cyrilzakka/clinical-trials huggingface dataset:
An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families

This study aims to examine the real-life experience and impact of using emicizumab in a cohort of patients with haemophilia and inhibitors, who were prescribed emicizumab as part of the early access to medicine schema (EAMS),those who have been in clinical trials and those now receiving emicizumab as part of routine haemophilia care.

The Investigators also intend to capture the impact of emicizumab use on the lives of close family members (parents/carers/children/partners/siblings). Each participant and his family members will be deemed a study 'dyad'.

This is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice.

The study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and therefore offers insight into how these patients and families cope with haemophilia.

Emicizumab offers patients protection from bleeding with fewer injections, this should reduce treatment burden. While patient expectation is high (as evidenced by the conversations on UK haemophilia social media sites) there are risks that patients may forget to treat and thus experience bleeds. During clinical trials participants have been monitored closely and, through questionnaire completion, have described improvement in quality of life \[Mancuso et al, 2018, Oldenburg et al 2019\].

What is also apparent to clinical teams is the dramatic change to life experience of not just the patient but his family - the ability to travel, to plan attending events knowing that bleeds will not occur, even potentially to have another child because of the reduction in treatment burden, particularly the time needed for treatment and rehabilitation.

This therapy represents a substantial shift in the entire life experience of living with and managing haemophilia with inhibitors. As such, there is a need to look beyond the quantitative data collected in clinical trials and to assess the real impact of therapy on the everyday lives of patients and their families, gathered using qualitative research techniques.

Emicizumab has been available in the UK in clinical trials and as part of an Early Access to Medicines Scheme (EAMS) for non-trial eligible patients since spring 2018. It was recently licensed in the UK and is now available for routine clinical care for any person with haemophilia A and an inhibitor.

We hypothesise that patients and their carers/families will be excited about this new treatment option and that for most, if not all, the promise of improved care and quality of life will be a reality.

Eligibility Criteria:
Inclusion Criteria:

* Males
* A diagnosis of Haemophilia A with inhibitors
* Using emicizumab (prescribed by treating clinicians) in usual clinical care.

Exclusion Criteria:

* No history of a Factor VIII inhibitor
* Not currently being treated with emicizumab,
* Does not speak English (for the interviews)
* Does not consent to take part.
Gut Microbiota Metagenomics for Chronic Liver Disease

Gut microbiota modulation has effect on chronic liver disease

Chronic liver disease has been known as a disease of the liver which lasts over a period of 6-month. It consists of inflammation, hepatitis, cirrhosis, hepatocellular carcinoma.

Gut microbiota plays an important roles in the chronic liver disease. It can suppress the progression of the liver disease by moulation gut microbiota.

The investigators are still evaluating the effect of gut microbiota on the liver disease.

Eligibility Criteria:
Inclusion Criteria:

* Age 19+
* Normal : AST or ALT level \< 50IU/L, BMI \< 25
* Liver disease : AST or ATL level ≥ 50IU/L, BMI ≥ 25
* Patients hospitalized with chronic liver disease (viral hepatitis, alcoholic liver disease, non-alcoholic liver disease, cirrhosis, liver cancer)

Exclusion Criteria:

* Normal : AST or ATL level ≥ 50IU/L, BMI ≥ 25
* Liver disease : AST or ALT level \< 50IU/L, BMI \< 25
* If the patient refuses
Safety and Tolerability of Combined Treatment With Nilotinib and Ruxolitinib in CML and Ph+ ALL Patients

In this study it was the rationale to evaluate the safety and tolerability of the combined administration of nilotinib and increasing dose of ruxolitinib in patients with chronic myeloid leukemia and patients with Philadelphia positive acute lymphoblastic leukemia.


Eligibility Criteria:
Inclusion Criteria:

Patients of the first stratum must have chronic myeloid leukemia receiving nilotinib first-line therapy or receiving second-line or subsequent-line treatment with nilotinib.

