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NCT ID: NCT06250595 Recruiting - Leukemia Clinical Trials

European Rare Blood Disorders Platform (ENROL)

ENROL
Start date: July 1, 2022
Phase:
Study type: Observational [Patient Registry]

ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform. ENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data. Accordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when / where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies. The standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources. Moreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal. ENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670

NCT ID: NCT06119685 Recruiting - Multiple Myeloma Clinical Trials

IDP-023 as a Single Agent and in Combination With Antibody Therapies in Patients With Advanced Hematologic Cancers

Start date: October 25, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

This is an open label, Phase 1/2, first-in-human, multiple ascending dose, and dose-expansion study of IDP-023 administered as a single agent and in combination with or without interleukin-2 (IL-2), and with or without daratumumab or rituximab to evaluate the safety, tolerability and preliminary antitumor activity in patients with advanced hematologic cancers.

NCT ID: NCT06116032 Recruiting - Cancer Clinical Trials

Immune Profiling for Cancer Immunotherapy Response

Start date: October 3, 2022
Phase:
Study type: Observational

In patients clinically treated with FDA-approved immunotherapy the investigators will assess the predictive value of pre- and on-treatment 1) immune-methylation profiling across cancer types, and 2) immune-methylation profiling and cytokine profiling within cancer types.

NCT ID: NCT05873205 Recruiting - Blood Cancer Clinical Trials

Open-Label, Phase II Trial of Isatuximab for Patients With Refractory Immune Cytopenias After Allogeneic Hematopoietic Cell Transplantation

Start date: July 21, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this study is to find out whether isatuximab is an effective treatment for people who developed immune cytopenias/ICs after allogeneic hematopoietic cell transplant/allo-HCT.

NCT ID: NCT05709522 Recruiting - Blood Cancer Clinical Trials

Neurocognitive and Psychosocial Outcomes Among Pediatric Brain Tumor and Blood Cancer Patients

PBT
Start date: November 20, 2020
Phase:
Study type: Observational

This will be the first in-depth study to evaluate pretreatment and 12 months post-treatment, neurocognitive and psychological outcomes of children with brain tumor and blood cancer in Pakistan. The investigators will also determine the socioeconomic burden of pediatric brain tumors in low middle-income region and the association of micro RNA and protein markers with neurocognitive outcomes in PBT and blood cancer children. A prospective cohort study with a follow-up of 12 months at the Aga Khan University Hospital, Karachi, Pakistan and Jinnah postgraduate Medical Centre , Karachi, Pakistan will be conducted. After taking consent/ assent the investigators will recruit 80, 5-21 years old children with newly diagnosed brain tumors presenting with any stage, who have not undergone any treatment. Trained psychologist will assess the neurocognitive outcomes by the Slosson scale, Raven's progressive matrices and Wechsler Intelligence Scale for children (WISC V) and Wechsler Adult Intelligence Scale (WAIS-IV) tools. The Quality of life and depression of the children will be determined by PedQL and Revised Children's Anxiety and Depression Scale (RCADS) and hospital anxiety and depression scale (HADs) respectively . The financial burden of the disease on the family will be measured on a visual analog scale ranging from no burden (0) to very large burden (100) and the parents QoL and disrupted schedule, financial problems, lack of family support, health problems and the impact of caregiving on caregiver's self-esteem will be assessed by Pediatric Quality of Life Inventory PedQl (family module) and Caregiver Reaction Assessment (CRA) tools respectively. The serum micro RNA (mi-21, mi-10b and mi-210) and protein markers (GFAP, NSE and S100β) will be assessed by qRT-PCR and ELISA.

NCT ID: NCT05692713 Recruiting - Leukemia Clinical Trials

Polyomic Biomarker Verification in Adult Chronic Graft-Versus-Host Disease (ABLE3.0/CTTC2201)

Start date: July 1, 2023
Phase:
Study type: Observational [Patient Registry]

Chronic graft-versus-host disease (cGvHD) is one of the most serious complications following BMT (Bone Marrow Transplantation). cGvHD occurs when donor immune cells "attack" the tissues and organs of the person receiving the BMT. cGvHD can be difficult to treat once it is established leading to poor quality of life for recipients of a BMT. The goal of this study is to determine if, by using biomarkers, the investigators can predict which patients are at the highest risk of developing cGvHD after BMT, before cGvHD develops. The ABLE3.0 / CTTC 2201 study will validate and potentially refine the initial predictive biomarker algorithm developed from the original ABLE/PBTMC 1202 study (ABLE1.0), allowing clinicians the ability to pre-emptively predict their patient's future risk of developing both late-acute and chronic GvHD. This will provide the foundation for the later development of clinical trials aimed at reducing immune suppression quicker after transplant for low-risk patients (<10% risk) and justifying more intensive approaches such as pre-emptive treatments before the onset of chronic GvHD in high-risk patients (>45% risk).

