cyrilzakka/clinical-trials huggingface dataset:
Long-term Safety and Efficacy of ESK-001 in Moderate to Severe Plaque Psoriasis

The objective of the ESK-001-018 long term extension is to evaluate the safety and efficacy of ESK-001 over time. The scientific questions it aims to answer are:

* How safe is taking ESK-001 long-term in people with moderate to severe plaque psoriasis?
* Does taking ESK-001 long-term reduce the severity of people's plaque psoriasis?

Patients will enter the long-term extension study following completion of one of the parent studies (ESK-001-016 or ESK-001-017) and will receive open-label ESK-001 twice daily for 24 weeks. After 24 weeks, the first 200 patients meeting at least PASI-75 clinical response will be randomly assigned to receive ESK-001 or placebo. At any point during this time, the patients losing the initial clinical response may return to the open-label ESK-001 treatment. Patients who complete Week 48 will return to open-label ESK-001 treatment and they will receive ESK-001 until the end of the study or discontinuation. All the remaining patients not meeting the entry criteria for the randomized withdrawal phase will continue to receive open-label ESK-001 for the remainder of the study.

Patients taking part in the study must be men or women aged at least 18 years old and have completed a previous (parent) study of ESK-001 in moderate to severe plaque psoriasis.

Patients must consent and agree to:

* ensure drug daily compliance until end of study or discontinuation.
* visit the clinic for checkups and assessments.
* provide blood and urine samples.


Eligibility Criteria:
Inclusion Criteria:

1. Males or females, age ≥18 years
2. Completed either of the two previous (parent) studies of ESK-001 (ESK-001-016 or ESK-001-017)
3. ESK-001 safety and tolerability were acceptable in the parent study
4. Women of childbearing potential (WOCBP) and males who are sexually active with WOCBP must agree to adhere to highly effective methods of contraception for the entirety of the study

Exclusion Criteria:

1. Pregnant, lactating, or planning to get pregnant during the study period
2. Any acute or chronic illness/condition or evidence of an unstable clinical condition that in the Investigator's judgment will substantially increase the risk to the patient if they continue to receive ESK-001 treatment in this extension study
3. Deemed by the Investigator to be inappropriate for the study or unable to comply with the protocol
G-CSF in Stimulating Peripheral Stem Cells for Autologous Stem Cell Transplant in Treating Patients With Chronic Phase Chronic Myeloid Leukemia in Complete Remission

RATIONALE: Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored until transplant.

PURPOSE: This phase I trial is studying the side effects of G-CSF in stimulating peripheral stem cells for autologous stem cell transplant in treating patients with chronic phase chronic myeloid leukemia in remission.

OBJECTIVES:

* Determine the feasibility and safety of harvesting adequate numbers of CD34-positive peripheral blood stem cells using filgrastim (G-CSF) in patients with chronic phase chronic myeloid leukemia in complete cytogenetic remission.
* Determine the safety of temporarily discontinuing treatment with imatinib mesylate and using G-CSF during the harvesting procedure, in terms of the percentage of Philadelphia chromosome (Ph)-positive cells before and after stem cell harvest, in these patients.

OUTLINE: Patients receive filgrastim (G-CSF) and then undergo apheresis for up to 5 days.

After completion of apheresis, patients resume treatment with imatinib mesylate off study. Patients may later undergo autologous peripheral blood stem cell transplantation, when deemed necessary.

PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study within 2 years.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Diagnosis of chronic phase chronic myeloid leukemia

  * In complete cytogenetic remission, confirmed by bone marrow biopsy within the past month
* Has been receiving imatinib mesylate for ≥ 3 months\* NOTE: \*Imatinib mesylate is held during the study harvesting procedure
* No myelofibrosis on bone marrow ≥ 3+
* Ineligible for or refused allogeneic stem cell transplantation

PATIENT CHARACTERISTICS:

