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Bone Marrow Failure clinical trials

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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: NCT04644016 Recruiting - AML Clinical Trials

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Start date: November 20, 2020
Phase: Phase 2
Study type: Interventional

This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.

NCT ID: NCT04232085 Recruiting - Clinical trials for Primary Immune Deficiency Disorder

Reduced Intensity BMT for Immune Dysregulatory and Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide

Start date: February 12, 2020
Phase: Phase 2
Study type: Interventional

This is a Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).

NCT ID: NCT04211714 Recruiting - Telomere Shortening Clinical Trials

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Start date: April 8, 2021
Phase: Phase 1
Study type: Interventional

This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.

NCT ID: NCT03622788 Recruiting - Clinical trials for Acute Myeloid Leukemia

Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant

Start date: August 8, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This phase I/II trial studies how well cytokine-treated veto cells work in treating patients with hematologic malignancies following stem cell transplant. Giving chemotherapy and total-body irradiation before a stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Cytokine-treated veto cells may help the transplanted donor cells to develop and grow in recipients without causing graft-versus-host-disease (GVHD - when transplanted donor tissue attacks the tissues of the recipient's body).

NCT ID: NCT03579875 Recruiting - Clinical trials for Myelodysplastic Syndromes

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Start date: November 13, 2018
Phase: Phase 2
Study type: Interventional

This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.

NCT ID: NCT03249129 Withdrawn - Trauma Clinical Trials

Identification of Autoantibodies and Autoantigens in the Cerebrospinal Fluid of Patients With Spinal Cord Trauma

TRAME
Start date: September 2018
Phase:
Study type: Observational

The purpose of the study is to detect the presence of autoantibodies and autoantigens in cerebrospinal fluid early (<48 hours) following spinal cord trauma. The study also aims to define the central or peripheral origin of autoantibodies by looking for their simultaneous presence at the blood level and to evaluate the prognostic value of the presence of autoantibodies within the cerebrospinal fluid, as well as on the initial clinical severity than on the recovery potential.

NCT ID: NCT03086252 Withdrawn - Leukemia Clinical Trials

Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study

Start date: July 24, 2017
Phase: N/A
Study type: Interventional

This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.

NCT ID: NCT02833805 Completed - Immunosuppression Clinical Trials

NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

Start date: September 2016
Phase: Phase 2
Study type: Interventional

Our primary objective is to determine if it is feasible for previously untreated severe aplastic anemia (SAA) patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide.

NCT ID: NCT01856582 Terminated - Bone Marrow Failure Clinical Trials

CD34+ Stem Cell Infusion to Augment Graft Function

Start date: October 2010
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine if infusing additional special donor cells will help to improve graft or immune function in previously transplanted children with immune deficiencies and bone marrow failures.