cyrilzakka/clinical-trials huggingface dataset:
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

This is an open-label, single arm, multi-center study. Approximately 28 participants aged 2 to \<18 years will be enrolled stratified as 2 to 5 years and 6 to \< 18 years. The study is comprised of 3 periods, Screening (up to 45 days), Treatment (1 day), and Follow-up (52 weeks).

During the Screening period and on Day -1 (Baseline), eligibility will be assessed, including confirmation that nusinersen (Spinraza®) or risdiplam (Evrysdi®) have not been used for the defined period (4 month and 15 days prior to Day -1 respectively). On Day - 1 (Baseline) participants will be admitted to the hospital for pre-treatment baseline procedures. Prednisolone (or equivalent) will be given and continued as per the study protocol.

Participants who meet eligibility criteria at Screening and Baseline will receive a single dose of OAV101 (1.2 x 10\^14 vector genomes) by lumbar intrathecal injection on Day 1 (Treatment) and will then undergo in-patient safety monitoring atleast the next 48 hours, after which the participant may be discharged according to Investigator judgement.

During Follow-up, safety monitoring will continue as per the visits defined in the Assessment Schedule. Safety for participants enrolled will be evaluated by the study team together with the Data Monitoring Committee (DMC).

Final analysis will be performed after all participants have completed Week 52 or discontinued prior to Week 52. At the end of study, participants will be invited to enroll in a Novartis-sponsored long-term follow-up study to monitor long-term safety and efficacy.

Eligibility Criteria:
Inclusion Criteria

* SMA diagnosis
* Aged 2 to \< 18 years
* Have had at least four loading doses of nusinersen (Spinraza®) or at least 3 months of treatment with risdiplam (Evrysdi®) at Screening
* Must have symptoms of SMA as defined in the protocol

Exclusion Criteria:

* Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated
* Clinically significant abnormalities in test results during screening
* Contraindications for lumbar puncture procedure
* At Baseline, participants are excluded if they received:

  * nusinersen (Spinraza®) or
  * risdiplam (Evrysdi®) within a defined timeframe
* Vaccinations 2 weeks prior to administration of OAV101
* Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.
* Presence of an infection or febrile illness up to 30 days prior to administration of OAV101
* Requiring invasive ventilation
Cardiovascular Risk in Growth Hormone Deficient Young Adult Males After Completing Growth Hormone Therapy

The purpose of this study is to compare the cardiovascular risks of growth hormone deficient young adult males who have completed growth hormone therapy to healthy young adult males.

Adults with growth hormone deficiency are at risk for cardiovascular disease. What age this begins is not known.

The objectives of this study are to compare the following measurements between growth hormone deficient young males 3-7 years post completion of growth hormone treatment and age related healthy controls:

1. Carotid artery imaging
2. Metabolic and cardiac parameters
3. Body composition measurements

Eligibility Criteria:
Growth Hormone Deficient Inclusion Criteria:

* Males, 18-25 years old
* Completed growth hormone therapy approximately 3-5 years prior to study enrollment
* History of multiple pituitary hormone deficiencies or growth hormone stimulation test \<7 at initiation of growth hormone therapy

Healthy Control Inclusion Criteria:

* Males, 18-25 years old
* No confirmed endocrine diagnosis including Diabetes and thyroid disorders
* BMI - z-score within 2 SD of mean

Exclusion Criteria:

* Known risk factors for cardiovascular disease (eg tobacco use, hypertension, diabetes)
* Family history of early cardiovascular disease (myocardial infarction or stroke \<55years of age) in first degree family members
* Any implanted device
* Medication known to interfere with glucose or lipid metabolism or to influence blood pressure
* Chronic organ illness (eg severe asthma, hepatic or nephritic conditions)
Hydroquinidine Versus Placebo in Patients With Brugada Syndrome

The specific aim of this study is to determine whether hydroquinidine administration can prevent heart from appearance of ventricular arrhythmia detected by the automatic implantable defibrillator (ICD).

