bioihbjkbjnl/mcl2 huggingface dataset:
CD34+ Stem Cell Infusion to Augment Graft Function
Condition: Waning Donor Chimerism; Waning Immune Function; Primary Immunodeficiency Disease(s); Bone Marrow Failure
MeSH terms: Bone Marrow Failure Disorders; Pancytopenia; Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: CD34+
Intervention description: CD34+ cells are selected using the CliniMACS System; without preparative regimen
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 23.0
Sex: All
Maximum age: 35 Years
Healthy volunteers: No
Keywords: Primary Immunodeficiency Disease(s); Mixed Donor Chimerism; bone marrow failure syndromes; allogeneic hematopoietic stem cell transplant (HSCT); children

Summary:

      The purpose of this study is to determine if infusing additional special donor cells will
      help to improve graft or immune function in previously transplanted children with immune
      deficiencies and bone marrow failures.
    

Eligibility criteria:

        Inclusion Criteria:

        To be eligible for this protocol, patients must have the following:

          1. Primary immunodeficiency (e.g. SCID, Wiskott-Aldrich and/or other more rare conditions
             and other bone marrow failure syndromes) with prior allogeneic stem cell transplant.

          2. Waning donor chimerism or immune function that is inadequate to correct their disease
             or clinical condition, for which primary transplant was given, as determined by their
             attending physician.

          3. Available primary donor.

          4. Must not have other organ dysfunction deemed by the attending physician to preclude
             this procedure.

          5. Age < 35 years at time of transplant

          6. One of the following must be true:

               -  Patients must have evidence of persistent or recurrent immunodeficiency or
                  thrombocytopenia.

        -OR-

        • Primary immunodeficiency disease with known potential to progress to malignant condition
        if untreated.

        -OR-

        • Debilitating secondary disease known to be a consequence of inadequate immune response to
        known agent or pathogen, uncontrollable by other available medical therapies (e.g. third
        patient described on page 5).

        Exclusion Criteria:

          1. Absence of an available original donor

          2. Failure to sign consent form, or inability to undergo informed consent process

          3. Pregnant or lactating female

          4. Uncontrolled GVHD
A Safety, Efficacy and Pharmacokinetics Study of CD11301 for the Treatment of Cutaneous T-Cell Lymphoma (CTCL)
Condition: Cutaneous T Cell Lymphoma
MeSH terms: Lymphoma; Lymphoma, T-Cell; Lymphoma, T-Cell, Peripheral; Lymphoma, T-Cell, Cutaneous
Intervention: Placebo; CD11301 0.03%; CD11301 0.06%
Intervention description: Non active ingredients of CD11301; Topical Gel; Topical Gel
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 86.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: T-Cell; Lymphoma; Cutaneous; CTCL

Summary:

      To assess the efficacy, safety and pharmacokinetics in participants treated with CD11301 gel
      vs. placebo for early stage CTCL (IA, IB, or IIA).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Clinical Diagnosis of CTCL stage IA, IB, or IIA with biopsy within last 3 months

          -  Have BSA involvement corresponding to stages IA, IB or IIA CTCL with at least 3
             distinct lesions

        Exclusion Criteria:

          -  CTCL that is stage IIB or great or stage IIA with stage N2 with >5% circulating Sezary
             cells or CD8+ or large cell transformation or Progressive CTCL

          -  History of autoimmune disease

          -  Laboratory test values at screening outside of the normal range and judged clinically
             significant by the investigator

          -  Current participation in another clinical trial of a drug or device or past
             participation within 4 weeks before Baseline or participant is in exclusion period
             from a previous clinical trial
Propranolol Dose Escalation in Lymphedema in Patients
Condition: Primary Lymphedema
MeSH terms: Lymphedema
Intervention: Propranolol
Intervention description: escalating doses of propranolol from 1mg/kg/day to 2mg/kg/day to 3mg/kg/day
Intervention MeSH terms: Propranolol
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 1.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: Accepts Healthy Volunteers

Summary:

      This is a study to investigate the safety and efficacy of propranolol in the treatment of
      patients with primary lymphedema. The primary goal is to assess patient tolerability of
      increasing doses of propranolol. The secondary goal is to assess lymphedema symptoms and
      signs in response to propranolol treatment.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Primary lymphedema

          -  Measurable disease

          -  Adequate functional status: Karnofsky >50% (>age 16), Lanky >50 (<age 16),

          -  No prior therapy within 4 weeks of enrollment

          -  Adequate bone marrow, renal function, cardiac, and pulmonary function, negative
             pregnancy test (for women).

