cyrilzakka/clinical-trials huggingface dataset:
Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep

The primary objective for this study is to evaluate the long-term safety and tolerability of NBI-827104 in pediatric participants with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS).

This study will enroll participants who completed treatment in the Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel-group Study NBI-827104-CSWS2010 (NCT04625101). Participants who did not participate in Study NBI-827104-CSWS2010 may also be eligible for enrollment.

Eligibility Criteria:
Key Inclusion Criteria:

For participants who enroll directly following the completion of the Study NBI-827104-CSWS2010:

* Completed 12 weeks of treatment in Study NBI-827104-CSWS2010.

For Participants Who Do Not Enroll Directly from or Did Not Participate in Study NBI-827104-CSWS2010:

* Have diagnosis of EECSWS confirmed by the Diagnosis Confirmation Panel (DCP).

Key Exclusion Criteria:

For participants who enroll directly following the completion of the Study NBI-827104-CSWS2010:

* Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.

For Participants Who Do Not Enroll Directly from or Did Not Participate in Study NBI-827104-CSWS2010:

* Body weight \<15 kg at Day 1.
* Clinically relevant findings related to cardiovascular or laboratory parameters at screening as determined by the investigator.
* Presence of relevant neurological disorders other than EECSWS and its underlying conditions as judged by the investigator. Symptomatic conditions underlying EECSWS (for example, neonatal strokes) have to be stable for at least 1 year prior to screening.
* Planned surgical intervention related to structural abnormalities of the brain from screening through the Week 6 Visit.
* Used any active investigational drug other than NBI-827104 in the context of a clinical study within 30 days or 5 half-lives (whichever is longer) before screening or plans to use such an investigational drug (other than NBI-827104) during the study.
* Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.
2nd AC+: New Village Model

This study evaluates implementation of the Village Model to support older people living with HIV.

Understanding that older people living with HIV are facing uniques challenges as they age (lack of peer set due to extreme loss during the AIDS epidemic, ongoing HIV and age stigma, high rates of multi-morbidity) this study explores the acceptability and feasibility of implementing the Village Model in Central San Diego.

The Village Model started when several older members of Boston's Beacon Hill realized they were aging and may need more help as they aged to thrive in their own homes. The Village Model involves members of a community paying annual fees to support a village administrator that organizes regular socialization events and helps coordinate needs of individual members using the skill sets of other members within the community. From that first experience a few decades ago, today over 200 Villages exist across the country to enhance the ability of older adults to age in place.

However limitations of this model exists that may threaten the long term implementation and sustainability of the Village Model. Specifically the requirement to pay annual fees for an administrator, the lack of diversity (in age, race, sexuality etc), and difficulty in meeting member's needs in real-time.

This project proposes to pilot the Village Model in Central San Diego and will implement the following adaptions to address limitations. Members will not be required to pay annual fees but instead depend on a well-funded, long-established diverse organization (The San Diego LGBT Center) to serve as the administrator. This Village will allow participation of persons within the community at all ages, race, economic status. Lastly, the project proposes to pilot the acceptability of using a website to display and respond to community needs in real time.

To achieve this, the investigators will 1) perform questionnaires to understand the needs in the community by collecting needs assessments. 2) The investigators will explore the meaning of community and explore barriers and facilitators of Village Model implementation. 3) Lastly the investigators will pilot the Village model using a website.

Eligibility Criteria:
Inclusion Criteria:

* Lives in Central San Diego
* Consents to participate
* Willing to sign Village social contract
* No plans to move out of Central San Diego in the next year
* Speaks English

Exclusion Criteria:

* Severely cognitively impaired as indicated by \< 10 on mini mental state examination
* History of a violent felony or fraud in the past 10 years
* Not willing to consent to participation
Brain Clock and Insulin Resistance

In this observational cohort study the investigators will determine the activity rhythm of the suprachiasmatic nucleus in humans with progressive stages of insulin resistance, using advanced functional brain imaging (7 Tesla functional MRI).

