cyrilzakka/clinical-trials huggingface dataset:
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only.

Funding Source - FDA OOPD


Eligibility Criteria:
Simplified Inclusion Criteria:

* Clinical diagnosis of Stargardt disease (STGD1)
* Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
* Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
* Healthy as judged by investigator
* Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
* Has been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
* Female of childbearing potential has signed the attestation on contraception requirements

Simplified Exclusion Criteria:

* Is lactating or pregnant
* Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
* Has abnormal laboratory result(s) at screening
* Has an ocular disorder that may confound ocular assessments
* Has a history of ocular intervention within 90 days of screening
Analysis of Immune Response In Bacterial Infection of Obese Subject

Improve knowledge of the immune response to sepsis in obese patients. Define immunological and genetic prognostic factors of severe infections which can motivate a change in the therapeutic attitude


Eligibility Criteria:
Inclusion Criteria:

* between18 and 80 years
* obesity: BMI \> 30
* presumed bacterial infection:

  * pneumonitis (documented or probably)
  * pyelonephritis / acute prostatitis
  * soft tissue / skin infection (except for bedsore)
  * cholecystitis / angiocholitis
  * meningitis
  * clostridium colitis
  * surgical site infection
* signed consent

Exclusion Criteria:

* patients with viral, parasitic or mycotic documented infection
* patients with bacterial infection with treatment\> 4 weeks (ex. endocarditis; osteo- articular infections)
* patients with prior antibiotic treatment (\< 14 days)
* intensive care unit patients
* patients with a history of cancer or malignant hematological disease within the 5 previous years that is currently clinically significant (non metastatic prostatic and basocellular malignancy are excluded)
* patients with systemic disease
* Known diagnosis of human immunodeficiency infection (DICV, HIV...)
* immunosuppression treatment during last month including corticotherapy
* pregnancy
* 3 months follow up not possible
* non signed consent
Great Saphenous Vein Anatomy at the Proximal Medial Thigh as a Potential Site for Rescue Venous Access

The purpose of this study is to assess the anatomy of the great saphenous vein at the level of the medial thigh in the general population in an effort to categorize this as a reliable target vessel for rescue peripheral IV access.


Eligibility Criteria:
Inclusion Criteria:

- Pediatric and adult Emergency Department patients and employees of all ages from all areas of the emergency department or additional training sites staffed by emergency medicine residents.

Exclusion Criteria:

* Critically ill patients as determined by the Emergency Medicine consultant of record for the patient.
* Legs not accessible due to dressing, wounds, or other factors.
* Overlying cellulitis/soft tissue infection or injury.
* Patients that are prison inmates and patients/patient's surrogates who are unable to give verbal consent/assent.
Developing a Culturally Adapted Intervention for Latino Youth Transitioning to Adulthood

The purpose of this study is to evaluate the acceptability, feasibility (e.g., satisfaction, completion rate, barriers to recruitment, treatment fidelity) of the culturally refined Launching! to Adulthood (¡Iniciando! la Adultez) program, to test for a preliminary signal of effect between baseline and post-treatment for the Launching! to Adulthood (¡Iniciando! la Adultez) program and to identify preliminary neural mechanisms of action, including biomarkers of brain structure and connectivity, in terms of treatment response for 15 Latino young adults participating in the ¡Iniciando! therapy program.

The Launching! to Adulthood (¡Iniciando! la Adultez) program uses a culturally sensitive therapy program that was translated into Spanish to help Latino young adults with Autism Spectrum Disorder (ASD) who are transitioning to adulthood (18-25 years old) and their parents. The program aims to make the transition to adulthood easier for young adults and their families, and it includes specific cultural values of Latino young adults and their family members. The ¡Iniciando! la adultez program helps autistic young adults manage mental health needs like depression and anxiety and also addresses several areas impacting transition, including mental health barriers, executive functioning, and motivation. The ¡Iniciando! program also helps parents manage their own needs and addresses several areas, including psychoeducation and mental health.