        Exclusion Criteria:

          -  Secondary lymphedema

          -  Patients already receiving other investigational drugs

          -  Patients with known contraindications to receiving propranolol

          -  Other medical comorbidities including but not limited to: pheochromocytoma,
             bradycardia, bronchospasm/reactive airway disease, decompensated heart failure, heart
             block, ongoing active infections.
Study to Evaluate the Safety and Efficacy of CTP-543 in Adults With Moderate to Severe Alopecia Areata
Condition: Alopecia Areata
MeSH terms: Alopecia; Alopecia Areata
Intervention: CTP-543; CTP-543 matching placebo
Intervention description: Administered as tablets.; Administered as tablets.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 149.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No

Summary:

      This study will evaluate the safety and efficacy of CTP-543 on hair loss in adults with
      chronic, moderate to severe alopecia areata.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Definitive diagnosis of alopecia areata with a current episode lasting at least 6
             months and not exceeding 10 years at the time of Screening. Total disease duration
             greater than 10 years is permitted.

          -  At least 50% scalp hair loss, as defined by a Severity of Alopecia Tool (SALT) score
             ≥50, at Screening and Baseline.

          -  Clinical lab results within the normal range

        Exclusion Criteria:

          -  Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical
             treatment to the scalp, significant trauma to the scalp, or untreated actinic
             keratosis on the scalp.

          -  Treatment with systemic immunosuppressive medications or biologics.

          -  Vaccination with herpes zoster vaccine or any live virus within 6 weeks of screening
             or during the study
Ibritumomab Tiuxetan (Zevalin)+ Rituximab Maintenance
Condition: Non-Hodgkin's Lymphoma
MeSH terms: Lymphoma; Lymphoma, Non-Hodgkin
Intervention: Ibritumomab Tiuxetan (Zevalin) + Rituximab
Intervention description: Subjects will receive the Ibritumomab Tiuxetan (Zevalin) therapeutic regimen; then rituximab consolidation and maintenance therapy every 3 months until disease progression
Intervention MeSH terms: Rituximab; Antibodies, Monoclonal
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 18.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Zevalin, High Tumor Burden Indolent Non-Hodgkin's Lymphoma, Ibritumomab Tiuxetan

Summary:

      Subjects will receive the Ibritumomab Tiuxetan (Zevalin) therapeutic regimen; then rituximab
      consolidation and maintenance therapy every 3 months until disease progression
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients with biopsy-proven non-hodgkins lymphoma of follicular grade 1, 2, or 3

          -  Meeting FLIPI criteria for intermediate or high risk.

          -  No prior chemotherapy, radiotherapy or immunotherapy for lymphoma;

          -  Patients may not have known HIV infection, and must not be Hepatitis B Surface Antigen
             positive.

        Exclusion Criteria:

          -  May not be pregnant or breastfeeding, have documented CNS (Central Nervous System)
             disease, G-CSF (Granulocyte Colony Stimulating Facto) or GM-CSF
             (Granulocyte/Macrophage Colony Stimulating Factor) within 2 weeks prior
An Open Label Pharmacokinetic, Safety And Efficacy Study Of Maraviroc In Combination With Background Therapy For The Treatment Of HIV-1 Infected, CCR5 -Tropic Children
Condition: Human Immunodeficiency Virus
MeSH terms: Acquired Immunodeficiency Syndrome; HIV Infections
Intervention: Maraviroc
Intervention description: Maraviroc will be administered twice daily either as a liquid or tablet formulation, depending on the age of the subject. The dosage administered will be dependent upon the subject's body surface area as well as the background therapy.
Intervention MeSH terms: Maraviroc
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 103.0
Sex: All
Minimum age: 2 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: Open label pharmacokinetic safety and efficacy in HIV-1 infected pediatrics

Summary:

      The primary purpose of this study is to determine the pharmacokinetic properties (what the
      body does to maraviroc) and to determine a suitable dosing schedule of maraviroc in HIV-1
      infected children and adolescents. This study will also determine whether maraviroc is safe
      to use in children and adolescents.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects who are 2-18 years of age, treatment experienced for 6 months or longer with
             at least 2 ARV drug classes, with HIV-1 RNA ≥1,000 copies/mL

        Exclusion Criteria:

          -  X4- or dual/mixed-tropic virus detected by the Trofile™ viral tropism assay

          -  Concomitant therapy with other investigational agents (other than experimental ARV
             agents available through pre-approval access programs)

          -  Known ≥Grade 3 of any of the following laboratory tests at Screening or within 30 days
             prior to Baseline Visit: Neutrophil count, hemoglobin, platelets, AST, ALT, and
             creatinine, lipase;

          -  Total bilirubin ≥Grade 3, unless ALL of the following are true: Current regimen
             includes atazanavir; ALT/AST < 2.5 X ULN; No symptoms other than jaundice or icterus.

