cyrilzakka/clinical-trials huggingface dataset:
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) with Fabry Disease

This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants


Eligibility Criteria:
Inclusion Criteria:

* Male or female subjects diagnosed with Fabry disease \> 12 years of age who completed Study AT1001-020
* Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
* Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable

Exclusion Criteria:

* Has moderate or severe renal impairment (eGFR \<60 ml/min/1.73 m2 at screening)
* Has advanced kidney disease requiring dialysis or kidney transplantation
* History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
* Has received any gene therapy at any time or anticipates starting gene therapy during the study period
* Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study
* Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
* Subject is treated or has been treated with any investigational/experimental drug, biologic or device within 30 days before screening
* Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
* Pregnant or breast-feeding
* Otherwise unsuitable for the study in the opinion of the investigator
Metabolic Cost of Walking With Passive vs. Powered Prosthetic Knees Among Persons With Limb Loss

The purpose of this industry-initiated research study is to test that a powered microprocessor controlled knee improves metabolic function during walking in level and sloped conditions as compared to the subject's physician prescribed prosthesis.

The Procedures include:

Initial Visit:

1. Informed consent
2. HIPAA authorization
3. Determine/verify ambulation potential
4. Take and record body measurements (height, weight, circumferences)
5. Activities-Specific Balance Confidence (ABC) scale
6. L.A.S.A.R. Posture alignment assessment with RxPx

Second Visit

1. Fit with heartrate monitor and metabolic analyzer mask
2. Treadmill Test (level-ground) with RxPx
3. Rest
4. Treadmill Test (incline) with RxPx
5. Fit subject with PMPK
6. L.A.S.A.R. Posture alignment assessment with PMPK
7. Subject uses PMPK for one week as their primary prosthesis

Third Visit (1 week later)

1. Fit with heartrate monitor and metabolic analyzer mask
2. Treadmill Test (level-ground) with PMPK
3. Rest
4. Treadmill Test (incline) with PMPK
5. Activities-Specific Balance Confidence (ABC) scale
6. Return PMPK and refit of RxPx
7. Verify alignment with L.A.S.A.R. Posture alignment
8. Check that all fasteners are secure

Eligibility Criteria:
Inclusion Criteria:

* Males and Females with unilateral knee disarticulation or transfemoral (KD/TF) amputation.
* a body mass greater than 49Kg and less than 117Kg
* 18-75 years of age
* Have an ambulation potential of K3 or K4
* Use a prosthesis daily for walking or sports activities
* No less than six months of experience with a prosthesis
* No socket issues or changes in the last six weeks
* No residual limb pain affecting functional ability.

Exclusion Criteria:

* Cannot walk at different speeds (MCFL K0-K2)
* Limb-loss below the knee or through the hip
* More than one amputation.
* Uses an assistive device for walking
* Uncontrolled edema in leg compartments
* Compromised skin of the residual limb or contralateral foot
* Are 180 days or less post-amputation
* Pregnant (self-reported)
Cholera-Hospital-Based-Intervention-for-7-days

The Cholera Hospital Based Intervention for 7 Days (CHoBI7) randomized controlled trial for transition to scale aimed to: (1) Develop and evaluate scalable approaches to integrate the CHoBI7 intervention into the services provided for hospitalized diarrhea patients at health facilities in Bangladesh; and (2) Evaluate the ability of the CHoBI7 intervention to lead to a sustained uptake of the promoted hand washing with soap and water treatment behaviors and significant reductions in diarrheal disease over time.

The findings from the recent randomized controlled trial of The Cholera Hospital Based Intervention for 7 Days (CHoBI7) demonstrated that this intervention was effective in significantly reducing symptomatic cholera infections in intervention households, and had significant sustained impacts on hand washing with soap behaviors and improved water quality 12 months post intervention. Next steps to transition to scale were: (1) Develop and evaluate scalable approaches to integrate the CHoBI7 intervention into the services provided for hospitalized diarrhea patients at health facilities in Bangladesh; and (2) Evaluate the ability of the CHoBI7 intervention to lead to a sustained uptake of the promoted hand washing with soap and water treatment behaviors and significant reductions in diarrheal disease over time.

