly for up to 3 years.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Maximum age: 8 Months
Healthy volunteers: No
Keywords: LIPA; Wolman Disease; Wolman Phenotype; Acid Lipase Deficiency; Acid Cholesteryl Hydrolase; Acid Lipase Disease Deficiency, type 2; Cholesteryl Ester Storage Disease (CESD); Cholesteryl Ester Hydrolase Deficiency; Early Onset Lysosomal Acid Lipase Deficiency (Wolman Disease); LAL Deficiency; Late Onset Lysosomal Acid Lipase Deficiency (CESD); Wolman Disease (early onset LAL Deficiency); Related Disorders:; Non-alcoholic Fatty Liver Disease (NAFLD); Non-alcoholic Steatohepatitis (NASH); Alcoholic Liver Disease; Cryptogenic Cirrhosis; Niemann-Pick Disease (NPD) Type C; Chanarin Dorfman Syndrome

Summary:

      This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly
      progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received
      once-weekly infusions of sebelipase alfa for up to 3 years.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Participant's parent or legal guardian (if applicable) consent to participation in the
             study

          2. Confirmation of documented decreased LAL activity relative to the normal range of the
             lab performing the assay or confirmation of LAL-D diagnosis as determined by a
             Sponsor-approved central laboratory

          3. Substantial clinical concerns, in the opinion of Investigator and Sponsor, of rapid
             disease progression requiring urgent medical intervention including, but not
             restricted to the following:

               -  Marked abdominal distension and hepatomegaly

               -  Failure to thrive

               -  Disturbance of coagulation

               -  Severe anemia

               -  Sibling with rapidly progressive course of LAL-D

        Exclusion Criteria:

          1. Clinically important concurrent disease

          2. Participant was > 8 months of age at the time of first dosing

          3. Participant received an investigational medicinal product other than sebelipase alfa
             within 14 days prior to the first dose of sebelipase alfa in this study

          4. Myeloablative preparation, or other systemic pre-transplant conditioning, for
             hematopoietic stem cell or liver transplantation

          5. Previous hematopoietic stem cell or liver transplant

          6. Known hypersensitivity to eggs
Evaluation of the Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Participants With Acute Respiratory Distress Syndrome (ARDS) Associated With at Least Severe COVID-19
Condition: Acute Respiratory Distress Syndrome; SARS-CoV-2
MeSH terms: Respiratory Distress Syndrome; Respiratory Distress Syndrome, Newborn; Acute Lung Injury; Syndrome
Intervention: PLN-74809; Placebo
Intervention description: PLN-74809; 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: 6.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      Evaluation of the safety, tolerability, and pharmacokinetics of PLN-74809 in participants
      with acute respiratory distress syndrome (ARDS) associated with at least severe COVID-19
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of ARDS (Berlin Criteria)

          -  Hospitalized with at least severe COVID-19 (FDA 2020)

          -  Receiving support for acute lung injury/respiratory distress via supplemental oxygen

          -  Serum aspartate aminotransferase (AST) concentration ≤ 120 U/L and serum alanine
             aminotransferase (ALT) concentration ≤ 150 U/L

          -  Serum total bilirubin ≤ 1.8 mg/dL, in the absence of Gilbert's syndrome or hemolysis

        Exclusion Criteria:

          -  Greater than 72 hours since time of onset of ARDS.

          -  Greater than 7 days since start of mechanical ventilation.

          -  Currently receiving or anticipated to receive extracorporeal life support (ECLS),
             extracorporeal membrane oxygenation (ECMO) or high-frequency oscillatory ventilation

          -  Unwillingness to follow lung protective ventilation strategy (i.e., tidal volume of 6
             mL/kg of predicted body weight and prone positioning) and fluid management protocol
             (Fluids and Catheters Treatment Trial [FACTT] Conservative or Lite) per local
             institutional standards (HFOV).
The HepQuant SHUNT Test for Monitoring Liver Disease and Treatment Effects by Measuring Liver Function and Physiology
Condition: NASH - Nonalcoholic Steatohepatitis
MeSH terms: Liver Diseases; Fatty Liver; Non-alcoholic Fatty Liver Disease
Intervention: HepQuant SHUNT Liver Diagnostic Kit
Intervention description: Serial testing over 48 weeks
Phase: Phase 2
Study type: Interventional
Primary purpose: Diagnostic
Intervention model: Sequential Assignment
Intervention model description: All subjects receive HepQuant SHUNT test and DSI measurement, but must first be enrolled into one of the three Gilead clinical trials listed above before they can be enrolled in the HepQuant study. Since the assigned intervention, the HepQuant SHUNT test, is the same for all participants in all three Gilead clinical trials, there is only one arm and intervention for the HepQuant study itself. Some analyses are conducted by grouping subjects into those who took the placebo in the parallel drug study versus those who were on active study drug in the parallel study.
Masking: None (Open Label)
Enrollment: 17.0
Sex: All
Minimum age: 18 Years
Maximum age: 70 Years
Healthy volunteers: No

Summary:

      The HepQuant SHUNT test, which is provided as a HepQuant SHUNT Liver Diagnostic Kit, is a
      minimally-invasive test of liver function and physiology which has been designated by the FDA
      as an investigational drug/device combination product.

