bioihbjkbjnl/mcl2 huggingface dataset:
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Condition: Retinal Degeneration
MeSH terms: Retinal Degeneration; Leber Congenital Amaurosis
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of the study is to determine whether gene therapy is safe and effective for the
      treatment of severe childhood blindness caused by mutations in RPE65.
    

Detailed description: 
      The main objective of the proposed trial is to determine the safety and efficacy subretinal
      administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at
      three different dosage levels in individuals with autosomal recessive severe early-onset
      retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring
      plan to investigate the safety and efficacy of vector delivery.
    

Intervention name(s): tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
Intervention type(s): Biological
Intervention description(s): Single subretinal injection of vector suspension; up to 3x10e12 vector particles
Other intervention name(s): rAAV 2/2.hRPE65p.hRPE65
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: retinal dystrophy; Leber congenital amaurosis; RPE65; gene therapy
Safety, Tolerability, and Pharmacokinetics of Naltrexone Implant (DLP-160)
Condition: Opiod Use Disorder
Study type: Interventional
Phase: Phase 1

Summary: 
      This is an Open-Label Study in Healthy Volunteers to Evaluate the Safety, Tolerability, and
      Pharmacokinetics of Switching from Oral Naltrexone HCL to DLP-160 (Naltrexone implant) to
      Intramuscular Vivitrol®
    

Detailed description: 
      This study is first-in-man clinical study of DLP-160, a Naltrexone implant product. The study
      is an open-label, single sequence design aimed at assessing the safety and tolerability of
      DLP-160 and evaluating its utility in achieving a PK profile that is comparable to the
      marketed oral and injectable forms of Naltrexone.

      Initially, a group of 2 healthy volunteers will receive the Naltrexone HCL followed by
      DLP-160 implant as a sentinel group. The safety and tolerability of the DLP-160 implant will
      be assessed in the sentinel group for at least 28 days prior to any other volunteers being
      implanted with DLP-160 in the study. Only if the safety and tolerability data, obtained from
      the sentinel group, are considered acceptable by the Investigator, will the remainder of
      healthy volunteers commence treatment in the study.

      Healthy volunteers will be enrolled and implanted with DLP-160 involving treatment of each
      study participant consisting of 3 sequential treatment periods with a washout period between
      treatments.

        1. Naltrexone HCL treatment (oral; 50 mg/day) (Day -6 to Day -2);

        2. Washout (Day -1);

        3. DLP-160 implant treatment (up to 123 days);

        4. DLP-160 removal and washout (1 day);

        5. Vivitrol® treatment (single intramuscular injection with a 30 day follow up);

        6. Follow up to completion of study participation (7 days).
    

Intervention name(s): Combination Product: Naltrexone
Intervention type(s): Combination Product
Intervention description(s): Naltrexone Implant
Other intervention name(s): DLP-160
Intervention MeSH terms: Naltrexone
Intervention model: Sequential Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Naltrexone; Addiction; Mental Health; Implant
Turkish Validity and Reliability Study of Virtual Reality Sickness Questionnaire
Condition: Sickness Behavior
MeSH terms: Illness Behavior
Study type: Observational

Summary: 
      This study is planned for the cultural adaptation and validity of the "Virtual Reality
      Sickness Questionnaire".
    

Detailed description: 
      Virtual reality (SG) creates a virtual environment that reflects a real environment and
      offers various experiences to users. Motion sickness can be observed in people after using
      virtual reality applications or simulation devices in education. There are questionnaires in
      the literature such as "Simulator Sickness Questionnaire" and "Motion Sickness
      Questionnaire". However, these questionnaires are specific surveys to evaluate symptoms that
      develop after vehicle handling or use of simulation devices for pilot training, while they
      are not ideal questionnaires to evaluate symptoms that may develop after virtual reality
      applications. Because there are important differences between virtual reality systems and
      simulation systems in terms of the appearance of motion disturbance symptoms. For this
      reason, "Virtual Reality Sickness Questionnaire" has been developed that can evaluate these
      symptoms.