Eligibility Criteria:
Inclusion Criteria:

Diarrhea patients will be defined as patients having acute watery diarrhea three or more loose stools over a 24-hour period in the past three days. Diarrhea patients admitted to ICDDRB Dhaka Hospital or Mugda General Hospital will be eligible for the trial if:

1. have had 3 or more loose stools over the past 24 hours
2. plan to reside in Dhaka for the next 12 months
3. have no basin for running water in their home
4. have a child under five years of age in their household (including themselves) that produced a stool sample at baseline
5. have a working mobile phone in the household.

Household members of the diarrhea patient will be eligible for the trial if:

1. they have shared the same cooking pot and resided in the same home with the diarrhea patient for the last three days
2. plan to reside in their current household with the diarrhea patient for the next 12 months

Exclusion Criteria:

(1) Children in foster care will be excluded from all research study activities.
Safety and Efficacy of ATG003 in Patients With AMD Receiving Anti-VEGF

This study is a double-masked, randomized, placebo-controlled study of the safety and preliminary efficacy of ATG003 (topical mecamylamine) in patients receiving maintenance injections of either ranibizumab or bevacizumab. Study Hypothesis: Mecamylamine could be effective in the treatment of neovascular AMD.


Eligibility Criteria:
Inclusion Criteria:

* \> 55 years of age
* clinical diagnosis of neovascular AMD

Exclusion Criteria:

* confounding ocular condition
Observational Study Regarding Adherence, Drug Use, Will to Take Part in Shared Decisioning in Asthma Outpatients

In a private practice setting typical for ambulatory medicine in Germany, asthma staging according to the GINA guidelines will be correlated with "real life" treatment and self management of adult outpatients suffering from asthma. Attitudes concerning adherence, awareness, interest in shared decision making and knowledge about the disease are assessed.

The study focuses on outpatients that have two different types of treatment at hand: on-demand reliever treatment and anti-inflammatory treatment. We will assess the correlation of number of devices and medications in regular or intermittent use with doctors recommendations and prescriptions and the parameters adherence, awareness, interest in shared decision making and knowledge about the disease.

The patients will be seen two to three times. The first visit is used to inform the patient about the study, to check with regard to inclusion and exclusion criteria, and finally to sign the informed consent, if the patient agrees to be enrolled in the study. The patient then will get the questionnaire, and she or he is asked to collect all asthma related medications in continuous or intermittent use and to bring them to the private practice staff.

In the second visit, the private practice staff will check the medications the patient has brought. This is a physical check, i.e. it is based on devices and medications on the desk, not on questions.

At least six months later, the private practice staff will check all asthma related prescriptions for the patient concerned, and for the last six months. The patient will be asked to give information if the questionnaire has not been answered completely.

In a previous study with 1.670 paediatric outpatients enrolled we have shown under-diagnosis, under-treatment and a low overall effectiveness of paediatric outdoor patient asthma care. In this previous paediatric study we compared suspected diagnosis and reported treatment. We did not assess parameters indicating low or high adherence, nor did we ask for interest in shared decision making or for awareness and indicators for knowledge about the disease.

Eligibility Criteria:
Inclusion Criteria:

* Age \> 18 years
* Asthma with irregular or regular use of short acting beta-agonists and/or:
* Asthma treatment with inhaled steroids
* Ability to understand the questionnaire

Exclusion Criteria:

* Age \< 18 years
* Significant airway disease other than asthma
* Asthma treatment with fixed combination of inhaled steroids with long or short acting beta-agonists
* Ability to understand the questionnaire must be doubted
Establishment and Validation of a Risk Prediction Model for Long-term Low Vision After Vitrectomy in PDR Patients

Diabetic retinopathy (DR) is the main cause of blindness among working-age adults in the world. Proliferative diabetic retinopathy (PDR) is the severe stage of DR, which is characterized by neovascularization of the retina. Vitreous hemorrhage and traction retinal detachment caused by PDR often require vitrectomy. The purpose of vitrectomy is to remove vitreous hemorrhage, peel off the preretinal proliferative membrane and help restore the retina. With the deepening of people's understanding of diabetes and the development and application of various hypoglycemic drugs, the life expectancy of patients with diabetes continues to prolong. Therefore, the goal of vitrectomy in patients with DR is not only to prevent blindness, but also to maintain good vision for a long time. The purpose of this study is to analyze the risk factors related to the occurrence of long-term low vision postoperatively and establish a risk prediction model, which can help patients adjust their psychological expectations reasonably and promote communication between doctors and patients.


