linjc16/ctgov huggingface dataset:
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Study Overview
=================
Brief Summary
-----------------
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

Detailed Description
-----------------
This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.

Official Title
-----------------
Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease

Conditions
-----------------
Pompe Disease

Intervention / Treatment
-----------------
* Biological: ATB200
* Drug: AT2221


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Subject must have a diagnosis of Pompe disease based on documentation of one of the following: deficiency of GAA enzyme GAA genotyping Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme) The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC. Exclusion Criteria: Patient, whether male or female, is planning to conceive a child during the treatment program. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221 Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements. Patient has received any gene therapy at any time. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)
Sexes Eligible for Study
-----------------
All

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: ATB200|nan|
|Drug: AT2221|nan|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
expanded access, compassionate use
Effect of Medical Treatment on Longitudinal Function of the Heart

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to determine the effect of medical treatment on longitudinal myocardial function. Patients who have been hospitalized for reasons other than heart disease will be examined with Transthoracic Cardiography before and after medical treatment to determine change in myocardial function.

Conditions
-----------------
Heart Function

Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Hospitalized patients receiving above-mentioned medications Exclusion Criteria: Patients suffering from heart conditions Patients unable to undergo Echocardiography Pregnancy

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 85 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients Without Acute Heart Conditions<br>Patients without acute heart conditions who were hospitalized for other medical indications. | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Longitudinal Myocardial Function | Analysis of longitudinal contractility of the heart by the calculation of the heart's strain factor before and after chronic medication treatment. | Two Years |
Concentrations of Salmeterol in Blood and Urine

Study Overview
=================
Brief Summary
-----------------
The purpose of the study is to assess the blood and urine concentrations of inhaled salmeterol.

Detailed Description
-----------------
The purpose of the study is to assess the serum and urine concentrations of salmeterol after inhalation of 100 microgram salmeterol in one dose. Furthermore, to evaluate any difference in three groups: 10 healthy men, 10 male asthmatic subjects and 10 male elite athletes with asthma.

Official Title
-----------------
Blood and Urinary Concentrations of Inhaled Salmeterol in Asthmatic Subjects and Elite Athletes With Asthma.

Conditions
-----------------
Asthma

Intervention / Treatment
-----------------
* Drug: inhaled salmeterol


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Physician-diagnosed asthma with positive reversibility or challenge test. Informed consent. Age between 18-45 years. Sex: male. Asthma classified as mild to moderate according to GINA guidelines. Used beta-2-agonist in minimum 12 months. Exclusion Criteria: Smokers or ex-smokers with a smoking history of 10 pack years or more. Respiratory tract infections within the last 2 weeks prior to study day. Subjects with other chronic diseases than asthma and/or allergy. Allergy towards the study medicine. Use of beta-2-agonist 10 days prior to study day.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 45 Years

Sexes Eligible for Study
-----------------
Male

Accepts Healthy Volunteers
-----------------
Accepts Healthy Volunteers

Study Plan
=================
How is the study designed?
-----------------

Design Details

Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Healthy<br>10 healthy men | Drug: inhaled salmeterol<br>* Inhalation of 100 micrograms salmeterol in one dose.<br>|
| Other: Asthmatics<br>10 male asthmatic subjects | Drug: inhaled salmeterol<br>* Inhalation of 100 micrograms salmeterol in one dose.<br>|
| Other: Elite athletes with asthma<br>10 male elite athletes with asthma | Drug: inhaled salmeterol<br>* Inhalation of 100 micrograms salmeterol in one dose.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Serum and urine concentrations of salmeterol |  | baseline, 4, 8, and 12 hours after medicine administration | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Asthma, Doping, Salmeterol, Beta-agonist, Urine concentrations
Prevention of Diabetes Progression Trial (PDPT)

Study Overview
=================
Brief Summary
-----------------
The primary purpose of this study is to determine the efficacy and safety of daclizumab in preventing the progression of diabetes in children who have just been diagnosed with type I diabetes. Additional purposes of the study are to investigate the amount of drug in the children, how quickly it is eliminated from the blood, and analyze the effect of the drug on the immune reaction to diabetes.

Official Title
-----------------
PD Initiated/Prevention of Diabetes Progression Trial (PDPT) ZEN119

Conditions
-----------------
Newly Diagnosed With Type 1 Diabetes

Intervention / Treatment
-----------------
* Drug: Zenapax


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Newly diagnosed type 1 diabetes without previous insulin or oral hypoglycemic therapy. ages 2 to 40 years at diagnosis enroll within 3 months of diagnosis test positive for at least one of the biochemical autoantibodies (ICA-512, GAD 65 or IAA) prior to enrolling Exclusion Criteria: Previous treatment with an anti-IL-2 directed monoclonal antibody or any other investigational agent that would interfere with the ability to evaluate the safety and efficacy of daclizumab. Other immunosuppressive drugs including, but not limited to, corticosteroids, cyclosporine, tacrolimus, rapamycin, mycophenolate mofetil, or azathioprine. Active significant infection Limited life expectancy because of disease other than diabetes Pregnancy

Ages Eligible for Study
-----------------
Minimum Age: 2 Years
Maximum Age: 40 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Zenapax|nan|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Area under the curve, Glucagon Stimulation Testing for c-peptide measurements at 0, +6min and +10min intervals over 2 years |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| HbA1c |  |  | 
| Insulin requirements (units/kg/day) |  |  |
A Study of the Safety and Pharmacokinetics of MEGF0444A Administered to Patients With Advanced Solid Tumors

Study Overview
=================
Brief Summary
-----------------
This is a Phase I, first-in-human, open-label, dose-escalation study of MEGF0444A administered by IV infusion to patients with advanced solid tumors for whom standard therapy either does not exist or has proven to be ineffective or intolerable.

