cyrilzakka/clinical-trials huggingface dataset:
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in participants with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.

This study was designed as a 3-part study to evaluate the safety and tolerability of IV administration of SBC-103. Participants enrolled in Part A (0.3, 1.0, or 3.0 milligrams \[mg\] per kilogram \[kg\] of SBC-103 administered every other week \[QOW\] for 24 consecutive weeks). Participants who completed Part A were eligible for Part B (an increase to 1.0 or 3.0 mg/kg QOW). Participants who completed Part B were eligible for Part C (5.0 and/or 10.0 mg/kg to continue through Week156; no participants received both 5.0 and 10.0 mg/kg). Due to the early termination of the SBC-103 development program, including this study, all participants withdrew from Part C at the sponsor's decision. As a result of the early termination of this program, this report provides only safety data.

Eligibility Criteria:
Key Inclusion Criteria:

* A participant was greater than or equal to 2 years of age but less than 12 years of age at the time of informed consent.
* Definitive diagnosis of MPS IIIB.
* Documented developmental delay.

Key Exclusion Criteria:

* Received treatment with gene therapy at any time.
* Previous hematopoietic stem cell or bone marrow transplant.
* Had any internal or non-removable external metal items that presented a safety risk for study assessments that utilized magnetic fields, or any other medical condition or circumstance in which magnetic resonance imaging was contraindicated according to local institutional policy.
* Known hypersensitivity to eggs.
Effect of Teleprevir in Triple Therapy on Intrahepatic Immunological Mechanisms

Chronic hepatitis C infection (HCV) is a disease that affects worldwide about 170 million people. The previous standard of care therapy of chronic HCV patients consists of pegylated-IFN-α combined with ribavirin, and results in sustained clearance of HCV-RNA in only about 50% of the HCV genotype 1 infected patients. Telaprevir, a NS3A-4A inhibitor, has previously proven to offer therapeutic options to previous non-responders to the standard of care. Although, not all chronic HCV patients benefit from telaprevir and it is still not known why certain patients are also non-responsive to this triple therapy. In this study we try to understand why certain patients are also non-responsive to telaprevir, how triple therapy modulates the responsiveness to IFN-α and what the immunological consequences are of treatment with telaprevir, either directly or as a result of telaprevir-induced reduction of HCV-RNA levels.


Eligibility Criteria:
Inclusion Criteria:

* Patients between 18 and 70 years of age, with a chronic hepatitis C - genotype 1 infection
* Patients are naive, non-responders or relapsers to previous treatment with peginterferon or conventional interferon plus ribavirin combination therapy
* High viral load (\>400,000 IU/ml)
* Indication for antiviral therapy of hepatitis C according to current clinical guidelines

Exclusion Criteria:

* Decompensated cirrhosis (Child-Pugh Grade B or C)
* Hepatic imaging (ultrasound, CT or MRI) with the evidence of hepatocellular carcinoma.
* Females who are pregnant or breast-feeding
* History or other evidence of severe illness, malignancy or any other condition which would make the patient, in the opinion of the investigators, unsuitable for the study
* Co-infections with human immunodeficiency virus (HIV) or Hepatitis B virus (HBV)
* Presence of contra-indications for antiviral therapy with telaprevir:
* Telaprevir is contraindicated when combined with drugs that are highly dependent on CYP3A for clearance and for which elevated plasma concentrations are associated with serious and/or life-threatening events. Telaprevir in contraindicated when combined with drugs that strongly induce CYP3A and thus may lead to lower exposure and loss of efficacy of Telaprevir. The contraindicated medications include the following:
* Alfuzosin
* Rifampicin
* Dihydroergotamine, ergonovine, ergotamine, methylergonovine
* Cisapride
* St John's wort
* Atorvastatin, lovastatin, simvastatin
* Pimozide
* Sildenafil or tadalafil
* Triazolam
* Presence of contra-indications for antiviral therapy with peginterferon or ribavirin
* Severe psychiatric disorder, such as major psychoses, suicidal ideation, suicidal attempt and/or manifest depression.
* Visual symptoms related to retinal abnormalities
* Pregnancy, breast-feeding or inadequate contraception
* Thalassemia, spherocytosis
* Interfering substance abuse, such as high alcohol intake (indicator: 28 drinks/ week)
Patient-Specific Versus Conventional Instrumentation in TKA

The aim of this prospective multicenter randomized clinical trial (RCT) is to compare the clinical and radiologic results of primary total knee arthroplasty (TKA) using patient-specific versus conventional instrumentation.