Patients of the second stratum must have CML in AP/BC or relapsed/refractory Ph+ ALL, or be Ph+ ALL patients with MRD with or without prior nilotinib pretreatment;

Patients must have adequate end organ function, as defined by:

* Creatinine \< 2.0 x upper limit of normal (ULN)
* Total bilirubin \< 1.5 x ULN (\< 3.0 x ULN if related to disease or polymorphism, such as Mb. Gilbert)
* ALT and AST \< 2.5 x ULN (\< 5.0 x ULN if related to disease)
* Serum lipase ≤ 1.5 x ULN
* Alkaline phosphatase ≤ 2.5 x ULN (\< 5.0 x ULN if related to disease);

Patients must have the following electrolyte values within normal limits or corrected to within normal limits with supplements prior to the first dose of study medication:

* Potassium
* Magnesium
* Phosphate
* Total calcium (corrected for serum albumin);

Female patients of childbearing potential (WOCBP) must have a negative serum pregnancy test within 7 days before initiation of study drug. All WOCBP must use highly effective contraceptive methods throughout and during 3 months after study;

Patient has an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 1 for patients in CP, ≤ 2 for patients in AP/BC or with relapsed/refractory Ph+ ALL or with Ph+ ALL with MRD;

Patient has the following laboratory values within 7 days of starting study drug:

- For CML and Ph+ ALL patients: platelet count \> 75 x 109/L and ANC \> 1.0 x 109/L

Exclusion Criteria:

Patient must not have evidence of active malignancy other than the existing CML or ALL

Patient must not receive drugs that interfere with coagulation or inhibits platelet function, with the exception of aspirin ≤ 150 mg per day or low molecular weight heparin.

Patient must not have history of platelet dysfunction, bleeding diathesis, and/or coagulopathy in the 6 months prior to screening;

Patient must not require treatment with any strong CYP3A4 inducer or inhibitor

Patient must not have history of hypersensitivity to any of the study drugs or to drugs of similar chemical classes and their excipients;

Patients must not take other investigational drugs within 28 days prior to screening;

Patient must not be pregnant or lactating at screening and/or baseline;

Patient must not have impaired cardiac functions

Other protocol-defined inclusion/exclusion criteria may apply
Partial Versus Total Knee Replacement in Bicompartmental Gonarthrosis (Medial and Patellofemoral): Prospective Functional Assessment Study

The study is, in accordance with current legislation, definable as a prospective single-center interventional randomized study.

The aim of the study is to evaluate the post-operative recovery of the group A patient undergoing the partial knee replacement surgical procedure compared to the group B patient undergoing the total knee replacement surgical procedure through clinical/functional scores.

The primary outcome is evaluate the difference in score of the KSS questionnaire, at 1 month after surgery, completed by the patient of group A compared to the patient of group B.

The population consists of 48 adult patients with gonarthrosis divided into two groups:

24 patients Group A experimental group: partial denture surgical procedure

24 patients Group B control group: total denture surgical procedure with patellar resurfacing

The medial unicompartmental prosthesis procedure associated with patellofemoral is reserved for active patients suffering from bicompartmental gonarthrosis in which total knee replacement is to be avoided. The advantage for the patient, compared to total prosthesis surgery, consists in the less invasive nature of the surgical gesture, in the total preservation of the cruciate ligaments and the external compartment and therefore of the native joint biomechanics with the possibility of remote revision with total prosthesis from first implant.

Patients keep "their knee" which is coated and not replaced with advantages in terms of joint feedback and functionality in daily activities and sports practices. This study aims to compare two groups of patients suffering from bicompartmental gonarthrosis (medial and patellofemoral gonarthrosis): Group A undergoing a bicompartmental prosthesis surgical procedure; Group B undergoing a total denture surgical procedure with patellar resurfacing. The two groups will be evaluated with clinical and functional scores aimed at evaluating the outcome in the two surgical procedures.