Type 2 diabetes mellitus (T2DM) has an increasing worldwide incidence. Insulin resistance is a key pathophysiological process in the development of hyperglycemia in patients with T2DM. Disruption of circadian synchrony leads to insulin resistance. Animal studies and post-mortem human brain studies suggest that the master brain clock in the hypothalamic suprachiasmatic nucleus (SCN) plays a role in the development of insulin resistance. Up to now, no-one has investigated whether the in vivo activity rhythm of the SCN is affected in patients with insulin resistance. The investigators hypothesize that the master brain clock has an important role in the development of human insulin resistance.

Eligibility Criteria:
Inclusion Criteria:

Group 1: obese people with normal insulin sensitivity

* age 25-65 years
* BMI\>30
* fasting plasma insulin ≤62 pmol/L
* fasting plasma glucose \<5.6 mmol/L
* Homeostatic Model Assessment for Insulin Resistance (HOMA-IR) ≤ 4.5

Group 2: obese people with insulin resistance

* age 25-65 years
* BMI\>30
* fasting plasma insulin \>62 pmol/L
* not fulfilling the American Diabetes Association (ADA) criteria for type 2 DM

Group 3: obese subjects with overt type 2 DM

* age 25-65 years
* BMI\>30
* diagnosis type 2 DM according to ADA criteria

Exclusion Criteria:

* An extreme chronotype (midpoint of sleep on free days (MSFsc) before 2:00 or after 6:00).
* Active psychiatric disorder (including circadian rhythm sleep disorder) as defined in Diagnostic and Statistical Manual of Mental Disorders (DSM) 5
* Disorders of the central nervous system (Early-onset dementia, stroke, epilepsy, Parkinson's disease, brain tumor)
* Severe visual impairment (WHO classification)
* Shift workers
* Crossing \> 2 time zones in the 3 months before the study
* Patients with type 2 DM receiving insulin treatment or glucagon-like peptide (GLP) 1 agonists
* MRI contraindications
A Study of Tecogalan Sodium

To evaluate the safety of different doses and dosing regimens of tecogalan sodium (DS-4152) and to establish the MTD at each of the different dosing schedules.

Patients receive intravenous DS-4152 by infusion twice weekly for 21 days, followed by 2 weeks of rest; courses may repeat. Patients undergo weekly follow-up. A punch biopsy will be obtained from patients with Kaposi's sarcoma.

Eligibility Criteria:
Inclusion Criteria

Patients must have:

* Kaposi's sarcoma plus HIV infection OR metastatic solid tumor.
* Life expectancy of at least 12 weeks.
* NO symptomatic AIDS-defining opportunistic infection within the past 4 weeks.
* Recovered from toxicity of any prior anticancer therapy.

Exclusion Criteria

Co-existing Condition:

Patients with the following symptoms or conditions are excluded:

* Leukemia or lymphoma.
* Current gastrointestinal bleeding by stool guaiac.
* Extensive bone metastases or significant radiographic osteoporosis in patients with solid tumors.
* Active heart disease such as uncontrolled angina, uncompensated congestive heart failure, or dysrhythmias requiring antiarrhythmics.
* Acute intercurrent infection other than genital herpes.
* Symptomatic or known central nervous system involvement (including brain metastases) unless stable and off therapy.

Concurrent Medication:

Excluded:

* Other anticancer therapy.
* Other investigational agents.

Patients with the following prior conditions are excluded:

* History of acute or chronic gastrointestinal bleeding or inflammatory bowel disease.
* History of myocardial infarction within past 6 months.

Prior Medication:

Excluded:

* Anticancer therapy within the past 3 weeks (6 weeks for nitrosourea or mitomycin C).
* Investigational agents within the past 4 weeks.
Scalable Expanded Access with Analysis of Neurofilament and Other Biomarkers for Ibudilast in ALS

The purpose of this Expanded Access Program is to provide MN-166 (ibudilast) to ALS patients who are not eligible for an enrolling ALS clinical trial. This Expanded Access Program will assess if MN-166 can help people with ALS in slowing down the progression of the disease.