Eligibility Criteria:
Inclusion Criteria:

* patients with primary or secondary osteoarthritis of the knee requiring total knee arthroplasty
* aged between 18 and 85 years
* able to understand information
* affiliated to social security.

Exclusion Criteria:

* active or suspected sepsis
* tumor around the knee
* previous partial or total knee replacement
* presence of hardware that could artifact MRI
* contraindication to MRI
* extra-articular deformation requiring osteotomy around the knee in conjunction with TKA
* social situation that could impair follow-up.
DNA Methylation and Lung Disease in Cystic Fibrosis

Lung disease progression is variable among cystic fibrosis (CF) patients and depends on DNA mutations in the CFTR gene, polymorphic variations in disease-modifier genes and environmental exposure. The contribution of genetic factors has been extensively investigated, whereas the mechanism whereby environmental factors modulate the lung disease is unknown. Because these factors can affect the epigenome, investigators hypothesized that DNA methylation variations at disease-modifier genes modulate the lung function in CF patients.

Lung disease progression is variable among cystic fibrosis (CF) patients and depends on DNA mutations in the CFTR gene, polymorphic variations in disease-modifier genes and environmental exposure. The contribution of genetic factors has been extensively investigated, whereas the mechanism whereby environmental factors modulate the lung disease is unknown. Because these factors can affect the epigenome, investigators hypothesized that DNA methylation variations at disease-modifier genes modulate the lung function in CF patients.

The investigators analyzed DNA methylation levels in the promoter of fourteen lung disease-modifier genes and showed that DNA methylation levels are altered in nasal epithelial and blood cell samples from CF patients. This study disclosed slightly, but significantly differentially methylated regions that collectively may modulate lung disease severity. It also highlighted that complex relationships between genetic and epigenetic factors contribute to the phenotypic variability of CF patients.

Eligibility Criteria:
Inclusion Criteria:

* \>18 years old
* homozygous for the F508del mutation

Exclusion Criteria:

* subjects who have an active CF exacerbation or a recent viral infection on the day of biological samples collection;
* pregnant women;
* patients who are included in interventional medical trials;
* patients who had lung transplantation.
In Vitro Study of Peripheral Blood Mononuclear Cell Function in Healthy Individuals and Patients

Peripheral blood mononuclear cells (PBMC) will be collected from healthy volunteers and patients who present with different diseases that involve or implicate the immune system dysregulation (HIV infection, autoimmune diseases and cancer). These PBMC will be studied in vitro for a number of functional parameters, including generating soluable factors that inhibit HIV infection, developing patterns of immune dysregulation, and inducing apoptotic T cell death. The purpose of such studies is to obtain insight into the mechanisms of natural resistance to viral infections, AIDS pathogenesis, and disease-induced immune dysregulation.

Peripheral blood mononuclear cells (PBMC) will be collected from healthy volunteers and patients who present with different diseases that involve or implicate the immune system dysregulation (HIV infection, autoimmune diseases and cancer). These PBMC will be studied in vitro for a number of functional parameters, including generating soluable factors that inhibit HIV infection, developing patterns of immune dysregulation, and inducing apoptotic T cell death. The purpose of such studies is to obtain insight into the mechanisms of natural resistance to viral infections, AIDS pathogenesis, and disease-induced immune dysregulation.

Eligibility Criteria:
* INCLUSION CRITERIA:

Greater than or equal to 18 years old.

Documented HIV infection by current DoD criteria (1 ELISA, 2 Confirmatory Western Blot) or by one of the following tests; DNA PCR, RNA PCR, p24 antigen test, viral culture assay.

CD4 lymphocyte cell count greater than 300 mm(3).

EXCLUSION CRITERIA:

Unable to provide informed consent.

Unable to meet the minimum (150 days) and maximum (395 days) research visit interval requirements.
Impact of Behavioral Compliance Between Residents and Surgeons

This study aims to evaluate the influence of perceived peer performance on the clinical skill of trocar placement during laparoscopic cholecystectomy among surgeons and surgical residents. It investigates whether exposure to incorrect trocar placements by peers affects the accuracy of subsequent placements.