During this double-blind randomized cross-over study, patient will receive during 18 months treatment 1 (hydroquinidine or placebo) and, after 7 days of wash-out, patient will receive treatment 2 (meaning for example hydroquinidine if treatment 1 was placebo). Time length before arisen of an appropriate shock registered on the defibrillator (meaning due to ventricular arrhythmia) will be assessed during treatment 1 period and treatment 2 period.We hypothesized that hydroquinidine administration will enhance time length before arisen of an appropriate shock and thus mean that hydroquinidine administration can prevent heart from appearance of ventricular arrhythmia. Patient's defibrillator recordings will be analysed every 6 months plus when patient experiences an ICD shock. If the shock delivered by the ICD is appropriate and happens during treatment 1 period, patient will switch to treatment 2 period after 7 days of wash-out. If the shock delivered by the ICD is appropriate and happens during treatment 2 period, study will be finished for this patient.Before starting the study, each patient will test which dose of hydroquinidine she/he requires to have an hydroquinidine concentration in her/his blood included between 3 and 6 µmol/L.

Planned enrollment: 200 subjects (60 being symptomatic with histories of aborted sudden cardiac death or of ventricular fibrillation, 70 being symptomatic with histories of syncope considered as of arrhythmic origin, 70 being asymptomatic with a spontaneous type 1 ECG and a positive electrophysiological exploration)

Eligibility Criteria:
Inclusion Criteria:

* Healthy adult (at least 18 years of age)
* Informed consent form signed
* Subject affiliated to French health insurance (Sécurité Sociale)
* Type 1 Brugada syndrome either symptomatic or asymptomatic
* Not pregnant, taking oral contraceptive measure if able to procreate
* If patient with asymptomatic type 1 Brugada, electrophysiological exploration must be positive at study inclusion
* No current intake of "betablocking" medicine used in cardiac insufficiency (bisoprolol, carvedilol, metoprolol)
* No current myasthenia
* No current treatment with halofantrine, pentamidine, moxifloxacin
* No current treatment with some neuroleptics
* Known hypersensitivity to hydroquinidine
* Intolerance to fructose, syndrome of glucose or galactose malabsorption, deficit in sucrase isomaltase- Cardiac insufficiency
* Histories of "torsades de pointe"
* Intake of medicine giving "torsades de pointe"

Exclusion Criteria:

* Subject not fulfilling inclusion criteria
* Subject being before study entry under hydroquinidine treatment but either at a dose \> 3 capsules per day or at a dose of 1, 2 or 3 capsules per day but with a plasmatic hydroquinidine concentration \>6µmol/L or \<3 µmol/L
Therapeutic Effect and Safety Study of Decitabine in Elderly Acute Myeloid Leukemia Patients

The purpose of this study is to confirm the safety and the therapeutic effect of Decitabine in the treatment of elderly Acute Myeloid Leukemia (AML) patients.

The outcome of the elderly AML patients is very poor. No obvious progress was achieved in this field. Decitabine is a kind of specific DNA methylation shift enzyme inhibitor. It can reverse the DNA methylation and induce the differentiation and apoptosis of the tumor cells. Recent studies about decitabine in the treatment for elderly AML patients had achieved inspiring results and indicated that low dose decitabine maybe a good choice for elderly AML patients. So in this research the investigators plan to evaluate the safety and the therapeutic effect of decitabine in the treatment of elderly AML patients.

Eligibility Criteria:
Inclusion Criteria:

1. Clinical diagnosis of acute myeloid leukemia.De novo or secondary AML.
2. Age \>= 60years,female and male.
3. Before the enrollment，WBC \< 40×10E9/L，Plt \> 20×10E9/L（Hydroxyurea is permitted.）
4. In 2 weeks before the enrollment，total bilirubin \< 1.5×ULN，ALT \< 2.5×ULN；GGT \< 2.5×ULN ，Scr \< 2.0×ULN or creatinine clearance rate ≥ 50 ml/min（Cockroft-Gault）.
5. Before the enrollment，patients must be free from the toxicity caused by the former treatment.Received no chemotherapy in the last 4 weeks and no nitrosourea in the last 6 weeks.
6. Contraception must be taken to avoid pregnancy during the study.
7. ECOG 0,1, or 2, predicted lifetime longer than 12 weeks.
8. Patients must sign the informed consent prior to any study related screening procedures being performed.

