cyrilzakka/clinical-trials huggingface dataset:
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.

The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution in children and adolescents with Phelan-McDermid Syndrome. The secondary purpose is to investigate measures of efficacy. Subjects will receive treatment with NNZ-2591 oral solution (50 mg/mL) doses for a total of 13 weeks.

Eligibility Criteria:
Inclusion Criteria:

1. Clinical diagnosis of PMS with a documented disease-causing genetic abnormality of SHANK3.
2. Males or females aged 3-12 years.
3. Body weight of 12 kg or higher at Screening.
4. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
5. Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
6. Each subject must be able to swallow the study medication provided as a liquid solution.
7. Caregiver(s) must have sufficient English language skills.

Exclusion Criteria:

1. Body weight \< 12kg at screening
2. Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
3. Abnormal QTcF interval or prolongation at Screening.
4. Any other clinically significant finding on ECG at the Screening visit.
5. Positive for severe acute respiratory syndrome coronavirus 2 (SARSCoV- 2) and previous COVID 19 infection with last 12 months that required hospitalization
6. Unstable or changes Psychotropic treatment 2 weeks prior to screening .
7. Excluded concomitant treatments.
8. Actively undergoing regression or loss of skills.
9. Unstable seizure profile.
10. Current clinically significant renal conditions and abnormalities
11. Current clinically significant cardiovascular, renal, hepatic, gastrointestinal, respiratory, endocrine disease, or clinically significant organ impairment.
12. Current clinically significant hypo or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes.
13. Has planned surgery during the study.
14. History of, or current, cerebrovascular disease or brain trauma.
15. History of, or current catatonia or catatonia-like symptoms.
16. History of, or current, malignancy.
17. Current major or persistent depressive disorder (including bipolar depression).
18. Significant, uncorrected visual or uncorrected hearing impairment.
19. Allergy to strawberry.
20. Positive pregnancy test
21. Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study
Stress Management and Resiliency Training Program for Parkinson's Disease

SMART-PD is an 8-session program designed to decrease the physiological, emotional, cognitive and behavioral effects of stress in participants with Parkinson's disease and their caregivers by teaching cognitive-behavioral techniques and relaxation skills to help participants learn to elicit the relaxation response, alter cognitive appraisals, improve healthy lifestyle behaviors and access social support. The SMART program has been shown to be effective for reducing mental health symptoms such as depression and anxiety as well as physical symptoms such as pain and in promoting positive health behaviors.

This is an interventional study sponsored by the Parkinson's Foundation designed to measure the feasibility, acceptability, and initial impact of the SMART-PD program on participants with a recent (less than five years) diagnosis of Parkinson's disease and their caregivers. This program provides information and tools to help patients and their caregivers cope with the new PD diagnosis and manage the stress and negative mental health symptoms (such as anxiety and depression) associated with the diagnosis.

SMART-PD is a manualized 8-week intervention that teaches cognitive-behavioral techniques and relaxation skills to help participants learn to elicit the relaxation response, alter cognitive appraisals, improve healthy lifestyle behaviors (i.e., sleep, nutrition, physical activity) and access social support.

Participants will take baseline and follow-up questionnaires to ascertain the impact of the program on outcomes such as quality of life, emotional health, coping skills, and healthy lifestyle behaviors.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of Parkinson's Disease within the past three years or caregiver of someone with a recent diagnosis of Parkinson's disease
* Ability to participate in 8 weekly program sessions via Microsoft Teams or in-person (as available)
* Ability to provide informed consent

Exclusion Criteria:

* Previous participation in the SMART program
Therapeutic Mechanisms of Epidural Spinal Cord Stimulation

The purpose of this study in patients undergoing routine care epidural spinal cord stimulation (SCS) is to determine 1) whether SCS reduces arterial blood pressure (BP) in patients which chronic low back pain and hypertension, 2) whether higher baseline BP (i.e., hypertension) predicts reductions in pain following SCS, and finally 3) whether different SCS waveforms elicits stimulus-evoked compound action potentials (ECAPs) in spinal cord and at the cortex (electroencephalography, and magnetoenchphalography).

