cyrilzakka/clinical-trials huggingface dataset:
Treatment of LSCD With DM

Limbal Stem Cell Deficiency (LSCD) is a blinding disease that accounts for an estimated 15-20% of corneal blindness worldwide. Current treatments are limited. Traditional corneal transplantation with penetrating keratoplasty (PKP) is ineffective in treating these patients. Without a healthy population of limbal stem cells (LSC) to regenerate the corneal epithelium, standard corneal transplants will not re-epithelialize and will rapidly scar over or melt.

The limbal niche is the microenvironment surrounding the LSCs that is critical for maintaining their survival and proliferative potential under physiologic conditions. Extracellular signals from the microenvironment are critical to the normal function and maintenance of pluripotent stem cells. Identifying an effective niche replacement is thus an important focus of limbal stem cell research and critical for advancing treatments for LSCD.

Descemet's membrane (DM), an acellular, naturally occurring, basement membrane found on the posterior surface of the cornea, is a promising niche replacement. DM is routinely isolated and transplanted intraocularly with associated donor corneal endothelium for treatment of diseases like Fuchs' dystrophy and corneal bullous keratopathy that specifically affect DM and corneal endothelium. However, its application on the ocular surface has not been explored. DM is optically clear and highly resistant to collagenase digestion. This makes it very attractive as a long-term corneal on-lay and niche replacement on the surface of the eye. The anterior fetal banded layer of DM shares key compositional similarities with limbal basement membrane, which is a major component of the limbal niche. These similarities include limbus-specific extracellular matrix proteins such as collagen IV that is restricted to the α1, α2 subtypes, vitronectin, and BM40/SPARC. Of these, vitronectin and BM40/SPARC are known to promote proliferation of LSCs and induced pluripotent stem cells (iPSC) in culture.

Because of this, DM is a promising biological membrane for establishing a niche-like substrate on the corneal surface in patients with LSCD. The purpose of this pilot study is to investigate the clinical efficacy of using DM as a corneal on-lay to promote corneal re-epithelialization in partial LSCD.


Eligibility Criteria:
Inclusion Criteria:

Arm 1:

* partial LSCD (involving less than 75% of the limbus, or \<75% of the corneal surface)
* Visually significant (best-corrected visually acuity 20/100 or worse)

Arm 2:

* total/near-total LSCD with recurrent erosions or PEDs (involving more than 75% of the limbus, or more than 75% of the corneal surface)
* Visually significant (best-corrected visually acuity 20/100 or worse) PLUS
* Persistent epithelial defects last \>2 weeks despite maximal medical therapy OR
* Recurrent erosions occuring at least once every month

Exclusion Criteria:

* Pregnant women
* Prisoners (vulnerable population)
* Adults lacking capacity to consent (vulnerable population)
* Adults unable to sign consent due to non-english speaking or illiterate (vulnerable population)
Per-Operative Radiotherapy by Papillon +TM in Localized Breast Cancer

Phase II study; open recruitment, multicentrique. The aim of this clinical research is to evaluate faisability and toxicity of the per-operative radiotherapy using PAPILLON + TM device for localized breast cancers patients over 65 years.


Eligibility Criteria:
Inclusion Criteria:

* Patient with invasive ductal adenocarcinoma \<=2cm, evaluate on all radiological exams;
* Women aged 65 years or older (patients 65 years of age in the year may be included);
* Grade 1 or 2 unifocal adenocarcinoma, all index KI67, positive HR, negative HER2 status;
* T0 or T1, N0 radio-clinic;
* Operable patient with breast volume compatible with conservative surgery;
* Patient with prior malignancy or other concurrent malignancies are eligible, including bilateral breast cancer
* Patients who have been made aware of the information sheet and have given their written signed informed consent;
* Patients benefitting from social health insurance coverage

Exclusion Criteria:

* Age less than 65 years (except if 65 years obtained during the year)
* Patient with an exclusive in situ carcinoma
* Patient with lymphatic invasion / peri-nerve involvement / vascular emboli
* Patient with a lobular adenocarcinoma
* Patient with metastatic disease
* Multifocal tumor
* Patient with grade 3 or N+ disease
* N1 proved by ultrasound guided
* patient unable to express her consent
* Patient deprived placed under the authority of a tutor
* Female patients who are pregnant or breastfeeding
* Vulnerable patient: as defined in article L1121-5 à -8
Motiva Flora® Aesthetic Breast Recon® Clinical Study

The present study will be based on a hybrid breast reconstruction approach with initial skin expansion using the Motiva Flora® Tissue Expander followed by a serial fat grafting session and a final step that includes the placement of a permanent breast implant Ergonomix2®.


Eligibility Criteria:
Inclusion Criteria:

1. Genetically female, aged 18 years or older.
2. Subjects who had provided written informed consent form.
3. The participant needs tissue expansion as part of breast reconstruction treatment, which may include immediate reconstruction.
4. Clinical condition to allow reverse expansion breast reconstruction, at the investigator's discretion.
5. Sufficient fat in donor sites (abdomen, gluteus, hips, and thighs) per plastic surgeon criteria.
6. Complete radiotherapy and chemotherapy at least one year before surgery.
7. BMI between 18.5 and 30.0 (average classified weight).
8. Physical and cognitive capacity to understand and follow the surgeon's recommendations.
9. To be able and willing to comply with all study requirements, including attending follow-up appointments.

   Only Sub study participants
10. Provide additional consent to undergo an MRI with contrast.

Exclusion Criteria:

1. Current pregnancy or lactation, or full-term pregnancy or lactation at any point during the clinical investigation.
2. Abnormal hematological and biochemical values after chemotherapy.
3. High surgical risk according to the investigator.
4. Breast width larger than 18 cm
5. Tumor residues in or near the area where tissue expansion is performed.
6. Subjects with metastatic breast cancer
7. Significant Breast ptosis or poor skin quality
8. Participants who do not have adequate tissue at the intended site for expansion, at the surgeon's discretion, due to previous radiotherapy, ulceration, vascular involvement, history of impaired wound healing, or mastectomy scar deformity.
9. Inadequate chest wall tissue due to damage caused by radiotherapy, tight skin grafts, or radical resection of the pectoralis major muscle.
10. Current or previous infection in the area where the expansion occurs.
11. Any condition that impedes magnetic resonance imaging (MRI), including implanted metal device, claustrophobia, or other ailments that would prohibit MRI scan.
12. Presence of autoimmune diseases such as lupus or scleroderma, or immunocompromised participants due to immunosuppressive or steroid therapy.
13. History of silicone sensitivity.
14. Active smokers
15. Previous attempts of breast reconstruction
16. Subjects who, in the opinion of the investigator, are considered part of any vulnerable population
17. Subjects with affiliation to the Sponsor, sites or investigators, including relatives.
18. Participants who do not live in the procedure's country make it impossible to assist in follow-up visits.
19. Subjects who are participating in other investigation(s) which may affect the outcomes or ability to comply follow-up requirements of this study
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

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

Eligibility Criteria:
Inclusion Criteria:

1. Subject must have a diagnosis of Pompe disease based on documentation of one of the following:

   1. deficiency of GAA enzyme
   2. GAA genotyping
2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
3. 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:

1. Patient, whether male or female, is planning to conceive a child during the treatment program.
2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
3. 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.
4. Patient has received any gene therapy at any time.
5. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)
Patient Education to Improve Pain Management in Older Adults With Acute Musculoskeletal Pain: A Pilot Randomized Trial

This is a three arm pilot randomized trial. Patients will be assigned to:

1. Usual care
2. Video education in the Emergency Department (ED)
3. Video education in the ED plus phone follow-up at three days with geriatric pain management specialist for all patients with pain \>=4/10

This is a three arm pilot randomized trial. Patients will be assigned to:

1. Usual care
2. Video education in the ED (10 minute interactive video)
3. Video education in the ED plus phone follow-up at three days with geriatric pain management specialist for all patients with pain \>=4/10