      There is no Turkish version of the "Virtual Reality Sickness Questionnaire" in the
      literature. For this reason, this study is planned for the cultural adaptation and validity
      of the "Virtual Reality Sickness Questionnaire".
    

Observational model: Case-Only
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Healthy people
      
Keywords: virtual reality; sickness; validity; reliability; turkish
High-Dose Chemotherapy and Radiation Therapy Followed by Peripheral Stem Cell Transplantation in Treating Patients With Acute Leukemia in Remission
Condition: Bone Marrow Ablation; Leukemia
MeSH terms: Leukemia
Study type: Interventional
Phase: Phase 2

Summary: 
      RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing
      so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation
      may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.

      PURPOSE: Phase II trial to study the effectiveness of high-dose radiation therapy and
      etoposide followed by peripheral stem cell transplantation in treating patients with acute
      leukemia.
    

Detailed description: 
      OBJECTIVES: I. Evaluate the efficacy of myeloablative total-body irradiation and etoposide
      followed by autologous peripheral blood stem cell (PBSC) transplantation in prolonging the
      disease-free survival of patients with acute leukemia. II. Investigate the ability of growth
      factor-primed PBSC to provide hematopoietic reconstitution following myeloablative therapy.

      OUTLINE: Patients are treated on Regimen A, then Regimen B. Regimen A: Stem Cell
      Mobilization. Granulocyte Colony Stimulating Factor (Amgen), G-CSF, NSC-614629. Regimen B:
      Radiotherapy and Myeloablative Chemotherapy with Hematopoietic Rescue. Total-Body
      Irradiation, TBI (high-energy electrons used for lung boost); and Etoposide, VP-16,
      NSC-141540; with Peripheral Blood Stem Cells, PBSC; G-CSF.

      PROJECTED ACCRUAL: Up to 45 patients will be studied over approximately 3 years.
    

Intervention name(s): filgrastim; etoposide; peripheral blood stem cell transplantation; low-LET electron therapy; low-LET photon therapy
Intervention type(s): Biological; Drug; Procedure; Radiation; Radiation
Intervention MeSH terms: Etoposide
Primary purpose: Treatment
Keywords: adult acute myeloid leukemia in remission; adult acute lymphoblastic leukemia in remission; adult acute erythroid leukemia (M6); adult acute myeloblastic leukemia without maturation (M1); adult acute myeloblastic leukemia with maturation (M2); adult acute promyelocytic leukemia (M3); adult acute myelomonocytic leukemia (M4); adult acute monoblastic leukemia (M5a); adult acute megakaryoblastic leukemia (M7); adult acute monocytic leukemia (M5b); bone marrow ablation; adult acute minimally differentiated myeloid leukemia (M0)
Clinical Trial to Evaluate of Efficacy and Safety of Ginsengberry Concentrate on Fatigue.
Condition: Fatigue
MeSH terms: Fatigue
Study type: Interventional

Summary: 
      The investigators conduct a 8-week, randomized, double-blind, placebo-controlled study to
      evaluate the efficacy and safety of ginsengberry concentrate on fatigue.
    

Detailed description: 
      Previous studies have indicated that ginseng root may have the ability to improve fatigue.
      Therefore, the investigators conduct a randomized, double-blind, placebo-controlled study to
      investigate the effects of ginsengberry concentrate on Improvement of Fatigue in adults; the
      safety of the compound are also evaluate. The Investigators examine FSS(fatigue severity
      scale), CIS, lactate, Ammonia, Exercise test at baseline, as well as after 4 and 8 weeks of
      intervention. 88 adults were administered either ginsengberry concentrate or a placebo each
      day for 8 weeks.
    