      Enrollment into one of the 3 Gilead Selonsertib clinical trials (GS-US-416-2124,
      GS-US-384-1943/1944) is required for enrollment into the HepQuant study
    

Eligibility criteria:

        Inclusion Criteria:

        The key inclusion criteria for this HepQuant Parallel Study are the inclusion criteria into
        one of the Gilead selonsertib trials (GS-US-416-2124, GS-US-384-1943 or 1944). Once that
        has been met, subjects must:

          -  Be able to ingest liquid by mouth

          -  Have venous access to support a peripheral IV and 6 blood draws

        Exclusion Criteria:

        The key exclusion criteria for this HepQuant Parallel Study are the exclusion criteria for
        one of the Gilead selonsertib trials (GS-US-416-2124, GS-US-384-1943 or 1944). If this has
        been met, the subject is not eligible to participate in HepQuant. If no exclusion criteria
        has been met for the Gilead selonsertib trial, ADDITIONAL exclusions for HepQuant are:

          -  Subject should not have had first dose of selonsertib

          -  Subject cannot take anything by mouth

          -  Subject cannot be hypersensitive to eggs, albumin preparations, any ingredient in the
             formulation, or component of the container

          -  Subject cannot have intercurrent medical or surgical illness (eg: acute MI, acute
             cerebral hemorrhage, sepsis)

          -  subject cannot have had extensive resection of large segments of small intestine
             (short gut)

          -  Subject cannot have severe gastroparesis
Dose Ranging Study To Assess Efficacy, Safety and Tolerability Of PF-06700841 Topical Cream In Psoriasis
Condition: Psoriasis
MeSH terms: Psoriasis
Intervention: PF-06700841; Vehicle (Placebo)
Intervention description: PF-06700841 topical cream; Vehicle topical cream
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: 344.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: psoriasis; topical

Summary:

      This is a Phase 2b, randomized, double blind, vehicle controlled, parallel group, multicenter
      study in participants with mild to moderate plaque psoriasis. The duration of study
      participation will be approximately 22 weeks, including up to a 6 week screening period, 12
      week treatment period, and approximately 4 week follow up period. Approximately 280
      participants are planned to be randomized into the study.
    

Eligibility criteria:

        Inclusion Criteria:

          -  plaque psoriasis for 6 months

          -  PGA score mild or moderate

          -  body surface area (BSA) 2-15%

        Exclusion Criteria:

          -  other skin conditions that would interfere with the evaluation of psoriasis

          -  history of herpes zoster or simplex

          -  Infected with Mycobacterium tuberculosis
Efficacy Of A PVP-I Fluoride Varnish
Condition: Dental Caries
MeSH terms: Dental Caries
Intervention: Test varnish; Standard varnish
Intervention description: Topical application of test varnish to the teeth 4 times per year.; Topical application of test varnish to the teeth 4 times per year
Intervention MeSH terms: Fluorides; Sodium Fluoride; Fluorides, Topical
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Single-center, double-blind, controlled Phase 2 study with parallel groups of children.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking description: All study personnel except for designated personnel in the data center will be blinded to the participant treatment assignment. In order to maintain blinding, the caries scoring examinations will precede the application of test varnishes at baseline. The staff member applying these varnishes will not perform the caries scoring exams in order to eliminate examiner bias and ensure blinding to treatment assignment. Test drug and comparator are packaged identically.
Enrollment: 284.0
Sex: All
Minimum age: 60 Months
Maximum age: 84 Months
Healthy volunteers: No
Keywords: sodium fluoride; povidone iodine; varnish

Summary:

      The purpose of the study is to determine the efficacy of Advantage Anti-Caries Varnish.
      Single-center, double-blind, controlled Phase 2 study with parallel groups of children.
      Subjects will be stratified by early childhood education center and then randomized to
      receive either test varnish or control varnish topically to the teeth. Treatment will be
      administered quarterly for up to 24 months. The primary outcome is surface-level primary
      molar caries increment (d2-4mfs) at 24- months post baseline.
    

Eligibility criteria:

        Inclusion Criteria:

          1. The subject's parent or legal guardian must provide signed and dated informed consent
             (parent permission form).

          2. The subject's parent or legal guardian of the subject must be willing and able to
             comply with study requirements.

