bioihbjkbjnl/mcl2 huggingface dataset:
Natural History Study of CEP290-Related Retinal Degeneration
Condition: Blindness; Leber Congenital Amaurosis 10; Vision Disorders; Eye Diseases; Eye Diseases, Hereditary; Eye Disorders Congenital; Retinal Disease; Retinal Degeneration
MeSH terms: Blindness; Vision Disorders; Eye Diseases; Retinal Diseases; Retinal Degeneration; Leber Congenital Amaurosis; Eye Diseases, Hereditary; Eye Abnormalities; Genetic Diseases, Inborn
Study type: Observational

Summary: 
      A prospective natural history study with systematic assessments and uniform follow-up to
      provide a high-quality dataset for assisting in the design of future clinical treatment
      trials involving patients with CEP290-related retinal degeneration caused by the common
      intron 26 mutation.
    

Detailed description: 
      The purpose of the study is to describe the natural history of CEP290-related retinal
      degeneration caused by a compound heterozygous or homozygous intron 26 c.2991+1655A>G
      mutation and to better understand the best assessments for evaluation of patients with this
      condition in a future interventional trial. Patients meeting the entry criteria will be
      enrolled in the study. Visits will occur at Screening, Baseline, and Months 3, 6, and 12, for
      a total duration of 1 year.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        The indication for this study is CEP290-related retinal degeneration caused by a compound
        heterozygous or homozygous intron 26 c.2991+1655A>G mutation.
      
Biospecimen description: 
      Blood sampling for genetic testing of the CEP290 gene, including coding and intron 26
      sequences, as part of a 280-gene retinal dystrophy gene panel.
    
Keywords: CEP290; LCA10; Retinal degenerative diseases (RDD); Leber congenital amaurosis (LCA); Congenital Retinal Blindness; p.Cys998X; c.2991+1655A>G; Eye Diseases Signs and Symptoms; Genetic Diseases, Inborn; Congenital Abnormalities; Eye Abnormalities
Low-dose Decitabine for the Treatment of Decreased Donor Chimerism After Allogeneic Stem Cell Transplantation
Condition: Response Rate
Study type: Interventional
Phase: Phase 2

Summary: 
      Decreasing donor chimerism is considered as an early sign of graft failure or relapse in
      patients undergoing allogeneic stem cell transplantation. The treatment option included
      tapering or stop of immunosuppression and or donor lymphocyte infusion (DLI) which may
      restore a full donor chimerism but subsequent graft versus host disease (GVHD) is the major
      complications. In this single arm prospective study, the investigator evaluate the effect and
      safety of low-dose decitabine alone or with DLI in patients with decreased donor chimerism
      after allo-HSCT.
    

Detailed description: 
      Decreasing donor chimerism is considered as an early sign of graft failure or relapse in
      patients undergoing allogeneic stem cell transplantation. The treatment option included
      tapering or stop of immunosuppression and or donor lymphocyte infusion (DLI) which may
      restore a full donor chimerism but subsequent GVHD is the major complications. In this single
      arm prospective study, the investigator plan to evaluate the effect and safety of low-dose
      decitabine treatment alone in patients with decreased donor chimerism after allo-HSCT. The
      investigators expect an overall response rate of 80% without serious toxicity such as grade
      III-IV aGVHD, ext cGVHD and lethal infection event associated with low-dose decitabine
      (LD-DAC) treatment. In case of donor chimerism decreasing, 5-day low-dose decitabine (5mg/m2)
      will given every 6 to 8 weeks until full donor chimerism is achieved (>98%). Fast withdraw of
      immuno-suppression or stop of immunosupression is not carried out in the study.
    

Intervention name(s): Decitabine
Intervention type(s): Drug
Intervention description(s): low-dose decitabine: 5mg/m2 daily for 5 days
Other intervention name(s): LD-DAC
Intervention MeSH terms: Decitabine
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: donor chimerism,; decitabine,; allo-HSCT
Evaluation of Effectiveness of Combined Intra-articular and Intra-osseus Injection VS a Single Intra-articular Injection of Bone Marrow Concentrate
Condition: Osteoarthritis, Knee
MeSH terms: Osteoarthritis; Osteoarthritis, Knee
Study type: Interventional
Phase: Phase 3

Summary: 
      Osteoarthritis (OA) is one of the most common diseases resulting in large burdens on society.