          -  Other laboratory values ≥Grade 3, must be reviewed by Pfizer.
Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE)
Condition: Acute Episode of Overt Hepatic Encephalopathy; Hepatic Encephalopathy
MeSH terms: Hepatic Encephalopathy; Brain Diseases; Hyperammonemia
Intervention: ornithine phenylacetate; placebo intravenous infusion
Intervention description: Ornithine phenylacetate for continuous IV infusion at dose levels predicated on level of hepatic decompensation; Placebo for continuous IV infusion that is visually identical to the experimental product
Intervention MeSH terms: Phenylacetic acid
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Masking description: While the study was double-blind (participant and investigator blinded), the care provider and outcomes assessor were also blinded.
Enrollment: 231.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: hepatic encephalopathy; hyperammonemia; cirrhosis; ornithine phenylacetate; OCR-002

Summary:

      The purpose of this study is to determine whether ornithine phenylacetate can speed recovery
      from an acute hepatic encephalopathy episode requiring hospitalization in cirrhotic patients.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Hospitalized with an acute episode of hepatic encephalopathy as complication of
             cirrhosis

          -  Elevated venous ammonia

        Exclusion Criteria:

          -  Renal failure with serum creatinine > 3 mg/dL or need for dialysis

          -  Molecular Adsorbent Recirculation System utilized

          -  Pregnancy or breastfeeding
Phase 2a Evaluation of PLN-74809 on αvβ6 Receptor Occupancy Using PET Imaging in Participants With IPF/
Condition: Idiopathic Pulmonary Fibrosis
MeSH terms: Pulmonary Fibrosis; Idiopathic Pulmonary Fibrosis
Intervention: PLN-74809; Knottin tracer
Intervention description: PLN-74809; Radiotracer
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Crossover Assignment
Intervention model description: Open label, incomplete crossover, up to 2 single doses administered at two different times
Masking: None (Open Label)
Masking description: No masking
Enrollment: 9.0
Sex: All
Minimum age: 40 Years
Healthy volunteers: No

Summary:

      A study in mild/moderate IPF patients using an αVβ6 PET ligand to evaluate target engagement.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of IPF, within 5 years prior to Screening,

          -  FVC %predicted ≥45%; historical FVC for entry in the study is permitted if within 1
             month of screening.

          -  DLco (hemoglobin-adjusted) ≥30%; historical DLco for entry in the study is permitted
             if within 1 month of screening.

          -  Participants receiving treatment for IPF with nintedanib or pirfenidone are allowed,if
             on a stable dose for at least 3 months

        Exclusion Criteria:

          -  Currently receiving or planning to initiate treatment for IPF (fibrosis) with agents
             not approved for that indication by the FDA

          -  Forced expiratory volume during the first seconds of the forced breath (FEV1)/FVC
             ratio <0.7

          -  Clinical evidence of active infection, including but not limited to bronchitis,
             pneumonia, sinusitis that can affect FVC measurement or IPF progression

          -  Known acute IPF exacerbation or suspicion by the Investigator of such, within 6 months
             of Screening

          -  Smoking of any kind within 3 months of Screening
A Safety and Efficacy Study of NS2 in Patients With Anterior Uveitis
Condition: Non-infectious Anterior Uveitis
MeSH terms: Uveitis; Uveitis, Anterior; Iridocyclitis
Intervention: NS2; Prednisolone acetate ophthalmic suspension (1%)
Intervention description: NS2 ophthalmic drops (0.5%)
Intervention MeSH terms: Prednisolone; Methylprednisolone Acetate; Methylprednisolone; Methylprednisolone Hemisuccinate; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Care Provider, Investigator, Outcomes Assessor)
Enrollment: 45.0
Sex: All
Minimum age: 18 Years
Maximum age: 85 Years
Healthy volunteers: No
Keywords: uveitis; acute uveitis; anterior uveitis; NS2; Aldeyra; prednisolone acetate

Summary:

      This is a randomized, multi-center, investigator masked, comparator controlled study. The
      purpose of this study is to determine the efficacy and safety of NS2 in patients with
      non-infectious acute anterior uveitis. Subjects will be randomized 1:1:1 to receive multiple
      doses of NS2 0.5%, NS2 0.5% and Pred Forte® 1%, or Pred Forte® 1%.

      Free aldehydes are thought to be related to inflammatory diseases such as uveitis. NS2, a
      small molecule aldehyde trap, may decrease inflammation by lowering aldehyde levels.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Non-infectious anterior uveitis

          -  Grade 1 - Grade 3 anterior chamber cell count

          -  Subjects must have Intraocular pressure <25 mmHg at baseline and may only administer a
             maximum of 1 topical medication to control Intraocular pressure in the study eye.