Eligibility Criteria:
Inclusion Criteria:

* Age 50 and older; ED visit for acute musculoskeletal pain

Exclusion Criteria:

* Cognitively impaired; chronic pain (daily opioid use prior to onset of pain or pain symptoms more than one month); prison; injury or pain condition requiring hospital admission.
User Performance Evaluation of the INRatio® Prothrombin Time (PT) Monitoring System With a New INRatio Test Strip Designed for Low Sample Volume and Heparin Insensitivity

This multi-center study is designed to evaluate the ability of intended lay users (patients on oral anticoagulation therapy, OAT, or their caregivers) to 1) operate the INRatio Prothrombin Time (PT) Monitoring System utilizing the INRatio test strip newly designed for low sample volume and heparin insensitivity, and 2) obtain accurate results for the quantitative determination of International Normalized Ratio (INR) when self-testing using fingerstick capillary blood. Patients will be trained by their healthcare provider using the instructions for use and product labeling provided. The accuracy of the patient INR results will be assessed by comparison to the INR results obtained by the site's trained healthcare professional using the same INRatio system (from a separate fingerstick collected from the same patient at the point-of-care), and with the INR results obtained on venous plasma obtained from the same patient and analyzed at a central laboratory with the Sysmex CA-560 System INR reference method.


Eligibility Criteria:
Inclusion Criteria:

* Adults (18 years of age or older);
* Ability to read and speak English;
* Subjects on stable oral anticoagulation therapy with warfarin (at least 3 months);
* Target INR interval between 2.0 and 4.5
* Willing and able to provide written informed consent and comply with study procedures;

Exclusion Criteria:

* Hematocrit less than 25 or greater than 55%
* Lupus or antiphospholipid syndrome (APS)
* Already participated in this study
* A current INRatio PT Monitoring System User / Patient Self Tester
Middle School Teacher Module for a Brief Bullying Intervention Program

This study evaluates the feasibility and short-term outcomes of a teacher training designed as a companion module to a bullying bystander intervention (STAC) for middle school students in rural communities. The aim of this project is to provide a brief, low-cost intervention that can be easily disseminated as part of a school-based bullying prevention program to address this important public health problem.


Eligibility Criteria:
Inclusion Criteria:

* Being a teacher of an adolescent enrolled in grades 6, 7, or 8 in a participating middle school in Idaho.
* Speaks and reads English.
* Consents to participate.

Exclusion Criteria:

* Speaks and reads only a language other than English.
* Does not consent for participation.
A Study Comparing Tretinoin Cream 0.025% and RETIN-A® Cream 0.025% in the Treatment of Acne Vulgaris

The bioequivalence and safety of Tretinoin Cream 0.025% (Taro Pharmaceuticals U.S.A, Inc.) and RETIN-A® (Tretinoin) Cream 0.025% in the treatment of acne vulgaris.

A Multi-center, Double-Blind, Randomized, Placebo Controlled, Parallel-Group Study, Comparing Tretinoin Cream 0.025% (Taro Pharmaceuticals U.S.A) and RETIN-A® (Tretinoin) Cream 0.025% and Both Active Treatment to a Placebo Control in the Treatment of Acne Vulgaris

Eligibility Criteria:
Inclusion Criteria:

* Healthy male or non-pregnant female aged ≥ 12 and ≤ 40 years with a clinical diagnosis of acne vulgaris.
* Subjects who are 18 years of age or older (up to the age of 40) must have provided IRB approved written informed consent. Subjects 12 to 17 years of age, inclusive, must have provided IRB approved written assent
* Subjects must have a definite clinical diagnosis of acne vulgaris severity grade 2, 3, or 4 as per the Investigator's Global Assessment (IGA)

Exclusion Criteria:

