cyrilzakka/clinical-trials huggingface dataset:
Effect of Lamotrigine on Cognition in NF1

The purpose of this study is to determine whether lamotrigine can improve cognitive and neurophysiological deficits in adolescents with Neurofibromatosis type 1.

Cognitive deficits in the autosomal dominant disorder Neurofibromatosis type 1 (NF1) typically consist of a lower than average IQ, impaired visual-spatial learning, attention problems and impaired executive functioning. These deficits have a substantial influence on the daily life of pediatric and adolescent individuals with NF1. One of the key underlying mechanisms of these deficits is an increased gamma-aminobutyric acid (GABA)-ergic inhibition and a subsequent decrease in synaptic plasticity. The ENCORE laboratory has recently shown that loss of the NF1-gene is associated with attenuated function of the hyperpolarization-activated cyclic nucleotide-gated channel 1 (HCN1). These channels, enriched in membranes of inhibitory interneurons, play an important role in the pathophysiology underlying the cognitive deficits in NF1. Lamotrigine, an HCN-agonist, restored function of HCN1, together with the electrophysiological and visual-spatial learning deficits in Nf1-mice. Thus, lamotrigine is a novel candidate drug for treating cognitive deficits associated with NF1.

Eligibility Criteria:
Inclusion Criteria:

* NF1 patients with a genetically confirmed diagnosis
* Age 12-17.5 years at inclusion
* Oral and written informed consent by parents and assent from participants

Exclusion Criteria:

* Segmental NF1
* Severe hearing problems or deafness
* Severe visual problems or blindness
* Use of the following medication, as of interaction with lamotrigine: phenytoin, carbamazepine, phenobarbital, primidon, rifampicin, atazanavir/ritonavir, lopinavir/ritonavir, oxcarbazepine, topiramate, oral contraceptive pill including stop-week (estrogen and progesterone) and valproic acid during 3 months before inclusion.
* Use of psycho-active medication other than methylphenidate
* Previous allergic reactions to anti-epileptic drugs
* Epilepsy or epilepsy in the past
* Suicidal thoughts or behaviour
* Renal insufficiency
* Liver insufficiency
* Pregnancy
* Brain tumour or other brain pathology potentially influencing the outcome measures
Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

This study is a Phase 3, randomized, double-blind, placebo-controlled study of DTX301 in patients with late-onset OTC deficiency 12 years of age and older.

Participants will be randomized 1:1 to DTX301 or placebo group and followed closely for 64 weeks. At week 64 eligible patients will crossover and receive DTX301 if they had previously received placebo or placebo if they had previously received DTX301.

The planned study duration is up to 324 weeks. Upon completion of this study or early withdrawal, all participants who received DTX301 are invited to enroll in the Disease Monitoring Program (DMP) for follow-up for up to an additional 5 years.

Eligibility Criteria:
Key Inclusion Criteria:

* Confirmed clinical diagnosis of late-onset OTC deficiency with historical documentation by enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic acid), or molecular testing (ie, OTC analysis)
* Free from symptomatic hyperammonemia and has not required emergent active intervention for hyperammonemia within 4 weeks before screening/baseline
* If on ongoing daily ammonia scavenger therapy, must be at stable daily dose(s) for ≥ 4 weeks prior to screening
* If on a protein-restricted diet, must be on a stable total daily protein intake that does not vary more than 20% for ≥ 4 weeks prior to screening
* From the time written informed consent through Week 128, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not father a child or donate sperm

Key Exclusion Criteria:

* Significant hepatic inflammation or cirrhosis
* Estimated glomerular filtration rate \< 60 mL/min/1.73 m2 at screening by the 2021 CKD-EPI creatinine-based formula (Inker et al., 2021) for patients ≥ 18 years of age or the Schwartz bedside formula (Schwartz and Work, 2009) for patients \< 18 years of age
* Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, documented by current use of antiviral therapy for HBV or HCV or by hepatitis B surface antigen (HBsAg) or HCV RNA positivity
* Active infection (viral or bacterial)
* Detectable pre-existing antibodies to the AAV8 capsid
* Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
* Participation (current or previous) in another gene transfer study