NCT ID: NCT05646576 Recruiting - Multiple Myeloma Clinical Trials

Defining the Role of Palliative carE for Patients With Hematologic Malignancies Undergoing Adoptive CEllular Therapy

PEACE
Start date: December 30, 2022
Phase: N/A
Study type: Interventional

The goal of this study is to determine whether a palliative care intervention (PEACE) can improve the quality of life and experiences of participants with Lymphoma, Leukemia, or Multiple Myeloma receiving adoptive cellular therapy (ACT). After completion of an open pilot, participants will be randomly assigned into one of two study intervention groups. The names of the study intervention groups involved in this study are: - Palliative care (PEACE) plus usual oncology care - Usual care (standard oncology care) Participation in this research study is expected to last for up to 2 years. It is expected that about 90 people will take part in this research study.

NCT ID: NCT05450367 Recruiting - Blood Cancer Clinical Trials

Predictive Markers of Response and Toxicity in Patients With a Haematological Malignancy Treated With Immunotherapy.

PRONOSTIM
Start date: August 1, 2017
Phase:
Study type: Observational

Immunotherapies have substantially improved the prognosis of patients with haematological malignancies. While clinical trial data suggest durable complete response rates, markers associated with non-response to treatment are still poorly described. The identification of predictive markers using demographic, physiologic, biologic, immunologic data as well as patients' treatment history, might enable the optimization of therapeutic sequences and the reduction of treatment toxicity. This study aim to assess markers of toxicity and response following an immunotherapy in patients with a haematological malignancy using real life data. It will allow the development of clinical and therapeutic benchmarks to guide medical decisions in relation to the therapeutic strategies to be implemented for patients benefiting from real-life conditions, in addition to the results obtained in randomized studies.

NCT ID: NCT05190653 Recruiting - Multiple Myeloma Clinical Trials

Early Integration of Palliative and Supportive Care in Cellular Therapy

PALS_CT
Start date: April 8, 2022
Phase: N/A
Study type: Interventional

Research has shown that early palliative care in cancer care is associated with improved symptom management, better prognostic understanding, improved quality of life for patients and family caregivers, and even improved survival. Yet, in spite of the proven benefits of integration of palliative care in oncology, it has been well established that patients with hematologic malignancies and those undergoing cellular therapy (hematopoietic stem cell transplantation (HSCT) and chimeric antigen receptor (CAR) T-cell therapy) do not routinely receive palliative care. Most of the published research on the early integration of palliative care in oncology describes studies that have involved patients with solid tumours. To date, only one randomized trial examining the impact of integrated palliative care among patients undergoing HSCT has been published and there have been no studies examining the impact of integrated palliative care for patients undergoing CAR T-cell therapy. The American Society of Clinical Oncology recommends early palliative care for patients with advanced cancers or for those with high symptom burden. Patients with blood cancers experience high symptom burden and in the last 30 days of life, compared to patients with solid tumours, patients with blood cancers are more likely to die in hospital, have more intensive care unit admissions, have prolonged hospitalizations (>14 days), and pass away in an acute care facility. There is an urgent need to proactively address suffering throughout cellular therapy trajectories, even before treatment starts, so that patients and caregivers are not inevitably waiting for symptoms to arise before they can be addressed and to optimize quality of life for patients undergoing transplant as well as their family caregivers. PALS_CT will compare early palliative care to standard care for patients and their family caregivers undergoing HSCT or CAR T-cell therapy for blood cancers.

NCT ID: NCT04955938 Recruiting - Clinical trials for Myeloproliferative Neoplasm

A Study of Fedratinib With IDH Inhibition in Advanced-Phase, IDH-Mutated Ph-Negative Myeloproliferative Neoplasms

Start date: October 29, 2021
Phase: Phase 1
Study type: Interventional

The purpose of this research is to gather information on the safety and effectiveness of fedratinib (a drug called a "jak inhibitor" ) in combination with ivosidenib or enasidenib (two anti-cancer drugs). While all three drugs are FDA-approved for various conditions, the US Food and Drug Administration (FDA) has not approved the combination of these drugs for the treatment of rare blood cancers that present Isocitrate dehydrogenase (IDH) mutations, and therefore these drugs can only be given in a research study.