cyrilzakka/clinical-trials huggingface dataset:
APL93: Timing of CxT and Role of Maintenance

Objectives of the trial were to assess the optimal timing of chemotherapy with or after ATRA and the role of maintenance therapy.

Induction treatment was stratified by age and initial WBC count. Patients ≤65 years of age with a WBC count less than 5,000/µL were randomized to receive the reference ATRA treatment of our previous trial (APL91 trial) {Fenaux, 1993 #2088}, ie, 45 mg/m2/d ATRA followed by CT (ATRA→CT group) or ATRA plus CT (ATRA+CT). In the ATRA→CT group, patients received 45 mg/m2/d ATRA orally until CR, with a maximum of 90 days. After CR achievement, they received a course of 60 mg/m2/d daunorubicin (DNR) for 3 days and 200 mg/m2/d AraC for 7 days (course I). However, course I was added to ATRA if the WBC count was increased to greater than 6,000/µL, 10,000/µL, or 15,000/µL by day 5, 10, and 15 of ATRA treatment, respectively, be-cause, from our experience, patients were at risk of ATRA syndrome above those thresholds{de Botton, 2003 #1127; De Botton, 1998 #1604}. Patients randomized to the ATRA+CT group received the same combination of ATRA and CT, with course I of CT starting on day 3 of ATRA treatment.

Patients with a WBC count greater than 5,000/µL at presentation (irrespective of their age) and patients 66 to 75 years of age with a WBC count ≤ 5,000/µL were not ran-domized but received ATRA plus CT course I from day 1 (high WBC group) and the same schedule as in the ATRA→CT group (elderly group), respectively.

Treatment of coagulopathy during the induction phase was based on platelet support to maintain the platelet count at a level greater than 50,000 /µL until the disappea-rance of coagulopathy. The use of heparin, tranexamic acid, fresh frozen plasma, and fibrinogen transfusions was optional.

CR patients received 2 CT consolidation courses, including course II (identical to course I) and course III, consisting of 45 mg/m2/d DNR for 3 days and 1 g/m2 AraC every 12 hours for 4 days. The elderly group only received course II.

Three to 4 weeks after hematological recovery from this consolidation CT, patients who were still in CR were randomized both to receive or not receive intermittent ATRA (45 mg/m2/d, 15 days every 3 months) and to receive or not receive continuous CT with 6 mercaptopurine (90 mg/m2/d, orally) and methotrexate (15 mg/m2/wk, oral-ly), according to a 2-by-2 factorial design stratified on the initial induction treatment group. Maintenance treatment was scheduled for 2 years. Randomizations for induc-tion and maintenance, stratified on center, were performed through a centralized tele-phone assignment procedure.

Eligibility Criteria:
Inclusion Criteria:

1. Diagnosis of APL, based on morphology criteria
2. Age 75 years or less; and
3. Written informed consent. Diagnosis had to be subsequently confirmed by presence of t(15;17) or PML-RAR gene rearrangement. In the absence of t(15;17) and if no analysis of the rearrangement could be made, review of initial marrow slides by an independent morphologist was mandatory.
Danon Disease Natural History Study

The goal of this international observational study is to learn about the natural history of Danon disease in male patients \>8 years of age and female patients \<30 years of age. The key objectives include assessing change over time in cardiac structure (left ventricular mass and wall thickness), cardiac biomarkers, symptoms, and quality of life and the incidence of clinical events such as HF hospitalization, cardiac transplantation, and death.

This is an international observational study with both retrospective and prospective data collection. The study is designed to describe the natural history of Danon disease which is a rare x-linked genetic disorder and one of the most severe and penetrant forms of inherited cardiomyopathy. This study will collect data about the clinical course of Danon disease including signs and symptoms, key clinical events, and the impact of the disease on quality of life managed with current standard of care. A hybrid (retrospective and prospective data collection) approach is being taken with the aim of achieving robust and longitudinal data generation.

Subset of patients will be used as an External Control Arm for comparison to RP-A501 Trial participants.