Exclusion Criteria:

1. Acute promyelocytic leukemia.
2. Chromosome and genetic abnormalities related with t（8; 21）、inv（16）、t（15; 17）.
3. Central nervous system leukemia.
4. Bone marrow dry tap.
5. Patients received stem cell transplantation or chemotherapy containing azacitidine,cytarabine or decitabine in last one year, radiation therapy in last 14 days,lenalidomide in 30 days before included.
6. Patients suffered from autoimmune hemolytic anemia or immune thrombocytopenia.
7. Patients suffered from non-leukemia related comorbidities that will cause dysfunction of organs.
8. Patients suffered from unstable angina or （NYHA）3/4 Congestive heart failure.
9. Patients suffered from chronic respiratory disease and needed continued oxygen.
10. Other active malignancy.
11. Active HBV,HCV or AIDS patients.
12. Uncontrolled virus or bacterium infection.
13. The investigator believe that patients who are not suitable for this trial.
14. Severe mental or body disorders which will interfere the research such as uncontrolled heart,lung diseases,diabetes,etc.
15. Allergic to decitabine or its accessory.
16. Patients received other researches in last 30 days.
17. Without contraception.
18. Complications causing organ dysfunction which are not caused by AML.
Three Different Transdermal Doses of Rotigotine in Subjects With Idiopathic Restless Leg Syndrome

The primary objective of this trial is to demonstrate that rotigotine (SPM 936) is efficacious in subjects with idiopathic restless leg syndrome (RLS). Additional objectives are to investigate the safety and tolerability of rotigotine.

The primary variables are the absolute change from Baseline in the International Restless Legs Severity Scale (IRLS) sum score and Clinical Global Impression-Global Improvement (CGI) Item 1 (severity of illness) score at the end of the Maintenance Period.

Subjects will be randomized to receive either placebo, 2.25, 4.5 or 6.75 mg/day rotigotine in a 1:1:1:1 (active:placebo) fashion. Approximately 450 subjects will be enrolled in this trial, participating at approximately 50 sites. The maximum duration of the trial is approximately 8 months (3-week Titration Period, 6-month Maintenance Period, 7-day Taper Period, and 30-day Safety Follow-Up Period). Subjects who complete the 6-month Maintenance Period will be eligible to participate in an open-label extension trial. Subjects who do not complete the 6-month Maintenance Period or who choose not to participate in the open-label extension trial will complete a 3-day Safety Follow-Up Period. Two different patch sizes will be used (5 and 10 cm2). Active patches will contain either 2.25mg (5cm2) or 4.5mg (10cm2) of rotigotine.


Eligibility Criteria:
Inclusion Criteria:

* Idiopathic RLS

Exclusion Criteria:

* History of sleep disturbances
Bronchial Thermoplasty for Severe Asthma With Dynamic Hyperinflation

Bronchial thermoplasty is a treatment for severe asthma that consist in decreasing the thickness of bronchial muscle by heat using a catheter inserted into the bronchi under direct vision with the help of an endoscope This treatment has shown efficacy on symptoms, quality of life and the number of exacerbations related to severe asthma.

This clinical study evaluates the efficiency of this treatment on the dynamic hyperinflation phenomenon (worsening of bronchial obstruction during exercise in patients with asthma contributing to worsening shortness of breath).

Bronchial thermoplasty is a recently validated bronchoscopic technique for the management of severe asthma that treats smooth muscle by radiofrequency to reduce its thickness. This procedure leads to improved asthma control and quality of life, but also to decreased exacerbations frequency and number of emergencies hospitalizations. This treatment requires 3 procedures every 3 to 4 weeks.

The pathophysiological mechanisms underlying its effectiveness need to be better understood for an improved selection of best candidates. In particular, there is a discrepancy between the improvement observed in symptoms (ACQ), quality of life (AQLQ) and the lack of improvement in forced expiratory volume in 1 second (FEV1). Investigator assume that this paradox is due to an efficiency appearing at effort, targeting dynamic hyperinflation phenomenon.

The objective of this study is to evaluate the influence of bronchial thermoplasty on dynamic hyperinflation in severe asthma. The secondary objectives are to assess the effectiveness of bronchial thermoplasty (ACQ, AQLQ) in a selected population of patients with dynamic hyperinflation and to describe bronchial wall structural changes by probe-based confocal LASER endomicroscopy (pCLE).