Eligibility Criteria:
Inclusion Criteria:

* Over 18 years old
* Spanish mother tongue
* Experience in laparoscopic cholecystectomy
* Consent to participate

Exclusion Criteria:

* Physical disability preventing understanding or response
* More than 3 years of suspension from surgical practice
Study in Rheumatoid Arthritis for Subjects Who Completed Preceding Study M13-390 With Adalimumab

A Phase 2b, open-label extension (OLE) study in rheumatoid arthritis (RA) patients designed to collect long-term safety, tolerability, efficacy, and immunogenicity data of the proposed new adalimumab formulation.

All participants who completed Study NCT01712178 had an opportunity to enroll into the study and to receive the new adalimumab formulation at a dose of 40 mg every other week (eow) for an additional 24 weeks.

Eligibility Criteria:
Inclusion Criteria:

1. Subject has completed the preceding Study M13-390 for rheumatoid arthritis and has not developed any discontinuation criteria from that study.
2. If female, subject is either not of childbearing potential, defined as postmenopausal for at least 1 year or surgically sterile (bilateral tubal ligation, bilateral oophorectomy and/or hysterectomy) or is of childbearing potential and is practicing an approved method of birth control throughout the study and for 150 days after last dose of study drug. Examples of approved methods of birth control include the following (see local informed consent for more detail):

   * Condoms, sponge, foams, jellies, diaphragm or intrauterine device (IUD);
   * Hormonal contraceptives for 90 days prior to study drug administration;
   * A vasectomized partner.
3. Subjects must be able and willing to self-administer subcutaneous (SC) injections or have a qualified person available to administer SC injections.
4. Subject is judged to be in good general health as determined by the Principal Investigator based upon the results of medical history, physical examination, laboratory profile performed at Baseline.
5. Subjects must be able and willing to provide written informed consent and to comply with the requirements of this study protocol.

Exclusion Criteria:

1. Ongoing infections at Week 0 that have NOT been successfully treated. Subjects with ongoing infections undergoing treatment may be enrolled BUT NOT dosed until the infection has been successfully treated.
2. Subject currently uses or plans to use anti-retroviral therapy at any time during the study.
3. Subject plans to use any live vaccine during the study.
4. Positive pregnancy test at Baseline (Week 0).
5. Subject is considered by the investigator, for any reason, to be an unsuitable candidate for the study.
The DEB Pilot Study

The purpose of the current study is to test if weight change is differentially affected by consumption of foods that are identical in carbohydrate (i.e. sugar) but vary in food form over 1 month.

Dose: Participants are to consume 1 unit per day of the study product.

Diet: Participants will receive no instructions for other dietary modifications; i.e., they will not be told to modify what or how much other food they consume.

Eligibility Criteria:
Inclusion Criteria:

* BMI 20 - 40

Exclusion Criteria:

* Diabetes (Type 1 or 2)
* Prediabetes
* Active cancer
* Serious digestive disorders
* Other conditions that affect metabolism or body weight
* Uncontrolled Thyroid disorder (controlled = 6 months of medication)
* Unable to consume study foods
* Non- weight stable (more than 10 lbs weight gain or loss in last 6 months)
* Pregnancy
* Intentions of becoming pregnant in the next 2 months
* Women who have undergone partial hysterectomy with intact ovary function
* Current or past alcohol or drug abuse problem
* Allergy or intolerance to study product ingredients.
* Any other medical, psychiatric or behavioral factors that in the judgment of the Principle Investigator that may interfere with study participation or the ability to follow the protocol
Posterior Assisted Levitation of Dropped Nucleus

Dropping of nucleus is a common complication during cataract extraction by phacoemulsification. The investigators describe a new technique to retrieve the dropped nucleus by using an instrument called Pars Plana Levitator (ASICO-LLC, USA).

Upon recognition of posterior capsular rupture and dropping of nucleus phaco prob will be withdrawn, status of the nucleus will be visualized by posterior segment non contact lens and if the nucleus found floating, pars plana levitator will be introduced through temporal pars plana, its prongs will be opened and nucleus will be grasped between prongs. Nucleus will be lifted up till the anterior chamber where phacoemulsification will be performed in a usual manner and a posterior chamber intraocular lens will be implanted if possible in the remaining bag.