Eligibility Criteria:
Inclusion Criteria:

Prospective Cohort:

1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
2. Patient or parent/legal guardian are capable and willing to provide signed informed consent
3. Age ≥ 8 years at enrollment

   Female Prospective Cohort:
4. Evidence of left ventricular hypertrophy with preserved systolic function in the 12 months prior to or at enrollment as defined by each of the following:

   Retrospective (only) Cohort:
5. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
6. Patient or parent/legal guardian are capable and willing to provide signed informed consent, as required by local regulations
7. Age ≥ 8 years at enrollment
8. Prior cardiac transplantation or prior mechanical circulatory support
9. At least 30 days of retrospective medical records available prior to cardiac transplantation or mechanical circulatory support

   Female Retrospective (only) Cohort:
10. Prior evidence of left ventricular hypertrophy.

Exclusion Criteria:

All Cohorts:

1. Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
2. Previous treatment with a gene therapy

   Prospective Cohort:
3. Prior mechanical circulatory support at time of enrollment to this study
4. Prior cardiac transplantation at time of enrollment to this study

   Female patients:
5. Age \>30 years at enrollment
Tapering From Long-term Opioid Therapy in Chronic Pain Population. Randomized Controlled Trial With 12 Months Follow up

This randomized controlled study compares tapering of long-term opioid therapy in a population with chronic non-cancer pain with control group constituted of waiting list. Half of participants receives intervention at baseline and the other half are controls but receives intervention after 4 months. Ethical approval to follow up excluded participants denying tapering at baseline.

Chronic non-cancer pain is a major problem in society and a common cause to seek health care. The suffering is complex as it, besides pain, often includes psychological symptoms and may decrease the ability to participate in work life. Pharmacological treatments have limited possibilities to ease suffering. Opioids used in this population have evidence to ease pain and to some extent increase physical ability when used in limited treatment periods and in selected patients. Medium to long-term opioid therapy, extended beyond three months, lack evidence of easing pain or increase physical ability.

The current study is conducted on three specialized pain care units (Skane University Hospital; Lund, Sahlgrenska University Hospital Gothenburg and University Hospital, Linkoeping) and aims at taper long-term opioid therapy with support from physician and nurse and study the effects of tapering.

Method: Randomized controlled study, without concealment. Waiting-list constitutes the control-group. Intervention means tapering with a motivated patient. Follow up at four and twelve months.

Primary end-point: collected opioid prescriptions registered in The National Board of Health and Welfare registry expressed in milligram of morphine equivalent.

Secondary end-point: Data will be retrieved from the Swedish Quality Registry for Pain Rehabilitation. The database contains participants' self-report of variables measuring the impact of rehabilitation on depressive symptoms, anxiety, rating of pain and acceptance to pain. Assessments are made prior to, at the end of and one year after discharge of the rehabilitation program.

As denying tapering or participation is shown to be as common as decision to taper drugs at baseline an extended ethical approval was acquired also retrospective. This approval covers obtaining collected prescribed medication and prescribed opioid replacement therapy (Buprenorphine or Methadone) after one year. The study may describe the excluded population better this way.

Eligibility Criteria:
Inclusion Criteria:

A long-term opioid therapy with daily intake due to chronic pain condition, by prescription. Willing to enter an intervention aiming at taper opioid therapy.

Exclusion Criteria:

No daily intake of opioids. Shorter duration of opioid therapy than 3 months. Repeated intake of non-prescribed opioids or other illegal drugs. Refusal to perform drug-screening Perceived medical risks of waiting with tapering of opioids (e.g: raising opioid doses after acute pancreatitis caused by excessive alcohol intake or kidney disease stage 4-5 in combination with excessive alcohol intake and high doses of opioids).
Does the Diacutaneous Fibrolysis Change Ultrasonographic Measurements in Patients With Carpal Tunnel Syndrome?

