The goal of this research study is to determine whether it is safe and practical to give CK0801 (a cord-blood derived T-regulatory cell product) to patients with Guillain-Barré Syndrome (GBS). Researchers also want to determine the highest possible dose that is safe to be given and to learn if CK0801 may improve the symptoms of GBS. There will be three doses of CK0801 given during this study. A minimum of three patients will be treated in each dose level. The dose a patient receives is dependent on the timing of when they enter the study, as after each dose level is completed the following patients will receive the next highest dose level.

开始日期2022-11-01

申办/合作机构

Cellenkos, Inc.初创企业

NCT03773393 / Active, not recruiting临床1期

Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome

The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome.
Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.

开始日期2019-05-30

申办/合作机构

Cellenkos, Inc.初创企业

100 项与 CK-0801 相关的临床结果

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100 项与 CK-0801 相关的转化医学

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100 项与 CK-0801 相关的专利（医药）

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项与 CK-0801 相关的文献（医药）

2024-05-28·NEJM evidence

Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes

Article

作者: Masarova, Lucia ; Lyu, Mi-Ae ; Verstovsek, Srdan ; Huang, Meixian ; Flowers, Christopher ; Borthakur, Gautam ; Yilmaz, Musa ; Garcia-Manero, Guillermo ; Abbas, Hussein A ; Jain, Nitin ; Kantarjian, Hagop ; Ly, Christopher ; Sadeghi, Tara ; Pemmaraju, Naveen ; Issa, Ghayas C ; Zeng, Ke ; Kadia, Tapan M ; Daver, Naval ; Jabbour, Elias ; Cook, Robin ; DiNardo, Courtney D ; Parmar, Simrit

BACKGROUND:

An inflammatory bone marrow microenvironment contributes to acquired bone marrow failure syndromes. CK0801, an allogeneic T regulatory (Treg) cell therapy product, can potentially interrupt this continuous loop of inflammation and restore hematopoiesis.

METHODS:

In this phase 1 dose-escalation study of CK0801 Treg cells, we enrolled patients with bone marrow failure syndromes with suboptimal response to their prior therapy to determine the safety and efficacy of this treatment for bone marrow failure syndromes.

RESULTS:

We enrolled nine patients with a median age of 57 years (range, 19 to 74) with an underlying diagnosis of aplastic anemia (n=4), myelofibrosis (n=4), or hypoplastic myelodysplasia (n=1). Patients had a median of three prior therapies for a bone marrow failure syndrome. Starting dose levels of CK0801 were 1 × 10⁶ (n=3), 3 × 10⁶ (n=3), and 10 × 10⁶ (n=3) cells per kg of ideal body weight. No lymphodepletion was administered. CK0801 was administered in the outpatient setting with no infusion reactions, no grade 3 or 4 severe adverse reactions, and no dose-limiting toxicity. At 12 months, CK0801 induced objective responses in three of four patients with myelofibrosis (two had symptom response, one had anemia response, and one had stable disease) and three of four patients with aplastic anemia (three had partial response). Three of four transfusion-dependent patients at baseline achieved transfusion independence. Although the duration of observation was limited at 0.9 to 12 months, there were no observed increases in infections, no transformations to leukemia, and no deaths.

CONCLUSIONS:

In previously treated patients, CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone marrow failure. (Funded by Cellenkos Inc.; Clinicaltrials.gov number, NCT03773393.).

项与 CK-0801 相关的新闻（医药）

2024-11-07

·PRNewswire

Cellenkos Announces Oral Presentation at ASH Annual Meeting 2024 Highlighting Phase 1b Clinical Data of CK0804 in Myelofibrosis