Note: Additional inclusion/exclusion criteria may apply, per protocol
ALzheimer and MUsic THerapy: Effects of Music Lessons on Brain Plasticity, Mood, and Quality of Life in Alzheimer Patients

Music has powerful effects on memory in patients with neurodegenerative diseases. However, although there is anecdotal evidence for beneficial effects of active music interventions in patients with Alzheimer's disease (AD), there is lack of high-quality research investigating this issue, and the cognitive, emotional, and social factors that contribute to potentially beneficial effects of music making in AD patients are largely unknown. In a randomised controlled intervention trial, a cohort of AD patients will undergo twelve months of music lessons specifically tailored for AD patients. Structural and functional magnetic resonance imaging (MRI) will be used to determine changes in brain age (as compared to two control groups), and voxel-based morphometry will be computed to determine contributions of different factors of the music intervention (cognitive, emotional, and social) to plastic changes of brain morphology, and a potential deceleration of brain atrophy. In addition, quality of life of patients. In cooperation with the Bergen municipality, and a strong network of national and international partners, the neurocognitive music therapy will be implemented, involving training of music therapists, and communication of results to patients, patient groups, and therapists.


Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of Alzheimer Disease
* Living independently at home (not in a care home or similar aged care facility)
* Able to complete questionnaires in Norwegian
* Able to undergo MRI scans
* Able to attend interventions and assessments in the area of Bergen, Norway
* Has given informed consent

Exclusion Criteria:

* Hearing impairment that cannot be mended by hearing aids
* Conditions that hinder MRI scans, such as claustrophobia or metal objects in body (e.g. pacemakers)
A Study to Evaluate the Long-Term Safety and Efficacy of Bimekizumab in Subjects With Psoriatic Arthritis

This is a study to assess the long-term safety and tolerability of bimekizumab in subjects with psoriatic arthritis


Eligibility Criteria:
Inclusion Criteria:

* In the opinion of the Investigator, the subject is expected to benefit from participation in an Open Label Extension (OLE) study
* Subject completed PA0008 without meeting any withdrawal criteria
* Female subjects must be postmenopausal, permanently sterilized or, if of childbearing potential, must be willing to use a highly effective method of contraception
* Male subjects with a partner of childbearing potential must be willing to use a condom when sexually active

Exclusion Criteria:

* Female subjects who plan to become pregnant during the study or within 20 weeks following the last dose of IMP. Male subjects who are planning a partner pregnancy during the study or within 20 weeks following the last dose
* Subjects with any current sign or symptom that may indicate a medically significant active infection (except for the common cold) or has had an infection requiring systemic antibiotics within 2 weeks of study entry
* Subjects who meet any withdrawal criteria in PA0008. For any subject with an ongoing Serious Adverse Event, or a history of serious infections (including hospitalizations) in the lead-in study, the Medical Monitor must be consulted prior to the subject's entry into PA0009
Establishment of a Early Risk Model of ECMO in Children With ARDS

The key problem in the treatment of ARDS is refractory hypoxemia. Extracorporeal Membrane Oxygenation (ECMO) is an extracorporeal oxygenation of venous blood to eliminate carbon dioxide and return to the body. It has been an important part of the rescue treatment for ARDS. This study intends to explore the timing of ECMO. The main research hypothesis is that the appropriate timing of ECMO treatment can improve the weaning success rate and survival rate of children with severe ARDS; it is expected to provide a basis for determining the best timing of ECMO treatment

Severe acute respiratory distress syndrome (ARDS), especially those caused by severe viral pneumonia, is still an important factor in the death of children, with a case fatality rate of 55%. The treatment includes lung protective ventilation and advanced ventilation support, but the effect is still not ideal for severe patients. ECMO can effectively support the respiratory system and provide good oxygen exchange. However, the survival rate of ECMO treatment in children with ARDS has a large bias.One of the key factors is the uneven timing of ECMO activation, which significantly affects the prognosis.This study intends to design a multi-center, prospective, non-randomized controlled trial , through retrospective research to find relevant factors affecting the prognosis of ECMO treatment, and to screen key indicators related to the timing of intervention; through a prospective cohort study to screen Good indicators and cut-off values suitable for starting ECMO , constructing and verifying comprehensive prediction models.