Official Title
-----------------
A Phase I, Open-Label, Dose-Escalation Study of the Safety and Pharmacokinetics of MEGF0444A, Administered Intravenously to Patients With Advanced Solid Tumors

Conditions
-----------------
Solid Cancers

Intervention / Treatment
-----------------
* Drug: MEGF0444A


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Histologically or cytologically documented, incurable, or metastatic solid malignancy that has progressed on, or failed to respond to regimens or therapies known to provide clinical benefit Exclusion Criteria: Inadequate hematologic and organ function Anti-cancer therapy within 4 weeks prior to initiation of study treatment Adverse events from prior anti-cancer therapy that have not resolved to Grade ≤ 1, except for alopecia Active infection or autoimmune disease Pregnancy (positive pregnancy test) or lactation

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: MEGF0444A<br>* Intravenous escalating dose<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and nature of dose-limiting toxicities (DLTs) |  | Days 1-21 of cycle 1 | 
| Incidence, nature, relatedness, and severity of adverse events |  | Day 1 to study completion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetic parameters including total exposure, minimum and maximum serum concentration, clearance, and volume of distribution |  | Following administration of study drug | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Solid Tumor, Cancer
Role of Mega Dose of Vitamin C in Critical COVID-19 Patients

Study Overview
=================
Brief Summary
-----------------
Vitamin C (ascorbic acid) is a water-soluble vitamin having anti-inflammatory, immunomodulatory, anti-oxidative, antithrombotic and antiviral properties. Considering these effects vitamin C should have beneficial impact in patients suffering from sepsis and acute respiratory distress syndrome (ARDS). The current study is designed to assess the beneficial effects of Vitamin C in COVID-19 infected patients.

Official Title
-----------------
Effects of Mega Dose Vitamin C in Critically Ill COVID-19 Patients: A Randomized Control Trial

Conditions
-----------------
Corona Virus Infection

Intervention / Treatment
-----------------
* Drug: Vitamin C


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: critically ill patients admitted in ICU Exclusion Criteria: allergic to Vitamin C didnt given the consent
Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: vitamin C<br>The dose would be 30 grams a day (10 grams TDS) for 3 days with standard treatment | Drug: Vitamin C<br>* mega dose is given to the selected critically ill patients<br>|
| No Intervention: Placebo<br>Distill water in the same dose with same standard treatment | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Partial pressure of Oxygen in arterial blood to fraction of inspired Oxygen (P/F ratio) |  | 1 and 4th day | 
| Survival analysis |  | 28 days follow up | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Length of Hospital stay |  | Admission to discharge | 
| Need for intubation | number of intubations needed in both groups. | 28 days | 
| Inflammatory markers | comparison of the inflammatory markers, CRP, D.Dimer, Procalcitonin, LDH, TLC and hematological variables at day four after the intervention started. | before the start of treatment (day 1) and on day 4 | 
| APACHE II score |  | before the start of treatment (day 1) and on day 4 |
Bicarbonates Versus Saline for Contrast Induced Acute Kidney Injury Prevention in Intensive Care Unit

Study Overview
=================
Brief Summary
-----------------
The aim of this study is to compare bicarbonates versus saline for prevention of contrast induced acute kidney injury in critically ill patients.

Conditions
-----------------
Contrast Induced Acute Kidney Injury

Intervention / Treatment
-----------------
* Drug: bicarbonates
* Drug: saline


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: patient > 18 years old in intensive care unit since 24h and for up to 48 hours with contrast medium injection Exclusion Criteria: patient under renal replacement therapy rise in creatinine > 26µmol/L within 48hours anuria within 12 hours cardiogenic pulmonary oedema pH > 7.50 or kaliemia < 3mmol/L pregnancy

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Factorial Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: bicarbonates<br>sodium bicarbonates 1,4% | Drug: bicarbonates<br>* sodium bicarbonates 1,4%<br>|
| Active Comparator: saline<br>sodium chloride 0,9% | Drug: saline<br>* sodium chloride 0,9%<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| contrast induced acute kidney injury |  | 72 hours | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
intensive care unit
The Influence of Meibomian Gland Expression on Corneal Aberrations in MGD Patients

Study Overview
=================
Brief Summary
-----------------
To evaluate the influence of corneal-aberrations before and after the meibomian gland thermal massage treatment.