Eligibility Criteria:
Inclusion Criteria:

* Severe asthma, uncontrolled despite optimal treatment according to GINA (global initiative for asthma) recommendations
* At least 2 exacerbations treated by systemic steroids in the last year
* FEV1 between 40 and 80% of predicted values and dynamic hyperinflation (defined as a decrease in inspiratory capacity by more than 500 ml during exercise)

Exclusion Criteria:

* Current asthma exacerbation or respiratory infection
* History of exacerbation after bronchoscopy
* FEV1 \< 40% of predicted values
* Oxygen saturation \< 90%
* Contra-indications to ALAIR catheter system : pacemaker or other electronic implanted device
* Allergy to Remifentanyl or Propofol
* pregnancy; breastfeeding
Non-Dispensing Evaluation of an Investigational Galyfilcon A Soft Contact Lens

The purpose of this investigation is to compare the fit characteristics and vision attributes of an investigational Galyfilcon A contact lens and an approved Galyfilcon A contact lens.


Eligibility Criteria:
Inclusion Criteria:

* Be of legal age (i.e., at least 18 years old)
* Sign a written informed consent
* Have contact lens distance sphere requirement in the range -1.00D to -6.00D
* Have spectacle astigmatism \<1.25D in each eye
* Currently wear ACUVUE® ADVANCE™ (for at least 6 months) with documentation of current prescription.
* Have normal eyes with no evidence of abnormality or disease.

Exclusion Criteria:

* Required concurrent ocular medication
* Any systemic illness which would contra-indicate lens wear or the medical treatment of which would affect vision or successful lens wear.
* Eye injury or surgery within eight weeks immediately prior to enrollment for this study.
* Abnormal lacrimal secretions
* Pre-existing ocular irritation that would preclude contact lens fitting.
* Keratoconus or other corneal irregularity.
* Pregnancy, lactating or planning a pregnancy at the time of enrolment.
* Participation in any concurrent clinical trial
* Known allergy to silver, silver ions, or silver containing compounds.
A Neuroprosthesis for Seated Posture and Balance

The purpose of this study is to evaluate a surgically implanted functional electrical stimulation (FES) system to facilitate stability of the trunk and hips. FES involves applying small electric currents to the nerves, which cause the muscles to contract. This study evaluates how stabilizing and stiffening the trunk with FES can change the way spinal cord injured volunteers sit, breathe, reach, push a wheelchair, or roll in bed.

In a single surgical procedure electrodes will be inserted into muscles of the trunk and hip musculature. The electrode leads are connected to a stimulator/telemeter located in the abdomen. After a typical post-operative period of two to five days, neuroprosthesis recipients will be discharged to home for two to six weeks of restricted activity to promote healing of all surgical incisions. After a period of exercise and training, functional use of the neuroprosthesis will begin. Laboratory assessments of strength, balance, and functional abilities with and without the system, as well as the technical performance of the implanted components will be evaluated.

Eligibility Criteria:
Inclusion Criteria:

* C4-T12
* ASIA Scale A through C
* Time post injury greater than 6 months
* Innervated and excitable trunk and pelvis musculature
* Absence of acute or chronic psychological problems or chemical dependency
* Range of motion within normal limits
* Controlled spasticity and absence of hip flexion and adduction spasm
* Height and weight within normal limits
* No history of balance problems or spontaneous falls
* No history of spontaneous fracture or evidence low bone density
* No acute orthopaedic problems
* No acute medical complications
* Adequate social support and stability
* Able to speak and read English

Exclusion Criteria:

* Pregnancy
* Non-English speaking
Breaking Through the Brain Fog: An Online Research Study

This study will investigate the benefits of distinct brain health trainings in breast cancer survivors (age 30-80) with cognitive concerns post-chemotherapy. This study can be completed from the comfort of participant's own home.

Participants will complete online assessments at 3 timepoints over 6 months (Month 0, 3, 6). During the first 3 months of the study, participants will be randomized into one of three online training programs to support cognitive function; training will end after the first 3 months. The third online assessment (Month 6) will measure maintained gains since ending training. Participants will not be aware of the interventions assigned to other participants. Participants may be reimbursed for their time.

Eligibility Criteria:
Inclusion Criteria:

Female breast cancer survivors who...

