cyrilzakka/clinical-trials huggingface dataset:
Ephedrine for the Treatment of Congenital Myasthenia

Previous research has demonstrated possible efficacy of Ephedrine in the treatment of congenital myasthenia caused by end-plate acetylcholinesterase deficiency.

The aim of the current study is to test the hypothesis that Ephedrine may be beneficial to these patients.

To test this hypothesis we will perform a double blind, placebo-controlled, crossover study clinical efficacy and safety study.

Drug naïve patients who agree to participate will be randomized to two groups. Each group will be treated in a blinded manner for 5 weeks with either placebo or Ephedrine HCl in an escalating dose up to 100 mg per day divided in two doses. After five weeks the groups will cross over and continue treatment or placebo for a further five weeks.

Evaluations of strength and fatiguability will be done at baseline, at the end of each five week period and after a further two weeks.

Safety will be assessed weekly by the investigators using interview and physical examination.

Outcome measures will include Barthel index, Quality of life questionnaire, Timed up and go, spirometry, timed elevation of limbs, and force measurements.

All patients will report to the clinic as per study schedule (See Appendix A). Specifically, the 12 clinic visits will include: baseline (1), safety and efficacy assessments(10) and closeout (1).


Eligibility Criteria:
Inclusion Criteria:

* Male or female patients , with congenital myasthenia, belonging to a previously reported kindred diagnosed with COLQ deficiency.

Exclusion Criteria:

* History of allergy to Ephedrine or any inactive component.
* Significant abnormalities in screening Cardiovascular parameters (blood pressure, pulse).
* Surgery within 6 weeks of screening.
* Concurrent use of any other medication except steroids.
* Pregnancy.
* Thyrotoxicosis.
* Co-morbid conditions or other neurological disorders that would confound assessment of clinical parameters.
* Participation in another clinical trial within 30 days of study start.
* Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form.
Establishment of a Brain and Spinal Tumor Tissue Bank

The purpose of this study is to establish a Brain and Spinal Tumor Tissue/Specimen repository to serve as a resource for current and future scientific studies.

Glioblastoma multiforme (GBM) is the most common and lethal primary Brain and Spinal Tumor in adults. It is nearly uniformly fatal, with a median survival of approximately one year, despite modern treatment modalities. Efforts to understand why some patients live longer or shorter than the average may provide insights into the biology of these neoplasms. For years, researchers have tried to identify independent predictors that could help in treatment planning. With the recent interest in molecular biology and molecular genetics, tumor markers are now being examined as potential predictors. A marker that could predict tumor response to therapy, the likelihood of recurrence, and survival would thus be quite useful.

Eligibility Criteria:
Inclusion Criteria:

* Patients must have a suspected or confirmed diagnosis of Brain and Spinal Tumor
* Age greater than 20
* Signed consent

Exclusion Criteria:

* Known HIV infection
* Any other clinical condition which, in the opinion of the principal investigator, would not allow completion of this study
Development of a Nanosecond Pulsed Electric Field System to Treat Skin Cancer

Human Basal Cell Carcinomas (BCCs) can be ablated by treating them with nanosecond pulsed electric fields (nsPEF)

The purpose of this study is to determine if low energy, non-thermal, 100 ns pulses can ablate BCCs. The investigators consider this a non-significant risk study because our PulseCure system only applies 0.4 watts to the skin compared to the 40 watts applied by the currently used electrosurgical unit to remove skin lesions, the Hyfrecator. Since the PulseCure applies 1/100 of the energy and does not even heat the skin significantly, it represents a non-significant risk to the patient. Our study will provide the first data on the response of BCCs to these 100 ns pulses. The investigators will first treat three BCCs on BCNS patients with different pulse numbers to determine the pulse number needed to ablate. Once the investigators have the optimal pulse number, the investigators will use it to treat 20 normal patients with sporadic BCCs on their trunks. This should provide sufficient data to conclude whether or not nsPEF can ablate BCCs on normal individuals.

Eligibility Criteria:
Inclusion Criteria:

1. Study subjects must have had diagnosed at least one BCCon their trunk
2. The subject is from 18-75 years of age, inclusive.
3. The subject must sign and date all informed consent statements.