* Female Subjects who are pregnant, nursing or planning to become pregnant during study participation
* Subjects with a history of hypersensitivity or allergy to tretinoin, retinoids, or any of the study medication ingredients.
* Subjects with the presence of any skin condition that would interfere with the diagnosis or assessment of acne vulgaris (e.g., on the face: rosacea, dermatitis, psoriasis, squamous cell carcinoma, eczema, acneiform eruptions caused by medications, steroid acne, steroid folliculitis, or bacterial folliculitis).
Comparative Responses to 15 Different Antidepressants in Major Depressive Disorder

Achieving results from RCTs with high internal and external validity is a major challenge within psychiatry due to the nature of psychiatric illnesses. The Investigators will conduct a "real world" naturalistic nation-wide population-based longitudinal register linkage study comparing long-term responses to all kinds of antidepressants in patients with major depressive disorder emulating a randomized trial.


Eligibility Criteria:
Inclusion Criteria:

All patients with a psychiatric contact (as inpatients or outpatients) in the period from 1995 to 2018 with a first main index diagnosis of a single depressive episode or recurrent depressive disorder (ICD-10 code: F32-F33.9) with an outpatient purchase of an antidepressant after the depression diagnosis (N=73.336).

Exclusion Criteria:

None.
Bridging Pediatric and Adult Biomarkers in Graft-Versus-Host Disease

This study is designed to collect longitudinal biological samples from patients after hematopoietic cell transplantation (HCT) cared for at multiple bone marrow transplant centers to validate biomarkers of both acute and chronic GVHD as well as for use in future unspecified research. The centers include Dana-Farber Cancer Institute and Boston's Children's Hospital, Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Fred Hutchinson Cancer Research Center, Texas Children's Hospital, Children's National Medical Center, and Indiana University Simon Cancer Center.

After informed consent is signed, this study will involve 1) collection of basic HCT data and clinical data available in the medical record and 2) providing blood (and saliva in occasional cases) samples for processing, storage, DNA extraction, and analysis (including a seven biomarker protein panel as well as future unspecified research purposes).

Pediatric and adult patients will be included (all adult patients will be from the Fred Hutchinson Cancer Research Center, the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, and the Indiana University Simon Cancer Center).

Primary Objective:

1. To confirm that ST2 alone or the seven-biomarker panel measured at initiation of GVHD therapy predict a) D180 post-therapy non-relapse mortality; b) D28 post-therapy non-response, and c) GVHD grade 1-4 onset D180 post-therapy non-relapse mortality.

Secondary Objective:

1. To demonstrate that ST2 alone or the seven-biomarker panel measured at day 14 or day 21 post-HCT (or a combination of these time points) predicts D180 post-HCT non-relapse mortality.
2. To demonstrate that ST2 alone or the seven-biomarker panel measured at initiation of GVHD symptoms/therapy diagnose acute GVHD as compared to other complications presenting with similar symptoms (drug rash, CMV, Clostridium enteritis).
3. To demonstrate that ST2 alone or the seven-biomarker panel measured at initiation of GVHD symptoms/therapy diagnose the severity of acute GVHD at onset and maximum.
4. To develop a repository of biospecimens linked to clinical data for future unspecified research.

Eligibility Criteria:
Inclusion Criteria: - All patients receiving an allogeneic hematopoietic stem cell transplant, cord blood transplant, bone marrow transplant, T cell depleted marrow, donor lymphocyte infusion (DLI), or donor cellular infusion (DCI) can be included.

Exclusion Criteria: - patients not receiving an allogeneic hematopoietic stem cell transplant, cord blood transplant, bone marrow transplant, T cell depleted marrow, donor lymphocyte infusion (DLI), or donor cellular infusion (DCI) will be excluded.
A Study of EOC237 in Patients With Advanced Solid Tumor

This research study is studying an investigational drug called EOC237 in Patients With Advanced Solid Tumor.

Part 1 Dose Escalation: an open, single/multiple dose, multi-center phase I study to evaluate the safety, tolerability and pharmacokinetics of EOC237 in Patients With Advanced Solid Tumor.

Part 2 Food influence research: to assess the effect of food on the pharmacokinetics of EOC237, by investigating the bioavailability following single dose administration under fed and fasted conditions.

