cyrilzakka/clinical-trials huggingface dataset:
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.

Spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) are genetic disorders that often result in progressive weakness and impaired function. Our recent findings suggest that novel machine-learning (ML)-based abstraction models may map noisy signals from foot-worn sensors (namely, instrumented insoles developed by the project team) into accurate and clinically relevant spatiotemporal and kinetic gait parameters. These gait parameters derived from instrumented insoles may serve as functional biomarkers to detect changes in real world function. All participants will be observed and measured while wearing the instrumented insoles in the lab and in real-life environments.

Eligibility Criteria:
Inclusion Criteria:

One of the following categories:

Genetic confirmation of disease (DMD, SMA) or healthy control Able to walk independently at least 25 meters

Cohort specific inclusion criteria include:

ongoing corticosteroids therapy or initiation of corticosteroid therapy in the previous 3 months for DMD stable dose of FDA approved SMN up-regulator therapy or in an open-label extension phase of a study treatment for at least 6 months for SMA or gene replacement at enrollment for SMA or DMD participants.

Exclusion Criteria

use foot orthoses or assistive devices for community ambulation or a mobility device for community navigation, use investigational medications intended for treatment of NMD within 30 days prior to study entry had an injury or surgery that would impact gait within the previous 3 months
Quality of Life Intermittent Catheter Study

The purpose of this study is to compare compact intermittent catheters with standard coated intermittent catheters with regard to quality of life, using the Intermittent Self-Catheterisation Quality of life Measure.


Eligibility Criteria:
Inclusion Criteria:

* Is at least 18 years of age
* Has given written informed consent
* Has neurogenic bladder dysfunction
* Has used coated intermittent catheter as primarily bladder emptying method for at least 6 months
* Is able to self-catheterise
* Is able to use SpeediCath® compact catheters
* If spinal cord injury subject, injury must have occurred more than 12 months ago
* Covered by Social security system

Exclusion Criteria:

* Has used SpeediCath® compact catheters (not including screening for this investigation)
* Is admitted to rehabilitation centre
* Subjects using primarily catheter sets
* Is pregnant or breast-feeding
ITP Registry and Accompanying Biospecimen Collection

The objective of this ITP registry is to collect clinical information, including biosampling, from consenting patients with a variety of ITPs at different points in the course of their disease.

Immune thrombocytopenia (ITP) is a rare hematologic disorder that can lead to a greater risk of bleeding or a prolonged bleeding time due to an autoimmune-mediated deficiency of platelets.

In recent years, new treatment options for patients with immune thrombocytopenia have emerged.

The results of published clinical studies on ITP can only be used for broad patient care to a limited extent, as they are designed for a patient population with clearly defined inclusion and exclusion criteria. Real world data collected from this registry will help to better understand the diagnosis and therapy of ITP patients in everyday treatment and to more effectively direct individual patients to optimal therapy, thus improving their outcomes.

By collecting biospecimens, this project will contribute new knowledge to the study of ITP through standardized, systematic, and high-quality collection and storage of patient samples and associated data.

The registry collects clinical data from patients diagnosed with ITP at defined points in the course of the disease. The Data collection includes a range of clinical measures, disease-related factors, treatment/treatment course and outcomes, complications during treatment and Qol, fatigue scoring and survival data (up to 5 years).

The data are collected prospectively. In addition, patients can be included retrospectively up to 12 months after the initial diagnosis if continuous documentation can be provided at the treatment centre. In both cases, a written declaration of consent is obligatory.

Eligibility Criteria:
Inclusion Criteria:

* Primary or secondary Immune Thrombocytopenia (ITP)
* Age ≥18 years
* signed declaration of consent

Exclusion Criteria:

* diagnoses that cannot be reconciled with the diagnosis of ITP (esp. heparin-induced thrombocytopenia, pregnancy-associated thrombocytopenia, pseudothrombocytopenia)
* no informed consent possible (this covers patients who are unable to understand the nature and scope of participation)
Study of V117957 in Overactive Bladder Syndrome

The purpose of this study is to evaluate the safety, tolerability and efficacy of V117957 in subjects with overactive bladder syndrome, compared to placebo.


Eligibility Criteria:
Key Inclusion Criteria include:

1. Female, age ≥18-70 years and capable of voiding independently. Able to comply with acceptable methods of contraception.
2. Has symptoms of overactive bladder including (OAB) urinary urgency and urinary frequency with incontinence for ≥3 months.
3. Willing to modify current OAB treatment regimen.