Exclusion Criteria:

1. The subject is exhibiting signs of a bacterial or viral infection, including fever.
2. The subject is unwilling to allow a biopsy of a malignant lesion for histological analysis.
3. The subject has a pace maker or other electronic device implanted
Transfusion of Biotinylated Red Blood Cells

The purpose of this research is to study how red blood cells (RBCs) survive in a person's circulation, and how that survival may be different in red blood cells that are donated and stored prior to being transfused. Investigators will study this by collecting blood samples from participants, "labeling" RBCs with a naturally occurring vitamin, biotin. The RBCs will then be re-infused back into the participant and blood samples will be taken weekly for 10 weeks to assess the number of labeled cells in the samples.

The purpose of this research is to study how red blood cells (RBCs) survive in a person's circulation, and how that survival may be different in red blood cells that are donated and stored prior to being transfused.

In this study, blood is collected from healthy subjects, processed into packed red blood cell units, and either immediately afterwards or 40-42 days later the packed red blood cells are labeled with biotin. The biotin-labeled red blood cells are then re-infused back to the donor (autologous transfusion). Blood samples are then taken from the subject every week for up to 70 days (10 weeks) to track survival of the labeled red blood cells.

This study also seeks to demonstrate that biotin-labeled RBCs can be safely transfused back to autologous subjects without any adverse reactions. All participants will be followed to watch for the development of antibodies against biotin-labeled RBCs.

Eligibility Criteria:
Inclusion Criteria:

* in good health

Exclusion Criteria:

* anemia (defined as hemoglobin \< 8 g/dL)
* chronic disease, including diabetes, heart or lung disease, poorly managed hypertension, and peripheral vascular disease
* ongoing consumption of biotin or raw egg supplements
* history of a bleeding disorder
* evidence of anemia at initial screening
* women who are pregnant or plan to become pregnant
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (144 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.


Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of DM1 with trinucleotide repeat size \>100.
* Age of onset of DM1 muscle symptoms ≥12 years.
* Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator.
* Hand grip strength and ankle dorsiflexion strength.
* Able to complete 10-MWRT, stair ascend/descend, and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.

Exclusion Criteria:

* History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study.
* History of anaphylaxis.
* Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments.
* Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments.
* Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator.
* Percent predicted forced vital capacity (FVC) \<50%.
* History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study.
* Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide.
* Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.
* Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers.

Note: Other inclusion and exclusion criteria may apply.
VDR SNPs & 25OHD Correlated With Dyslipidemia

The project was aimed to explore the associations of single nucleotide polymorphisms (SNPs) of vitamin D receptor gene with specific circulating lipids in adult Chinese. The circulating 25-hydroxyvitamin D concentration was also tested to explore its interactions with SNPs in the associations.


Eligibility Criteria:
Inclusion Criteria:

* 1) ≥ 20 years old Han Chinese;
* 2) living in Shenzhen for \> 2 years;
* 3) free of liver diseases, renal diseases, and any cancers in the past 6 months; and
* 4) not in pregnancy for women.

Exclusion Criteria:

* 1) had severe organic diseases,
* 2) had acute infection symptoms, allergic diseases, and malignant tumors,
* 3) had a family history of genetic diseases,
* 4) took VD supplements in the past 6 months,
* 5) took medicines to control lipid levels within 12 hours, or
* 6) took diuretics, did strenuous exercise, or overate within 24 hours before the test.
Effectiveness of Focal Vibration in Shoulder Pathology

We hypothesize that focal vibration therapy will reduce pain, increase the mobilization of the glenohumeral joint, and will improve functionality in patients with adhesive capsulitis.

The underlying mechanism of focal vibration therapy in individuals with adhesive capsulitis still remains unclear. Considering the physiological mechanism of focal vibration therapy, the effect of focal vibration therapy on pain, function, and resting muscle tone in patients with adhesive capsulitis will be evaluated. There are no studies examining the effect of focal vibration therapy on shoulder muscle tone in individuals with adhesive capsulitis.

Eligibility Criteria:
Inclusion Criteria:

* Patients aged \> 18 years
* Unilateral adhesive capsulitis of the shoulder
* The symptom duration \> 3 months
* Shoulder pain
* The limited glenohumeral joint active range of motion of greater than 50% in at least three specific movements among abduction, flexion, internal rotation, and external rotation

Exclusion Criteria:

* Previous shoulder surgeries
* Massive, minor, and major rotator cuff tears
* Pain and muscle strength loss due to neurological causes
* History or presence of malignancy
* Osteoporosis
* Diabetes
Operative Versus Non-operative Management of Rotator Cuff Tear

This study aims at determining the effectiveness and cost-effectiveness of surgical management of full-thickness rotator cuff tears compared to conservative treatment. The research setting is prospective, randomised, and controlled.

