cyrilzakka/clinical-trials huggingface dataset:
Phase 1/2 Study of MRTX1719 in Solid Tumors With MTAP Deletion

This is a Phase 1/2, open-label, multicenter, study of the safety, tolerability, PK, PD, and anti-tumor activity of MRTX1719 patients with advanced, unresectable or metastatic solid tumor malignancy with homozygous deletion of the MTAP gene.

This first-in-human clinical trial will begin with an exploration of MRTX1719 dose and regimen. As potentially viable regimens are identified, Phase 1b expansion cohorts may be implemented to ensure sufficient safety experience, PK information, compare food effect and relative bioavailability between capsules and tablets, and early evidence of clinical activity are available to recommend Phase 2 regimens. In Phase 2, separate cohorts of patients by histological diagnosis and/or baseline characteristics will be evaluated for the clinical activity and efficacy of MRTX1719.

Eligibility Criteria:
Inclusion Criteria

* Histologically confirmed diagnosis of a solid tumor malignancy with homozygous deletion of the MTAP gene detected in tumor tissue or ctDNA.
* Unresectable or metastatic disease.
* Presence of a tumor lesion amenable to mandatory biopsy for pharmacodynamic evaluation at baseline and on-study unless Sponsor-confirmed as medically unsafe or infeasible.
* Age ≥ 18 years.
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
* Adequate organ function.

Exclusion Criteria

* Prior treatment with a PRMT5 or MAT2A inhibitor therapy.
* Active brain metastases or carcinomatous meningitis.
* History of significant hemoptysis or hemorrhage within 4 weeks of the first dose of study treatment.
* Major surgery within 4 weeks of first dose of study treatment.
* History of intestinal disease, inflammatory bowel disease, major gastric surgery, or other gastrointestinal conditions (eg, uncontrolled nausea, vomiting, malabsorption syndrome) likely to alter absorption of study treatment or result in inability to swallow oral medications.
* Cardiac abnormalities.
* Other protocol-defined Inclusion/Exclusion criteria apply.
Nerve Transfer After Spinal Cord Injuries

Nerve Transfer surgery can provide improved hand function following cervical spinal cord injuries

Current treatment strategies of acute cervical spinal cord injuries remain limited. Treatment options that provide meaningful improvements in patient quality of life and long-term functional independence will provide a significant public health impact.

Specific Aim: Measure the efficacy of nerve transfer surgery in the treatment of patients with complete cervical spinal cord injuries with no hand function. Optimize the efficiency of nerve transfer surgery by evaluating patient outcomes in relation to patient selection and optimal timing the the surgery.

Hypothesis: Peripheral nerve transfers in patients with spinal cord injuries will improve hand function and provide improvement in patient quality of life and functional independence.

Eligibility Criteria:
Inclusion Criteria:

1. 18-65 years of age
2. Informed Consent Document (ICD) signed by patient
3. Cervical spinal cord injury resulting in arm \& hand functional impairment, with at least preserved elbow function
4. International Classification of Surgery of the Hand in Tetraplegia (ICSHT) category 0 - 4
5. Patients with a stable American Spinal Injury Association (ASIA) grade of A, B, or C, or with a diagnosis of central cord syndrome, showing minimal to no evidence of functional improvement in motor examination after at least 6 months of non-operative therapy post-injury
6. Appropriate candidate for nerve transfer study
7. Willing and able to comply with the study protocol
8. \< 48 months from injury

Exclusion Criteria:

1. Active infection at the operative site or systemic infection
2. Any return or ongoing clinical recovery of distal motor function within 6 months after injury
3. Physically or mentally compromised
4. Currently undergoing long-term steroid therapy
5. Significant joint contractures and/or limitations in passive range of motion in the arm or hand
6. Active malignancy
7. Systemic disease that would affect the patient's welfare or the research study
8. Pregnant
9. Immunologically suppressed or immunocompromised
10. Significant pain or hypersensitivity
11. Previous or current injury preventing use of tendon transfers to restore upper extremity function
12. Affective disorder of a degree that would make outcome assessment and study participation difficult
13. History of brachial plexus injury or systemic neuropathic process
Comparison of Two Exercise Interventions to Improve Gait in Older Persons

The purpose of this research study is to compare two different exercise treatments for walking problems in older adults. The investigators want to determine if participation in the exercise programs for 3 months will improve walking abilities by improving balance and strength.