Key Exclusion Criteria include:

1. Significant stress incontinence or mixed stress/urge incontinence where stress is the predominant factor.
2. Urinary tract infection (UTI) within past 30 days, or history of recurrent UTI.
3. Hematuria associated with bladder malignancy or other significant pathology.
4. Had surgical procedure that affected bladder function.
5. Received intravesical therapy within past 12 months or had bladder hydrodistention within past 6 months.
6. Grade III/IV pelvic organ prolapse with/without cystocele or urethral diverticulum.
7. Clinically significant kidney disease or nephrolithiasis.

Other protocol-specific inclusion and exclusion criteria may apply.
Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients

Many human immunodeficiency virus (HIV)-positive children are afflicted with diffuse flat warts that have been recalcitrant to multiple treatments.The aim of this prospective study was to evaluate the efficacy, safety and tolerability of 15% glycolic acid lotion (NeoStrata) for treatment of flat warts in HIV-positive children in Botswana. Additionally, the investigators characterized the flat warts in this population, including Human Papillomavirus (HPV) type(s) and degree of immunosuppression within the patients.


Eligibility Criteria:
Inclusion Criteria:

* Male or female HIV-positive patients 7 years of age or older with at least 15 flat warts, diagnosed clinically by one dermatologist (RLM), on the face and/or dorsal hands were included in the study.

Exclusion Criteria:

* Key exclusion criteria included current use of potential wart treatments or other topical OTC products on the affected area(s).
The Effects of Neuraminidase Inhibitor Oseltamivir in Patients With Chronic Heart Failure

Heart failure (HF) is a complex syndrome with increasing incidence and high rates of mortality and hospitalization. Although inhibitors of angiotensin converting enzyme (ACE), β-blockers and aldosterone-receptor blockers have improved the treatment of heart failure, mortality of HF remains unacceptably high.

Recently, we identified a key metabolite N-acetylneuraminic acid (Neu5Ac) increased in the plasma of patients with heart failure. Also, elevated plasma Neu5Ac, independent of other traditional risk factors, is associated with poor prognosis in patients with HF in long-term follow up. Neu5Ac levels, the most common sialic acid in mammals, generated from sialylated glycoconjugates by neuraminidase. Neu5Ac and its regulatory enzyme neuraminidase play a key role in heart failure. We found neuraminidase inhibitor could reduce Neu5Ac levels and improve heart failure in mice model, providing opportunity for a novel therapeutic strategy in HF. Neuraminidase inhibitor oseltamivir is also an old anti-influenza drug. Using oseltamivir may be a new therapeutic strategy in heart failure.

Based on above information, we designed the randomized, open-label, blank-controlled study in patients with chronic HF to receive either oseltamivir or placebo, in addition to standard HF therapy to Identify the effect of oseltamivir on serum Neu5Ac level in patients with heart failure and assess the clinic outcomes of level in patients with heart failure using oseltamivir.


Eligibility Criteria:
Inclusion Criteria:

1. 18 of age or older, regardless of gender;
2. Patients with NYHA II-IV, LVEF less than 40%;
3. Levels of NT-proBNP more than 450 pg/ml
4. Willing to participate in this study and sign an informed consent, and receive follow-up contact for at least 6 months.

Exclusion Criteria:

1. Onset of acute myocardial infarction within the last 1 month;
2. Had cardiac surgery or onset of cerebrovascular accident within the last 6 months;
3. Plan to have cardiac transplantation or CRT, or already having CRT therapy;
4. Females with pregnancy or plan to have pregnancy;
5. Have participate in any other clinical trial within the last 3 months;
6. Severe neurological disorders (Alzheimer's disease, progressive stage of Parkinson's syndrome), disabilities of lower limbs or deaf-mute;
7. had a history of tumour, or precancerous lesions confirmed by pathological tests (ductal carcinoma in situ, cervical dysplasia, etc.);
8. had malignant tumours (by physical examinations, X-ray, type-B ultrasonic imaging or other methods), or had hyperplastic glands or adenoma which possess endocrine activity and affect cardiac or endocrine functions (pheochromocytoma, goitre, etc.);
9. The subjects who refuse to comply with the items of the research protocol;
10. The subjects who are not available to fulfil the follow-up plan (due to project management or other reasons), according to the judgement of researchers.
Measurement Variability of Liver Metastases From Neuroendocrine Tumors on Different Magnetic Resonance Imaging Sequences

The aim of our study is to assess variability of measurements of liver metastases from neuroendocrine tumors (NET) on different magnetic resonance imaging (MRI) sequences.