Eligibility Criteria:
Inclusion Criteria:

* Advanced Solid Tumors for which standard curative or palliative measures do not exist or are no longer effective.
* Expected survival≥ 3 months.
* ECOG performance status 0-1.
* Good organ and marrow function.

Exclusion Criteria:

* Patients with a history of severe drug allergic reaction.
* Pregnant or lactating female subjects.
* Uncontrolled, significant intercurrent or recent illness.
* Corrected QT interval calculated by the Fridericia formula (QTcF) \> 480 ms per electrocardiogram (ECG)
* Concomitant use of certain medications
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography

This pilot study aims to assess subcellular muscle structure in patients with Duchenne X-linked progressive Duchenne muscular dystrophy (DMD) in comparison to healthy volunteers using multispectral optoacoustic tomography (MSOT). During MSOT, a transducer is placed on the skin similar to a conventional sonography and instead of sound, energy is supplied to the tissue by means of light flashes. This leads to a constant change of minimal expansions and contractions (thermoelastic expansion) of individual tissue constituents or molecules. The resulting sound waves can then be detected by the same examination unit.

Duchenne X-linked progressive Duchenne muscular dystrophy (DMD) is one of the most common progressive childhood muscle diseases with an incidence of 1 in 3500 male newborns and is associated primarily with decreased life expectancy. From the age of 4-5 years manifest motor problems in everyday life, typical signs of proximal muscle weakness, with lab-chemical increase of the muscle enzyme (creatinine kinase, CK). Within a few years, relevant muscle and tendon shortening leading to joint malpositions and instability, as well as scoliosis and loss of walking around the age of 10 are formed. Supportive therapies can not curatively affect complications and progression of the disease. Pathogenetically, there is a deficiency of dystrophin, a structural protein of the sarcolemma, which is caused by mutations (usually deletions) of the dystrophin gene (Xp21.3-p21.2). The result of dystrophin deficiency is a necrosis of muscle cells that are replaced by connective tissue and adipose tissue. Clinical scores (6-minute walk test, 6MWT) and MRI studies to characterize the degenerative changes of skeletal muscle in the early stages are available for the quantitative assessment of the disease progression as well as therapy effects, the significance of which is controversially discussed. However, the highly sensitive assessment of gene therapy effects (e.g., PTC 124) will become increasingly important in the future. Sensitive, non-invasive methods for the detection of early muscle degeneration and muscle function in the course are of great clinical and scientific importance. The purpose of this first pilot study is to investigate whether the differences in skeletal muscle composition of healthy volunteers and ambulatory patients with early stage DMD can be quantified and characterized using multispectral optoacoustic tomography (MSOT). This could in the future generate a completely new, non-invasive method to develop non-invasive biomarkers of disease progression or therapy response.

Eligibility Criteria:
DMD-Patients

Inclusion Criteria:

* Histologic or genetically proven DMD
* Age 3-10 years

Exclusion Criteria:

-

Healthy controls

Inclusion Criteria:

* Male
* Age 3-10 years

Exclusion Criteria:

* Suspected muscular disease/myopathia
* missing informed consent
The Therapeutic Effect of Dysthyroid Optic Neuropathy

Thyroid associated ophthalmopathy (TAO) is a common autoimmune disorder. The pathogenesis of TAO is unclear, and studies found that T cell, B cell and monocytes, macrophages and mast cells are located in the orbital tissue of TAO. Dysthyroid optic neuropathy (DON) is the most serious complication of TAO, which can cause blurred vision, color vision and vision function damage, and affects the quality of life. Investigation of the therapeutic effect of orbital decompression may provide some clues to make the policy at treatment of DON. We explore the therapeutic effect of orbital decompression in patients with DON in both eyes.


Eligibility Criteria:
Inclusion Criteria:

- Ability to perform visual fields and VEP examination Without any serious systemic diseases Without any surgery on eyes in six months

Exclusion Criteria:

- With any serious systemic diseases With any surgery on eyes in six months