Eligibility Criteria:
Inclusion Criteria

* ALS by "Gold Coast" diagnostic criteria
* Age \> 18 years
* Either I) Ineligibility for other interventional ALS clinical research participation due to at least one of the following standard exclusion criteria:

  * Time since onset of weakness due to ALS \> 36 months
  * Vital Capacity less than 50% of predicted capacity for age, height, and sex measured (by Slow Vital Capacity (SVC) or Forced Vital Capacity (FVC))
  * Cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
  * Geographic isolation from nearest actively enrolling research trial location, defined as either \>75 miles or \>120-minute travel time
  * OR II) Completed assigned regimen as participant in COMBAT-ALS clinical trial.

Exclusion Criteria

* Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to treating physician's judgment (e.g., psychiatric, cardiovascular instability, systemic infection, untreated thyroid dysfunction, or clinically significant laboratory abnormality or ECG changes).
* Clinically significant lab abnormalities including, but not limited to: hemoglobin \< 10 g/dL, white blood cells \< 3.0 x 103/mm3, neutrophils, absolute ≤ 1000/mm3, eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), low platelet counts (\< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN), eGFR \< 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels \>10 mIU/L or \<0.01 mIU/L.
* Active drug or alcohol abuse
* Concomitant use of another investigational medical product for treatment of ALS
Live Long Walk Strong (LLWS) Rehabilitation Program: What Features Improve Mobility Skills

This study is being performed to evaluate the efficacy of the Live Long Walk Strong rehabilitation program in Veterans 50 years and older. This study will also examine the features of the program that contribute to improved gait speed.

Due to COVID-19, currently completed a virtual version of the LLWS intervention study.

The investigators are currently recruiting for the original study design in-person.

This study is a 4 year phase II randomized clinical trial study design. This study will provide important information about the mechanisms that contribute to meaningful improvement in mobility, the benefits of a novel mode of physical therapy care, and the duration of treatment effect.

Eligibility Criteria:
Inclusion Criteria:

* Veteran
* Aged 50 and older
* Community dwelling
* Ability to speak and understand English
* Usual gait speed 0.5 m/s- 1.0 m/s Activity modifications and access to telehealth rehabilitation

Exclusion Criteria:

* Presence of a terminal disease
* A major medical problem, unstable chronic condition, or psychiatric disorder that interferes with safe and successful testing and training
* Myocardial infarction or major surgery in previous 3 months
* Planned major surgery
* Baseline short physical performance battery score less than 4
* Use of a walker
* Modified mini mental status examination score less than or equal to 77
* Presence of a significant disease specific impairment such as:

  * peripheral neurologic impairment
  * orthopedic impairment
  * visual impairment
Proton MR Spectroscopy and 18F-Fluorocholine PET for Breast Cancer Diagnosis

The application of FCH PET in breast cancer diagnosis has not been reported.

We hypothesize that FCH reveals choline metabolic profiles of breast cancers, and shows the similar pathophysiological mechanism to choline on proton MRS, and our study goals are:

1. To investigate and compare the diagnostic performance of proton MRS and FCH PET for localized findings on mammography and breast ultrasound.
2. To investigate whether FCH PET findings are correlated with choline signals on proton MRS.
3. To evaluate if choline, water and lipid signals on proton MRS, FCH PET are associated with factors related to clinical outcome and prognosis- that is, molecular markers, tumor staging, histologic grade of breast cancers.
4. For localized advanced breast cancer, to investigate the treatment response to NAC using proton MRS and FCH PET, and to evaluate which modality is more sensitive.
5. To investigate the usefulness of FCH PET for whole body staging for breast cancer patients.

In recent years, the application of 18F-FDG PET (positron emission tomography) and MRI for breast lesion diagnosis was emerging. Imbriaco et al compared the diagnostic performance of 18F-FDG PET and MRI for suspicious breast lesions, with PET showing a sensitivity of 80% and specificity 100%; and MRI revealed a sensitivity of 95% and specificity of 98%. Lim et al studied 18F-FDG PET-CT with MRI to monitor treatment response for breast cancer patients receiving NAC, with a sensitivity 70% and specificity 72%, and can detect the treatment response earlier than conventional imaging.