          3. The subject is aged 60-84 months at the time of enrollment.

          4. The subject must be in good general health as evidenced by parent report.

        Exclusion Criteria:

          1. Known allergy to iodine

          2. Known allergy to seafood

          3. Known hypersensitivity to fluoride varnish

          4. Diagnosis of thyroid disease

          5. Chronic, prophylactic use of antibiotics

          6. Treatment with another investigational drug or other intervention within 30 days
             preceding the Baseline Visit.
Comparison of A Single Dose Combination of Methadone and Morphine With Morphine Alone for Treating Post-operative Pain
Condition: Pain
MeSH terms: Pain, Postoperative
Intervention: Methadone; methadone and morphine
Intervention description: Upon the 1st request for analgesic medication morphine 2 mg, upon the 2nd request for analgesic medication morphine 2 mg; Upon the 1st request for analgesic medication morphine 2 mg, upon the 2nd request for analgesic medication morphine 1 mg and methadone 1 mg
Intervention MeSH terms: Morphine; Methadone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 50.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Pain; Analgesics; Retroperitoneal lymph node dissection

Summary:

      This is a randomized Phase II study testing the effectiveness of the combination of morphine
      and methadone versus morphine alone in relieving pain. A second goal is to further evaluate
      any side effects of the combination of morphine and methadone.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Retroperitoneal lymph node dissection

          -  Planned post-operative analgesia with PCA at 1 mg continuous infusion and 1 mg every
             10 minutes

          -  18 years of age or older

          -  English-speaking

          -  Give informed consent to participate in this study

        Exclusion Criteria:

          -  Known hypersensitivity to methadone or morphine

          -  Patients with past or present history of substance abuse

          -  Patients with a history of methadone treatment

          -  Patients with a history of chronic pain requiring daily analgesic use for more than 3
             months

          -  Patients treated with opioids within one month from the scheduled surgery

          -  Creatinine clearance less than 50 mg/kg (using Cockcroft-Gault Equation).

          -  Neurologic or psychiatric disease sufficient, in the doctor's opinion, to compromise
             data collection
Efficacy Study of Intranasal Insulin to Treat Tobacco Abstinence Syndrome
Condition: Tobacco Abstinence Syndrome
MeSH terms: Syndrome
Intervention: Intranasal Insulin; Placebo
Intervention MeSH terms: Insulin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 51.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: Accepts Healthy Volunteers

Summary:

      This study will evaluate safety and efficacy of intranasal insulin in abstinent smokers.
      Groups' (placebo vs. insulin) cognitive function and stress response will be compared.
    

Eligibility criteria:

        Inclusion Criteria:

          -  smokers (>10 cig/day) for the past 1 year

          -  normosmic olfactory function

        Exclusion Criteria:

          -  previous/current use of insulin

          -  current Diagnostic and Statistical Manual-IV-Revised (DSM-IV-R) Axis I disorder

          -  current pregnancy (or lactation)

          -  lifetime history of endocrine disease

          -  excessive alcohol use (>25 standard units of alcohol/week)

          -  current use of illicit drugs

          -  current use of a smoking cessation aid (NRT, Chantix or Wellbutrin), or a psychotropic
             agent

          -  local infections, inflammation, structural abnormalities, or other nasal pathology

          -  current use of any medications administered intranasally, including intranasal
             steroids
Efficacy Study in Patients With Acne Vulgaris.
Condition: Acne Vulgaris
MeSH terms: Acne Vulgaris
Intervention: ADPS topical product; Placebo Control
Intervention description: topical product; topical product
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 294.0
Sex: All
Minimum age: 9 Years
Healthy volunteers: Accepts Healthy Volunteers

Summary:

      Safety and efficacy study in patients with acne vulgaris
    

Eligibility criteria:

        Inclusion Criteria:

          -  Healthy male or non pregnant female aged ≥ 9 years with a clinical diagnosis of acne
             vulgaris

        Exclusion Criteria:

          -  Female Subjects who are pregnant, nursing or planning to become pregnant during study
             participation.
A Safety Extension Study to Evaluate the Long-term Safety of QGE031 in Chronic Spontaneous Urticaria (CSU) Patients
Condition: Chronic Spontaneous Urticaria
MeSH terms: Urticaria; Chronic Urticaria
Intervention: Ligelizumab
Intervention description: QGE031 240 mg s.c. q4w
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 226.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: QGE031; chronic; spontaneous; Urticaria; CSU; adults; Safety; ligelizumab

Summary:

      A safety extension study to evaluate the long-term safety of QGE031 240 mg s.c. given every 4
      weeks for 52 weeks in Chronic Spontaneous Urticaria (CSU) patients who completed study
      CQGE031C2201
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients eligible for inclusion in this study have to fulfill all of the following
             criteria:

               1. Written informed consent must be obtained before any assessment is performed.

               2. Patients who complete the treatment epoch in study CQGE031C2201 and complete at
                  least Visit 203 (Week 32 of the follow-up epoch, ≥16 weeks after last injection)
                  and present with active disease as defined by UAS7 ≥12.