The purpose of this research study is to compare two different exercise treatments for walking problems in older adults. We want to determine if participation in the exercise programs for 3 months will improve walking abilities by improving balance and strength.

Adults aged 65 years or older, who are able to walk without the help of another person, are being asked to participate. If you are able to walk independently and you have medical clearance from your physician to participate in activities requiring low to moderate physical activity, you are invited to participate. We plan to enroll 40 older adults, all who have small problems with walking due to balance or strength, in this research study.

Eligibility Criteria:
Inclusion Criteria:

1. 65 years of age and older
2. Ambulatory without an assistive device or the assistance of another person
3. Usual 4 meter gait speed ≥1.0 m/s
4. Completes a figure-of-eight walk in \> 8.0 seconds

Exclusion Criteria:

1. Inability to participate in testing:

   1. persistent lower extremity pain that is present on most days of the week and crepitus, tenderness or enlargement of joints of the lower extremity (arthritis).84
   2. back pain that is present on most days of the weeks and interferes with walking and activities of daily living or back pain that increases with walking (lumbar stenosis)
   3. calf pain or cramping which worsens with walking and is relieved by rest (PAD)
   4. refusal to walk on a treadmill
2. Safety concerns:

   1. dyspnea at rest or during activities of daily living or use supplemental oxygen (CHF, COPD)
   2. any acute illness or medical condition that is not stable according to the approving physician
   3. resting systolic blood pressure ≥ 200 mm Hg or diastolic blood pressure ≥ 100 mm Hg or resting heart rate \> 100 or \< 40 beats per minute85
   4. diagnosed dementia or cognitive impairment defined as a MMSE score \< 24
   5. hospitalized in the past 6 months for acute illness or surgery, other than minor surgical procedures
   6. severe visual impairment with visual acuity \< 20/70 with best correction
   7. history of stroke
   8. fixed or fused lower extremity joints such as hip, knee or ankle
   9. lower extremity strength \<4/5 on manual muscle testing
   10. lower extremity amputation
   11. progressive movement disorder such as MS, ALS or Parkinson's disease
Caspofungin as Prophylaxis in High Risk Liver Transplantation Recipients

The aim of the study is to determine viable use of caspofungin in post-OLTx patients, and to demonstrate in particular the effectiveness, understood as the ability to reduce the incidence of invasive fungal infections, and to assess the ability to reduce the risk and incidence of side effects (toxicity) which may arise in transplant patients treated with other drugs, especially in individuals recognized as high risk (e.g. renal failure).

The prophylactic use of anti-fungal drugs is crucial in order to decrease the incidence of invasive fungal infections in transplantation patients.

Invasive fungal infections (IFI) ---organ-related or systemic infections--- are in fact one of the most important causes of morbidity and mortality in patients undergoing solid organ transplantation (respectively, 70% and 100%).

The rationale of the proposed study is to evaluate a new protocol for the prevention of IFIs through the use of a newly introduced anti-fungal, caspofungin (commercial name: Cancidas), to be used for primary prophylaxis of fungal infections post-OLTx and to compare to drugs already in use (eg. amphotericin B, fluconazole), until 21 days after liver transplantation.

The aim of this study is determine viable use of caspofungin in post-OLTx patients, and to demonstrate in particular the effectiveness, understood as the ability to reduce the incidence of invasive fungal infections, and to assess the ability to reduce the risk and incidence of side effects (toxicity) which may arise in transplant patients treated with other drugs, especially in individuals recognized as high risk (e.g. renal failure).

The possibility of reducing the risk of fungal infections in liver transplant patients (usually between 7 and 42%) is therefore an important clinical goal.