      Among the emerging treatments, Bone Marrow Concentrate (BMC) intra-articular injections are a
      promising regenerative approach. However, they offer only a temporary benefit since they
      target synovial and chondral tissues but fail to address the osteochondral interface, which
      plays a key role in the onset and progression of joint degeneration.

      This project will investigate the efficacy of combined BMC injections, targeting both
      intra-articular tissues and subchondral bone, to treat OA in a Randomized Controlled Trial
      (RCT).
    

Detailed description: 
      The main aim is to evaluate in a RCT the potential of a new combined BMC treatment for OA,
      through the use of scores for the clinical outcome, the study of imaging and biological
      samples for tissue regeneration and systemic effects, and the identification of BMC and
      patient characteristics predictive of better results.

      All patients will undergo an arthroscopic procedure to debride degenerated tissues (this can
      offer a temporary benefit but not long lasting results) before BMC intra-articular injection.
      Half patients will receive also the injection of BMC at the subchondral level (bone samples
      obtained to allow BMC placement will be sent to the Lab).

      The same minimally invasive incisions will be used for all patients, ensuring patient
      blinding. Evaluations, performed by medical staff not involved in the treatment to ensure
      double blinding, will document subjective clinical improvement, functional measurements,
      biomarker study, and imaging with the use of modern 3TMRI and qCT technology. BMC
      characterization and patient features will be analysed to identify factors predictive of a
      better outcome.
    

Intervention name(s): Bone Marrow Concentrate
Intervention type(s): Biological
Intervention description(s): Bone Marrow Concentrate injection
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Bone Marrow Comcentrate; intra-articular; intra-osseus
Comparison of Exsufflation Versus Drainage in Primary Spontaneous Pneumothorax
Condition: Pneumothorax
MeSH terms: Pneumothorax
Study type: Interventional

Summary: 
      Management of primary spontaneous pneumothorax (PSP) remains unclear. Primary therapeutic
      goals for PSP include removal of air from the pleural space an prevention of recurrences. The
      absence of generally accepted and methodologically sound recommendations may account for the
      extensive variation in practice for air evacuation techniques. Air evacuation may be achieved
      by simple aspiration (exsufflation) or conventional chest tube drainage. Chest tube
      thoracotomy remains the most popular technique.Aspiration is a more simple technique, that
      allows possibility of ambulatory management. The purpose of the present study is to compare
      simple aspiration performed with a specific thoracentesis device, versus conventional chest
      tube drainage. Comparison will be performed on immediate efficacity of resolution of the
      pneumothorax.The hypothesis is that simple aspiration performed with a specific device is not
      inferior to chest tube drainage for management of a first episode of large size primary
      spontaneous pneumothorax. A second goal is to measure the efficacy at one week, and the
      recidive at one year. The trial is randomized, controlled and will include 200 patients for
      each group. The patients will be enrolled in 29 hospitals in France. Emergency department,
      thoracic surgery and pulmonary departments of each hospital were primarily contacted for
      agreement to participate to the study.
    


Intervention name(s): exsufflation; thoracic tube drainage
Intervention type(s): Procedure; Procedure
Intervention description(s): exsufflation will be performed with a specific thoracentesis system after introducing the device into chest pneumothorax, aspiration will be performed during 30 minutes; thoracic tube drainage will be performed with a tube Ch 16 or ch 20
Other intervention name(s): Turkel Kit; Monod Trocar; Chest Tube
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: spontaneous; primary; first episode
Comparison of Pharmacokinetic Parameters Between Adopters of Electronic Cigarettes and a Historical Sample of Combustible Cigarette Smokers
Condition: Smoking
Study type: Observational

Summary: 
      This study assesses the nicotine pharmacokinetic (PK) parameters in adopters of electronic
      cigarettes following a 12-hour tobacco and nicotine abstinence. The PK results of this study
      will be compared to historical data on smokers obtained in prior studies, as well as on naïve
      and short-term users of electronic cigarettes.
    