The number and type of exams are as follows:

* Randomization to Group A or Group B (ad hoc)
* Knee replacement surgery (as usual)
* Compilation of KSS questionnaire at 1, 2, 6, 12 months of follow-up with clinical and functional scores (as a matter of practice)
* Questionnaires and scales (WOMAC, Tegner, VAS and patient satisfaction) at 2, 6, 12 months post intervention (as a matter of practice)
* Recording of any adverse events at 1, 2, 6, 12 months after surgery (as a matter of practice)

Eligibility Criteria:
Inclusion Criteria:

* Grade 3-4 sec Kellgren-Lawrence medial gonarthrosis
* Patellar symptoms
* Primary gonarthrosis
* Patients with ligament integrity
* Age between 40 and 85 years inclusive
* Signing of the Informed Consent and consent to cooperate in all study procedures.

Exclusion Criteria:

* Cognitive impairment
* Psychiatric disorders
* Neuromuscular disorders
* Age \> 85 years and \<40 years
* Lateral gonarthrosis grade 4 sec Kellgren-Lawrence
* Secondary medial gonarthrosis
* Revisions
* Minors
* Pregnant women self-declaration
Serial CT Scans in Fractionated Stereotactic Radiotherapy

This study aims at providing a quantitative evaluation of the accuracy of two different fixation systems, where one system is also evaluated with two different set-ups.

Stereotactic conformal radiotherapy (SCRT) combines the precision of the stereotactically guided tumor localisation and the radiobiological advantages of the fractionation. This implies an highly accurate repositioning of the isocenter of the cerebral target at the isocenter of the linac gantry and couch + 30 times. In the use of stereotactic conformal radiotherapy (SCRT) accuracy is the mainstay for the treatment of brain tumours. The reason are reduced safety margins around the tumour (or no margins) in order to reduce normal tissue dose.

This study aims at providing a quantitative evaluation of the accuracy of two different fixation systems, where one system is also evaluated with two different set-ups. The first approach will be based on the BrainLAB thermoplastic masque (the masque with (= standard fixation) and without a custom made bite-block), the second will use the BrianLAB relocatable frame with the bite- block and a home made fixation system.

Repositioning accuracy will be evaluated in a randomized manner patient-dependent and independent with the two fixation systems mentioned above.

The rationale is to determine the margins to define the planning target volume (PTV), it is necessary to know the set-up accuracy of the immobilization system. An overestimation of the PTV would lead to possible toxicity and an underestimation could lead to a geographical miss.

The main endpoint of this trial will be the accurate reproducibility of the fixation system evaluated by repeated CT scan.

Eligibility Criteria:
Inclusion Criteria:

* Clinical diagnosis of brain tumor
* Fractionated stereotactic radiotherapy

Exclusion Criteria:

-
A Phase 1 Study of ARQ 197 in Patients With Solid Tumors

This is a Phase 1, open label, dose escalation study of ARQ 197 in patients with advanced/recurrent solid tumors. The purpose of this study is to determine the safety, tolerability and recommended Phase 2 dose (RP2D) of ARQ 197.


Eligibility Criteria:
Inclusion Criteria:

* Patients who did not respond or are refractory to available therapy or for whom no standard effective systemic therapy exists.
* Karnofsky performance status (KPS) ≥ 70%, or Eastern Cooperative Oncology Group (ECOG) performance status 0 to 1
* Patients with adequate organ function

Exclusion Criteria:

* Anti-cancer chemotherapy, hormonal therapy, radiotherapy, immunotherapy, or investigational agents within 4 weeks prior to the first dose of ARQ 197
* Known symptomatic brain metastases
* Pregnant or breastfeeding
* Uncontrolled intercurrent illness
Cognitive Effects of Theta-burst Stimulation (TBS) in Suspected Non-Alzheimer Disease Pathophysiology (SNAP)

This is a pilot study to identify a homogeneous cohort of pathologically specific dementia with SNAP for clinical trial of brain stimulation.