Eligibility Criteria:
Inclusion Criteria:

* patients shortlisted for liver transplantation
* negative pregnancy test for fertile female patients 7 days prior enrollment
* patients who can adequately communicate with study responsibles, who can understand and answer to protocol requirements

At least one of the following criteria:

* MELD score ≥25
* liver transplantation for acute liver failure
* liver re-transplantation
* fever without bacterial or viral infection
* biliodigestive
* re-laparatomy after LTx
* post LTx pancreatitis
* post LTx dialysis or renal insufficiency

Exclusion Criteria:

* Patients enrolled in other clinical trial or those having received other experimental drugs 4 weeks prior to enrollment
* Patients with a known fungal infection (based on the EORTC/MSG criteria)
* Patients with history of hypersensitivity to the drug, or other counterindications
* Patients with a diagnosed Severe Hepatic insufficiency (CTP \>9)
* Physical or hematochemical alterations
* Clinically relevant psychological alterations in the 2 weeks preceding enrollment such as to interfere, in the researchers opinion, with the goal of the study
* Patients being treated with Ciclosporin A

Subjects being removed from the trial shall be replaced.
Vascular Access Complications for Therapeutic Apheresis in Nephrology

The aim of this study is to describe vascular access complications which are used for therapeutic apheresis in Nephrology.

The aim of this study is to describe vascular access complications which are used for therapeutic apheresis in Nephrology.

Complications may include arterio-veinous fistula or graft (AVF or AVG) thrombosis with or without known stenosis; AVF or AVG or central veinous catheter (CVC) infections and CVC dysfunctions.

The investigators may enroll all patients receveing therapeutic apheresis for nephrologic indications in North East France.

Nephrologics indications include :

* acute or active antibody mediated rejection (ABMR),
* Goodpasture disease,
* ANCA vasculitis,
* thrombotic microangiopathy (TMA),
* Focal segmental glomerulosclerosis (FSGS),
* HLA or ABO desensitization for kidney transplantation.

Data inclusion period is from June 2020 to June 2022.

The aim of the study is to describe the proportion and type of complication in the population of patient receving therapeutic apheresis for nephrologic disease in the North East France. Te investigators will study the risk factors that are associated with this complications.

The second aim of the study is to describe the population of patient receving therapeutic apheresis in our unit and in our region.

Eligibility Criteria:
Inclusion Criteria:

* patients in nephrology receiveing apheresis treatment by filtration or double filtration

Exclusion Criteria:

* apheresis treatment for other indications
* centrifugation apheresis treatment
Assessment of Optune Therapy for Patients with Newly Diagnosed Glioblastoma Using Advanced MRI

The purpose of this prospective, nonrandomized controlled phase IV study is to compare standard and advanced MRI for their ability to predict response to Optune therapy.

Protocol Summary: Optune therapy is a newly approved treatment for patients with glioblastoma. This therapy demonstrated comparable outcomes for patients with recurrent GBM, in comparison to standard therapies, but better quality of life. More recently, in an interim analysis of 315 patients with GBM, adding Tumor Treating Fields (TTFields) to maintenance temozolomide chemotherapy significantly prolonged progression-free and overall survival. It also has demonstrated promise in other cancers such as pancreas, mesothelioma, ovarian and lung. However, in order to optimize the treatment regimen, and potentially predict which patients are most likely to respond, reliable ways of evaluation are necessary. Advanced perfusion and diffusion MRI methods have shown utility with other treatments such as bevacizumab. Preliminary data suggests the same might be true for the evaluation of Optune therapy. The team therefore proposes to determine the utility of these methods to predict response to Optune therapy. A successful result will have wide-ranging implications not only for the optimization of Optune treatment of brain cancer but also other cancers shown to benefit from this novel therapy.

Objective: To compare standard and advanced MRI for their ability to predict response to Optune therapy. The team's approach will be to obtain both standard and advanced MRI in patients with newly diagnosed glioblastoma undergoing adjuvant chemotherapy plus Optune therapy. The ability of standard and advanced MRI to predict response will be assessed. The hypothesis is that perfusion and/or diffusion imaging metrics can be used to predict response to and elucidate mechanisms of action for Optune treatment.