Intervention name(s): Ginsengberry concentrate; Placebo
Intervention type(s): Dietary Supplement; Dietary Supplement
Intervention description(s): Ginsengberry concentrate; Placebo
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Vinorelbine and Gemcitabine Versus Capecitabine in Pretreated Metastatic Breast Cancer
Condition: Breast Cancer
MeSH terms: Breast Neoplasms
Study type: Interventional
Phase: Phase 3

Summary: 
      The combination of vinorelbine and gemcitabine seems to be an important part of the
      chemotherapy regimens used in metastatic breast cancer patients following treatment failure
      with the combination of a taxane and an anthracycline. Capecitabine rescue monotherapy in
      metastatic breast cancer patients following treatment failure with the combination of a
      taxane and an anthracycline, also seems to be an important part of the chemotherapy regimens
      used in metastatic breast cancer patients. Whether the combination of vinorelbine and
      gemcitabine or capecitabine administration is preferable is not yet known, especially in
      patients with metastatic disease.
    

Detailed description: 
      This trial will compare the efficacy of combination treatment with Vinorelbine and
      Gemcitabine versus Capecitabine monotherapy in metastatic breast cancer patients following
      treatment failure with the combination of a taxane and an anthracycline.
    

Intervention name(s): Gemcitabine; Capecitabine; Vinorelbine
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Gemcitabine at the dose of 1000 mg/m2 intravenously (IV) every 2 weeks for 12 consecutive cycles; Capecitabine at the dose of 1250 mg/m2 twice daily orally (os), from day 1 to day 14, every 3 weeks for 6 consecutive cycles; Vinorelbine at the dose of 25 mg/m2 IV on day 1 every 2 weeks for 12 consecutive cycles
Other intervention name(s): Gemzar; Xeloda; Navelbine
Intervention MeSH terms: Gemcitabine; Capecitabine; Vinorelbine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Breast cancer; Chemotherapy; Vinorelbine; Gemcitabine; Capecitabine
Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Puberty, Delayed
Study type: Observational

Summary: 
      "Observational study of clinical outcomes for testosterone treatment of pubertal delay in
      Duchenne Muscular Dystrophy" is a single centre observational study that aims to follow the
      progress of 20 adolescents with Duchenne Muscular Dystrophy (DMD) and delayed puberty who are
      treated by the Newcastle muscle team, as they are treated with testosterone to induce
      puberty. The participants will all be treated with the standard stepwise regimen of
      testosterone injections every 4 weeks and data will be collected to help determine the
      effectiveness and tolerability of the current treatment regimen. The investigators will use
      the data to explore the effect of testosterone on pubertal development, growth, muscle
      strength and function, bone mineral density and body composition and characterise any side
      effects. Semi-structured interviews will also be carried out to learn the boys' views on the
      tolerability of the regimen. The study will last up to a maximum of 27 months in total for
      each participant, but may be less if they are happy with pubertal development before this
      time. It is important to do this study because from the investigator's limited experience in
      this group, testosterone treatment seems to be well liked and tolerated but the best
      treatment regimen to use remains unknown and there is no current consensus. It is not
      currently part of the standard of care in DMD but it would be important to include it if this
      study can show that it is an effective treatment for pubertal delay.
    


Intervention name(s): Sustanon (testosterone)
Intervention type(s): Drug
Intervention description(s): Standard incremental regimen of Sustanon
Intervention MeSH terms: Testosterone
Observational model: Case-Only
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        This is a single-centre, prospective, observational, study on clinical outcomes of
        testosterone replacement therapy in adolescents with DMD and delayed puberty. Being an
        observational study of routine care, there will be no specified end point. We will aim to
        recruit any adolescent with DMD and delayed puberty who is reviewed by the muscle team at
        the John Walton Muscular Dystrophy Research Centre from the study approval date.
      