Eligibility Criteria:
Inclusion Criteria:

* confirmed case of ASD
* a score of \>14 on the Social Communication Questionnaire-Lifetime (SCQ-L110) completed with the mother or father
* meet DSM-5 criteria for ASD based on a Diagnostic and Statistical Manual of Mental Disorders (DSM)-5 ASD symptom checklist
* previous diagnosis of ASD from a licensed mental health or medical professional
* speak English
* have a parent willing to participate (treatment group only)
* score \>85 on verbal intelligence quotient (IQ) on the Kaufman Brief Intelligence Test (KBIT-2)

Exclusion Criteria:

* history of a psychotic disorder or current psychotic symptoms
* suicidal ideation with intent or plan
* current alcohol or other substance use disorder rated severe
* concurrent enrollment in another clinical trial for autism spectrum disorder
* expression of unwillingness to complete study procedures
* For fMRI, unable to undergo fMRI scanning due to metallic devices or objects (cardiac pacemaker or neurostimulator, some artificial joints, metal pins, surgical clips, or other implanted metal parts) or claustrophobic to the scanner.
Correlation Between Dental Inclination and Bone Thickness in Patients With Class III Dentofacial Deformities

The correlation between tooth inclination and bone thickness and the comparison between thickness measurements and buccolingual inclination before and after dental decompensation will be evaluated through tridimensional images in individuals with class III dentofacial deformities.

Class III dentofacial deformity is associated with important functional, psychological and aesthetic changes and thus individuals affected by this type of deformity are motivated in seeking treatment. The sample will consist of three-dimensional images of 40 individuals with Class III dentofacial deformities. From these images generated by CBCT scans, the buccolingual inclinations of all teeth and maxillo-mandibular alveolar bone thickness in three different heights will be measured (3, 6 and 8 mm) from the cementoenamel junction through the Dolphin 3D® program. The 3D scans of the sample will be evaluated in two stages: the initial stage and after dental decompensation at the end of pre-surgical orthodontic phase. Pearson correlation test will be used to verify the relationship between tooth inclination and bone thickness. The thickness measurements and buccolingual inclination will be compared between treatment times (initial and pre-surgical) using paired Student t-test.

Eligibility Criteria:
Inclusion Criteria:

* Brazilian nationality;
* Class III malocclusion, for molars and canines;
* Concave facial profile;
* Indications for orthodontic-surgical treatment;
* Craniofacial growth finalized ;
* Absence of tooth extractions;
* No local and / or general contraindications for surgery;
* No previous orthodontic-surgical treatment;
* Good dental and periodontal conditions.

Exclusion Criteria:

* Facial asymmetry;
* Absence of teeth (except the third molars);
* Periodontal disease and vertical or horizontal bone resorption;
* Extensive metal restorations;
* Previous orthodontic treatment;
* Root anomalies.
Jaw Clenching on Anaerobic Ability and Ventilatory Flows

The latest findings on the ergogenic effects of a dentistry-design, bite-aligning mouthpiece require additional research to assess its impact on anaerobic ability and ventilatory parameters. This study was aimed at determining the ergogenic acute effects of wearing a custom-made mouthpiece on oral airflow dynamics, Wingate Test performance parameters.

The objectives of this study are to investigate the acute effects of a customized, bite-aligning mouthpiece on the different parameters assessed during a Wingate Test, as compared to clenching the jaw without a mouthpiece, in a physically-active, male population. The study also investigate the influence of wearing a mouthpiece on non-forced and forced airflow dynamics, as compared to open mouth and a jaw clenching without mouthpiece. Mouthpieces are made using a new scanning method that simplified the fitting process and lowered its cost.

A nonrandomized study was designed to compare the effect of the occlusion condition on the anaerobic power and airflow dynamics. Conditions of wearing or not wearing mouthguard (MOUTHG and NO-MOUTHG, respectively) are randomly distributed in all tests. Each subject participate in three sessions. The first session is used to obtain informed consent assess anthropometric measurements and to scan the subject's mouth structure. In the second session, subjects are familiarized with the test protocols via a learning session that included demonstration of Wingate Test and the airflow dynamics measurements. Subjects also perform the airflow tests and the first Wingate Test trial. In the third session, subjects perform the second Wingate Test trial. Conditions are randomly distributed in all tests. Wingate Test trials were separated by three days and subjects were not allowed to perform intensive training workloads.