Eligibility Criteria:
Inclusion Criteria:

* Acute onset; within 7 days of clinical insult
* Chest imaging (radiograph or computed tomography) findings of new infiltrates (unilateral or bilateral) consistent with acute parenchymal disease
* Edema not fully explained by fluid overload or cardiac failure
* May present as new acute lung disease in setting of chronic lung disease and/or heart disease
* OI≥16
* Viral etiology is clear

Exclusion Criteria:

* Perinatal lung disease
* Large intracranial bleed with mass effect or need for neurosurgical intervention
* Hypoxic cardiac arrest without adequate CPR
* Irreversible underlying cardiac or lung pathology (and not a transplant candidate)
* Pulmonary hypertension and chronic lung disease
* Chronic multiorgan dysfunction
* Incurable malignancy
* Allogenic bone marrow recipients with pulmonary infiltrates
* Hepatic or renal failure
* Pertussis infection in infants
* Fungal pneumonia
* Immunodeficiency
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.


Eligibility Criteria:
Inclusion Criteria:

* Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003

Exclusion Criteria:

* Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
* Future or current participation in another investigational device or drug study, scheduled to occur during this study
* Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
Combined Breast Cancer Risk Study

A prospective, non-interventional study in women 18 to 84 years of age. Subjects will provide a sample for genetic testing and information about their medical and family history. The results of the genetic test will be combined with clinical data to validate a method of predicting breast cancer risk.

This is a prospective, non- interventional study. Women presenting at imaging centers for routine breast cancer screening or breast cancer diagnostic assessment and who provide written informed consent will undergo genetic testing. Subjects will also provide information about their personal medical and cancer history and family cancer history. The results of the genetic test will be combined with the subject's clinical information, family history, and a risk assessment model to validate a new method of predicting breast cancer.

Eligibility Criteria:
Inclusion Criteria:

Women without breast cancer:

* 18 to 84 years of age
* Western/Northern European, Central/Eastern European, or Ashkenazi ancestry
* No history of invasive breast cancer

Women with a history of breast cancer:

* 18 to 84 years of age
* Western/Northern European, Central/Eastern European, or Ashkenazi ancestry
* Pathologically confirmed invasive breast cancer diagnosed within the past 12 months

Exclusion Criteria:

* Unwilling to provide written informed consent
* Women with history of ductal carcinoma in situ (DCIS).
* Patient has had a prior breast biopsy, exclusive of a breast biopsy diagnostic of breast cancer, that showed either hyperplasia, atypical hyperplasia, lobular carcinoma in situ (LCIS), or the specific histologic result is unknown to the patient
Clinical Value of Left Colic Artery in Laparoscopic Radical Rectectomy

Colorectal cancer is one of the most common tumors in Asia. According to the recent research, surgical procedure could provide more treatment benefit in rectal cancer. Therefore, it was consider that important to standardized and improved the surgical procedure for rectal cancer. With the development of anatomical technique, minimally surgery with laparoscopy had become the trend for surgical treatment. There were several studies has been done to evaluate the safety and feasibility of laparoscopic surgery. In order to achieve better surgical outcome and reduce operative complications, the investigators design stratified randomization, double blinded, muti - center clinical trail to investigate the value of left colic artery in laparoscopic radical rectectomy.