The purpose of this study in patients undergoing routine care epidural spinal cord stimulation (SCS) is to determine 1) whether SCS reduces arterial blood pressure (BP) in patients which chronic low back pain and hypertension, 2) whether higher baseline BP (i.e., hypertension) predicts reductions in pain following SCS, and finally 3) whether different SCS waveforms elicits stimulus-evoked compound action potentials (ECAPs) in spinal cord and at the cortex (electroencephalography, and magnetoenchphalography).

The Investigators will identify patients with chronic pain who are scheduled for SCS implant at the University of Alabama at Birmingham for management of chronic neuropathic pain as part of routine care. The research team will assess BP with an arm cuff arm cuff before and after the implant among patients providing written informed consent.

The Investigators' central clinical hypothesis is that SCS decreases blood pressure in patients with chronic pain and comorbid hypertension. The Investigators further hypothesize that the SCS will improve serological markers of sympathetic nerve activity and kidney function, possibly due to reductions in spinal cord sympathetic nerve activity. A secondary hypothesis is that higher baseline blood pressure predicts larger reductions in blood pressure following SCS implant.

Additionally, this project seeks to expand knowledge on therapeutic mechanisms of SCS via recording electrophysiological responses at the spinal cord (via adjacent, unused SCS contacts) and from EEG scalp recordings over the cerebral cortex.

Eligibility Criteria:
Inclusion Criteria:

1. Male or Female, age 18-89
2. Chronic pain for more than 3 months
3. Willing to visit a research lab
4. Willing to undergo a blood draw
5. Able to provide written informed consent

Exclusion Criteria

1. History of neurological disease (e.g., dementias, Parkinson's)
2. History of stroke
3. Current diagnosis of cancer
4. Subject is unwilling or unable to comply with the protocol
An ObserVatIonal STudy of the Effectiveness of AdaLimumab on Health and Disability Outcomes in New Zealand Patients With Immune-Mediated InflammaTorY Diseases (VITALITY)

For public health purposes disability is becoming increasingly important as an outcome measure. Despite this, there are few data on the effectiveness of adalimumab on disability outcomes in patients with immune-mediated inflammatory diseases (IMIDs), particularly in the Phase IV setting. There are even less data available in New Zealand, which did not have the opportunity to participate to a major extent in large, multinational, Phase III pivotal studies of adalimumab in IMIDs.

The World Health Organisation Disability Assessment Schedule (WHODAS) 2.0 is a simple, validated, free and easy-to-use generic assessment instrument for health and disability. It is applicable across cultures, in all adult populations. It is a responsive measure that can show what difference a treatment makes.

Results from study of effect of adalimumab on WHODAS scores and other patient-reported outcomes (PROs) of work activity and well-being will be of interest to a variety of stakeholders in the healthcare system including patients, healthcare practitioners and payers.


Eligibility Criteria:
Inclusion Criteria:

* Patients with a diagnosis of rheumatoid arthritis, Crohn's disease or psoriasis who have made a decision with their physician to commence treatment with adalimumab in accordance with routine medical practice and with the approved adalimumab New Zealand Datasheet.
* Patients who have been evaluated for tuberculosis risk factors/exposure for active/latent tuberculosis infection (per local requirements and according to the approved adalimumab New Zealand Datasheet).
* Adult subjects, 18-75 years old, who have voluntarily signed and dated an informed consent form prior to any study-specific procedures.

