Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

people enrolled in this arm will receive a typical haploidentical donor HCT

people enrolled in this arm will receive a coinfusion of cord blood unit in addition to a typical haploidentical donor HCT

HCT will be performed with a haploidentical donor

haplo-HCT

Haploidentical donor HCT will be performed with coinfusion of unrelated cord blood unit

haplo-cord HCT

Depei Wu, M.D., Ph.D.

Patients who have acute myeloid leukemia and will undergo haplo-identical donor hematopoeitic cell transplantation (haplo HCT) are potential candidates of this trial. Participants will randomized into two arms: Arm A will undergo a typical haplo HCT, while Arm B will receive an coinfusion of an unrelated cord blood unit (haplo-cord HCT) in addition to Arm A. Progression-free survival, overall survival, cumulative incidence of relapse and nonrelapse mortality will be recorded as endpoints.

Haplo HCT vs Haplo-cord HCT for Patients With AML

An Open-label, Multicenter, Randomized, Phase 3 Trial to Assess the Efficacy and Safety of Coinfusion With Unrelated Cord Blood Unit for Patients With Acute Myeloid Leukemia Undergoing Haploidentical Hematopoietic Cell Transplantation

estimated progression-free survival at 3 year

estimated cumulative incidence of nonrelapse mortality at 3 year

Zhejiang University

The Second People's Hospital of Huai'an

Soochow Hopes Hematology Hospital

Hygeia Suzhou Yongding Hospital

The First Affiliated Hospital of Soochow University

Myeloproliferative Disorders

Primary Myelofibrosis

Bone Marrow Diseases

Thrombocytosis

Bone Marrow Neoplasms

Chronic Myeloproliferative Disorders

CK0804 will be administered intravenously (IV) 100 million Treg Cells every 28 days up to 6 infusions.

CK0804 is a cryopreserved, allogeneic T-regulatory cell product that is manipulated to traffic to the bone marrow.

CK0804

Tara Sadeghi

Stacy Minor

Trisha Yassear, CCRC

Mehrdad Abedi, MD

Clinical Research Coordinator

Swati Goel, MD

Beatriz Raposo Corrandini, MSc

Mark Heaney, MD

Alleyne Genevieve

Lucia Masarova, MD

To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib

1. Safety Run-in

   The study will employ a 3+3+3 design to assess the safety and tolerability of the treatment based on treatment-limiting toxicities (TLTs) occurring up to 1 Cycle (28 days) after the first infusion.
2. Expansion

After a total of 9 participants completed 28 days and are evaluated for tolerability in the safety run-in phase, additional participants may be included in the expansion cohort in order to have approximately 24 evaluable myelofibrosis

This is a single-arm study consisting of a safety run-in (3+3+3 design) followed by an expansion cohort after completion of safety run-in data is evaluated for tolerability in the safety.

Leading in MPNs Beyond Ruxolitinib in Combo With T-Regs

Phase Ib, Open-label Study of Add on Therapy With CK0804 in Participants With Myelofibrosis, With Suboptimal Response to Ruxolitinib

* severe (grade 3 or 4) infusion-related toxicity within 24 hours (NCI-CTCAE V5.0) of exposure that does not resolve with standard of care treatment within 72 hours.
* regimen related death within 28 days

To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib

Cellenkos, Inc.

Symptoms and General Pathology

Immune System Diseases

Syndrome

Preleukemia

Myelodysplastic Syndromes

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Lymphoma

Hematologic Neoplasms

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Precancerous Conditions

Chronic Disease

Disease Attributes

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Chronic Graft Versus Host Disease

Lymphosarcoma

Chronic Myeloid Leukemia

NiCord® is a cryopreserved stem/progenitor cell based product comprised of:

1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF))
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells.

Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.

NiCord® (omidubicel)

Cord Blood Unit

Mitchell Horwitz, MD

This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.

Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion.

A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low stem cell dose can compromise the chances of engraftment and contributes to delayed kinetics of neutrophil and platelet recovery, as well as other transplant outcomes.

The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers of cells that have rapid and robust in vivo neutrophil and platelet producing potential to enable successful transplantation.

NiCord® is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of UCB. NiCord® utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of Hematopoietic Progenitor Cells (HPC) expanded in ex vivo cultures. The chief aim of the study is to compare the safety and efficacy of NiCord® single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy.

Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS

A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, UCB-derived, Stem and Progenitor Cells, vs. Unmanipulated UCB for Patients With Hematological Malignancies

The time to engraftment of neutrophils \>500/μl was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.

First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population

Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population

Platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count \> 20 × 10\^9/L with no platelet transfusions during the preceding seven days (counting day of engraftment as one of the preceding seven days) for the Intent to Treat Population

Gamida Cell ltd

Safety population (N=108). Serious Adverse Events (SAEs) \& Adverse Events (AEs) relate to safety population (SP) (N=108).

omidubicel is a cryopreserved stem/progenitor cell based product comprised of:

1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.

Omidubicel

Unmanipulated CBU(s)

NiCord® is a cryopreserved stem/progenitor cell based product comprised of:

1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF))
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells.

Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.

NiCord® (omidubicel)

NiCord® (Omidubicel)

Total

Age, Categorical

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Netherlands

Singapore

United States

Brazil

United Kingdom

Israel

Spain

Region of Enrollment

Kelly Myers, CRA

NiCord® (omidubicel) is a cryopreserved stem/progenitor cell based product comprised of:

1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) Non-cultured Fraction (NF) consisting of mature myeloid and lymphoid cells.

Cord blood unit: Cord blood unit 1-2 units

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Haplo HCT vs Haplo-cord HCT for Patients With AML	NCT03719534	Hematopoietic S... Haplo-identical... Cord Blood Unit Acute Myeloid L...	haplo-HCT haplo-cord HCT	18 Years - 60 Years	The First Affiliated Hospital of Soochow University
Haplo HCT vs Haplo-cord HCT for Patients With AML	NCT03719534	Hematopoietic S... Haplo-identical... Cord Blood Unit Acute Myeloid L...	haplo-HCT haplo-cord HCT	18 Years - 60 Years	The First Affiliated Hospital of Soochow University
Leading in MPNs Beyond Ruxolitinib in Combo With T-Regs	NCT05423691	Myelofibrosis	CK0804	18 Years -	Cellenkos, Inc.
Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS	NCT02730299	Hematological M... Acute Lymphobla... Acute Myelogeno... Chronic Myeloge... Myelodysplastic... Lymphoma Acute Leukemia	NiCord® (omidub... Cord Blood Unit	12 Years - 65 Years	Gamida Cell ltd

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