Age

* Over 18

Performance status

* ECOG 0-1

Life expectancy

* Not specified

Hematopoietic

* WBC \> 3,000/mm\^3
* Platelet count \> 100,000/mm\^3

Hepatic

* Adequate hepatic function for stem cell transplantation

Renal

* Adequate renal function for stem cell transplantation

Cardiovascular

* Adequate cardiovascular function for stem cell transplantation

Pulmonary

* Adequate pulmonary function for stem cell transplantation

Other

* HIV negative

PRIOR CONCURRENT THERAPY:

Biologic therapy

* No other concurrent biologic therapy

Chemotherapy

* More than 4 weeks since prior chemotherapy
* No other concurrent chemotherapy

Endocrine therapy

* Not specified

Radiotherapy

* No concurrent radiotherapy

Surgery

* No concurrent surgery

Other

* No other concurrent experimental therapy
Outcomes Following Pulmonary Valve Replacement in Adults

One of the most common residual lesions in adult survivors of pediatric cardiac surgery is pulmonary valve disease, particularly regurgitation.

Multiple studies have demonstrated that placement of a pulmonary valve in such patients, results in improved ventricular function and resolution of symptoms. However, the optimal prosthetic valve for use in the pulmonary position has not been defined. There are essentially three alternatives available: the stented bioprosthetic valve, stentless bioprosthetic "tube" grafts used to replace the entire right ventricular outflow tract and mechanical valves.

All three of these valve options have been used in adults with congenital heart disease at Emory Healthcare and at Children's Healthcare of Atlanta at Egleston.

The goal of this study is to evaluate and compare the indications and short and mid-term outcomes for these alternative therapies.


Eligibility Criteria:
Inclusion Criteria: Adult patients who have received a pulmonary valve replacement for congenital heart disease at Children's Healthcare of Atlanta at Egleston or are in the Emory Healthcare System between 1976 and 2004.

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Exclusion Criteria:

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A Study of Motexafin Gadolinium for the Treatment of Chronic Lymphocytic Leukemia (CLL)

The purpose of this study is to determine if the drug motexafin gadolinium will be an effective treatment for patients who have chronic lymphocytic leukemia (CLL) that has come back after treatment or that has stopped responding to treatment.


Eligibility Criteria:
Inclusion Criteria:

* At least 18 years old
* Refractory or relapsed CLL
* ECOG performance status score of 0, 1, or 2
* Each patient must sign a study-specific informed consent form

Exclusion Criteria:

Laboratory values of:

* Platelet count \<30,000/uL
* AST or ALT \>2 x the upper limit of normal (ULN)
* Total bilirubin \>2 x ULN
* Creatinine \>2.0 mg/dL
* Chemotherapy, radiation therapy, immunotherapy, systemic corticosteroids, or systemic biologic anticancer therapy within 21 days before beginning study treatment
* Women who are pregnant or lactating
Phase-1 Study of Folinic Acid to Modulate MGMT Gene in Glioblastoma

O6-méthylguanine méthyltransférase (MGMT) is the main repair gene after DNA lesion induced by Temozolomide in combination with radiation therapy of Glioblastoma (GBM) in Stupp.R et al published regimen. In preclinical models, it has been demonstrated that MGMT methylation (which is silencing the DNA repair process) is achievable by folic acid. About half of the patients with operated GBM have an un-methylated MGMT gene status and therefore a poorer prognosis. A phase-1 dose escalation study is proposed with pharmacologic doses of folinic acid in combination with temozolomide and radiotherapy of operated GBM.

Glioblastoma treated by Stupp regimen (Temozolomide + radiation therapy) have a different outcome depending on the methylation status of MGMT gene: when the gene is unmethylated, the repair process is active and the prognostic poor. In pre-clinical models, it has been demonstrated that Folic acid could re-methylate the MGMT gene and therefore the repair process to radiation and temozolomide could be limited, allowing a better prognosis. The proposed phase-1 study will explore the safety and efficacy of escalated doses of oral Folinic acid concomitantly with Stupp regimen. To determine the MTD is the main objective of the study, then the toxicty profile, the RDP2 and the methylation process efficacy at the MGMT gene level.