Exclusion Criteria:

* Previous treatment with adalimumab.
* Previous treatment with any biologic.
* Severe infection including sepsis, active tuberculosis or opportunistic infection.
* Moderate to severe heart failure (New York Heart Association Class II/III).
* Concurrent administration with anakinra.
* Hypersensitivity to adalimumab or its excipients.
* Any condition that in the opinion of the investigator would compromise the subject's well-being or ability to perform the study requirements.
Prospective Study to Validate the Clinical Accuracy of the Norbert Device to Measure Oxygen Saturation

The purpose of this study is to evaluate the SpO2 accuracy and performance of the Norbert Device during non-motion conditions over the range of 70-100% SaO2 as compared to arterial blood samples assessed by CO-oximetry for SpO2 validation.

The study is a single center, prospective, non-randomized study to measure the accuracy of an investigational device compared to blood reference values. The design of the study will be conducted in accordance with the recommendations of ISO 80601-2-61:2011 Annex EE.2 Procedure for invasive laboratory testing on healthy volunteers. The purpose of this study is to evaluate the SpO2 accuracy and performance of the Norbert Device during non-motion conditions over the range of 70-100% SaO2 as compared to arterial blood samples assessed by CO-oximetry for SpO2 validation. The end goal is to show the SpO2 accuracy performance of the Norbert Device. It is expected that the Accuracy Root Mean Square (Arms) performance of pulse oximeters will meet a specification of 3.5 or better in non-motion conditions for the range of 70-100% SaO2 thereby demonstrating an acceptable SpO2 accuracy performance specification.

Eligibility Criteria:
Inclusion Criteria:

* Healthy, male or female subjects between the ages of 18 to 50 years
* Completion of a health screening for a medical history by a licensed physician, nurse practitioner, or physician assistant
* Previously measured hemoglobin electrophoresis with normal result recorded in Duke Epic EHR
* Minimum weight 40kg; BMI within range 18.0 - 35.0
* Assigned American Society of Anesthesiologists (ASA) Physical Status 1 by Principal Investigator or delegate

Exclusion Criteria:

* Prior or known allergies to lidocaine (or similar pharmacologic agents, e.g., Novocain)
* Prior known severe allergies to medical grade adhesive/tape (Band-Aid)
* Taking any medication other than birth control
* Is currently participating in, or has recently participated in (discontinued within 30 days prior to the hypoxia procedure for this study) in an investigational interventional drug, device, or biologic study
* Has a negative Allen's Test to confirm non patency of the collateral artery
* Has made a whole blood donation or has had at least 450 ml of blood drawn within 8 weeks prior to the study procedure
* Is female with a positive urine pregnancy test, or is female and is unwilling to use effective birth control between the time of screening and study procedure or is breast feeding
* Has anemia
* Has heparin allergy
* Has a history of sickle cell trait or thalassemia \[self-reported\]
* Has a positive urine cotinine test or urine drug screen or oral ethanol test
* Has a room air saturation less than 95% by pulse oximetry
* Has a clinically significant abnormal EKG
* Has a COHb greater than 3%, or MetHb greater than 2%
* Students and Employees under the direct supervision of PI or Sub-I
Restart Anticoagulation in Patients With Spontaneous Intracerebral Hemorrhage and Mechanical Heart Valves

The investigators retrospectively reviewed the electronic medical records of the patients with spontaneous cerebral hemorrhage and mechanical valves from 2015 to 2020 in the second affiliated hospital of Zhejiang university medical college. Medical documents and patient data were reviewed, including patient age, gender, type of mechanical heart valve, replacement time, replacement site, underlying disease, anticoagulant drugs at admission, type of cerebral hemorrhage, treatment mode, time without anticoagulant, length of stay, mortality, etc


Eligibility Criteria:
Inclusion Criteria:

* patients with spontaneous cerebral hemorrhage and mechanical valves

Exclusion Criteria:

* age\<18y
* pregnant patients
* death in 24 hours without anticoagulant
Exercise Capacity in Kidney Transplant Recipients

This study aimed to determine the determinants of 6 minute walk test distance in patients with kidney transplant recipients.