The aim of the study is to search out evidence based data of indications for rotator cuff repair. The investigators also aim at offering patients the most efficient and effective treatment and reduce the number of operations that do not have sufficient effectiveness. The data obtained will facilitate developing guidelines for referrals to a specialist when rotator cuff tear is suspected.

The investigators hypothesize that there are subgroups of patients suffering from rotator cuff tears that benefit from surgery whereas other subgroups are best treated conservatively.

Subgroup analyses:

* age
* sex
* duration of symptoms
* presence of trauma before symptoms
* size of tear
* degenerative findings
* other findings in MRI or arthroscopy
* type of operation
* co-morbidities
* occupation
* pain (VAS)
* objective shoulder function
* activities of daily living

Eligibility Criteria:
Inclusion Criteria:

* age over 35 years old
* duration of symptoms at least three months despite of non-operative treatment
* the patient accepts both treatment options (operative and conservative)
* a full-thickness rotator cuff tear in MRI arthrography

Exclusion Criteria:

* previous shoulder operations
* too high risk for operation
* any disease or social problem reducing the ability to co-operate
* rheumatoid arthritis
* severe arthrosis of the glenohumeral or acromioclavicular joint
* irreparable rotator cuff tear (including rotator cuff tear arthropathy)
* progressive malign disease
* adhesive capsulitis
* high-energy trauma before symptoms
* cervical syndrome
* shoulder instability
Predictors for Analgesic Effect to Standardized Exercise Therapy for Osteoarthritic Pain

Pain sensitization has been associated with pain severity in people with knee osteoarthritis (KOA) and a neuropathic pain component has been identified in up to 30% of KOA patients. In addition, exercise-induced hypoalgesia (EIH), a measure of descending pain control, has previously been found dysfunctional in a subgroup of people with painful KOA and has also been utilized as a predictive factor for pain progression following total knee replacement.

Measures of pain sensitization have been used prognostic to identify responders to treatment but EIH as a prognostic tool for Physiotherapy-guided Evidence-based Exercise Therapy (PEET) has not been investigated. The primary aim of this explorative study is to investigate if EIH assessed pre-PEET was associated with changes in pain, disability and PainDETECT (PDQ) score post-PEET. The secondary aim is to investigate if PEET changes EIH and PDQ score in patients with painful KOA.

Part 2:

Cross-sectional studies indicate, that daily level of activity influence the effectivity of the EIH mechanims, while pain patients (e.g. KOA patients) can have normal of dysfunctional EIH. It has not been investigated if EIH changes after prolonged exercise in healthy subjects. The aim of this secord part of the study, is to investigate if EIH changes after 6-9 weeks of intensive military training in healthy subjects


Eligibility Criteria:
Inclusion Criteria:

* The American College of Rheumatology for clinical knee osteoarthritis (excluding radiological OA assessment)

Exclusion Criteria:

* Known factors to influence pain and pain sensitization.

Part 2:

Inclusion Criteria

* starting as recruits in Defence Command Denmark

Exclusion Criteria:

* Known factors to influence pain and pain sensitization.
OPTAMISE: Clinical Effectiveness of Teriparatide After Alendronate or Risedronate Therapy in Osteoporotic Postmenopausal Women

To determine how prior therapy with alendronate or risedronate in postmenopausal women with osteoporosis influences the clinical effectiveness of teriparatide; The primary objective of the study is to compare the teriparatide (human, recombinant PTH\[1-34\])-associated change from baseline in a marker of bone formation, N-terminal propeptide of type I collagen (P1NP), between subjects previously treated with risedronate and those previously treated with alendronate.

All subjects will be treated with teriparatide (human, recombinant PTH\[1-34\])(human, recombinant PTH\[1-34\]), 20 microgram subcutaneously daily for 12 months. Subjects will consist of 290 postmenopausal women previously treated with either risedronate or alendronate for at least 24 months. An approximately equal number of subjects will have been previously treated with risedronate and alendronate, and the subjects will be balanced with regard to duration of previous treatment.