Detailed description: 
      This is an observational, open-label study to assess the nicotine pharmacokinetic (PK)
      parameters over a 6-hour period in adopters of electronic cigarettes with respect to an
      in-clinic 10-minute ad libitum period following a 12-hour tobacco and nicotine abstinence. In
      addition to PK parameters, subjective effects of urge for product will be assessed over a
      6-hour period concurrently with the collection of the PK blood samples following a protocol
      defined schedule. The results of this study will be compared to historical PK and urge for
      product data obtained from smokers as well as naïve and short-term users of electronic
      cigarettes. In addition to the PK parameters and urge for product outcomes, plasma will be
      assessed for cotinine concentration prior to the 12-hour abstinence period.
    

Intervention name(s): Electronic Cigarette #1; Electronic Cigarette #2
Intervention type(s): Other; Other
Intervention description(s): VUSE® Digital Vapor Cigarettes (original flavor, 29 mg nicotine); VUSE® Digital Vapor Cigarettes (menthol flavor, 29 mg nicotine)
Other intervention name(s): VUSE® Original; VUSE® Menthol
Intervention MeSH terms: Menthol
Observational model: Cohort
Time perspective: Cross-Sectional
Sampling method: Non-Probability Sample
Study population: 
        Generally healthy males and females 21 to 60 years of age who are exclusive users of
        electronic cigarettes.
      
Biospecimen description: 
      Plasma, urine, buccal cells
    
Keywords: electronic cigarettes; e-cigarette; combustible cigarette; cigarette; menthol; cig-alike; subjective measures; pharmacokinetics; craving
Combination Chemotherapy Followed by Stem Cell Transplant in High-risk Neuroblastoma Patients
Condition: Neuroblastoma
MeSH terms: Neuroblastoma
Study type: Interventional
Phase: Phase 3

Summary: 
      Improvement of event free survival of high-risk neuroblastoma patients by introduction of two
      additional topotecan containing chemotherapy cycles into the multimodal standard treatment
      (induction chemotherapy, myeloablative therapy, radiation, surgery as indicated, and
      consolidation therapy).
    

Detailed description: 
      Experimental intervention (6 weeks + duration of the control intervention):

      2 x N8 cycle (topotecan, cyclophosphamide, and etoposide) followed by standard arm treatment
      (i.e., control intervention)

      Control intervention (total duration 70-76 weeks):

      3 x N5 cycle (cisplatin, etoposide, and vindesine) 3 x N6 cycle (vincristine, dacarbacine,
      ifosfamide, and doxorubicine), myeloablative chemotherapy with autologous stem cell
      transplantation (melphalan, carboplatin, etoposide) 9 x retinoic acid cycles (6 months, 3
      months break, 3 months) supportive care (PCP/fungal prophylaxis, transfusions, antibiotics,
      G-CSF)
    

Intervention name(s): Cycles N8, N5 and N6; Cycles N5 and N6
Intervention type(s): Drug; Drug
Intervention description(s): two chemotherapy cycles N8 followed by standard arm therapy; Standard arm six chemotherapy cycles (3xN5 and 3x N6) followed by myeloablative therapy with stem cell support and isotretinoin
Other intervention name(s): topotecan, cyclophosphamide, etoposide; cisplatin, vindesine, ifosfamide, dacarbacine, doxorubicine
Intervention MeSH terms: Cyclophosphamide; Ifosfamide; Cisplatin; Doxorubicin; Etoposide; Topotecan; Vindesine
Allocation: Randomized
Intervention model: Sequential Assignment
Intervention model description: Randomized, multi-center, non-blinded, prospective, controlled trial
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: neuroblastoma; chemotherapy; autologous stem cell transplantation; radiotherapy; mIBG; tumor resection
A Phase I/II Study of Chondrogen Delivered by Intra-Articular Injection Following Meniscectomy
Condition: Recovery Following Partial Medial Meniscectomy
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this study is to determine whether Chondrogen is a safe and effective
      post-operative treatment of the knee following menisectomy (the surgical removal of all or
      part of a torn meniscus).
    

Detailed description: 
      Chondrogen is a preparation of adult mesenchymal stem cells (MSCs) in a solution containing
      hyaluronic acid. Preclinical studies have shown that injection of Chondrogen aids in the
      repair of meniscal tissue following meniscectomy. In Chondrogen treated subjects, surgically
      removed meniscal tissue was regenerated, cartilage surface was protected, and joint damage
      was decreased in comparison to control subjects. These benefits persisted at least one year.