In this institutional review board-approved retrospective study from January 2011 to December 2012, all liver MRIs performed at our department in patients with proven liver metastases from NETs and with at least one measurable lesion according to Response evaluation criteria in solid tumors (RECIST 1.1) were included.

The aim of our study is to assess variability of measurements of liver metastases from neuroendocrine tumors (NET) on different magnetic resonance imaging (MRI) sequences.

In this institutional review board-approved retrospective study from January 2011 to December 2012, all liver MRIs performed at our department in patients with proven liver metastases from NETs and with at least one measurable lesion according to Response evaluation criteria in solid tumors (RECIST 1.1) were included. Up to two lesions were selected by an independent radiologist on T2-weighted images and marked by an arrow. Three reviewers (attending radiologist R1, fellow R2, senior R3) independently obtained long axis measurements of 135 metastases in 30 patients, on T2, diffusion-weighted imaging (DWI) with three b values (50, 400, 800), arterial, portal and late phases after gadolinium ), and during two distant separate sessions. Intraclass correlation coefficients and Bland-Altman plots were used to assess intra-and interobserver variability.

Eligibility Criteria:
inclusion criteria :

* Patients with liver metastasis from neuroendocrine tumor
* Patients with a magnetic resonance imaging in Reims university hospital
* Patients with at least one measurable lesion according to Response evaluation criteria in solid tumors
Safety and Efficacy Study of Tenalisib (RP6530) in Combination With Pembrolizumab in Relapsed or Refractory cHL

To characterize safety, tolerability and to establish the maximum tolerated dose (MTD) for Tenalisib in combination with Pembrolizumab in patients with cHL.


Eligibility Criteria:
Inclusion Criteria:

1. Age ≥18 years on the day of signing informed consent.
2. Histologically confirmed diagnosis of cHL.
3. Disease status as defined as.

   * Refractory patients who are naïve to anti-PD-1/PDL-1 therapy OR Relapsed after 3 or more lines of therapies; and are naïve to anti-PD-1/PDL-1 therapy OR
   * Patients currently on Pembrolizumab and achieve a less than complete response
4. Must have ECOG performance status of 0 or 1
5. At least one bi-dimensional measurable lesion with minimum measurement of \> 15 mm in the longest diameter.
6. Toxicities related to prior therapy must have returned to Grade 1 or less, except for alopecia.

   1. Adequate bone marrows, liver and renal function as assessed by the following laboratory requirements. Hemoglobin ≥8.0 g/dL (may not be transfused or treated with erythropoietin in preceding week to maintain or exceed this level)
   2. Absolute neutrophil count (ANC) ≥1,000/µL
   3. Platelet count ≥75,000/μL
   4. Total bilirubin ≤1.5 times the ULN (or ≤3 x ULN, if patient has Gilbert syndrome)
   5. ALT and AST ≤2.5 x ULN
   6. Serum creatinine ≤ 1.5 x ULN or CrCl \> 60 ml/min (Cockcroft-Gault formula)
7. Use of an effective means of contraception for women of childbearing potential and men with partners of childbearing potential
8. Provide written informed consent prior to any study-specific screening procedures.
9. Willingness and capability to comply with the requirements of the study.

Exclusion Criteria:

1. Patient receiving anticancer therapy (e.g. chemotherapy, biologic therapy, hormonal therapy, surgery and/or tumor embolization) ≤3 weeks or 5 half-lives (whichever is shorter) prior to C1D1,
2. Prior therapy with an anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CD137 or anti CTLA-4 antibody or any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathways)
3. Radiotherapy within the last 21 days prior to C1D1 (limited field palliative radiation is allowed if ≥ 14 days prior to C1D1);
4. Investigational drug therapy outside of this trial during or within 3 weeks prior to C1D1.
5. Patients with Allo-SCT on active GVHD or immunosuppression therapy within 3 months prior to C1D1.
6. Patient with active autoimmune disease or any medical condition requiring the use of systemic immunosuppressive medications .
7. Pregnancy or lactation.
8. Known clinically active CNS involvement.
9. Evidence of active Hepatitis B, active Hepatitis C infection (HCV) or cytomegalovirus (CMV) or known history of HIV.
10. Subjects with concomitant second malignancies
11. Patient with any active immune toxicity of Grade 1 or greater or any other severe or Grade 3 treatment-related adverse event.
12. History of Grade 4 anaphylactic reaction to monoclonal antibody therapy.
Colonoscopy Outreach for Rural Communities Aim 2