- CK0804 is an off-the-shelf, allogeneic, non-HLA matched, CXCR4 enriched, T regulatory cell therapy product. - Multiple doses of CK0804 can be safely infused in myelofibrosis patients - CK0804 reduces symptom burden and induces spleen volume reduction in patients with myelofibrosis who have suboptimal response to prior lines of therapy HOUSTON, Nov. 7, 2024 /PRNewswire/ -- Cellenkos® Inc., a clinical-stage biotechnology company focused on developing allogeneic, off-the-shelf, T regulatory (Treg) cell therapies for inflammatory disease areas of high unmet needs and autoimmune disorders, today announced that its CK0804 Phase 1b study data in myelofibrosis has been selected for oral presentation at the 66th Annual Meeting & Exposition of the American Society of Hematology (ASH), December 7-10, 2024, in San Diego, California. The oral presentation will feature the safety and efficacy data from Cellenkos' Phase 1b trial evaluating CK0804 in patients with myelofibrosis who have suboptimal responses to prior therapies, including ruxolitinib. CK0804 is an off-the-shelf, allogeneic, non-HLA matched, CXCR4-enriched Treg cell therapy and is administered in fixed dose of 100 million cells infused every 28 days for 6 doses. CK0804 has shown early promise in addressing this high unmet medical need. Oral Presentation Details () Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Advancing MPN Care: Innovative Therapies and Clinical Breakthroughs in Myelofibrosis Date: Monday, December 9, 2024 Time: 5:00 PM (Presentation) Location: Manchester Grand Hyatt San Diego, Harbor Ballroom DEFG Publication Number: 999 Title: A Phase Ib, Open-Label Study of Add-on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib Submission ID: 211587 The study data to be presented demonstrates the safety and efficacy of CK0804 in patients with myelofibrosis who had previously failed standard treatments. Key findings from the trial include significant improvements in symptom burden, spleen volume reduction, blood transfusion dependence, and systemic inflammatory cytokine levels. Importantly, CK0804 was well-tolerated, with no significant drug-related adverse events observed, aside from one patient with a sulfa drug allergy who had an infusion reaction. Study Highlights Nine patients, with a median of two (range, 1-6) prior lines of treatment, were treated with CK0804. Improvements in fatigue and overall symptom burden were reported by all patients, with four out of six evaluable patients showing spleen volume reduction (SVR). Two patients who were transfusion-dependent showed a reduction in their monthly need for transfusions by the end of the treatment period. Longitudinal data indicates sustained benefits, including improvements in hemoglobin levels and inflammatory markers. Following the initial success of the Phase 1b trial, the Data Safety Monitoring Board has approved an expansion cohort to further explore the safety and efficacy of CK0804. This expansion includes an induction phase of four weekly doses, followed by five monthly doses, and active participant enrollment is ongoing (NCT05423691). About Myelofibrosis Myelofibrosis is a rare, chronic, and progressive blood cancer that causes scar tissue to form in the bone marrow, disrupting the production of normal blood cells. Patients with myelofibrosis often experience debilitating symptoms such as fatigue, spleen enlargement, and night sweats. Approximately 16,000 to 18,500 people in the U.S. are living with myelofibrosis, and those who fail to respond adequately to current treatments including ruxolitinib, face limited options and a poor prognosis. Inflammation is a key driver for disease pathogenesis and progression in myelofibrosis. About CK0804 CK0804 is an investigational, allogeneic, off-the-shelf Treg cell therapy that leverages the CXCR4/CXCL12 axis to suppress inflammatory cytokines implicated in myelofibrosis pathogenesis. CXCR4 enriched Tregs home faster to bone marrow compared to unmanipulated Tregs. Derived from clinical-grade umbilical cord blood and manufactured using Cellenkos' proprietary CRANE® process, CK0804 does not require HLA matching, making it an ideal therapeutic option for patients in need of prompt intervention. The therapy is administered intravenously and can be infused in an outpatient setting. About Cellenkos, Inc. Cellenkos is a clinical-stage biotechnology company based in Houston, Texas, focused on developing off-the-shelf Treg cell therapies for rare inflammatory diseases and autoimmune disorders. The company's portfolio includes four clinical-stage candidates: CK0801 for aplastic anemia: durable transfusion independence. CK0802 for COVID-19 ARDS: survival benefit CK0803 for amyotrophic lateral sclerosis (ALS): slowing of ALSFRSR score decline and decrease in neurofilament CK0804 for myelofibrosis: decrease in symptom burden and spleen volume reduction Cellenkos' Treg therapies, derived from umbilical cord blood, are designed to provide powerful anti-inflammatory effects and long-lasting immune modulation without the need for donor matching. The company is committed to advancing the development of these promising therapies to improve the lives of patients with rare and underserved conditions. For more information, please visit . Contact: Ting Huang [email protected] SOURCE Cellenkos, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

细胞疗法临床1期ASH会议临床结果

2024-04-01

·PRNewswire

Cellenkos® enters into Sponsored Research Agreement with Icahn School of Medicine at Mount Sinai, New York.