          -  Visual acuity ≥ 20/200 in the study eye

        Exclusion Criteria:

          -  Severe/serious ocular pathology

          -  Active intermediate or posterior uveitis.

          -  Previous anterior uveitis episode in the study eye within 4 weeks of Visit 1

          -  Oral corticosteroids within 14 days of Visit 1

          -  Intravitreal or sub-Tenon corticosteroid treatment in the study eye within the past 6
             months.
Safety and Efficacy of RAD001 (Everolimus) in Combination With Letrozole in the Treatment of Postmenopausal Women With Locally Advanced or Metastatic Breast Cancer
Condition: Postmenopausal Women; Locally Advanced Metastatic Breast Cancer; Metastatic Breast Cancer
MeSH terms: Breast Neoplasms
Intervention: Everolimus; Letrozole
Intervention description: Everolimus 10 mg (2 tablets of 5 mg) once daily; Letrozole 2.5 mg once daily
Intervention MeSH terms: Everolimus; Letrozole
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 72.0
Sex: Female
Minimum age: 18 Years
Healthy volunteers: No
Keywords: RAD001 (Everolimus); Letrozole; Breast cancer; Metastatic Breast cancer; Estrogen receptor positive; Failure of Tamoxifen; Anastrozole or Examestane

Summary:

      This was a multi-center, Israeli phase II open label study evaluating treatment with RAD001
      (10 mg daily) combined with letrozole (2.5 mg daily) in postmenopausal women after recurrence
      or progression on Tamoxifen, Anastrozole or Examestane.

      There were no treatments specifically approved after recurrence or progression on AIs.
      Available options, based on common clinical practice and several treatment guidelines (e.g.
      NCCN treatment guidelines 2008), included fulvestrant.

      Combining RAD001 with letrazole was a rational approach to the treatment of advanced Brest
      Cancer, offering the potential for inhibition of tumor cell growth\ proliferation and anti
      angiogenesis while at the same time potentially preventing the development of letrazole
      resistance.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Postmenopausal women with estrogen receptor positive locally advanced or metastatic
             breast cancer after documented recurrence or progression on Tamoxifen, Anastrozole or
             Examestane.

               -  Refractory disease to hormonal therapy is defined as:

                    1. Recurrence while on, or within 12 month of end of, adjuvant treatment with
                       Tamoxifen , Anastrozole, or Exemestane.

                    2. Recurrence while on, or within 24 month of end of, adjuvant treatment with
                       Letrozole.

                    3. Progression while on Tamoxifen, Anastrozole or Exemestane treatment for
                       locally advanced or metastatic breast cancer.

        Exclusion Criteria:

          -  Prior use of chemotherapy and letrozole for Advanced Breast Cancer and mTOR inhibitors
             as the last anticancer treatment prior to study entry.

          -  Patients must have radiological evidence of recurrence or progression on last therapy
             prior to study entry.
A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Urothelial Carcinoma - (FIGHT-201)
Condition: UC (Urothelial Cancer)
MeSH terms: Carcinoma; Carcinoma, Transitional Cell
Intervention: pemigatinib; pemigatinib
Intervention description: Pemigatinib once a day by mouth for 2 consecutive weeks and 1 week off therapy.; Pemigatinib once a day by mouth continuously.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 263.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Urothelial carcinoma; fibroblast growth factor (FGF); fibroblast growth factor receptor (FGFR); FGF/FGFR alterations

Summary:

      The purpose of this study is to evaluate the overall response rate (ORR) of pemigatinib as a
      monotherapy in the treatment of metastatic or surgically unresectable urothelial carcinoma
      harboring FGF/FGFR alterations.
    

Eligibility criteria:

        Inclusion Criteria:

          -  20 years and older in Japan

          -  Histologically documented metastatic or surgically unresectable urothelial carcinoma;
             may include primary site from urethra, ureters, upper tract, renal pelvis, and
             bladder.

          -  Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

          -  Life expectancy ≥ 12 weeks.

          -  Radiographically measurable per RECIST v1.1.

          -  Documented FGF/FGFR alteration and have either 1a) failed at least 1 previous
             treatment for their metastatic or surgically unresectable urothelial carcinoma (ie,
             chemotherapy, immunotherapy) or 1b) have not received chemotherapy due to poor ECOG
             status or 2) have insufficient renal function.

        Exclusion Criteria:

          -  Prior receipt of a selective FGFR inhibitor.

          -  Use of any potent CYP3A4 inhibitors or inducers within 14 days or 5 half-lives
             (whichever is shorter) before the first dose of study drug.

          -  Inability or unwillingness to swallow pemigatinib or significant gastrointestinal
             disorder(s) that could interfere with the absorption, metabolism, or excretion of
             pemigatinib.