The carpal tunnel syndrome (CTS) is a common entrapment neuropathy caused by compression of the median nerve at the wrist.Clinically electroneuphysiological assessment is not accessible to all clinicians. In this way ultrasounds (US) is a more accessible and economical tool and many studies have reported that US has high sensitivity and specificity in the diagnosis of CTS.Diacutaneous Fibrolysis (DF) is a physiotherapeutic technique derived from Cyriax deep friction massage principles.DF technique could assist in improving changes in the connective tissues adjacent to the median nerve, especially the thickness of TCL and this could be reflected in a decreasing of TCL. Moreover, the neurophysiological and mechanical effect described by this technique may improve the nerve compression decreasing the CSA of the median nerve.

The purpose of this study is to quantify changes in the cross-sectional area of the median nerve in the carpal tunnel and the thickness of transversal carpal ligament measured by US and the changes in the intensity of the numbness and the subjective assessment of clinical change after DF treatment in forearm, wrist and hand area compared to placebo.


Eligibility Criteria:
Inclusion Criteria:

- Diagnosed medically with neurophysiological test of carpal tunnel syndrome (low and moderate intensity)

Exclusion Criteria:

* Severe carpal tunnel syndrome
* Previous surgery on the hand
* hormonal factors: diabetes, thyroids pathologies, pregnant
* Cervical disfunctions
* Ulcerations or skins disorders
Immediate Effects of Hamstring Stretching Alone or Combined With Myofascial Release

To assess the immediate effects of hamstring stretching alone or combined with myofascial release of the hamstring muscle on masseter muscle activity, pressure pain threshold, active mouth opening, hamstring flexibility, and forward head flexed posture in recreationally active participants. The young, healthy, recreationally active adults aged 18-25 years were included in the study. The participants were randomly separated into two groups of equal number. Static stretching alone was applied to one group and this was combined with myofascial release techniques (SSMR) for the other group. Masseter muscle activity was assessed using a NORAXON® surface electromyography device. Both pre-and post-intervention, mechanical pressure algometer was used to measure the pressure pain threshold, hamstring flexibility was evaluated using the maximum hip flexion and active knee extension test, the maximum mouth opening (MMO) was measured with the millimetric ruler, and forward flexed posture was evaluated using the tragus to wall distance test.


Eligibility Criteria:
Inclusion Criteria:

* Individuals who had no previous hamstring injury
* Individuals had a Straight Leg Raise test \<80°
* Individuals had a pressure-pain threshold difference between two symmetrical points not exceeding 2 kg/cm2
* Individuals who had no previous hamstring injury had no deviation during mouth opening and closing

Exclusion Criteria:

* acute low back pain or musculoskeletal pain in the lower extremities
* recent spinal surgery
* previous whiplash injury
Sealing of Occlusal Dentin Caries in Primary Molars.

BACKGROUND A simple method for arresting dentin caries with a tight seal of the cavity would exclude the need for injection and excavation, reduce the risk for dental fear and possibly postpone or eliminate the need for further treatment.

AIM The aim is to evaluate success-rate and children's subjective experience of resin-based sealing (RBS) and resin-modified glass ionomer cement (GIC) for treatment of occlusal dentin caries in primary molars.

DESIGN Children aged 2-9 years will be recruited to undergo RBS- or GIC-treatment treatment for occlusal dentin caries in primary molars. A parent will answer the Short Form of Children's Fear Survey (CFSS-DS; parental version). No excavation but etching with 38% phosphoric acid will precede RBS. Topical and local anaesthesia and excavation with high speed bur will precede GIC. Time for treatment will be estimated. The children will rank their experience of each treatment on a 7-grade face scale. The dentist will evaluate the child's experience, cooperation and the overall experience of the treatment on a four-grade scale. The treatment will be evaluated as "without remark" or "defective" after 3, 6, 12, 24 and 36 months. For statistical analysis the Chi-2-test, Wilcoxon-Signed-Rank-test and Paired t-test will be used.

BACKGROUND A simple method for arresting dentin caries with a tight seal of the cavity would exclude the need for injection and excavation, reduce the risk for dental fear and possibly postpone or eliminate the need for further treatment.

AIM The aim is to evaluate success-rate and children's subjective experience of resin-based sealing (RBS) and resin-modified glass ionomer cement (GIC) for treatment of occlusal dentin caries in primary molars.