Detailed Description
-----------------
62 MGD patients treated with meibomian gland thermal massage once a week for 3 to 4 weeks were recruited.The Ocular Surface Disease Index questionnaire; tear film assessment encompassing tear meniscus height and non-invasive keratograph breakup time ; meibography score,corneal fluorescein staining; the Schirmer I test ; expressible meibomian gland, and quality ,anterior and total corneal aberrations were evaluated before and after treatment.

Official Title
-----------------
The Effect of Meibomian Gland Thermal Massage on Corneal Aberrations in Meibomian Gland Dysfunction Patients

Conditions
-----------------
Meibomian Gland Dysfunction

Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: tear break-up time (BUT) ≤10 seconds or Schirmer I test (SIT) ≤10 mm, the presence of dry eye symptoms, and fluorescein staining (FL) score <3 points and at least one lid margin abnormality and poor meibomian gland expressibility Exclusion Criteria: eyelid structure abnormal(Entropion , ectropion); having received any other treatment for MGD within three months history of ocular surgery contact lens use ocular infection any type of corneal scarring

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 90 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------
What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| corneal aberration corneal aberrations | Corneal aberrations were measured with a Pentacam。 The anterior corneal wavefront aberration was expanded with normalized Zernike polynomials.The corneal aberration from 3rd to 6th order were recorded as the root mean square (RMS) for analysis. | baseline and 1 week after treatment | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
thermal massage, corneal aberrations, physical therapy
A Comparison of SLx-4090 in Combination With Metformin Versus Metformin Therapy Alone in Patients With Type 2 Diabetes

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to determine whether SLx-4090 in combination with metformin therapy will reduce HbA1c in patients with Type 2 Diabetes more effectively than metformin therapy alone.

Detailed Description
-----------------
HbA1c after 12 weeks FPG after 12 weeks Safety and tolerability Plasma levels of SLx-4090

Official Title
-----------------
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of SLx-4090 in Combination With Metformin in Subjects With Type 2 Diabetes

Conditions
-----------------
Type 2 Diabetes

Intervention / Treatment
-----------------
* Drug: SLx-4090
* Other: Placebo
* Drug: Metformin


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: HbA1c 7-11% On a stable dose of metformin for at least 6 weeks Exclusion Criteria: Type 1 Diabetes Antidiabetic medication other than or in addition to metformin FPG > 270 mg/dL

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>SLx-4090 in combination with Metformin | Drug: SLx-4090<br>* SLx-4090<br>Drug: Metformin<br> <br> |
| Other: 2<br>Placebo | Other: Placebo<br>* matching tablets<br>Drug: Metformin<br> <br> |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Reduction in HbA1c |  | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events |  | 12 weeks |
Pharmacokinetics of Sedatives and Analgesics During Extracorporeal Membrane Oxygenation (ECMO) Support

Study Overview
=================
Brief Summary
-----------------
This study will measure plasma concentrations of dexmedetomidine, fentanyl, morphine and midazolam in pediatric patients supported with extracorporeal membrane oxygenation (ECMO) aiming to understand the pharmacokinetics of these drugs in this setting.

Official Title
-----------------
Pharmacokinetics of Sedatives and Analgesics During Extracorporeal Membrane Oxygenation (ECMO) Support

Conditions
-----------------
Drug Effect

Intervention / Treatment
-----------------
* Drug: Dexmedetomidine
* Drug: Fentanyl
* Drug: Midazolam
* Drug: Morphine


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: supported on ECMO receiving one or a combination of the following drugs dexmedetomidine, fentanyl, midazolam or morphine as part of sedation management Exclusion Criteria: -none

Ages Eligible for Study
-----------------
Minimum Age: 0 Days
Maximum Age: 17 Years

Sexes Eligible for Study
-----------------
All

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pediatric Patients on ECMO Support<br> | Drug: Dexmedetomidine<br>* Administered for sedation management per hospital protocol<br>Drug: Fentanyl<br>* Administered for sedation management per hospital protocol<br>Drug: Midazolam<br>* Administered for sedation management per hospital protocol<br>Drug: Morphine<br>* Administered for sedation management per hospital protocol<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in plasma concentrations of dexmedetomidine |  | Throughout the duration of sedation management with dexmedetomidine at baseline, 30, 60, 120 minutes and then at 4, 6, 12 hours after each bolus or change in infusion and then at 30,60, 90, 120, 240minutes and 4 hours after the drug is stopped | 
| change in plasma concentrations of fentanyl |  | Throughout the duration of sedation management with fentanyl at baseline, 30, 60, 120 minutes and then at 4, 6, 12 hours after each bolus or change in infusion and then at 30,60, 90, 120, 240minutes and 4 hours after the drug is stopped | 
| change in plasma concentrations of morphine |  | Throughout the duration of sedation management with morphine at baseline, 30, 60, 120 minutes and then at 4, 6, 12 hours after each bolus or change in infusion and then at 30,60, 90, 120, 240minutes and 4 hours after the drug is stopped | 
| change in plasma concentrations of midazolam |  | Throughout the duration of sedation management with midazolam at baseline, 30, 60, 120 minutes and then at 4, 6, 12 hours after each bolus or change in infusion and then at 30,60, 90, 120, 240minutes and 4 hours after the drug is stopped |