* are between ages of 30-80
* were diagnosed with Stage I-III
* received chemotherapy
* completed chemotherapy at least 6 months ago but not more than 5 years
* endorse changes in thinking since end of chemotherapy treatment
* have working internet and a device
* consider themselves fluent in English

Exclusion criteria:

* Males
* A re-occurrence of breast cancer or a diagnosis of another
* An uncorrected hearing or vision issue that would hinder ability to read and/or listen to training material
* Participation in a cognitive training program in the last 12 months.
* A reported substance abuse problem
* Diagnosis of a neurodegenerative disease or psychotic disorder
* History of stroke, concussion, or brain injury since completing chemotherapy
* Inability to function independently due to Autism Spectrum Disorder diagnosis
Predict + Protect Study: Exploring the Effectiveness of a Predictive Health Education Intervention on the Adoption of Protective Behaviors Related to ILI

The goal of this prospective, digital randomized controlled trial is to evaluate the effectiveness of a predictive ILI detection algorithm and associated alerts during influenza season for adults living in the contigent United States. The main study objectives are to assess the effectiveness of predictive ILI detection algorithm and associated alerts on protective behaviors related to ILI and assess the accuracy of a predictive ILI detection algorithm using participant self-reported ILI symptoms and diagnosis.


Eligibility Criteria:
Inclusion Criteria:

* Member of the Evidation platform
* 18 years or older
* Lives in the U.S.
* Currently owns and uses a consumer wearable activity tracker (Apple Watch, Garmin, or Fitbit) linked to their Evidation account
* Meets data density requirements for wearable data: Steps and heart rate data present for 15% of the last 60 days (or no fewer than 2 total days for Evidation accounts less than 60 days old)

Exclusion Criteria:

* Does not have an Evidation account
* Not 18 years or older
* Does not live in the U.S.
* Does not have an activity tracker linked to their Evidation account
* Enrolled in an Evidation supported ILI monitoring and engagement program, or clinical study (e.g., FluSmart)
Development and Validation of a Novel Functional Eye-Tracking Software Application for Neurological Disorders

This study aims to develop and validate a sensitive and non-invasive eye-tracking software application.

This study will obtain participant responses to brief cognitive tests designed to evaluate several key functions known to be affected by neurological disorders and non-invasive eye movement measurements in response to visually presented stimuli during specifically designed eye-tracking tests. The study data will be used to develop machine learning algorithms and validate a software application intended to track the progressive component of neurological disorders and associated cognitive changes.


Eligibility Criteria:
Inclusion Criteria:

* Aged 18 years old and above
* A score of ≥ 26 on the Montreal Cognitive Assessment (MoCA)
* Able to provide informed consent
* Visual acuity of 20/100 in at least one eye (corrective glasses, contact lenses, surgery etc are permitted).

Exclusion Criteria:

* Evidence or medical history of psychiatric issues, which are known to also affect movements and oculomotor control.
* Evidence or history of significant neurological disorder (e.g. MS, PD, ALS, Dementia)
* Use of prescription drugs known to influence ocular motor visual function, such as benzodiazepines, antipsychotics, and anticonvulsants.
* Diagnosed with an active substance use disorder.
* History of stroke.
* Recent traumatic brain injury (within the last 6 months).
Treatment Adherence With Twin-Blocks for Correction of Class II Malocclusion

To compare the effect of two control intervals (once every 4th week or once every 6th week) on the wear time of a Twin Block removable appliance.

If the patient fulfill the inclusion criteria, they will be asked to participate in the study. After being informed about the study and the planned treatment, participants and their guardians will give their written consent to participate in the study.

Therafter they will be consecutively randomized into one of two groups with different control intervals, Group 1 (every 4th week) and Group 2 (every 6th week). They will recieve a clinical and radiological examination and then start treatment with a Twin Block appliance.

The Twin Block appliance will be fitted with a digital timer module to measure the wear time of the appliance objectively.

Eligibility Criteria:
Inclusion Criteria:

* Healthy patients (ASA 1 and 2)
* Overjet of 6 mm or more
* Incomplete lip closure

Exclusion Criteria:

* Severe chronic conditions such as asthma or allergies
* Neuroopsychiatric disorders
* Craniofacial syndromes
* Previous orthodontic treatment