Eligibility Criteria:
Inclusion Criteria:

* Newly diagnosed glioblastoma (GBM), World Health Organization (WHO) grade IV.

Exclusion Criteria:

* Optune compliance \< 75%; they would be excluded from the final analyses.
* History of craniectomy or significant skull defect (contraindication to Optune).
* Active implantable medical device (e.g., deep brain stimulator (DBS), spinal cord stimulator, pacemaker, defibrillator, vagus nerve stimulator, programmable shunt).
* Karnofsky Performance Status (KPS) \< 60.
Biological, Prospective Study Evaluating the Dosage of Plasma Cytokines Including the FLT3 Ligand and IL6 of Patients Treated With Non-intensive Chemotherapy

There are 2 possible treatments for the treatment of Acute Myelogenous Leukemia (AML), high-risk myelodysplastic syndromes (HR-MDS) or chronic myelomonocytic leukemia (CMML): intensive curative chemotherapy , and for over-aged or co-morbid patients , non-intensive palliative chemotherapy with a hypomethylating agent (Azacytidine) associated or not with venetoclax.

Pro-inflammatory cytokines and in particular IL-6 (Interleukin 6) seem to play a key role in the chemoresistance of solid cancers and AML : it would be associated with a poor prognosis of AML , would promote the proliferation of leukemic blasts , and would promote the progression of MDS to AML .

In AML treated with intensive chemotherapy, researchers demonstrated that a particular kinetic profile of the FLT3 ligand and IL6 at day 22 could very significantly predict the survival of patients with AML .

It therefore seems interesting to study the plasma cytokine profiles in patients with AML, HR-MDS or CMML treated non-intensively, and to see if researchers observe the same prognostic correlation as during intensive chemotherapy.

Patients will be divided into 2 groups treated according to a non-intensive chemotherapy :

Group 1 : 40 patients treated with Azacytidine and Venetoclax +/- another molecule according to the following schedule:

* Azacytidine 75 mg/m² D1 to D7 SC
* Venetoclax: 400 mg/day orally in 1 dose between 14 and 28 days per cycle. The cycles will be 28 days long and will be carried out until relapse or death. Cytokine assays will be carried out on D1, D8, D15 and D22 of each cycle for 2 cycles.

Group 2 : 20 patients treated with Azacytidine +/- another molecule according to the following schedule:

• Azacytidine 75 mg/m² D1 to D7 SC The cycles will be 28 days long and will be carried out until relapse or death. The cytokine assays will be carried out on D1, D8, D15 and D22 of each cycle for 3 cycles.

The duration of follow-up for a patient is 12 months from day 1 of the first cycle.

Eligibility Criteria:
Inclusion Criteria :

* Age \>=18 years
* AML or SMD-HR or CMML in first line or in relapse receiving a hypomethylating agent +/- another molecule or a hypomethylating agent in combination with venetoclax +/- another molecule.
* Patient having agreed to participate in the study (information note signature) and having signed the biocollection consent

Exclusion Criteria :

* No social security or any other regime
* Pregnant women or patient unable to take contraception in case of fertility
* Breastfeeding women
* Minors
* Adults under guardianship, curators or safeguard of justice
TANDem-2: Closing the Gap to Interventions for TAND

The goal of this pilot study is to determine the feasibility, acceptability and impact of a brief behavioral intervention - the "Well-Beans for Caregivers" program, for adult caregivers of individuals with tuberous sclerosis complex (TSC) of any age. The main questions of the study are:

1. Can this innovative, ultra-brief caregiver well-being intervention be delivered feasibly to caregivers via an online modality?
2. How do the caregivers experience the program (acceptability)?
3. Does limited efficacy testing suggest the program has an impact on caregiver's well-being?