Eligibility Criteria:
Inclusion Criteria:

* Voluntary and able to sign an informed consent form
* Age ≥18 years
* Documented diagnosis of diabetes mellitus (type I or type II diabetes) defined by the American Diabetes Association or World Health Organization criteria.
* Unabsorbed vitreous hemorrhage lasted for more than 2 weeks, with or without tractional retinal detachment examen by slit lamp, B-ultrasound and fundus color photography.

Exclusion Criteria:

* Patients with less than 12 months of follow-up
* Previous intraocular surgery (eg. corneal transplantation, glaucoma filtering, vitrectomy, except cataract surgery)
* Complicated with other retinal diseases
* Underwent surgery (eg. intravitreal injection, cataract surgery) of the study eye within 3 months
* Lack of medical records.
Efficacy of Computer Controlled Intraosseous Anesthesia in Pediatric Dental Patients

To evaluate the effectiveness of intra-osseous anesthesia using Quicksleepr5 in pain elimination during dental treatment in maxillary and mandibular primary molars, compared to conventional anesthesia.

The study will be an eight-armed randomized controlled trial, parallel design. A total of 120 pediatric dental patients aged from 5 to 9 years with good health, will be selected from the Pediatric Dentistry and Dental Public Health Department clinic, Faculty of Dentistry, Alexandria University, Egypt. They will be recruited among those who need local anesthesia to perform either quadrant restorative treatment or extraction of their primary molars. Participants will be allocated into 2 groups according to the local anesthetic technique to be used. Children in the test group will receive intraosseous anesthesia using QuickSleeper5, while inferior alveolar nerve block or infiltration anesthesia will be used for those who are in the control group. All the procedures will be videotaped. Pain response upon needle prick and dental treatment will be assessed subjectively using the visual analogue scale, and objectively using Face, Leg, Activity, cry, Consolability scale. Physiological parameters will also be recorded to determine pain reaction. Efficacy, latency period and duration of anesthesia will be recorded for both injection techniques. Child behaviour toward dental treatment in both injection techniques will be evaluated using Frankl rating scale. Dental anxiety of pediatric patients will be evaluated from the videotapes using VENHAM Clinical Anxiety Scale. Postoperative assessment for the occurrence of any adverse events is planned.

Eligibility Criteria:
Inclusion Criteria:

* Cooperative children during preoperative assessments.
* Patients selected in the restorative group: presenting with at least one primary molar indicated for restorative treatment or vital pulp therapy, after thorough clinical and radiographic examination.
* Patients selected in the extraction group: presenting with at least one primary molar indicated for extraction.

Exclusion Criteria:

* Any physical or mental disability, or psychological problems.
* Immunocompromised patients.
* Hypersensitivity to local anesthetic drugs used
* History of previous dental local anesthesia.
* Taking analgesics or other medications that would alter the child behavior or awareness of pain 12- hours pre-operative
A United States Study of Corlux for Psychotic Symptoms in Psychotic Major Depression

Corlux (mifepristone) is a new medication that modulates the body's use of a hormone called cortisol. Under normal conditions, cortisol and other hormones are created by the body in response to physical and emotional stress, triggering a healthy stress response. People who suffer from psychotic major depression may have unusually high levels of cortisol circulating within them or abnormal patterns of cortisol levels, overloading the stress response mechanism and causing symptoms of psychosis such as delusional thoughts or hallucinations. If Corlux can keep the body's cortisol receptors from being overloaded, the stress response system may return to normal function, which may result in improvement of symptoms. The purpose of this 56 day study is to learn the safety and effectiveness of Corlux in patients who have been diagnosed with psychotic major depression (PMD).