However, 18F-FDG is not tumor specific, and searching for an alternative tracer agent as a tumor biomarker is necessary. 18F-Fluorocholine (FCH), a choline analog, was used for diagnosis of prostate cancer, hepatocellular carcinoma (HCC), bronchioloalveolar carcinoma, brain tumor, since malignant tumors show higher choline metabolites than benign lesions; it was reported that FCH PET showed higher sensitivity (88-94%) than 18F-FDG PET (59-68%) in detecting HCC. FCH PET was used for staging for prostate cancer, with FCH PET-CT can provide sufficient information for management with an accuracy of 88%.

Eligibility Criteria:
Inclusion Criteria:

* Women aged 20-75 years old.
* with localized findings on mammography or breast ultrasound in recent 3 months and who will receive breast biopsy or excision for the findings; or with recently diagnosed LABC who will receive NAC.

Exclusion Criteria:

* unable to cooperate with the examinations
* pregnant or planning to be pregnant
* estimated GFR (eGFR) \< 60 ml/min/1.73m2 or with recent acute renal failure, past history of renal dialysis.
* Past history of claustrophobia
* Past history of anaphylactoid reactions to MRI contrast agents or PET tracer agents.
* with cardiac pacemaker, aneurysmal clip, mechanical valve replacement, recently applied coronary artery stent (\<2 months).
* Past history of breast cancer or other malignancy (treated within 5 years).
* lactating.
Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Open Label

The goal of this study is to target the neurobiology of restricted and repetitive behaviors in children with autism spectrum disorder using N-acetylcysteine (NAC), a well-tolerated nutritional supplement that has shown promise for reducing symptom severity in recent small-scale trials. The findings from this research will shed light on the mechanisms of action underlying the clinical benefits of NAC and the effects of NAC on altering restricted and repetitive behavior symptom severity in children with autism spectrum disorder.


Eligibility Criteria:
Inclusion Criteria:

* participation in an associated single-dose (IRB-54931, NCT04278898) or randomized, controlled trial of NAC (IRB-63262, NCT05664789).
* stable medication regimens (≥ 30 days) with no anticipated changes during the trial.

Exclusion criteria:

• the presence of significant medical problems that reduce medical stability.
Human Intravenous Interferon Beta-Ia Safety and Preliminary Efficacy in Hospitalized Subjects With COVID-19

This double-blinded, randomized study is being conducted to see if the investigational new drug called FP-1201-lyo - intravenous Interferon beta-1a, hereafter IV IFN beta-1a, can help patients recover more quickly from COVID-19 and prevent worsening of the condition. To understand if IV IFN beta-1a can help treat patients with COVID-19, this study drug will be compared to dexamethasone.

Study subjects will be treated daily with IV IFN beta-1a 10 μg or IV dexamethasone for 6 consecutive days while hospitalized and will undergo daily assessments while in hospital for a maximum of 28 days. Study specific assessments will be collected at pre-dose Day 1 through Day 28 (PD and PIM assessments), in addition, clinical routine assessments will be utilized for safety and efficacy assessments.


Eligibility Criteria:
Inclusion Criteria:

1. Age ≥18 years
2. Positive SARS-CoV-2 test by PCR (polymerase chain reaction) or other diagnostic method within the past 7 days
3. Admission to hospital with respiratory symptoms of COVID-19 requiring hospital care and oxygen supplementation (≤ 8L/min)
4. Respiratory symptom onset no more than 7 days prior to hospital arrival
5. Informed consent from the subject or the subject's personal legal representative or a professional legal representative must be available

Exclusion Criteria:

1. Unable to screen, randomize and administer study drug within 48 hours from arrival to hospital
2. Systemic corticosteroid, baricitinib or tofacitinib (or other JAK-STAT signalling pathway inhibitors) therapy within 7 days prior to arrival to hospital or planned for the next days
3. Known hypersensitivity or contraindication to natural or recombinant IFN-beta-1a or its excipients, or to dexamethasone or its excipients
4. Currently receiving IFN-beta-1a therapy
5. Home assisted ventilation (via tracheotomy or non-invasive) except for Continuous Positive Airway Pressure (CPAP) / Bilevel Positive Airway Pressure (BIPAP) used only for sleep-disordered breathing
6. Participation in another concurrent interventional pharmacotherapy trial during the study period
7. Decision to withhold life-sustaining treatment; patient not committed to full support (except DNR after cardiac arrest only)
8. Woman known to be pregnant, lactating or with a positive pregnancy test (urine or serum test)
9. Subject is not expected to survive for 24 hours
10. Subject has liver failure (Child-Pugh grade C)
11. Any clinical condition that in the opinion of the attending clinician or Investigator would present a risk for the subject to participate in the study
Retrobulbar Block Versus Ketamine Infusion for Post-enucleation Analgesia

This study compares the efficacy and safety of two techniques, retrobulbar block versus intra-operative ketamine infusion, for control of post-operative pain occurring in patients undergoing ocular enucleation or evisceration performed under general anesthesia.


Eligibility Criteria:
Inclusion Criteria:

* Ocular enucleation candidate patients
* Ocular evisceration candidate patients

Exclusion Criteria:

* Any condition contra-indicating general or local anesthesia
* Allergy to any of the drugs used during the procedure
Diet and Hereditary Haemorrhagic Telangiectasia

Hereditary Haemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people. The purpose of this study is to provide data regarding the diet and nosebleed frequency using a questionnaire.

This will be filled in by people with HHT.

The questionnaire has been designed in paper format.

Hereditary Haemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people, usually causing nosebleeds, skin blood spots, and/or anaemia as a result of bleeding from the nose or gut. The majority of people with HHT also have abnormal blood vessels (arteriovenous malformations) in internal organs such as the lungs, liver and brain. Management of this multisystem disorder is highly challenging.

The Lead Applicant has spent 20 years working on this rare disease, and identified multiple areas where more evidence is required to assist clinicians and patients with this lifelong condition. A particular issue is whether the diet influences HHT or its complications in any way.

In this study, people will fill in two questionnaires, one giving details of their diet, and another details of their nosebleeds. They will also be asked to consider participating in an accessory study arm which includes weighing food for one week and providing a food diary, in addition to having a single set of blood test.

Eligibility Criteria:
Inclusion Criteria:

* A diagnosis of hereditary Haemorrhagic Telangiectasia (HHT)

Exclusion Criteria:

* Unable to provide informed consent
* Presence of another major organ disorder that may affect nutritional status, such as inflammatory bowel disease, or celiac disease.
Incontinence and MRI Results in Children with Cerebral Palsy

More than 50 % of children and adults with cerebral palsy experience lower urinary tract symptoms (LUTS). This can negatively influence quality of life of the child and the total social environment.

Previous research demonstrated that the brain lesion, characteristic for cerebral palsy (CP), is a prognostic factor for the functional abilities of a child with CP. Previous research within our department demonstrated functional impairment as a risk factor for incontinence. This demonstrates an indirect connection between the brain lesion and incontinence of a child with CP. However, this connection isn't alway correct as some children with high functionality also demonstrate incontinence.

As bladder control is also regulated by higher brain centers, the present study wants to investigate a direct connection between the brain lesion of the child, visible by means of MRI, and the incontinence status of the child. .

The current study is a cross-sectional observational study in which a correlation is investigated between known anatomical MRI results and the continence status of a child with CP.

Although neuroimaging isn't seen as a requirement for the definition and diagnosis of CP2, MRI imaging is often part of diagnostic evaluation. The Surveillance of Cerebral Palsy in Europe (SCPE) working group composed the MRI classification system or MRICS classifying neuroimaging results in maldevelopments, predominant white matter injury, predominant grey matter injury, miscellaneous and normal. These results will be correlated with results of the validated Vancouver symptoms score for dysfunctional elimination syndrome.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of cerebral palsy
* MRI investigation in history
* Dutch or French speaking child and parent

Exclusion Criteria:

* No MRI investigation in history
* Child or parent not speaking Dutch or French