Eligibility Criteria:
Inclusion Criteria:

* Patients with kidney transplantation
* At least 6 months after the kidney transplant
* Being aged 18-65
* Not having regular exercise habits
* Voluntary participation in the study

Exclusion Criteria:

* Having a musculoskeletal problem that may interfere with the tests
* Having neurological impairment
* Having chronic lung disease
* Having had any cardiovascular event in the last 6 months
The Effect of Asthma Education Given by Taking Health Literacy Into Account

The aim of this randomized controlled trial was to examine the effect of education given to asthmatic children and their parents by taking health literacy into account on disease self-management.

The study was conducted with 88 children and their parents between October 2018 and July 2019. While education and booklets were given to both children and parents in intervention group 1, only the children were given the education and the booklets in intervention group 2. There was no intervention applied for the control group. Post-test was performed 3 weeks after the education and then follow-ups were carried out in the next three months over the phone.


Eligibility Criteria:
Inclusion Criteria:

* Having asthma children with a physician diagnosis,
* Studying in one of the public primary schools in the district where the study was conducted,
* Using asthma medication,
* Having parents without any reading difficulties.

Exclusion Criteria:

* Being in the 1st grade in their school,
* Not using any medication for asthma,
* Having parents with reading difficulties.
Objectives and Measures Dimensions Nasal Resistance of Preterm and Term (MODERN)

During the resuscitation of preterm infants, withdrawal of non-invasive ventilation is difficult. In a recent study in children gestational age \<30 weeks of amenorrhea (SA), definitive withdrawal rate from the first attempt to stop the non-invasive ventilation was 32% . In the same study, the median number of attempts before achieving a final withdrawal was 3 for children born before 28 weeks. The reasons for weaning failure are multiple and little studied. It seems that the type of interface used, mask or cannula, having an influence on the effectiveness of non-invasive ventilation . The nasal lesions induced by non-invasive ventilation are not uncommon, regardless of the interface used . In a recent randomized trial, they ranged from 40% to 50% depending on the type of nasal cannula and non-invasive ventilation mode. It is in this case external damage. But we assume that the breakdown causes internal lesions dependent on the pressure, humidity and flow. These factors are likely to generate a nasal obstruction, source intervention nurses who aspire nasal passages more or less traumatic.

The nasal cavities are often abused and, because of their key role in breathing, could be involved in the withdrawal of ventilatory failure

The measurements of the nasal cavity dimensions are carried out by acoustic rhinometry (about 10 seconds for an acquisition). Measurement of nasal resistance is performed by rhinomanometry earlier (about a minute to a measure). They may be made to wakefulness or sleep since they do not require their participation. These measures will be carried at birth and then every 7 to 10 days (before the age of 28 days), if the child is still hospitalized.

During these measurements, the heart rate and oxygen saturation are collected by a measuring sensor transcutaneously.

Eligibility Criteria:
Inclusion Criteria:

* Affiliation to social security

  * Informed Parental Consent
  * Newborns term and preterm
  * Age less than or equal to 28 days
  * Signature of informed consent by both parents

Exclusion Criteria:

* craniofacial malformation

  * Dyspnea (Silverman score\> 3)
  * severe sepsis
  * life-threatened
ACE2016 in Adult Subjects With Locally Advanced or Metastatic Solid Tumors Expressing Epidermal Growth Factor Receptor (EGFR)

ACE2016 is an off-the-shelf, allogeneic gamma delta T (gdT) cell therapy derived from healthy donors, that is under investigation for the treatment of Locally Advanced or Metastatic Solid Tumors Expressing Epidermal Growth Factor Receptor (EGFR).

The ACE2016-001 study is an open-label, Phase I, first-in-human (FIH) study that aims to evaluate the safety and tolerability, persistency, pharmacodynamics and efficacy of ACE2016 in patients with Locally Advanced or Metastatic Solid Tumors Expressing Epidermal Growth Factor Receptor (EGFR).