The investigators hypothesize that an innovative, ultra-brief caregiver wellbeing intervention developed for caregivers of children with developmental disabilities can be adapted as a universal caregiver wellbeing intervention in tuberous sclerosis complex (TSC), that it can be delivered feasibly in an online modality, and will result in changes in key caregiver wellbeing factors. To test these hypotheses, the researchers will adapt the 3-session 'Well-Beans for Caregivers' program through a participatory method with the TAND consortium (the TAND consortium is an international group of clinical and research professionals as well as family members living with individuals with TSC - TAND stands for TSC-Associated Neuropsychiatric Disorders - see tandconsortium.org). The input from the TAND Consortium will be augmented by members of TSCi (Tuberous Sclerosis International), the global network of TSC non-profit organizations.

For step 2 (pilot intervention), two specialist facilitators and four trainee observers will deliver the 3-session program using ZoomPro, a secure online platform to two groups of 10-12 caregivers. Caregivers are those who provide long-term, day-to-day support and care, and this group includes parents, partners, relatives, or other family members (not paid professional caregivers). These sessions will not be recorded to ensure that participants can join the program without feeling concerned about being recorded. Data collection will use a quasi-experimental pre-post-test design. Eligible and consented participants will therefore complete baseline data as 'pre-test' and then 'post-test' data after the 3-week intervention. Following this, they will complete 3-month and 6-month data collection. Caregiver wellbeing measures will only include open access and freely available measures. The program as well as data collection will be conducted in English.

Eligibility Criteria:
Inclusion Criteria:

* Adult caregiver of an individual with Tuberous Sclerosis Complex (TSC) of any age

Exclusion Criteria:

* Not an adult caregiver of an individual with TSC
Virtual Reality as a Treatment Tool for Chronic Neck Pain in Patients With Fibromyalgia

Fibromyalgia (FM) is a disease characterized by widespread pain, fatigue and cognitive alterations, among other symptoms, neck pain being the most frequent and debilitating. Virtual reality (VR) has emerged as a breakthrough for the treatment of such chronic conditions. The objective of this study is to evaluate the efficacy of VR in the treatment of chronic neck pain in patients with FM. Material and methods: Single-blind randomized clinical trial study. Patients with FM who meet the inclusion criteria will be recruited and will be distributed into three groups (GC, G1, G2). G1 (VR + cervical mobility exercises), G2 (cervical mobility exercises), control group (CG). The treatment will be administered twice a week for 4 weeks. Measurements of variables such as FIQ, EQ-5D, kinesiophobia, pain (VAS and algometer), range of motion (ROM), fatigue and adherence to treatment will be performed.

A follow-up is intended to be carried out 15 days and a month after the end of the study.

RESEARCH QUESTIONS: Can virtual reality combined with exercise improve the quality of life and impact of fibromyalgia? Can exercise performed with VR improve kinesiophobia, cervical range of motion, and pain? Will VR lead to improvements in patients' exercise functional capacity?

The aim is to collect a sample of more than 50 patients with fibromyalgia and cervical pain lasting more than three months. Variables to be collected include cervical range of motion (measured with goniometry), pain (measured with a visual analog scale and pressure algometry at two cervical points), kinesiophobia (TSK questionnaire), impact of fibromyalgia (FIQ), quality of life (EQ-5D), fatigue (BORG visual scale), disability caused by cervical pain (questionnaire), central sensitization (questionnaire), and functional capacity (measured with the "timed-up and go" physical test).

Recruitment was conducted through the Fibromyalgia and Chronic Pain Association of Mostoles. A one-month treatment will be administered with two sessions per week (with at least one day in between), totaling 8 sessions lasting approximately 30 to 40 minutes each. Measurements will be taken before starting the treatment, at the end of the treatment, 15 days after completion, and 30 days after completing the entire treatment.

Subjects will be randomized into three groups: a control group (CG) that will not receive treatment, Group 1 (G1) that will receive cervical mobility exercise combined with exercise using virtual reality (VR), and Group 2 (G2) that will receive cervical mobility exercise alone.

Eligibility Criteria:
Inclusion Criteria:

* Fibromyalgia condition
* present neck pain during the last 3 months

Exclusion Criteria:

* present any pathology that contraindicates the use of virtual reality
* being pregnant at the moment of the study.
* not accepting the informed consent.