DESIGN Up to 300 children aged 2-9 years will be recruited to undergo RBS- or GIC-treatment treatment for occlusal dentin caries in primary molars. A parent will answer the Short Form of Children's Fear Survey (CFSS-DS; parental version). No excavation but etching with 38% phosphoric acid will precede RBS. Topical and local anaesthesia and excavation with high speed bur will precede GIC. Time for treatment will be estimated. The children will rank their experience of each treatment on a 7-grade face scale. The dentist will evaluate the child's experience, cooperation and the overall experience of the treatment on a four-grade scale. The treatment will be evaluated as "without remark" or "defective" after 3, 6, 12, 24 and 36 months. For statistical analysis the Chi-2-test, Wilcoxon-Signed-Rank-test and Paired t-test will be used.

Eligibility Criteria:
Inclusion Criteria:

• primary occlusal dentin caries in two primary molars

Exclusion Criteria:

* Language problems or disabilities
* Less than 3 years until expected exfoliation
* Hypomineralization
* Previous filling or dentin caries
* Carious cavity \>1/3 of occlusal surface
* Unable to cooperate
* Appearing caries or filling (on studied tooth) not originating from the studied cavity
* Tooth exfoliating before end of study
* Leaving Public dental service or moving out of the region during follow up period
Absorption, Metabolism, Excretion, and the Determination of Absolute Bioavailability of Niraparib in Subjects With Cancer

This is an open-label study with 2 parts, plus an extension study following completion of Parts 1 or 2, that is being conducted in approximately 12 subjects (6 subjects in Part 1; 6 subjects in Part 2) with cancer to examine the absorption, metabolism, excretion, and absolute bioavailability of niraparib.

Part 1: After an overnight fast of at least 10 hours, subjects will receive a single, oral dose of niraparib (unlabeled active pharmaceutical ingredient) on Day 1. Two hours after the oral dose, subjects will receive a 15-minute IV infusion of niraparib (labeled pharmaceutical ingredient).

Part 2: After an overnight fast of at least 10 hours, subjects will receive a single, oral dose of niraparib, labeled active pharmaceutical ingredient.

Eligibility Criteria:
Inclusion Criteria:

* Subject, male or female, is at least 18 years of age.
* Subject has histologically or cytologically confirmed diagnosis of metastatic or locally advanced solid tumors that have failed to respond to standard therapy, have progressed despite standard therapy, refuse standard therapy, or for which no standard therapy exists, and that may benefit from treatment with a poly (adenosine diphosphate \[ADP\]-ribose) polymerase (PARP) inhibitor. The diagnosis must be confirmed with a previous computed tomography (CT) scan.
* The subject has adequate organ function:
* Subject must have an ECOG performance status of 0 to 2.
* Female subjects of childbearing potential must have a negative serum pregnancy test (beta human chorionic gonadotropin \[hCG\]) within 72 hours prior to receiving the first dose of study drug.

Exclusion Criteria:

* Subject has undergone palliative radiotherapy within 1 week of study drug administration, encompassing \>20% of the bone marrow.
* Subject has persistent \>Grade 2 toxicity from prior cancer therapy.
* Subject has known hypersensitivity to the components of niraparib.
* Subject has had major surgery within 3 weeks of study drug administration or has not recovered from all effects of any major surgery.
* Subject is considered a medical risk due to a serious, uncontrolled medical disorder; nonmalignant systemic disease; or active, uncontrolled infection. Examples include, but are not limited to, uncontrolled ventricular arrhythmia, recent (within 90 days of the Screening Visit) myocardial infarction, uncontrolled major seizure disorder, unstable spinal cord compression, superior vena cava syndrome, or any psychiatric disorder that prohibits obtaining informed consent.
* Subject has participated in a radioactive clinical study and has received an investigational radiolabeled drug within 6 months prior to study drug administration (for subjects participating in Part 2)
Evaluation of Spinal Mobility Reeducation in Patients Treated for a Lumbar Spinal Stenosis After Epidural Infiltration

In actual practice the patients with mild or moderate lumbar spinal stenosis symptoms receive an epidural infiltration and participate in kyphosis reeducation in first intention. Yet the kyphosis reeducation did not show a real profit in the time compared with the natural evolution of the pathology. The study assume that the spinal mobility reeducation will reduce the incidence of pain recurrences compared with the classic kyphosis reeducation.