Research exploring CK0804 (CXCR4-enriched, allogeneic, cord blood-derived T-regulatory cells) for treatment of myelofibrosis patients. HOUSTON and NEW YORK, April 1, 2024 /PRNewswire/ -- Cellenkos®, a clinical stage biotech cell therapy company focused on treating autoimmune diseases and inflammatory disorders, today announced that it has entered into a sponsored research agreement with the Icahn School of Medicine at Mount Sinai, New York, to explore the application of CK0804, CXCR4 enriched, allogeneic, cord blood T regulatory (Treg) cells, for treatment of myelofibrosis. This research will be conducted under the guidance of Ronald Hoffman, MD, Albert A. and Vera G. List Professor of Medicine and Director of the Myeloproliferative Disorders Research Program at The Tisch Cancer Institute- Mount Sinai. "We are very excited by our collaboration with Mount Sinai and eagerly anticipate gaining a deeper understanding of mechanisms of CK0804 Tregs work in myelofibrosis, especially to decrease inflammation," said Tara Sadeghi, Chief Operating Officer, Cellenkos Inc. "CK0804 is already in phase 1 clinical trial to examine its safety and efficacy in patients with myelofibrosis who have suboptimal response to the JAK2 inhibitor, ruxolitinib. We are particularly intrigued by the possibility of using CK0804 in treatment naïve myelofibrosis patients as well as in combination with other approved therapies including ruxolitinib, momelotinib, pacritinib and fedratinib." "We are eager to find out if this novel cell therapy product has the potential to transform the care of myelofibrosis patients," said Dr. Ronald Hoffman, principal investigator, "We will investigate the role of Treg cells in the pathogenesis of the myeloproliferative neoplasms and examine the effectiveness of CK0804, especially in patients with low blood counts." CK0804 is a novel allogeneic, CXCR4 enriched, Treg cell therapy product that utilizes Cellenkos' proprietary CRANE® technology to generate disease specific products. CK0804 engage with CXCL12 ligand and preferentially home to the inflamed bone marrow. When compared to control, CK0804 significantly decrease TGFβ1 and TGFβ2, in vivo. The ongoing, multicenter, first-in-human, Phase 1b trial, shows safety of multiple doses of CK0804 in myelofibrosis patients in the ambulatory setting as well as improvement in their blood transfusion requirements, spleen volume reduction and symptom burden. The ability to administer CK0804 cells in ambulatory setting without requiring lymphodepletion or immune suppression, positions this potentially lifesaving treatment to be made available to patients in the community setting. The trial is open for accrual at Albert Einstein Hospital, Bronx, NY, MD Anderson Cancer Center, Houston, TX and University of California Davis, Sacramento, CA. ClinicalTrials.gov ID NCT05423691. Dr. Ronald Hoffman serves as a paid consultant for Cellenkos. About Cellenkos®, Inc. Cellenkos is a clinical-stage biotechnology company located in Houston, Texas, USA, dedicated to the development and commercialization of the allogeneic, "off-the-shelf" cell-based products for the treatment of rare inflammatory diseases and autoimmune disorders. Cellenkos' T regulatory cells (Tregs) are derived from umbilical cord blood and as such are naïve, bonafide suppressor cells that do not require HLA or ABO matching and resolve inflammation through multiple direct and indirect mechanisms. Cellenkos' proprietary CRANE® technology platform tailors Tregs to home to target tissue. Cellenkos' current clinical portfolio includes CK0801 (bone marrow failure); CK0802 (COVID associated acute respiratory distress syndrome); CK0803 (Amyotrophic Lateral Sclerosis) and CK0804 (myelofibrosis). For more information, please visit . Contact: Stacy Minor [email protected]. SOURCE Cellenkos, Inc.

细胞疗法临床1期

2024-02-19

·PRNewswire

Cellenkos Approved to Proceed with CK0803 Neurotrophic T regulatory Cell therapy to Treat Second Cohort in ALS Trial