      Three groups of recent meniscectomy patients will be followed in this study, including
      patients that will receive placebo and patients that will be treated with one of two possible
      doses of Chondrogen.
    

Intervention name(s): Mesenchymal Stem Cells; Hyaluronan
Intervention type(s): Drug; Drug
Intervention MeSH terms: Hyaluronic Acid
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: meniscectomy; Chondrogen; Mesenchymal Stem Cells; Osiris
Assessing the Safety and Ability of SG1002 to Overcome Deficits in Hydrogen Sulfide in Heart Failure Patients
Condition: Heart Failure
MeSH terms: Heart Failure
Study type: Interventional

Summary: 
      Patients with heart failure are reported to have lower levels of hydrogen sulfide in their
      blood, even though sulfur is available naturally in the diet. Hydrogen sulfide is a molecule
      that has been shown to have a number of beneficial effects and thus the low levels may
      contribute to the disease. This trial is testing whether a medical food product of synthetic
      sulfur molecules, SG1002, can overcome this deficit in blood levels of hydrogen sulfide.
    

Detailed description: 
      Subjects with heart failure have been shown to have a deficit in circulating hydrogen sulfide
      levels, which, since sulfur is not readily bioavailable and is declining in food substances,
      cannot be treated adequately by diet alone. This deficit in circulating hydrogen sulfide is
      thought to lead to increases in oxidative stress and with this, associated problems that can
      contribute to heart failure. This is a study to evaluate the safety and ability of multiple
      doses of oral SG1002 in subjects with heart failure to reverse the deficits in circulating
      hydrogen sulfide. The primary objective is to assess the safety and the ability of multiple
      doses of twice daily administration of SG1002 compared with placebo to increase circulating
      levels of hydrogen sulfide. Initially, a dose escalation study will be carried out for 21
      days in 4 normal subjects, randomized 3:1 active:placebo. Subjects will receive a 200 mg dose
      BID for 7 days and in no adverse events, escalate to 400 mg for 7 days then 800 mg for 7
      days. Safety parameters will be assessed for each dose, along with pharmacokinetic analysis
      and markers of oxidative stress and heart failure. Following completion of the normal healthy
      subjects, 10 heart failure subjects will be randomized 4:1 active:placebo and tested as
      described above.
    

Intervention name(s): SG1002; Placebo
Intervention type(s): Other; Other
Intervention description(s): 200 mg capsules will be administered BID for 7 days, then doubled to 2 capsules BID for 7 days and doubled again to 4 capsules BID for the final 7 days.; 200 mg capsules containing placebo will be administered BID for 7 days, then doubled to 2 capsules BID for 7 days and doubled again to 4 capsules BID for the final 7 days.
Other intervention name(s): alpha sulfur/sodium sulfate
Intervention MeSH terms: Sodium sulfate
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Other
Keywords: Heart Failure; Congestive Heart Failure; Oxidative Stress; Hydrogen Sulfide
An Open-Label Safety Study Of DIC075V (Intravenous Diclofenac Sodium) In Patients With Acute Post-Operative Pain
Condition: Pain, Postoperative
MeSH terms: Pain, Postoperative
Study type: Interventional
Phase: Phase 3

Summary: 
      This is an open-label, multiple-dose, safety study of DIC075V in patients with acute
      post-operative pain following abdominal or orthopedic surgery.
    

Detailed description: 
      This is an open-label, multiple-dose, multiple-day, single-arm safety study of repeat-doses
      of DIC075V in patients with acute post-operative pain following abdominal (i.e.,
      non-laparoscopic abdominal surgeries) or orthopedic (e.g., hip or knee joint replacement)
      surgery. Eligible patients will receive DIC075V IV bolus q6 hours. Safety assessments will be
      collected at baseline (immediately prior to starting DIC075V therapy) and at study discharge
      or early termination.
    