Eligibility Criteria:
Inclusion Criteria:

Individuals eligible for enrollment into this study are male and female adult patients who:

* Are 18 to 75 years of age
* Have a diagnosis of major depressive disorder with psychotic features (DSM-IV 296.24 or 296.34)
* Are able to provide written informed consent

Exclusion Criteria:

Individuals not eligible to be enrolled into the study are those who:

* Have a major medical problem
* Have previously participated in a Corlux (C-1073, mifepristone) clinical trial
* Have a history of an allergic reaction to Corlux (C-1073, mifepristone)
Effect of Photodynamic Therapy on Gingival Crevicular Cytokines in Periodontitis Patients

Systemically healthy periodontitis patients were recruited in this single-blind, split-mouth, randomized controlled clinical trial. After SRP, PDT was applied at probing depth (PD) ≥ 4 mm at test quadrant. At baseline, 3rd month and 6th month, clinical parameters and cytokine pattern in gingival crevicular fluids (GCFs) were analysed.

Periodontal parameters and cytokine IL-1β, IL-6, IL-8, TNF-α, MMP-8 levels were analysed.

Eligibility Criteria:
Inclusion Criteria:

* Adults who were medically fit or have medical condition that did not alter the outcome of periodontal treatment.
* Females who were not pregnant or nursing.
* Controlled diabetes mellitus with HbA1c \< 7.5 mmol/L.
* Not on any antibiotics or steroids 3 months prior to periodontal treatment.
* Diagnosed to have either localized or generalized chronic or aggressive periodontitis (Armitage, 1999) with all 4 quadrants having at least 2 sites with probing depth ≥ 4 mm.
* Consented to follow the non-surgical periodontal treatment (scaling and root planing) with PDT throughout the period of study, i.e. approximately 6 months.

Exclusion Criteria:

* Allergic to photosensitizer or dye
* Pregnant lady and nursing mothers
* Uncontrolled diabetes
Topical Ocular Mecamylamine in Diabetic Macular Edema (DME)

This is an open-label study evaluating the safety and tolerability of topical ocular mecamylamine given twice a day in patients with diabetic macular edema (DME). Patients will be treated for 12 weeks.


Eligibility Criteria:
Inclusion Criteria:

* macular edema due to diabetic retinopathy

Exclusion Criteria:

* vision loss from other ocular disease
* intraocular surgery within 3 months
* intraocular anti-VEGF or steroids within 3 months
* HbA1c \>12
SBI for PSM and PSD

A pilot randomized controlled trial to assess the feasibility, acceptability, and impact of screening and brief intervention to address prescription stimulant misuse and diversion among college students.

A screening and brief intervention including a control group, face-to-face group, and e-intervention group where the focus is prevention or brief intervention or referral to treatment related to students prescription stimulant misuse and diversion behaviors. Students will complete assessments at three time points, and the health providers who implement the intervention will also complete assessments.

Eligibility Criteria:
Inclusion Criteria:

Age 18 and student at the campus serving as the intervention site; Passive recruitment: Student has a scheduled appointment for a physical/wellness exam. Active recruitment: Student has a past 3 month history of PSM or PSD.

Exclusion Criteria:

Any eligible student who, during the course of the session, demonstrates the need for immediate mental health services, will have the study session terminated so that participant can receive needed care.
A Safety and Efficacy Study of ADS-5102 in Patients With Multiple Sclerosis and Walking Impairment

This study assessed the long-term safety and tolerability of ADS-5102 in subjects with MS and walking impairment who had completed the double-blind, placebo-controlled study of ADS-5102 in subjects with MS (ADS-AMT-301).

This was a multicenter, open-label study of ADS-5102 (amantadine) extended-release capsules in subjects with MS and walking impairment who completed study drug treatment for 16 weeks and completed a Week 16 visit in Study ADS-AMT-MS301.