Eligibility Criteria:
Inclusion Criteria:

* Locally advanced unresectable or metastatic solid tumors that have failed at least two lines of therapy (one of which must be targeted therapy)
* At least one measurable lesion as defined by RECIST v1.1 criteria
* Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1
* Adequate hematologic and renal, hepatic and cardiac function
* Oxygen saturation via pulse oximeter ≥92% at rest on room air

Exclusion Criteria:

* Prior treatment with a genetically modified cell therapy product targeting EGFR
* History of allogeneic transplantation
* Subjects with active CNS metastases
* History or presence of clinically relevant Central Nervous System (CNS) disorder (e.g. epilepsy)
* Clinically significant active infection
* Human Immunodeficiency Virus (HIV) infection, active hepatitis B infection, or hepatitis C infection.
* History of malignancies with the exception of certain treated malignancies with no evidence of disease.
* Primary immunodeficiency disorder
* Pregnant or lactating female
* Any medical, psychological, familial, or sociological conditions that, in the opinion of the Investigator or Sponsor Medical Monitor, would impair the ability of the subject to receive study treatment or comply with study requirements, including understanding and rendering of informed consent
Quality of Sexual Function in BRCA Mutated Women

Quality of sexual function in BRCA mutated women is investigated using SFQ28 questionnaire.

We aim to investigate and compare the impact of diagnosis of BRCA, the impact of risk reducing surgery (RRS) and oncological diagnosis.

Eligibility Criteria:
Inclusion Criteria:

* TBC

Exclusion Criteria:

* TBC
Motivational Therapy for Substance Users With Depression

The primary objective of the study is to test the incremental efficacy and outcomes of an aftercare program of Cognitive Behavioral Therapy combined with motivational therapy (CBT-MT) relative to treatment as usual (TAU) in improving depression, substance use, and healthcare outcomes in a population with drug dependence and comorbid major depressive disorder (MDD). The investigators expect that among drug-dependent patients with comorbid MDD, CBT-MT will yield better clinical outcomes relative to TAU in reducing depressive symptoms and substance use and improving healthcare outcomes during treatment.

Secondary Objectives:

1. Test efficacy and outcomes of CBT-MT and TAU
2. Evaluate the differential effect of CBT-MT versus TAU on HIV-risk behavior of participants,
3. To evaluate the impact of cognitive functioning on treatment retention and outcomes, and
4. To explore additional psychosocial, demographic, and diagnostic factors (e.g., age, gender, education level, motivation for change, social support) that may be associated with treatment outcome and retention in this high need population.

This study will include approximately 80 participants randomized to either 12 weeks of motivational therapy or treatment as usual. Both treatments will meet once weekly for 60 minutes. Participants will be recruited from the Adult Partial Hospitalization program at University of California, Los Angeles. During the active treatment phase, participants will attend clinic weekly for collection of data and urine specimens.

1. For those randomly assigned to CBT-MT, they will meet with a therapist in a group format for 60-minute sessions once weekly.

   a. Each CBT-MT session will begin with 20 minutes of motivational therapy (MT), followed by 40 minutes of CBT content.
2. Those randomly assigned to TAU, they will attend a weekly 60-minute Dual Recovery Anonymous self-help group.

Eligibility Criteria:
Inclusion Criteria:

1. Age 18 or over
2. DSM-IV diagnosis of Substance Dependence (cannabis, stimulant, opioid, or prescription drug) or DSM-IV diagnosis of Alcohol Dependence and abuse of cannabis, stimulant, opioid, or prescription drug
3. Diagnostic and Statistical Manual -IV diagnosis of lifetime Major Depressive Disorder (MDD)
4. BDI-II score of 13 or greater

Exclusion Criteria:

1. Presence of life threatening or unstable medical illness
2. Lack of proficiency in English
3. Current homelessness (unless residing in a recovery home for which contact information can be provided)
4. Psychiatric symptoms warranting safety concerns or inpatient treatment, including acute suicide risk
5. Present diagnosis of Bipolar Disorder, Schizophrenia, or Schizoaffective Disorder.