This study aims to test the effectiveness of a patient navigation program for increasing colonoscopy completion for colorectal cancer screening, among rural populations. The study will partner with geographically disparate primary care organizations to recruit patients aged 45-75 to the study. The patient navigation program will be delivered through a community organization. This project is critical in advancing our knowledge of the effectiveness of patient navigation for increasing colonoscopy in this patient population as well as for understanding factors that can support long term implementation and sustainability of effective interventions.

Colorectal cancer (CRC) is a leading cause of cancer death in the United States and its burden is greater in rural and low-income communities. Failure to complete recommended colonoscopy contributes to later-stage CRC diagnosis and worse CRC outcomes, which means timely and successful colonoscopy is a key opportunity to reduce CRC mortality. Using a Type 1 Hybrid Implementation-Effectiveness design, we will randomize 480-600 subjects from 6-8 primary care clinical organizations in a randomized controlled trial (RCT) to accomplish the following:

* Test the effectiveness of a patient navigation program delivered by a community-based organization, for increasing colonoscopy completion rates for colorectal cancer screening among rural patients.
* Assess implementation and sustainability of the patient navigation program from the perspectives of both community and clinical stakeholders.

Eligibility Criteria:
Inclusion Criteria:

* Aged 45-76.25 on the date of their referral for colonoscopy
* Referred for colonoscopy as part of colorectal cancer screening in the last 3 months. This includes patients referred for colonoscopy after an abnormal stool screening test (e.g., FIT/FOBT/Cologuard) and patients referred for colonoscopy as a primary screening test for colorectal cancer.

Exclusion Criteria:

* Deceased
* Colonoscopy procedure since colonoscopy referral
* Colonoscopy procedure scheduled less than or equal to 7 days from study enrollment date
* Colonoscopy procedure scheduled more than 9 months from colonoscopy referral date (if known at enrollment)
* Have diagnosis or past history of total colectomy or colorectal cancer (malignant neoplasm) at any time
* Has hospice
* Aged 66 and older living long-term in an institution for more than 90 consecutive days during the year prior to the query
Pharmacokinetic Study of STN1012600 Ophthalmic Solution in Healthy Adult Males Volunteers

To evaluate the safety and plasma pharmacokinetics of STN1012600 ophthalmic solution 0.002% (1 drop once daily for 7 days) in healthy adult male subjects.


Eligibility Criteria:
Inclusion Criteria:

* Subjects who can comply with scheduled visits and the examination and observation as specified in the clinical study protocol

Exclusion Criteria:

* Subjects have a disease and/or abnormal laboratory value which is considered inappropriate from safety evaluation perspective
* Subjects who are inappropriate as participants in this study in the opinion of the investigator or sub investigator
Assess Screening for Diabetic Retinopathy Using Retinal Camera in Family Medicine Clinic

The long-term goal for this study is to improve compliance of screening for diabetic retinopathy among subjects with diabetes. Researchers are also doing this research to determine the ability of appropriately trained family physicians to screen for and identify Diabetic Retinopathy using a retinal camera in addition to determining an overall patient perspective of the convenience and cost-effectiveness of retinal imaging within a primary care setting.


Eligibility Criteria:
Inclusion Criteria:

* Patient ≥ 18 years of age
* Diabetes Mellitus Type I or II
* Patient is willing and able to provide informed written consent

Exclusion Criteria:

• Known Diabetic Retinopathy
Effect of Teriparatide on Fracture Healing in Patients With Incomplete Atypical Femur Fractures

Atypical femur (thigh bone) fractures (AFFs) are uncommon low-trauma fractures which often occur without warning, are highly debilitating, and are slow to heal. As physicians learn more about them, AFFs are increasingly being identified prior to a total break. However, there's no evidence on the best way to treat these non-displaced AFFs. One option is teriparatide, an osteoporosis medication that can build new bone. Investigators will conduct a double blind randomized placebo controlled trial to examine whether teriparatide use in AFF patients with an incomplete break will help accelerate fracture healing and prevent surgical intervention. Co-primary outcomes include change in WOMAC scores from baseline to 12 months and number of participants requiring surgical intervention at 12 months. Investigators will recruit 60 women and men over the age of 30 who have experienced an incomplete AFF. Participants will be randomly assigned to either teriparatide (20 mcg/day) or placebo (using an identical injection pen) for up to 2 years. As AFFs are highly debilitating, a trial examining the use of Teriparatide in this population is urgently needed.