Eligibility Criteria:
Inclusion Criteria:

* Healthy physical active males

Exclusion Criteria:

* Acute or chronic injury that caused pain during testing procedures
* Temporomandibular joint disorders
Long-term Efficacy and Safety of Gabapentin Enacarbil in Japanese Restless Legs Syndrome Patients

The objective of this study is to evaluate the efficacy and safety of ASP8825 (gabapentin enacarbil) for long-term treatment of restless legs syndrome patients.


Eligibility Criteria:
Inclusion Criteria:

* Patients who diagnosed with RLS according to the diagnostic criteria established by the International RLS Study Group
* International Restless Legs Syndrome Scale (IRLS) score ≥15 presence of RLS symptoms on ≥15 days per month and ≥4 days per week preceding inclusion in this study

Exclusion Criteria:

* Patients using dopamine agonists or gabapentin within 1 week before or any anti-RLS treatment within 2 weeks before the start of the pretreatment observation period
* Patients with an estimated creatinine clearance \<60 mL/min determined using the Cockcroft-Gault formula
* Pregnant or lactating women
* Individuals with serum ferritin \<20 ng/mL were also excluded from the trial
* Individuals with movement disorders and/or abnormal neurological findings
Blood Markers in Adult Patients With Sudden Sensorineural Hearing Loss (SSNHL)

The roles of thrombophilia and cardiovascular risk factors in sudden sensorineural hearing loss (SSNHL) remain controversial. Cochlear micro-thrombosis has been hypothesized as a possible pathogenic mechanism of SSNHL. The objective was thus to measure the levels of markers of macrovascular thrombosis and microvascular risk factors

To recruit adult consecutive outpatients referred for SSNHL to the otolaryngologist clinic of our university hospital starting June 2016 and prospectively until december 31th 2018.

Plasma sampling and biomarkers quantification : After a 48-hours diet excluding serotonin- and tryptophan-rich food, fasting blood samples were collected into vacuum tubes containing 109 mM sodium citrate (Becton-Dickinson, Le Pont de Claix, France) with a 9:1 blood-to-anticoagulant ratio. After removing 0.5 mL of whole blood for serotonin measurements, the remainder was centrifuged at 1,000 x g for 10 minutes, and the supernatant was then centrifuged at 3,000 x g for 15 minutes to isolate platelets and obtain platelet-poor plasma. Importantly, the time between blood sampling and platelet/plasma isolation was less than one hour. Aliquots of whole blood, platelets, and plasma were kept frozen (-20°C) until serotonin and homocysteine measurements performed within one week after congelation. Whole blood, platelet and plasma 5-HT as well as plasma homocysteine (Hcy) levels were measured by high pressure liquid chromatography coupled to fluorimetric detections .

Thrombophilia screening included measurements of antithrombin , protein C, protein S, factor V Leiden, prothrombin G20210A, methylene tetrahydrofolate reductase (MTHFR) C677T, antiphospholipid antibodies anticardiolipin IgG and IgM and anti-beta2 glycoprotein 1 IgG), dilute Russell viper venom time , Rosner index, factor VIII, von Willebrand factor (vWF) activity and antigen. Tests were performed on citrated plasma, serum or DNA as appropriate and as previously described

Eligibility Criteria:
Inclusion Criteria:

* adult idiopathic SSNHL

Exclusion Criteria:

* secundary hearing loss
A Study in Healthy Subjects to Assess How Dosing of Rifampin Affects What the Body Does to a Dose of GW679769 (Casopitant).

This study will examine what the body does while taking GW679769 alone and together with rifampin in healthy adult subjects.