Eligibility Criteria:
Inclusion Criteria:

* Operated GBM (complete or near complete resection)
* Un-methylated MGMT gene

Exclusion Criteria:

* Non operable GBM
* Methylated MGMT
Simulated Patient and AI-based Roleplay for History-taking

The goal of this clinical trial is to evaluate whether AI-based simulated patient training can improve clinical reasoning and history-taking skills in medical students. The main questions it aims to answer is:

Does GPT-based simulated patient training improve medical students' history-taking skills compared to traditional role-playing methods?

Participants will:

Participants in the intervention group perform medical history-taking conversations with an AI-simulated patient. Receive AI-generated structured feedback on their performance. The control group participated in role-playing exercises with instructors who acted as patients, receiving feedback after each session. Complete standardized assessments to evaluate clinical reasoning and decision-making skills.


Eligibility Criteria:
Inclusion Criteria:Medical education enrollment:

Students enrolled in a medical or health-related professional program.

Clinical coursework completion:

Students who have completed basic clinical coursework, including foundational topics in patient communication and clinical reasoning.

Age group:

Participants aged 18-30 years to reflect typical medical student demographics.

Language proficiency:

Adequate proficiency in the language used for the study (e.g., English) to effectively interact with the AI-simulated patient and understand feedback.

Willingness and availability:

Students who provide informed consent and are willing to participate in all required study activities, including training sessions and assessments.

Access to technology:

Ability to access and use the necessary technology, such as a computer and internet connection, to complete the simulated patient interactions.

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Exclusion Criteria:Non-medical background:

Students not enrolled in medical or related health professions programs. Students in early years of study who have not completed basic clinical coursework.

Technical limitations:

Inability to access or use the required technology (e.g., computer or online platforms).

Time constraints:

Inability to complete all required training and assessments within the study timeline.

Language barriers:

Insufficient proficiency in the language used for the study (e.g., English) to effectively communicate or understand the simulated patient interactions.

Health-related factors:

Conditions that may impair participation, such as significant hearing or vision impairment, or severe psychological conditions.

Prior participation in similar studies:

Students who have already participated in studies involving similar AI-based simulated patient training or feedback.

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Safety and Immunogenicity of Oral Cholera Vaccine in Kolkata

The purpose of the study is to confirm the safety and immunogenicity of the oral killed bivalent cholera vaccine in adult and pediatric volunteers in Eastern Kolkata, West Bengal, India.

Cholera remains to be a serious public health problem worldwide. In the mid-1980s following technology transfer from Sweden, Vietnamese scientists developed and produced an oral killed monovalent cholera vaccine for Vietnam's public health programs. A 2-dose regimen of this vaccine has been shown to be safe and efficacious. Subsequently, a bivalent vaccine was developed containing the newly emergent O139 V. cholerae. This vaccine has several advantages over the existing Swedish vaccine. It confers protection against the El Tor biotype in younger children, is considerably less expensive, does not require a buffer during administration and does not require strict cold chain requirements. However, this vaccine is not licensed for use in countries other than Vietnam.

Through IVI, an agreement between VABIOTECH in Hanoi and Shantha Biotechnics PVT, LTD in India has been reached that will make the bivalent vaccine available in India. A double-blind randomized phase III trial in a cholera-endemic area would be necessary to demonstrate the efficacy of this vaccine in other settings. This would pave the way for the introduction of the vaccine into the national immunization programme in India and the internationalization of this vaccine and licensure in other countries where it is needed. Prior to the phase III trial, a phase II study will be performed among adults and children.

Eligibility Criteria:
Inclusion Criteria:

* Healthy non-pregnant adults aged 18-40 years and children aged 1-17 years

Exclusion Criteria:

* Diarrhea during the past week
* Antibiotic and anti-diarrheal medicine use during the past week
* One or more episodes of diarrhea or abdominal pain lasting for 2 weeks during the past 6 months
* Abdominal pain, nausea, vomiting, loss of appetite or generalized ill feeling for the past 24 hours
* Pregnancy
Mid-HDF Randomized Controlled Study on Outcome

The purpose of this study is to determine whether mid-dilution hemodiafiltration is effective in the reduction of the crude mortality risk in patients who have been undergoing renal replacement treatment for less than 1 year. Patients will be randomized since the beginning of the study in two groups: standard HF dialysis and mid-dilution HDF.