Biospecimen description: 
      Samples to be collected as part of Biobank
    
Keywords: testosterone; pubertal delay
Cannabis Use and Epigenetic Alterations in Human Male Sperm Cells
Condition: Cannabis Use
MeSH terms: Marijuana Abuse
Study type: Observational

Summary: 
      This is a follow-up project to a proof-of-concept study to determine if Cannabis use in male
      humans is associated with changes in deoxyribonucleic acid (DNA) methylation profile of
      sperm. This proposal will examine the epigenome of sperm from men actively using Cannabis
      before and after a period of Cannabis abstinence. The results will be compared to control
      non-users.
    

Detailed description: 
      This project involves recruitment of 72 healthy males between the ages of 18 and 40 years.
      Forty eight of the participants will be regular cannabis users with a self-reported frequency
      of at least once weekly over the past 6 months, and a control group of twenty four cannabis
      non-users. Cannabis users will be asked to quit using cannabis for 11 weeks and will be
      required to attend 25 visits during the 11 week abstinence/contingency management period.
      Non-users will be asked to return to the clinic once a week for 11 weeks. Abstinence will be
      determined by self-report, qualitative urine rapid screening test at the clinic and
      quantitatively via Enzyme Immunoassay (EIA) analysis of THC (cannabinoids) and, by Liquid
      Chromatography Tandem Mass Spectrometry (LCMSMS) analysis of 11-nor-9-carboxy-∆
      9-tetrahydrocannabinol (THCCOOH) performed by an analytical lab contracted by the study team.
      Semen sample for both groups will be collected prior to the start of abstinence and after 11
      weeks. Semen analysis and DNA methylation of sperm cells will be compared.
    

Intervention name(s): Cannabis use abstinence for 11 weeks
Intervention type(s): Other
Intervention description(s): Cannabis user group will be asked to abstain from any form of Cannabis use for 11 weeks. During their contingency management phase/abstinence phase, they will be required to come to the clinic for 25 visits over an 11 week period of abstinence.
Observational model: Other
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        72 healthy males between the ages of 18 and 40 years. Forty eight - regular Cannabis users
        with a self-reported frequency of at least once weekly over the past 6 months, Twenty four
        Cannabis non-users - control group
      
Keywords: Cannabis Abstinence Sperm Cells
Ascending Doses of Autologous FDP vs FFP
Condition: Freeze Dried Plasma in Healthy Volunteers
Study type: Interventional
Phase: Phase 1

Summary: 
      Assess the safety of single infusions with RePlas FDP product at increasing fixed doses
    

Detailed description: 
      This is a single-site, partial double-blind study in healthy volunteers designed to assess
      the safety of infusing ascending doses of reconstituted autologous freeze dried plasma (FDP)
      in 3 fixed-dose cohorts. Beginning with Cohort 1, subjects will receive a single infusion of
      1 unit (approximately 270 mL) of either FDP manufactured from fresh frozen plasma (FFP)
      derived from autologous whole blood (WB) collection(s) that use citrate phosphate dextrose
      (CPD) as the anticoagulant (FDP-CPD) or FDP manufactured from FFP units from autologous
      plasmapheresis where acid citrate dextrose (ACD) is used as the anticoagulant (FDP-ACD).
      Recruitment of subjects for Cohort 2 follows the completion of infusions in Cohort 1.
      Subjects in Cohort 2 will receive a single infusion of 2 units (approximately 540 mL) of
      either FDP-CPD or FDP-ACD before recruitment for Cohort 3 is initiated. Subjects enrolled in
      Cohort 3 will receive the highest study dose, a plasma infusion dose of 3 units
      (approximately 810 mL) of FDP-ACD at one infusion visit and the same dose of autologous FFP
      at another infusion visit. Cohort 3 subjects will only be infused with FDP and FFP products
      sourced from autologous plasmapheresis. Randomization of Cohort 3 subjects to a treatment
      sequence determines whether they will be infused with 3 units of FDP or 3 units of FFP at
      their first infusion visit followed by infusion with the alternate product at the second
      infusion visit. A 2-week interval will be maintained between infusion visits in Cohort 3.
      Crossover of FDP and FFP enables comparison of infusion safety and select coagulation factor
      recoveries within the same subjects between FDP and FFP at this higher dose.
    