The standard treatment of lumbar spinal stenosis is the lumbar canal recalibration surgery which presents co-morbidity factors and risks of post-operative complications. The non-invasive methods are a good alternative compared with the surgery : the patients medically treated present few damages and the results of the postponed surgery are equivalent to the immediate surgery. That is why a non-surgical treatment is proposed in first intention to the patients with mild or moderate symptoms : this treatment associates an epidural infiltration and a kyphosis reeducation.

Yet the kyphosis reeducation did not show a real profit in the time compared with the natural evolution of the pathology. The benefits of the infiltration are lost three months after this one in 80 % of the cases. A return to physical activity and a restored spinal mobility would improve the duration of the infiltration effect. The objective is then to compare the efficiency of a spinal mobility reeducation program versus a kyphosis reeducation program in patients with acquired and central lumbar spinal stenosis. It is a prospective, monocentric, randomized, superiority and parallel-group study :

* group C (control group) : kyphosis reeducation + patient education + auto-reeducation at home,
* group M (test group) : spinal mobility reeducation + patient education + auto-reeducation at home.

Eligibility Criteria:
Inclusion Criteria:

* age \> 50 years,
* central, acquired and multi-staged lumbar spinal stenosis,
* pain since at least three months,
* radicular pains higher than the lumbar pains,
* walking distance \< 1000 m,
* oral consent.

Exclusion Criteria:

* intercurrent pathology limiting the walking distance (arteriopathy, cardio-respiratory insufficiency, disabling arthrosis affecting the lower limbs...),
* history of spinal surgery for lumbar spinal stenosis,
* extended lumbar arthrodesis (equal to or greater than 2 levels),
* monosegmental and degenerative spondylolisthesis,
* foraminal stenosis,
* motor deficit of the lower limbs or Cauda equina syndrome ("Cauda equina" syndrome (CES) is a serious neurologic condition in which damage to the cauda equina causes acute loss of function of the lumbar plexus, (nerve roots) of the spinal canal below the termination (conus medullaris) of the spinal cord),
* bleeding disorders or allergies contraindicating the epidural infiltration.
Trial of Caffeine to Alleviate Dyspnea Related to Ticagrelor

Ticagrelor treatment is associated with increased rates of dyspnea, where previous studies have implied a possible role of adenosine. The purpose of this study is to determine if the caffeine-antagonist is effective in reducing dyspnea related to ticagrelor.


Eligibility Criteria:
Inclusion Criteria:

* Age ≥ 18 years
* Recent acute coronary syndrome with ongoing ticagrelor treatment
* Stabilized clinical condition with no plans of additional revascularization
* Dyspnea with onset after start of ticagrelor treatment
* Willingness to abstain from caffeine intake (e.g. coffee, tea, cola-type beverages; other foods, nutritional supplements or medications containing caffeine) for the duration of the study
* Provision of signed informed consent form

Exclusion Criteria:

* Chronic obstructive pulmonary disease, asthma or other known pulmonary disease requiring daily medical therapy
* Obstructive sleep apnea syndrome requiring therapy
* Ongoing signs and symptoms of heart failure\*, or evidence of moderately to severely reduced left ventricular (LV) function
* Renal failure, glomerular filtration rate (GFR) \<30 or on dialysis
* Pregnancy or lactation
* Known allergy to ticagrelor, or caffeine, or known intolerance of caffeine
* Ongoing treatment with any of the following: quinolone antibiotics, fluvoxamine, phenylpropanolamine, carbamazepine, clozapine, lithium, non-steroidal anti-inflammatory drugs (NSAIDs); or any drug containing theophylline or caffeine.
* Any condition that seriously increases the risk of non-compliance or loss of follow-up