Eligibility Criteria:
Inclusion Criteria:

* Patients during cataract surgery whose nucleus will be dropped during phacoemulsification

Exclusion Criteria:

* Completely dropped nucleus touching the posterior pole
Comparison of Oral Ferrous Sulfate to Intravenous Ferumoxytol in Antepartum Iron Deficiency Anemia

Evaluate the extent to which treatment of iron deficiency anemia beyond 24-34 weeks' gestation of pregnancy with intravenous iron increases hemoglobin compared to oral iron. The investigators will test the hypothesis that pregnant women who are anemic in the second and third trimester are more likely to significantly increase their hemoglobin with intravenous iron as opposed to the usual standard of care, oral iron.


Eligibility Criteria:
Inclusion Criteria:

1. Pregnant women 18 years old and above
2. Hemoglobin \< 11 g/dL, serum ferritin \< 30 ug/dL and or transferrin saturation (TSAT) \< 20%
3. Between 24-34 weeks' pregnancy
4. Singleton pregnancy
5. Receiving prenatal care at Stanford/LPCH OB clinic and planning to deliver at LPCH
6. Hemodynamically stable

Exclusion Criteria:

1. Patients unable to give informed consent
2. Known allergy/hypersensitivity to IV iron
3. Inflammatory Bowel Disease or history of gastric bypass surgery
4. Dialysis-dependent Chronic Kidney Disease/ ESRD
5. Known Hemoglobinopathies such as sickle cell disease, beta-thalassemia, alpha thalassemia
6. Folate/Vitamin B12 deficiency
7. Known malignancy
8. Medication allergy to Tylenol (acetaminophen)
9. Hemoglobin above 12 or less than 7 g/dL
10. Patients with complex past medical histories which may include history of multiple medication allergies (greater than 2 allergies), connective tissue disorder, etc.
11. Diagnosis of placenta previa
Association Between Ankle Dorsiflexion and Frontal Projection Angle in PFPS

The study will investigate an association between ankle dorsiflexion and altered frontal knee kinematics during step down test in patients with PFPS.

Altered frontal and transverse plane hip kinematics during single leg weight-bearing tasks are thought to be important contributors to patellofemoral pain (PFP). The closed chain nature of single leg tasks means that hip kinematics can be influenced by more distal mechanics, such as foot pronation.

One of the often-studied distal movements theorized to cause PFPS is pronation of the subtalar joint. Pronation is a tri-planar movement that includes dorsiflexion, eversion, and abduction of the foot. Many studies have examined eversion characteristics of PFPS patients, but the dorsiflexion aspect of the movement has been shown to be a possible risk factor, restricting dorsiflexion was shown to increase medial knee displacement in young healthy adults. Conversely, when available dorsiflexion ROM is increased, medial knee displacement is thought to decrease.

Patients with PFPS were observed to have a decreased DFROM (dorsiflexion range of motion) as compared to normal individuals, though this topic has not thoroughly been investigated.

Eligibility Criteria:
Inclusion Criteria:

1. Anterior or retropatellar knee pain from at least 2 of the following Activities : (1) prolonged sitting; (2) stair climbing; (3) squatting; (4) running; (5) kneeling; and (6) hopping/jumping.
2. Insidious onset of symptoms unrelated to a traumatic incident and persistent for at least 6weeks.
3. VAS equal to or greater than 3.
4. Age of the subject 18-35 years to limit the possibility that PFPS over age 35 may have been complicated by arthritic changes, and also the subjects should have closed epiphyseal growth plates.
5. BMI under 30 kg/m2, both gender

For the control group, subjects were recruited to this study if they had:

1. No previous history or diagnosis of knee pathology.
2. No pain with any of the above-mentioned provocative activities.
3. No history of lower limb or spinal pathology.

Exclusion Criteria:

1. A history of any of the following condition: meniscal or other intraarticular pathologic conditions; cruciate or collateral ligament involvement.
2. A history of traumatic patellar subluxation or dislocation.
3. Previous surgery in the lower extremities within the 12 months prior to participation in the study.
4. Any balance impairments are secondary to a vestibular or neurological disorder or secondary to the use of medication.
5. Any lower limb bony/congenital deformity
Compare Polyethylene Glycol and Sodium Picosulfate Alone or Combined

Sodium picosulfate/magnesium citrate (SPMC) is generally better tolerated than PEG, its cleansing effect remains uncertain. While most studies showed SPMC was non-inferior to PEG, some studies reported that SPMC was less effective than PEG. To improve the bowel cleansing effect of SPMC, splitting the dose by using one sachet the evening before colonoscopy and the other sachet 4 to 5 hours before colonoscopy in the morning, has been proposed.Adding bisacodyl to the regimen also has been shown to be helpful. Some side effects, such as hyponatremia, dehydration and sleep disturbance, were reported to be more commonly associated with SPMC than with PEG.