The investigators apply the NIA-AA criteria to identify the individuals of biomarker-defined SNAP endophenotype. This is a prospective, randomized controlled clinical trial for the first two weeks, followed by an open-label trial for the next two weeks, and combined functional neuroimaging study of 18F-FDG-PET to further explore the potential mechanisms. A total of 20 SNAP dementia patients will be recruited for consecutive two years, and be subjected to iTBS for 5 daily interventions per week for four consecutive weeks. Cognitive evaluation will be performed before and immediately after TBS intervention, and 8 weeks after TBS. Data on functional neuroimaging will be also collected before and after TBS protocol.

Eligibility Criteria:
Inclusion Criteria:

1. Subjects aged 50-90 year.
2. Patients meet NIA-AA research criteria for suspected non-Alzheimer disease pathophysiology (SNAP) (Petersen, Smith et al. 1999; Jack, Bennett et al. 2018), and DSM-5 criteria for major neurocognitive disorder of dementia (Regier, Narrow et al. 2013).
3. Amyloid PET should ever be performed.
4. The CDR of subjects can be 0.5-2.

Exclusion Criteria:

1. Any subject has a definite diagnosis of epilepsy or history of seizure attack.
2. Current or past history of clinically significant neurological insults affecting brain structure or function like completed stroke, head injury or brain tumor. (cerebrovascular disorders, hydrocephalus and intra-cranial mass, documented by MRI; a history of traumatic brain injury or another neurological disease).
3. Any subject has clinically significant or unstable medical diseases including metabolic, renal, liver, lung or cardiovascular disorders including metabolic, renal, liver, lung or cardiovascular disorders.
4. Any subject has current alcohol or other substances abuse and/ or dependence within the recent one year, or prolonged history of major psychiatric disorder like schizophrenia, bipolar disorder, and previously prolonged substances abuse.
5. Any females who is pregnant or lactating.
6. General MRI, TMS and/or PET exclusion criteria including subjects who had received brain aneurysm surgery, or implanted pacemaker, mechanical valves, cochlear implant or other metal devices/ objects that are not MR compatible in the body.

Withdrawal criteria

1. Complications onset after intervention that affect efficacy and safety judgments.
2. New onset or progression of disease that may affect outcomes.
3. Use of other therapies or drugs during the intervention period to change cognitive functions.
4. Any subjects who are recognized as high risk of adverse effects by principle investigator.
Adjuvant Therapeutic Cancer Vaccine (AST-201, pUMVC3-hIGFBP-2) in Patients With Advanced Ovarian Cancer

The purpose of this phase 2 study is to assess the efficacy and safety for adjuvant therapeutic cancer vaccine AST-201 (pUMVC3-hIGFBP-2) in patients with newly diagnosed homologous-recombination proficient(HRP) advanced ovarian cancer (Stage III) after debulking surgery. Patients will receive AST-201 with rhuGM-CSF(Colony Stimulating Factor) or placebo with rhuGM-CSF in combination with standard adjuvant chemotherapy(Paclitaxel/Carboplatin).

The study will comprise a screening period of -28 Days prior to initiation of study treatment (Day 1); an enrollment period of 24 months; the treatment duration will be approximately of 5 months.

The study will evaluate whether the addition of AST-201/rhuGM-CSF to the standard adjuvant chemotherapy will extend the Progression Free Survival(PFS) rate. Survival follow-up will be performed every 3 months (±14 days) after the End of treatment (EOT) visit for 2 years after randomization and every 6 months (±28 days) thereafter until disease progression or death from any cause or withdrawal of consent whichever comes first. Survival follow-up visits will be conducted by telephone, in-person visit, or chart review. The end of study (EOS) is defined as 2 years after the date of last patient enrollment.