Eligibility Criteria:
Inclusion Criteria:

i. Female or male; ii. Over the age of 30; iii. Experienced an incomplete AFF that satisfies the diagnostic criteria as set forth by the American Society for Bone and Mineral Research (ASBMR) International Task Force on AFFs:

1. Atraumatic or low trauma incomplete stress fractures, defined as fractures sustained with minimal force, such as a fall from standing height;
2. From distal to the lesser trochanter to proximal to the supracondylar flare along the femoral shaft.
3. Radiographic confirmation of AFF features is required for a definitive diagnosis. Incomplete AFF features include cortical thickening, cortical beaking, and lucency line. Patients with cortical beaking without lucency line can be included.

Exclusion Criteria:

i. Peri-prosthetic fractures, ii. High trauma fractures, iii. Pathological fractures secondary to metastases or metabolic bone diseases other than osteoporosis, iv. Fractures that are not confirmed by radiological investigations, v. Contraindication to use of teriparatide such as:

1. pregnant or nursing mothers, or women who are planning on pregnancy within 1 year of completing teriparatide therapy,
2. hypersensitivity to teriparatide,
3. severe renal impairment,
4. multiple myeloma,
5. active cancer in past 5 years (other than non-melanoma skin cancer),
6. primary hyperparathyroidism,
7. hypercalcemia,
8. Paget's disease or other conditions that may increase the risk for osteosarcoma,
9. Prior radiation therapy involving the skeleton, vi. Concomitant use of other osteoporosis therapies including bisphosphonates or denosumab (except calcitonin).

AFF patients with complete fractures on the contralateral side or patients with secondary osteoporosis will not be excluded.
ROTEM Assessment of Modern Crystalloid, Hydroxyethyl Starch and Gelatin Effect on Coagulation

Modern crystalloid and colloid solutions are balanced solutions which are increasingly used in perioperative period. However, studies investigating their negative effect on whole blood coagulation are missing. The aim of our study was to assess the effect of modern balanced crystalloid and colloid solutions on whole blood coagulation in vivo using rotational thromboelastometry.

Blood samples were obtained from 30 patients during knee arthroscopy before and after administration of 500 ml of modern balanced crystalloid, hydroxyethyl starch (HES) or gelatin. Rotational thromboelastometry (EXTEM, INTEM and FIBTEM tests) were performed to assess negative effect of fluid solutions on whole blood coagulation.

Eligibility Criteria:
Inclusion Criteria:

* healthy patients undergoing knee arthroscopy

Exclusion Criteria:

* receiving antiplatelet drugs
Effect of Early Enteral Tube Feeding Nutrition With an Immune Enhancing Diet in Severe Burn Patients

Compare clinical and biological effects of two enteral tube feeding nutrition formula (immune enhancing diet versus polymeric diet)in severe burn hospitalized patients .


Eligibility Criteria:
Inclusion Criteria:

* Thermic burn from 20% to 80%
* 15 \< age \< 70 years
* written informed consent

Exclusion Criteria:

* diabetes mellitus
* corticoid or immuno-suppressive therapy
* HIV
* evolutive cancers
* pregnancy
* abdominal lesion
* hepatic or renal failure
Treatment of Upper Ureteric Stones

This study will be conducted to compare the success and complication rates of ureteroscopy, and shockwaves lithotripsy for treatment of upper ureteric stones ≤1 cm.

This study will be conducted to compare the success and complication rates of Flexible, Semi-rigid URS, and SWL approaches for proximal ureteric stones with size ≤1 cm.

Eligibility Criteria:
Inclusion Criteria:

* Radio-opaque stones
* less than or equal 1 cm

Exclusion Criteria:

* Active UTI
* Coagulopathy
* Pregnancy
* Ureteric stricture