To enhance the efficacy and reduce the side effects, two studies have evaluated the combination of SPMC and PEG, with conflicting results.The effect of combining PEG and SPMC should be best appreciated with head-to-head comparison with PEG and SPMC alone at the same time.

Therefore we designed this head-to-head comparison study for 2 L PEG, 1L PEG plus one sachet of SPMC and 2 sachet of SPMC, all with split-dose and the addition of 10 ml bisacodyl. Our hypothesis is the bowel cleansing effect of the combination regimen was not inferior to PEG alone. The tolerability, acceptability and side effects of the 3 regimen will also be evaluated.

Patients will be randomly assigned to either PEG, PEG plus SPMC or SPMC group, in a 1:1:1 ratio using a computer-generated sequence. The treatment allocation will be concealed and revealed by non-research medical personnel at the screening visit.


Eligibility Criteria:
Inclusion Criteria:

* Eligible patients will include those anticipated to undergo colonoscopy, aged 20 to 80 at the time of enrollment.

Exclusion Criteria:

* previous colon resection, gastrointestinal obstruction, ileus, intestinal perforation, toxic megacolon, active stage of inflammatory bowel disease, congestive heart failure (New York Heart Association Class III or IV),acute cardiovascular disease, uncontrolled arterial hypertension (systolic pressure \>170 mmHg, diastolic pressure\>100 mmHg), severe liver cirrhosis (Child-Pugh score C) or renal failure (creatinine clearance\<30 mL/minute) or any allergy to, PEG, or sodium picosulfate solution, phenylketonuria, and glucose-6-phosphate dehydrogenase deficiency.
A Study to Look at How Safe NNC0268-0965 is in Healthy People and People With Type 1 Diabetes

This study is investigating the safety and tolerability of the new medicine NNC0268-0965 (referred to as insulin 965), its concentrations in the blood and its effect on blood sugar for the treatment of diabetes. This first part of the study is conducted in healthy people, while there is a second part involving people with type 1 diabetes. The study will test how insulin 965 is tolerated by the participants' body, how it is taken up in the participants' blood, how long it stays there and how blood sugar is lowered. Participants will either get the new insulin 965 or placebo (an injection that does not contain active medicine) - which treatment you get is decided by chance. It is the first time that insulin 965 is tested in humans. Participants will get one injection of either insulin 965 or placebo under the skin of the left thigh. The study will last for about 5 weeks. Participants will have 6 clinic visits with the study doctor. People cannot be in the study if the study doctor thinks that there are risks for their health.


Eligibility Criteria:
Inclusion Criteria:

* Part 1 (healthy subjects):

  1. Male, aged 18-55 years (both inclusive) at the time of signing informed consent.
* Part 2 (subjects with type 1 diabetes mellitus):

  1. Male or female, aged 18-55 years (both inclusive) at the time of signing informed consent.
  2. Diagnosed with type 1 diabetes mellitus equal to or more than1 year prior to the day of screening.
  3. Male subject or female subject of non-child bearing potential. Non-child bearing potential being surgically sterilized (i.e. documented hysterectomy, bilateral salpingectomy or bilateral oophorectomy or being postmenopausal (as defined as no menses for 12 months without an alternative medical cause).
  4. Current daily total insulin treatment between 0.2 (I)U/kg/day and 1.2 (I)U/kg/day (both inclusive).
  5. HbA1c equal to or below 8.5%.
  6. Fasting C-peptide below 0.30 nmol/L.

Exclusion Criteria:

* (Part 1 and Part 2) 1. Male of reproductive age who or whose partner(s) is not using adequate contraceptive methods (adequate contraceptive measures as required by local regulation or practice). Adequate contraceptive measures include that the male subject uses a condom during intercourse and that the partner practices adequate contraception (risk of pregnancy must be lower than 1%). In addition, subjects must not donate sperm for the duration of the trial.