Eligibility Criteria:
Inclusion Criteria:

* Aged more then 18 years old;
* Diagnosed as rectal cancer with colonoscopic biopsy;
* Without metastasis;
* No Invasion of surrounding tissues;
* Limited operation;
* Underwent laparoscopic radical proctectomy(L-Dixon);
* BMI 18\~30kg/m2;
* Without multiple primary tumors;
* Sign on the Medical informed Consent.

Exclusion Criteria:

* Simultaneous or simultaneous multiple primary colorectal cancer;
* Preoperative imaging examination results show: (1) Tumor involves the surrounding organs and combined organ resection need to be done; (2)distant metastasis; (3)unable to perform R0 resection;
* History of any other malignant tumor in recent 5 years;
* Patients need emergency operation;
* Not suitable for laparoscopic surgery;
* Women during Pregnancy or breast feeding period;
* Informed consent refusal
Evaluation of Three SS-OCT and One PCI Optical Biometers in Eyes With Dense Cataract

To describe and compare the acquisition rate between different biometers in eyes with advanced cataracts

To describe and compare the acquisition rate between different biometers in eyes with advanced cataracts

Eligibility Criteria:
Inclusion Criteria:

* LOCS III cataract classification with a grade of ≥3 for nuclear, cortical or posterior subcapsular.
* DLI index, from the ITrace device, should be ≤ 5.

Exclusion Criteria:

* Previous continuous contact lens wear
* Ocular trauma
* Intraocular lens surgery
* Poor fixation
* Corneal disease (i.e. keratoconus)
Lipiflow Treatment Alone vs. Lipiflow + Dextenza Treatment for Dry Eye Disease

To evaluate the benefit of treatment with a physician administered intracanalicular dexamethasone insert in evaporative dry eye disease (DED) patients with meibomian gland disfunction (MGD) and underlying inflammation undergoing LipiFlow Thermal Pulsation.

This prospective, open-label, single-center, non-randomized, investigator-sponsored clinical study seeks to investigate the benefit of managing patients with DED secondary to MGD with a sustained release intracanalicular dexamethasone (0.4 mg) insert in addition to LipiFlow Thermal Pulsation compared to LipiFlow Thermal Pulsation alone. In addition, this study will evaluate the ease of Dextenza insertion and the patient preference for therapy.

After screening a given patient for inclusion and exclusion criteria, and gaining informed consent, each eye will undergo LipiFlow Thermal Pulsation on the same day. The most symptomatic eye will be selected to receive DEXTENZA® insertion on the day of the procedure (study eye), while the other eye will be assigned to a receive a sham punctum dilation (control eye). If there is no obvious symptomatic difference, the right eye will receive the intracanalicular insert. Per patient, the study period will last for approximately 12 weeks after the LipiFlow procedure, consisting of one screening visit, one treatment v4isit and 3 post-procedure follow-up visits (week 1, week 4 and week 12). At week 1, week 4 and week 12, primary and secondary endpoints will be assessed alongside standard-of-care procedures. Adjusting for enrollment period, the study will last a total of approximately 4 months.

Eligibility Criteria:
Inclusion Criteria:

A patient's study eye must meet the following criteria to be eligible for inclusion in the study:

* 18 years of age or older
* Evaporative DED with MGD and clinically significant inflammation
* Willing and able to comply with clinic visits and study related procedures
* Willing and able to sign the informed consent form

Exclusion Criteria:

A patient who meets any of the following criteria will be excluded from the study:

* Patients under the age of 18.
* Pregnancy (must be ruled out in women of child-bearing age with pregnancy test)
* Active infectious systemic disease
* Active infectious ocular or extraocular disease
* Altered nasolacrimal flow of either acquired, induced, or congenital origin
* Hypersensitivity to dexamethasone
* Patient being treated with either topical, oral, or intravenous steroids
* Patients with severe disease that warrants critical attention, deemed unsafe for the study by the investigator
Managing Diabetes in Pregnancy Using Cell Phone/Internet Technology

To compare compliance and satisfaction between a traditional method of blood glucose reporting using voicemail (control) and a novel method using cell phone /internet (Confidant) technology in the management of diabetic pregnant women.