Eligibility Criteria:
ELIGIBILITY CRITERIA include:

* Post-menopausal women who have used risedronate or alendronate continuously for at least 24 mos prior to enrollment
* Dosing regimens allowable are continuous (ie, uninterrupted) daily or weekly formulations of risedronate (5 mg once daily \[OD\] or 35 or 30 mg once a week \[OAW\]) or alendronate (10 mg OD or 70 mg OAW), for a minimum of 24 months prior to enrollment into study
* Lumbar spine or total hip BMD T-score 1ess than or equal to -2.0 and \>/= 1 prevalent osteoporotic fracture, or lumbar spine or total hip BMD T-score less than or equal to -2.5 with or without and \>/= 1 prevalent osteoporotic fracture. The qualifying values must be documented prior to enrollment
* Vitamin D (25-hydroxyvitamin D) between 16 ng/ml and 80 ng/ml
* Urine NTX \<50 nmol/mmol creatinine (to assure treatment compliance and bone turnover is in the pre-menopausal range)

EXCLUSION CRITERIA include:

* Impaired renal function, demonstrated by creatinine clearance \< 30 ml/min
* Any condition or disease that may interfere with the evaluation of at least 2 lumbar vertebrae (not necessarily contiguous), determined in a screening radiograph by a radiologist at the central facility (eg, confluent aortic calcifications, severe osteoarthritis, spinal fusion, lumbar spine fractures)
* Depot injection vitamin D \>10,000 IU in the past 9 months prior to starting the investigational product
* Treatment with antiresorptive agents other than risedronate, alendronate, and hormone replacement therapy within the last 36 months before study entry (ie, ibandronate, pamidronate, etidronate, raloxifene, clodronate, or zoledronate)
* Use of combination alendronate and risedronate, either simultaneously or sequentially, within 60 months prior to enrollment, or use of any anti-resorptive agent in combination with risedronate or alendronate
Intranasal Transmucosal Fentanyl Pectin for Breakthrough Cancer Pain in Radiation-induced Oropharyngeal Mucositis

An open-label, non-randomized study to assess the titration, safety and efficacy of intranasal fentanyl pectin nasal spray for the treatment of secondary breakthrough pain secondary to radiation-induced mucositis in patients with confirmed tolerance of opioid therapy for chronic pain.

Study objectives include assessment of breakthrough pain episodes related with food intake in patients with mucositis secondary to radiotherapy or radio-chemotherapy for head and neck tumors

An open, non-randomized study design was used to assess the titration, safety and efficacy of intranasal fentanyl in pectin for the treatment of secondary breakthrough pain in patients with confirmed tolerance of opioid therapy for chronic pain secondary to radiation-induced mucositis. The study plans to include 30 evaluable patients from multiple centers throughout the country. The trial comprises a screening period, an open titration period, and an open-label treatment period in which at least 12 breakthrough pain episodes are to be treated.

Eligibility Criteria:
Inclusion Criteria:

A histologically confirmed diagnosis of head and neck carcinoma Treatment in the form of radiotherapy or radio-chemotherapy Breakthrough pain related to swallowing (one to four daily episodes) Treatment with controlled release opioids at the time of inclusion: at least 60 mg of morphine sulfate daily, at least 25 microg of transdermal fentanyl per hour, at least 30 mg of oral oxycodone daily, at least 8 mg of oral hydromorphone daily

Exclusion Criteria:

* Patients without controlled release opioid treatment.
An International Study of the Safety and Tolerability of Corlux for Psychotic Symptoms in Psychotic Major Depression

Corlux (mifepristone) is a new medication that modulates the body's use of a hormone called cortisol. Under normal conditions, cortisol and other hormones are created by the body in response to physical and emotional stress, triggering a healthy stress response. People who suffer from psychotic major depression may have unusually high levels of cortisol circulating within them or abnormal patterns of cortisol levels, overloading the stress response mechanism and causing symptoms of psychosis such as delusional thoughts or hallucinations. If Corlux can keep the body's cortisol receptors from being overloaded, the stress response system may return to normal function, which may result in improvement of symptoms. The purpose of this 56 day study is to learn the safety and effectiveness of Corlux in patients who have been diagnosed with psychotic major depression (PMD).


Eligibility Criteria:
Inclusion Criteria:

* Are 18 to 75 years of age
* Have a diagnosis of major depressive disorder with psychotic features (Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition \[DSM IV\] 296.24 or 296.34)
* Are able to provide written informed consent.

Exclusion Criteria:

* Have a major medical problem
* Have previously participated in a Corlux (C-1073, mifepristone) clinical trial
* Have a history of an allergic reaction to Corlux (C-1073, mifepristone).