Eligibility Criteria:
Inclusion criteria:

* healthy
* female subjects who cannot become pregnant
* able to swallow and keep down oral medication
* can understand and follow the protocol requirements and instructions

Exclusion criteria:

* smokes at least 4 packs per day in the past 12 months
* use of prescription or non-prescription drug(s)
* herbal or dietary supplements or vitamins with 14 days prior to first dose of study medication
* a clinically relevant abnormality medical condition or circumstance making the subject unsuitable for the study per the study doctor
* blood donation in excess of 1 pint within 56 days before dosing of medication
* iron deficient
* history or drug allergy of study medication
* history of drug or alcohol abuse or dependency within the past 6 months
* subjects cannot use any nicotine-containing products within the last 6 months
* positive for HIV, Hepatitis B or C
* active peptic ulcer disease
* uncontrolled nausea and vomiting
* active infection
* heart failure
* female who is lactating
* female who has a positive pregnancy test
A Study of Telitacicept for the Treatment of Moderately to Severely Active Systemic Lupus Erythematosus (REMESLE-1)

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of moderately to severely active SLE.

Systemic Lupus Erythematosus (SLE) is a chronic autoimmune disease with heterogeneous manifestations and disease course. Despite advances in medical care, there are still significant unmet needs in SLE with diminished health-related quality of life (HRQoL), persistent disease activity, disease flares, intolerance to standard of care (SoC) therapies, and development of organ damage and co-morbidities.

Telitacicept is a fully human TACI-Fc fusion protein that targets B lymphocyte stimulator (BLyS) and a proliferating-inducing ligand (APRIL). Blocking the interaction of BLyS and APRIL with their cell membrane receptors (TACI, B-cell maturation antigen (BCMA), and B-cell activating factor receptor (BAFF-R)) would inhibit B cell proliferation and maturation, suppresses immune responses, and may alleviate autoimmune symptoms.

This Phase 3 study is a 2-stage study to evaluate the efficacy and safety of telitacicept compared to placebo in patients with moderately to severely active SLE while receiving SoC treatment in a global patient population with active SLE disease.

* Stage 1: a study to evaluate the efficacy, safety, pharmacokinetics (PK) and PD of two treatment arms of telitacicept compared to placebo in patients with moderately to severely active SLE while receiving SoC treatment.
* Stage 2: a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of telitacicept added to SoC compared to placebo with SoC therapy in patients with moderately to severely active SLE.

Eligibility Criteria:
Inclusion Criteria:

1. Age 12-70 years at screening.
2. Has a diagnosis of SLE for at least 6 months prior to the screening visit.
3. Meets the 2019 EULAR/ACR Classification criteria for SLE.
4. Moderately to severely active SLE defined by the following:

   1. SELENA SLEDAI total score ≥6 points with clinical SLEDAI score ≥4 points at screening;
   2. BILAG organ system scores of at least 1A or 2B at screening.
5. Clinical SLEDAI score of ≥4 at Day 0 prior to randomization.
6. At least one positive serologic parameter within the screening period.
7. Currently receiving at least one of the SOC SLE medications: oral corticosteroid, antimalarial and/or immunosuppressive agent.

Exclusion Criteria:

1. Active lupus nephritis undergoing induction therapy or unstable renal diseases within 12 weeks prior to screening.
2. Active or unstable neuropsychiatric SLE.
3. Autoimmune or rheumatic disease other than SLE
Intraoperative Bleeding During Endoscopic Sinus Surgery

The purpose of this study is to compare the blood loss during sinus surgery looking at two different types of anesthesia: either intravenous anesthesia with propofol or inhalational anesthesia with sevoflurane.


Eligibility Criteria:
Inclusion Criteria:

* male or female patients who are 18 years of age or older,
* ASA grade 1 (normal otherwise healthy patient),
* ASA grade 2 (patient with mild systemic disease),
* patients who have chronic sinusitis with or without nasal polyps.

Exclusion Criteria:

* pregnancy,
* abnormal coagulation panel
* preoperative use of NSAIDS or ASA medications within 7 days,
* end-stage renal disease,
* allergy to any of the used medications,
* maximal body mass index over 35
Exposure to General Anaesthesia in Infancy and Neurocognitive Development

This prospective cohort study aims to determine if apparently healthy children with no significant co-morbidities who undergo general anaesthesia for minor surgery before 15 months of age will develop measurable deficits in neurocognitive development compared to apparently healthy children with no significant co-morbidities who do not undergo general anaesthesia or surgery.