               3. Patients must not have any missing eDiary entries in the 7 days prior to Visit
                  301 (patients are allowed to repeat until this criterion is met).

               4. Willing and able to complete a daily symptom eDiary for the duration of the study
                  and adhere to the study visit schedules.

        Exclusion Criteria:

        Clearly defined underlying etiology for chronic urticaria other than chronic spontaneous
        urticaria

          -  Evidence of parasitic infection

          -  Any other skin diseases than chronic spontaneous urticaria with chronic itching

          -  Contraindications to or hypersensitivity to fexofenadine, loratadine, cetirizine, or
             epinephrine

          -  History of anaphylaxis

          -  History or current diagnosis of ECG abnormalities indicating significant risk of
             safety for patients participating in the study

          -  History of hypersensitivity to any of the study drugs or its components of similar
             chemical classes

          -  Pregnant or nursing (lactating) women Other protocol-defined inclusion/exclusion
             criteria may apply
BI 655066 (Risankizumab) Compared to Placebo in Japanese Patients With Moderate to Severe Chronic Plaque Psoriasis
Condition: Psoriasis
MeSH terms: Psoriasis
Intervention: risankizumab; placebo for risankizumab
Intervention description: Risankizumab pre-filled syringe, administered by subcutaneous (SC) injection; Placebo for risankizumab pre-filled syringe, administered by subcutaneous (SC) injection
Intervention MeSH terms: Antibodies, Monoclonal
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 182.0
Sex: All
Minimum age: 20 Years
Healthy volunteers: No
Keywords: BI 655066; ABBV-066; risankizumab

Summary:

      This is a randomized double blind, double dummy, placebo controlled, parallel design study
      that is being performed to assess the safety and efficacy of BI 655066 (risankizumab).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Have a diagnosis of chronic plaque psoriasis (with or without psoriatic arthritis) for
             at least 6 months before the first administration of study drug. Duration of diagnosis
             may be reported by the patient.

          -  Have stable moderate to severe chronic plaque psoriasis with or without psoriatic
             arthritis at both Screening and Baseline (Randomisation):

               1. Have an involved body surface area (BSA) ≥10% and

               2. Have a Psoriasis Area and Severity Index (PASI) score ≥12 and

               3. Have a Static Physician Global Assessment (sPGA) score of ≥3.

        Exclusion Criteria:

          -  Patients with

               1. non-plaque forms of psoriasis (including guttate, erythrodermic, or pustular)

               2. current drug-induced psoriasis (including an exacerbation of psoriasis from beta
                  blockers, calcium channel blockers, or lithium)

               3. active ongoing inflammatory diseases other than psoriasis and psoriatic arthritis
                  that might confound trial evaluations according to investigator's judgment

          -  Previous exposure to BI 655066
A Study of RPL554 Drug Administered by Metered Dose Inhaler to Treat Chronic Obstructive Pulmonary Disease
Condition: COPD
Intervention: Part A: RPL554; Placebos; Part B: RPL554
Intervention description: Single dose RPL554 via metered dose inhaler.; Part A: Single dose placebo via metered dose inhaler. Part B: Repeat doses of placebo via metered dose inhaler in crossover fashion. One dose administered twice daily over 7 days.; Part B: Repeat doses via metered dose inhaler in crossover fashion. One dose administered twice daily over 7 days.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Double (Participant, Investigator)
Masking description: Double blind
Enrollment: 40.0
Sex: All
Minimum age: 40 Years
Maximum age: 80 Years
Healthy volunteers: No
Keywords: COPD; Adults; Phase II; RPL554; Ensifentrine

Summary:

      The purpose of this study is to investigate 5 doses of RPL554 and placebo, administered by
      pressurized metered dose inhaler (pMDI), in patients with moderate to severe chronic
      obstructive pulmonary disease (COPD).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male and female patients with moderate to severe COPD, with a post bronchodilator FEV1
             of 40 to 80% of predicted and FEV1/FVC ratio of ≤0.70.

          -  They must have a baseline increase in FEV1 of >150 mL following four puffs of
             salbutamol.

          -  They must have at least a 10 pack-year smoking history, and may be either a current or
             former smoker.

        Exclusion Criteria:

          -  Patients must be clinically stable without recent COPD exacerbations or
             hospitalisations.

          -  They must not have uncontrolled disease or chronic heart failure.