Control of blood glucose levels is beneficial during pregnancy, however, the ability to effectively monitor patient's blood glucose levels is challenging and time consuming for patients and care providers. Patients must keep track of their own glucose levels and take the initiative to check blood glucose multiple times per day. Traditionally, patients record their glucose levels in a logbook, which they bring with them during an office visit or use to report their glucose levels over the phone. Non compliance and factitious reporting are not uncommon. Obstetricians and or diabetes program staff spend hours communicating with patients to obtain and record glucose values therefore alternatives to phone calls are needed.

Investigators performed a prospective randomized cross over study comparing a conventional voicemail (control) system for home glucose reporting with a cell phone/internet reporting system (Confidant). The primary outcome measure was compliance with home blood glucose fingerstick reporting. Compliance was determined by the number of home glucose fingersticks reported / expected (%). The secondary outcome was patient satisfaction. Subjects were participants in the Kapiolani Medical Center for Women and Children's diabetes in pregnancy program known as "A Sweeter Choice". IRB approval was obtained before starting the study and all subjects received informed consent. The study was funded by Hawai'i Medical Service Association, a medical insurance company in Hawaii. The Confidant equipment was supplied by Hookele Personal Health Planners, LLC. None of the Ho'okele or Hawaii Medical Service Association staff were involved in data collection or analysis. Study subjects were randomized to Confidant or the control system at the entry into the diabetes program during consultation with the Maternal Fetal Medicine physician. After randomization, subjects performed the assigned monitoring system for 3 weeks. After three weeks, they switched to the other monitoring system. A satisfaction survey was completed after three weeks on the second system was completed.

Eligibility Criteria:
Inclusion Criteria:

-Pregnant women \>=18 years with gestational diabetes or pregestational Type 2 diabetes referred to the diabetes in pregnancy management program before 30 weeks 1 day gestation.

Exclusion Criteria:

* Age less than 18 year
* Pregnancy \>=30 weeks 1 day
* Type I pregestational diabetes
* Inability to speak English
Chronic Obstructive Pulmonary Disease (COPD) and Domestic Endotoxin (CODE)

This project aims to characterize the independent effect of domestic endotoxin exposure on health status, as well as pulmonary and systemic inflammation, in former smokers with COPD. Positive findings from this study would be clinically relevant, as they would provide evidence to support aggressive reduction of ongoing endotoxin exposure in patients with COPD. The investigators also hope to make a methodological advance in the field of endotoxin exposure assessment by elucidating whether settled dust and/or airborne endotoxin measurements are the more relevant exposure of interest in epidemiological studies of respiratory disease. To fulfill the specific aims, the investigators will conduct a longitudinal study, including 75 former smokers with COPD. All subjects will have indoor air monitoring, in-home settled dust collections, home inspections as well as assessments of health status, quality of life (QOL), lung function and pulmonary and systemic inflammation.


Eligibility Criteria:
Inclusion Criteria:

* Patient reported physician diagnosis of COPD/Chronic Bronchitis/Emphysema
* Cigarette Smoking \> 10 pack-years
* Age \> 40 years
* Has not smoked \> 1 year
* Post BD FEV1/FVC ≤ 70%
* Post BD FEV1 (% predicted) 30-80%
* Ability and willingness to provide consent

Exclusion Criteria:

* Non- English speaking
* Planning to move or live away from the home during the study period
* Co-morbid illnesses such as:

  * Current physician diagnosis of Asthma
  * Other pulmonary diseases
  * Systemic disease requiring treatment with immunosuppressive agents
  * Known alpha-1 antitrypsin deficiency
  * Occupational exposure with high endotoxin exposure
* Within the last 6 months Use of oral corticosteroids