It is a matter of fact that in the last decades the Chronic Kidney Disease (CKD) population has widely changed. In Italy, for example, more than 50% of the incident dialysis patients are more than 70 year old, with diabetic and hypertension being the major underlying diseases; moreover a great percentage of the patients starts dialysis with a burden of at least 1-2 comorbidities \[1\]. Online hemodiafiltration (online HDF) has been recently associated with better patient survival in comparison with standard hemodialysis in two large trials \[2,3\]; the overall relative risk of mortality was found to be approximately 33% lower in patients treated with online HDF \[2,3\]. These impressive results were not obtained anyway on the whole population, but in a sub-group analysis. A strong correlation was found between the total convective volume obtained and the mortality risk reduction; HDF was found to be significantly better than standard hemodialysis when a total convective volume of 19-22 L/session (in 4 hours sessions of post-dilution HDF) was achieved.

These results support the importance of the "adequate convective dose" concept in order to improve the patient outcomes especially in frail patients, as recently demonstrated by a large randomised control trial, the MPO study \[4\], comparing High Flux versus Low Flux dialysis in patients with plasma albumin levels equal to or less than 4 gr/dl ( as a marker of patient comorbidities) and, in a post hoc analysis, in diabetic patients \[4\].

Mid-dilution HDF is a variant of classical HDF combining simultaneous pre- and post.-dilution in order to maximise middle and large solutes removal.

The MILESTONE study would aim to fully demonstrate for mid-dilution HDF the significant mortality risk reduction observed in the recent mentioned studies.

Eligibility Criteria:
Inclusion Criteria:

* On a thrice/week RRT for at least 3 months
* Dialysis vintage \> 3 months
* Signed informed consent
* Blood flow \> 300 mL/min

Exclusion Criteria:

* On waiting list for living-donor transplant
* Residual diuresis \> 500 mL/day
* Inability, as judget by the investigator, to follow or understand the protocol instructions
* Active neoplastic disease
* Single needle treatment
* Patients with expectancy life lower than 6 months
* Inclusion to other studies
* Pregnancy
Assessing the Ability of the T-SPOT®.TB Test (IQ)

The primary objective of this clinical research study is to demonstrate performance of the normalized TB specific lymphocyte response (NTBSLR) in identifying patients with active TB disease. The secondary exploratory objective is to demonstrate that active TB cannot be identified using an antigen-to-mitogen ratio derived from an ELISA-based IGRA.

This study will enroll up to 202 subjects (assumes 15 % exclusion/drop-out rate). The target number of subjects is 75 active TB (recruited from subjects presenting at TB clinics) and 100 active TB excluded (recruited from subjects in the community).

All subjects enrolled in this study will be men or women, aged 18 years or older.

Duration: 1 year

Eligibility Criteria:
Subject Inclusion Criteria: Cohort 1A and 1B

* Must be at least 18 years of age
* Must be able to provide informed consent
* Must be able to provide a minimum of 10 mL of whole blood at each visit
* Must be T-SPOT.TB positive
* First visit suspect TB subjects with no prior history of TB diagnosis Subject exclusion criteria: Cohort 1A and 1B
* Negative in the T-SPOT.TB test
* Previous or pre-existing confirmed TB diagnosis
* On anti-TB treatment for less than 1 week\*
* Not meeting inclusion criteria

Subject inclusion criteria: Cohort 2A and 2B

* Must be at least 18 years of age
* Must be able to provide informed consent
* Must be able to provide a minimum of 10 mL of whole blood at each visit
* Must be T-SPOT.TB positive
* No prior history of TB diagnosis

Subject exclusion criteria: Cohort 2A and 2B

* Negative T-SPOT.TB test
* Previous or pre-existing confirmed TB diagnosis
* On anti-TB treatment
* Symptoms of active TB
* Not meeting inclusion criteria
Natural History Studies of Mucopolysaccharidosis III

The purpose of this study is to assess rates of decline in motor and cognitive functional measures, and to assess potential biomarkers, in order to identify potential outcome measure appropriate for use in therapeutic clinical trials.