Intervention name(s): DIC075V (intravenous diclofenac sodium)
Intervention type(s): Drug
Intervention description(s): multiple doses up to 5 days
Intervention MeSH terms: Diclofenac
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: safety; diclofenac; pain, postoperative
Clinical Outcomes of Zirconia-reinforced Lithium Silicate Partial Coverage Crowns Compared to Lithium Disilicate Partial Coverage Crowns
Condition: Partial Coverage Restoration
Study type: Interventional

Summary: 
      The purpose of this randomized controlled clinical trial is to evaluate the clinical outcomes
      of Partial Coverage Restorations fabricated with zirconia-reinforced lithium silicate ceramic
      system compared to partial coverage restorations fabricated with lithium disilicate ceramic
      system.
    

Detailed description: 
      This study is constructed to assess in patients with carious vital posterior teeth whether
      the use of Vita Suprinity® (VITA Zahnfabrik H. Rauter GmbH & Co.KG- Germany) Zirconia
      reinforced lithium silicate partial coverage restorations will result in higher survival
      rates and better patient related outcome scores compared to IPS-e.max® CAD (Ivoclar Vivadent
      AG, Schaan - Liechtenstein) lithium disilicate partial coverage restorations.
    

Intervention name(s): Vita Suprinity; IPS e.max
Intervention type(s): Other; Other
Intervention description(s): zirconia reinforced lithium silicate partial coverage restorations; lithium disilicate partial coverage restorations
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: split-mouth design
Masking: Double (Participant, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Partial coverage restoration; Crown; Posterior teeth; All ceramic; Zirconia reinforced lithium silicate; Lithium disilicate; CAD/CAM
Physiological Effects of Expiration With the Glottis Open in Lateral Posture (ELTGOL) and Flutter Valve in Patients With Bronchiectasis
Condition: Bronchiectasis
MeSH terms: Bronchiectasis
Study type: Interventional

Summary: 
      This study aimed to evaluate the acute physiological effects of expiration with the glottis
      open in lateral posture (ELTGOL) and Flutter valve in dynamic and static lung volumes in
      patients with bronchiectasis and, secondarily, to study the effect of these techniques in
      sputum production.
    


Intervention name(s): Flutter valve; expiration with the glottis open in lateral posture (ELTGOL); Control
Intervention type(s): Other; Other; Other
Intervention description(s): Flutter valve is a physiotherapy device commonly used for the treatment of pulmonary hypersecretive conditions. The patients is taught to exhale through the device, producing positive expiratory pressure and flow oscillations. In this study the intervention took 15 minutes. During this period the patients were free to cough.; This technique is a maneuver of slow expiration with the glottis open, with the subject in lateral posture. The patients performed three sets of 10 repetitions in left and right lateral decubitus.; Patients were instructed to stay comfortably seated during 15 minutes. After this period they were stimulated to cough during a five minutes period.
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Single (Participant)
Primary purpose: Treatment
Effects of Dexmedetomidine on Quality of Recovery and Analgesia After Video-assisted Thoracoscopic Surgery
Condition: Lung Cancer; Video-assisted Thoracoscopic Surgery
Study type: Interventional

Summary: 
      Dexmedetomidine is a selective α2 adrenergic agonist that can be considered analgesics,
      anxiolytic,and anti-stress effect . This randomized, double-blind placebo-controlled trial of
      intraoperative dexmedetomidine for improvement of quality of recovery and analgesia from
      surgery. Patients scheduled to undergo video-assisted thoracoscopic surgery (VATS) will be
      enrolled. Patients will be recruited before the date of their surgery and will complete the
      Quality of Recovery-40 (QoR-40) questionnaire before their surgery and at 24 and 48 hours
      post op. They will also complete clinically significant pains score and oxygenation from the
      postanesthetic recovery unit (PACU).
    


Intervention name(s): Dexmedetomidine; Normal saline
Intervention type(s): Drug; Drug
Intervention description(s): Formula for administrated were randomized by computer-generated randomization. The patients and anesthesiologist, who administered and conducted all this trial, were blinded to the medication allocation. An anesthesiologist, who responsible for preparation of medication was the only person, recognized of the randomization code during trial. After one-lung ventilation, dexmedetomidine group would start the loading of dexmedetomidine (1μg/kg) during 20 minutes.; Saline placebo infusion will be adjusted the same fashion as the active comparator drug.
Other intervention name(s): Precedex; Saline 0.9%
Intervention MeSH terms: Dexmedetomidine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Keywords: dexmedetomidine; quality of recovery