DSMB approval to proceed to second cohort of REGALS trial (T Regulatory Cells for ALS). Ongoing Phase 1/1b study evaluating safety and efficacy of CK0803 (Neurotrophic T regulatory cells) HOUSTON, Feb. 19, 2024 /PRNewswire/ -- Cellenkos® today announced encouraging safety data from its innovative CK0803 neurotrophic T regulatory (Treg) cell therapy, being developed to help treat individuals with Amyotrophic Lateral Sclerosis (ALS). The company can now begin treating the second group of patients in the trial (clinicaltrials.gov: NCT05695521) The company was given the green light to move ahead following a review of safety data by the Data Safety Monitoring Board (DSMB). The DSMB found none of the participants experienced any serious adverse events or dangerous side effects from the therapy. "We are very excited and encouraged by this development," says Tara Sadeghi, Chief Operating Officer of Cellenkos. "This recommendation from the DSMB is an important step in bringing this potential disease modifying therapy to the patients and takes us closer to our ultimate goal of curing ALS." CK0803 is a neurotrophic, allogeneic, umbilical cord blood-derived T regulatory (Treg) cell therapy that preferentially homes towards inflamed microglia, developed by using Cellenkos' proprietary CRANE® technology, to generate disease-specific products. The completion of cohort 1 dosing allows for the continued enrollment on the Phase 1 Safety Run-in Study, to be followed by a Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803 in patients with ALS. The treatment will include four weekly infusions followed by five monthly infusions. Regulatory T Cells for Amyotrophic Lateral Sclerosis (REGALS) is a multicenter study including Columbia University, New York; Michael E. DeBakey VA Medical Center, Houston and Baylor College of Medicine, Houston. The primary objective of this study is to establish safety and tolerability of multiple doses of CK0803 in ALS patients. This study will also provide preliminary efficacy data of CK0803 in ALS using the primary endpoint of combined assessment of function and survival (CAFS) that ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, and secondary endpoint of longitudinal measurement of neurofilament light chain levels in serum and cerebrospinal fluid (CSF). About Amyotrophic Lateral Sclerosis (ALS) According to the National Institute of Neurological Disorders and Stroke, amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die and stop sending messages to the muscles. Unable to function, the muscles gradually weaken and waste away (atrophy). Eventually, the brain loses its ability to initiate and control voluntary movements. The disease is progressive, meaning the symptoms get worse over time. Majority of ALS cases (90 percent or more) are considered sporadic. This means the disease seems to occur at random with no clearly associated risk factors and no family history of the disease. In a small (approximately 10%) of cases, ALS is caused by a mutation in a known ALS-associated gene. Most people with ALS eventually die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. However, about 10 percent of people with ALS survive for 10 or more years. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. About CK0803 CK0803 is a novel allogeneic cell therapy product consisting of T regulatory cells, with a high cell surface expression of CD11a, that leverage CXCR3/CXCL10 axis to engage with the inflamed microglia. CK0803 is derived from clinical-grade umbilical cord blood units and manufactured using Cellenkos' proprietary CRANE® process. There is no requirement for HLA or ABO matching of CK0803 to the recipient. Multiple doses of CK0803 can be manufactured from a single umbilical cord blood unit, where the final cryopreserved product is readily available for use, at the point of care, which makes it an ideal therapy that can be infused intravenously, in the outpatient setting. About Cellenkos®, Inc. Cellenkos is a clinical-stage biotechnology company located in Houston, Texas, USA, dedicated to the development and commercialization of the allogeneic, "off-the-shelf" cell-based products for the treatment of rare inflammatory diseases and autoimmune disorders. Cellenkos' T regulatory cells (Tregs) are derived from umbilical cord blood and as such are naïve, bonafide suppressor cells that do not require HLA or ABO matching and resolve inflammation through multiple direct and indirect mechanisms. Cellenkos' proprietary CRANE® process allows for tailoring of Tregs to become disease specific. Cellenkos' current clinical portfolio includes CK0801 (bone marrow failure); CK0802 (COVID ARDS); CK0803 (ALS) and CK0804 (myelofibrosis). For more information, please visit . Contact: Stacy Minor [email protected] SOURCE Cellenkos, Inc.

细胞疗法临床1期免疫疗法

100 项与 CK-0801 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
骨髓衰竭疾病	临床2期	-	Cellenkos, Inc.初创企业	-
格林-巴利综合征	临床1期	-	Cellenkos, Inc.初创企业	2022-11-01
移植物抗宿主病	临床1期	美国	Cellenkos, Inc.初创企业	2017-12-24
1型糖尿病	临床前	美国	Cellenkos, Inc.初创企业	-
1型糖尿病	临床前	美国	The University of Texas MD Anderson Cancer Center	-
多发性硬化症	临床前	美国	Cellenkos, Inc.初创企业	-
多发性硬化症	临床前	美国	The University of Texas MD Anderson Cancer Center	-
银屑病	临床前	美国	The University of Texas MD Anderson Cancer Center	-
银屑病	临床前	美国	Cellenkos, Inc.初创企业	-