Mucopolysaccharidosis (MPS) type III (Sanfilippo syndrome) is a group of four devastating genetic diseases that result in the abnormal accumulation of glycosaminoglycans in body tissues. In MPSIII the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death.

In anticipation for future therapeutic studies for MPSIIIA and MPSIIIB, we are performing a natural history study to identify the following:

1. Individual rates of decline in motor and cognitive function in a cohort of potential clinical trial patients
2. The natural history of outcome measures in order to assess their appropriateness as outcomes in an eventual trial
3. Baseline functional data in patients who will be potential candidates for an eventual trial
4. Biomarkers of disease progression over a 12-month interval, including changes in brain MRI and in cerebrospinal fluid

Patients in this study will need to come to Nationwide Children's Hospital in Columbus, Ohio, three times. At each of these three time points, cognitive outcome measures will be assessed: at baseline (visit 1), 6 months (visit 2), and at 12 months (visit 3). At baseline (visit 1) and 12 months (visit 3), an MRI and a lumbar puncture will be performed.

Eligibility Criteria:
Inclusion Criteria:

1. Age 2 years old or greater
2. Confirmed diagnosis of MPSIIIA or MPSIIIB by either of two methods:

   1. No detectable or significantly reduced NAGLU (MPSIIIB) or SGSH (MPSIIIA) activity in serum or leukocyte assay
   2. Genomic DNA mutation analysis demonstrating a homozygous or compound heterozygous mutations in the NAGLU (MPSIIIB) or SGSH (MPSIIIA) genes
3. Clinical history of or examination features of neurologic dysfunction.

Exclusion Criteria:

1. Inability to participate in the clinical evaluations
2. Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
3. Inability to be safely sedated in the opinion of the clinical anesthesiologist
Supportive and Supportive-Expressive Treatment for Depression

This study will assign patients to two types of psychotherapies in treating people with a major depression disorder, expressive versus supportive techniques, and will examine their ability to benefit from treatment based on their attachment orientation. This is a four month protocol, with a year follow up period, will compare patients receiving supportive-expressive treatment with either expressive focus or supportive focus.

One hundred patients suffering from major depression will participate in 16 sessions of supportive-expressive treatment. Patients will be randomized to one of two conditions: one that places a greater emphasis on supportive techniques, or one that places a greater emphasis on expressive techniques. These two conditions (supportive vs. expressive) hold the potential of either complementing or counter-complementing patients' attachment orientations (e.g., for a patient with higher levels of attachment anxiety, the supportive condition is complementary while the expressive is counter-complementary; the reverse is true for a patient with higher levels of attachment avoidance). Importantly, this study will employ multiple complementary methods, which will include session-by-session self-report alliance questionnaires from both patient and therapist, as well as a cognitive task assessing patients' relationship expectations, and behavioral observations of therapist-patient interactions. This study will be the first to utilize such a combination of methodologies in psychotherapy research and the first to examine the proposed mediation model. It will also be the first to manipulate the use of techniques in order to experimentally examine whether therapeutic techniques can be utilized to develop more efficient treatment models, based on the two transdiagnostic concepts of attachment and alliance. The findings will contribute both to our understanding of the relevance of attachment theory to psychotherapy research, and to the growing empirical literature on targeting transdiagnostic concepts (here, attachment and alliance) that cut across many disorders and treatment orientations. These transdiagnostic concepts can be utilized in the move towards tailoring existing psychological interventions to specific individuals according to their attachment orientations.

Eligibility Criteria:
Inclusion Criteria:

* Meeting MDD diagnostic criteria using the structured clinical interviews for DSM-V and scoring more than 14 on the 17-item Hamilton rating scale for depression at two evaluations (one week apart).
* If on medication, patients' dosage must be stable for at least three months prior to entering the study, and they must be willing to maintain stable dosage for the duration of treatment
* Age between 18 and 60
* Hebrew language fluency
* Provision of written informed consent.

Exclusion Criteria:

* Current risk of suicide or self-harm
* Current substance abuse disorders
* Current or past schizophrenia or psychosis, bipolar disorder, or severe eating disorder requiring medical monitoring
* History of organic mental disease
* Currently in psychotherapy