Eligibility Criteria:
Inclusion Criteria:

* Newly diagnosed stage III (FIGO classification) epithelial ovarian cancer including primary peritoneal cancer, fallopian-tube cancer
* Has received upfront surgery and optimally debulked(a residual tumor less than 1 cm)
* Can start adjuvant therapy within 6 weeks of debulking surgery
* Has Homologous Recombination Proficiency (HRP) tumor defined by FDA-approved testing
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
* Demonstrates adequate organ function.

Exclusion Criteria:

* Has a history of hypersensitivity or other contraindications to rhuGM-CSF
* Has a history of active malignancy ≤5 years prior to first administration of investigational drug except for adequately treated non-melanoma skin cancer or epithelial carcinoma without evidence of disease
* Is on immune suppression therapy or has a history of immune suppression therapy ≤4 weeks prior to the first administration of investigational drugs
* Has active or prior autoimmune disease or inflammatory disease
* Has active infectious disease including tuberculosis, hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
* Is pregnant or breastfeeding or expecting to conceive children within the projected duration of the study
Effect of Core Exercises on Patellofemoral Pain Syndrome

the goal of this interventional study is to Investigate the effect of core strengthening exercises on pain, strength, function, and balance in patients with PFPS.

Purpose: Patellofemoral pain syndrome is a frequent musculoskeletal disorder, which can result from core muscles instability that can lead to pain, function and altered dynamic balance. The objective of this study is to assess the effect of core muscle strengthening on pain, function, strength and dynamic balance in patients with patellofemoral pain syndrome.

Subjects and Methods: Forty-four patients with age ranging from 18 to 35 years with patellofemoral pain syndrome will be divided into study (N=22) and control (N=22) groups. Both groups will be given 6 weeks of conventional physical therapy program and an additional core muscle strengthening for the study group. The tools are being used to assess the outcome are 1- Visual Analogue Scale,2- handheld dynamometer,3-AKPS 4- BIODEX Balance.

Hypotheses:

It will be hypothesized that:

1. There is no effect of core strengthening exercises on pain in treating PFPS.
2. There is no effect of core strengthening exercises on muscle strength in treating PFPS.
3. There is no effect of core strengthening exercises on function in treating PFPS.

Eligibility Criteria:
Inclusion Criteria:

1. Patients of both genders with an age range of 18- 35 years old.
2. Gradual development of pain in the front of the knee lasting more than 12 weeks.
3. Patients with body mass index 18- 25 kg m2.
4. Pain is aggravated by at least 2 of the following activities: jumping, running, prolonged sitting, stair climbing, kneeling, and squatting.

   1. Positive patellar grinding test.
   2. Insidious onset of symptoms, unrelated to a traumatic damage.

Exclusion Criteria:

1. Traumatic patellar subluxation or dislocation.
2. A history of any of the following conditions: meniscal or other intra articular pathologic conditions; cruciate or collateral ligament involvement.
3. Any lower limb bony or congenital deformities.
4. Previous knee and hip joint surgeries.
5. Osteoarthritis in the ankle, knee, or hip joints.
6. Conditions that affect muscle strength such as diabetes mellitus or rheumatoid arthritis.
Real-world Study of UTD1 in Chinese Advanced Breast Cancer

To evaluate the efficacy and safety of UTD1 in patients with advanced breast cancer.


Eligibility Criteria:
Inclusion Criteria:

1. Patients aged over 18 years old.
2. Metastatic breast cancer included unresectable locally advanced breast cancer, de novo stage IV breast cancer, and recurrent metastatic breast cancer.
3. Plan to receive or has received UTD1 monotherapy or UTD1 based therapy
4. Available medical history.

Exclusion Criteria:

1.Incomplete medical history.
Resistance Training in Elderly

In the present study the investigators aim to determine whether protein supplementation can augment the response to a 12 week resistance training program in healthy elderly men.


Eligibility Criteria:
Inclusion Criteria:

* Living independently

Exclusion Criteria:

* Diabetes
* Cardiovascular disease
* Orthopedic limitations
* Any disorder known to compromise ability for resistance training
* History of resistance training in past 5 years