Intervention name(s): Autologous Freeze Dried Plasma (FDP); Fresh Frozen Plasma (FFP)
Intervention type(s): Biological; Biological
Intervention description(s): Safety of Ascending Doses of Autologous Freeze Dried Plasma (FDP) in Healthy Volunteers; Controlled FFP in cohort 3 only
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Open-label, Flexible-dose Study of Vortioxetine in Patients With Depression in India
Condition: Major Depressive Disorder
MeSH terms: Depressive Disorder; Depression; Depressive Disorder, Major
Study type: Interventional
Phase: Phase 4

Summary: 
      The purpose of this study is to evaluate the safety and tolerability of flexible doses of
      vortioxetine (5 - 20 mg/day) over a period of 12 weeks in patients with depression in India
    


Intervention name(s): Vortioxetine
Intervention type(s): Drug
Intervention description(s): 5 - 20 mg/day flexible-dose vortioxetine, tablets, orally
Intervention MeSH terms: Vortioxetine
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Dynamic Effects of Online HDF on Cardiac Function and Myocardial Perfusion Utilising Cardiac MRI (CAMRID)
Condition: Other Postprocedural Cardiac Functional Disturbances; Myocardial Perfusion
Study type: Interventional

Summary: 
      The purpose of this study is to characterise, in detail, the cardiac structure, function and
      perfusion of those on haemodialysis and haemodiafiltration, comparing the two modalities to
      ascertain if haemodiafiltration provides relative myocardial protection compared to
      conventional haemodialysis.
    

Detailed description: 
      Trial Configuration: Non-blinded crossover pilot study Setting: Secondary care Sample size
      estimate: This is a pilot study therefore power calculations are not appropriate. A group
      size of 12 participants has been selected for feasibility.

      Number of participants: 12 patients to complete study Description of interventions

        -  6 sessions of conventional haemodialysis each lasting 4 hours

        -  6 sessions of online haemodiafiltration each lasting 4 hours

        -  2 sessions of Cardiac MRI each lasting 6 hours in total to take place around 6th session
           of dialysis Randomisation and blinding: This is an open study. Statistical methods: All
           continuous variables will be tested for normality. The primary and continuous secondary
           endpoints will be compared between the two modalities using a paired t test or Wilcoxon
           signed rank test as appropriate. The association between categorical variables will be
           tested using the Chi squared or Fischer's Exact test. An alpha error at 0.05 will be
           judged as significant.
    

Intervention name(s): online Hemodiafiltration; Hemodialysis
Intervention type(s): Other; Other
Intervention description(s): Effects of high efficiency online hemodiafiltration on cardiac structure, function and myocardial perfusion were compared with standard haemodialysis.; Effects of high efficiency online hemodiafiltration on cardiac structure, function and myocardial perfusion were compared with standard haemodialysis.
Other intervention name(s): Hemodiafiltration; Haemodialysis
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Primary purpose: Health Services Research
A Study to Evaluate the Effect of Tacrolimus and Corticosteroid Combination Therapy in Patients With Minimal Change Nephrotic Syndrome
Condition: MCNS; Minimal Change Nephrotic Syndrome (MCNS)
MeSH terms: Nephrotic Syndrome; Nephrosis; Nephrosis, Lipoid; Syndrome
Study type: Interventional
Phase: Phase 4

Summary: 
      To compare the therapeutic effect of tacrolimus in combination with low-dose corticosteroid
      with high-dose corticosteroid alone in patients with minimal-change nephrotic syndrome.
    


Intervention name(s): Tacrolimus; Prednisolone
Intervention type(s): Drug; Drug
Intervention description(s): Oral; Oral
Other intervention name(s): FK506; Prograf Capsule
Intervention MeSH terms: Prednisolone; Tacrolimus
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Glomerulonephritis; Tacrolimus; Nephrotic syndrome