All enrolled subjects were to receive ADS-5102 at 137 mg for the first week, 205.5 mg for the second week, and 274 mg for the remainder of the 52-week open-label treatment period.

Subjects returned to the clinic for safety and efficacy assessments at Weeks 4, 24, and 52. In addition, a telephone visit for safety assessments was conducted at Week 2 and Week 38. Subjects who withdrew from the study prior to completion of the Week 52 visit had an early termination (ET) visit that included safety and efficacy assessments. Subjects who completed 52 weeks of open-label treatment had a final visit for post-treatment safety follow-up and efficacy assessment at Week 54.

All study visits and efficacy assessments were to be scheduled to occur at approximately the same time of day for each individual subject. Each subject's efficacy assessment was to be performed by the same clinical rater, if possible.

Eligibility Criteria:
Inclusion Criteria:

* Signed a current IRB-approved informed consent form
* Successful completion of a prior double blind study of ADS-5102 in patients with MS walking impairment.

Exclusion Criteria:

* Based on the judgment of the investigator or Medical Monitor, participation in the study would jeopardize the safety of the subject.
* If female, is pregnant or lactating
* If a sexually active female, is not surgically sterile or at least 2 years post-menopausal, or does not agree to utilize a highly effective hormonal method of contraception (an IUD, or vasectomized male partner is also acceptable), in combination with a barrier method, from baseline through at least 4 weeks after the completion of study treatment. If a sexually active male, does not agree to utilize condoms from screening through at least 4 weeks after the completion of study treatment.
* Anticipated treatment with any amantadine formulation other than ADS-5102
* Planned participation in another interventional clinical trial
Transplantation of Fibroblast for Correction of Nasolabial Folds

The study evaluated the safety and efficacy of the Autologous cultured fibroblast for correction of Nasolabial folds and inhibition of skin aging

Autologous cultured fibroblast which is obtain from the patient's normal skin is harvested and expanded in an in vitro. Biopsies samples are obtained from the behind of the patient's ear as a source of fibroblasts, then fibroblast cells isolated and expanded through cell culture, and used for the correction of Nasolabial folds, deep wrinkles of the forehead. Prior to final packaging, cell viability is assessed to be at least 85%

Eligibility Criteria:
Inclusion Criteria:

* Available and willing to attend all follow-up visits.
* Age \> 18 years.
* Able and willing to give informed consent

Exclusion Criteria:

* Known allergy or sensitivity to any cellular products
* The subject has received a soft tissue augmentation to the area to be treated within the last 6 months.
* Subject is unwilling to forgo any cosmetic augmentation procedures for the duration of the study.
* The subject has received autologous fat transfer in the last 6 months.
* Subject is suffering from facial Kaposi's sarcoma.
* The subject has active skin diseases or inflammation on or near the area of injection
* positive HIV, hepatitis B virus (HBV), hepatitis C virus (HCV)
Endosleeve in Adolescents

In this study, the investigators assess the safety and efficacy of endoscopic sleeve gastroplasty in adolescents with obesity.

Endoscopic bariatric therapy (EBT) is an emerging, rapidly evolving field that aims to serve as a middle ground between safe, albeit poorly effective medical weight loss strategies and drastic but effective bariatric surgery. Endoscopic sleeve gastroplasty (ESG) is a procedure that restricts the stomach to a sleeve-like configuration by utilizing full-thickness sutures that plicate the greater curvature of the stomach. Emerging data from adult populations shows an acceptable safety, tolerability and efficacy profile. Based on analysis that confirmed the safety and efficacy of ESG in adult patients who undergo the procedure under a standardized protocol and clinical care pathway, the investigators planned to study its effects on adolescents in a specialized center that offers surgical, endoscopic, and medical management of obesity under a standardized protocol and care pathway.

Eligibility Criteria:
Inclusion Criteria:

* BMI that is \>120% of the 95th percentile for age and gender
* Written informed assent/consent, with concomitant parental consent for patients aged ≤17 years

Exclusion Criteria:

* Age \<10 years
* Age \>21 years
* Presence of large hiatal hernia
* Previous gastric surgery
* Cognitive impairment