We hypothesize that healthy children exposed to general anaesthesia for minor surgery before 15 months of age will have significantly lower neurocognitive scores at 6,18, 24 and 36 months of age compared with age, and gender matched healthy children from the GUSTO cohort without prior exposure to general anaesthesia or surgery. (GUSTO: Growing Up Towards Healthy Outcomes in Singapore: a prospective longitudinal study providing normative data from the local population).

Eligibility Criteria:
Inclusion Criteria:

* Infants born \> 36 weeks gestation
* No significant co-morbidities
* Who undergo general sevoflurane anaesthesia for minor surgery lasting \<150 minutes (including herniotomies, orchidopexies, circumcisions and pyloromyotomies) before 15 months of age

Exclusion Criteria:

* Prematurity \<36 weeks gestation
* Birth weight less than 2400g
* Presence of a genetic or chromosomal disorder
* Disorder of the central nervous system, including any seizure disorder
* Major congenital cardiac defects
The Incredible Years Parents and Babies Program in Denmark

The purpose of the pilot study is to examine the effects of the Incredible Years Baby Programme (IY baby) offered as a universal intervention compared to Usual Care (UC) for parents with infants aged 0-12 months.

A randomised controlled effectiveness pilot trial of the Incredible Years Parents and Babies Program (IY baby)offered as a universal intervention in two municipalities in Denmark. 120 families will be randomised to IY Baby (intervention) og Usual Care (UC- control). Families recruited to the study have infants aged 0-4 months old. All first time mothers in one municipality and all mothers in another municipality are recruited. Outcomes are measured at 20 weeks post baseline and when the child is 18 months old. The aim of this trial is to estimate the effectiveness of the IY Baby program on parent and infant well-being, development and relationship and establish parameters for a definitive trial. In addition, the pilot study will also gather information on parent recruitment and participation to inform future research in this area.

Eligibility Criteria:
Inclusion Criteria:

* Parents with a child aged 0-4 months
* Parents are able to understand Danish at a basic level

Exclusion Criteria:

* Serious physical disability
* serious mental disability
* Child can not be placed in out of home care
Admixture Mapping of Ethnic and Racial Insulin Complex Outcomes

The main objective of this study is to investigate the effect of genetic and environmental parameters on racial/ethnic differences in aspects of insulin secretion and action. The genetic component will be modeled by estimating the degree of European genetic admixture (ADM) obtained from approximately 50 ancestry informative markers (AIMs). The environmental components will be modeled by measuring energy intake (food intake), energy expenditure (physical activity) and socioeconomic status.


Eligibility Criteria:
Inclusion Criteria:

* African American, European American, or Hispanic American, aged 7-12, healthy, weight is equal to or greater than 25 Kg

Exclusion Criteria:

* Type 1 or 2 diabetes, polycystic ovary disease, disturbances in glucose or lipid metabolism disturbances
* Use of medication known to affect body composition or physical activity, diagnosis of genetic disorders known to affect body composition or fat distribution
* Use of tobacco, alcohol consumption in excess of 400 g per week
* Menstruated for more than a year prior to the recruitment; OR
* A medical history that counter-indicates inclusion of the study
Summative Usability Study of Ciprofloxacin Dry Powder for Inhalation Using Placebo

The rationale of this study is to conduct a summative (i.e., validation) usability test of Ciprofloxacin Dry Powder for Inhalation (DPI) and the associated Instructions for Use.


Eligibility Criteria:
Inclusion Criteria

* Subjects (male and female) with non-cystic fibrosis bronchiectasis (NCFB) or chronic obstructive pulmonary disease (COPD)
* Subjects must be aged ≥40
* Subject must be able to independently manage and administer their NCFB/COPD medications

Exclusion Criteria:

* Subjects with recent exacerbation
* Subjects with recent significant hemoptysis in the four weeks before screening (and/or during the screening period)
* Subjects allergic to quinine
* Known chronic bronchial asthma