Cellular Therapies: Clinical Studies Open For Enrollment
- Allogeneic Stem Cell Transplant to Induce Mixed Donor Chimerism in Patients with Sickle Cell Disease & Thalassemia NYMC 556
- Cancer and Blood & Marrow Transplantation Wellness & Survivorship Research Registry
- Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents and Young Adults with Sickle Cell Disease Following Myeloablative Conditioning (MAC) & Haploidentical Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback
- Pilot Study in the Treatment of Refractory Adenovirus (ADV) Infections with Related Donor ADV Specific Cytotoxic T-Cells (CTLs) in Children, Adolescents and Young Adult Recipients- NYMC 579
- A Pilot Study in the Treatment of Refractory Cytomegalovirus (CMV) Infections with Related Donor CMV Specific Cytotoxic T-Cells (CTLs) in Children, Adolescents and Young Adult Recipients- NYMC 580
- A Pilot Study in the Treatment of Refractory Epstein-Barr (EBV) Infections with Related Donor EBV Specific Cytotoxic T-Cells (CTLs) in Children, Adolescents and Young Adult Recipients- NYMC 581
- A phase II pilot trial to estimate survival after a non-total body irradiation (TBI) based conditioning regimen in patients diagnosed with B-acute lymphoblastic leukemia (ALL) who are pre-allogeneic hematopoietic cell transplantation (HCT) next-generation-sequence (NGS) minimal residual disease (MRD) negative
- Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing alpha/beta T cell and CD19+ B Cell Depletion: NYMC 588
- A Pilot Study in the Treatment of Refractory BK Infections with Related Donor BK Specific Cytotoxic T-cells (CTLs) in Children, Adolescents and Young Adult Recipients – NYMC 590
- A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC)
- J12106 A Phase II Trial of Reduced Intensity Conditioning of Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplant for High-risk Solid Tumors
- ALXN1210-TMA-314 A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to < 18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)
- ALXN1210-TMA-313 A PHASE 3, OPEN-LABEL, RANDOMIZED, MULTICENTER STUDY OF RAVULIZUMAB IN ADULT AND ADOLESCENT PARTICIPANTS WHO HAVE THROMBOTIC MICROANGIOPATHY (TMA) AFTER HEMATOPOIETIC STEM CELL TRANSPLANT (HSCT)
- A Phase II Pilot Study of Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults with High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant: A Multicenter Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) Study (EXCEL Trial)
- A Pilot Study of SARS-CoV-2 Specific Cytotoxic T Lymphocytes (SARS-CoV-2-CTLs) for Treatment of Mild to Moderate Coronavirus Disease 2019 (COVID-19)
- A PHASE I MULTI-DOSE STUDY OF HUMAN PLACENTAL HEMATOPOIETIC STEM CELL DERIVED NATURAL KILLER CELLS (CYNK-001) WITH OR WITHOUT RECOMBINANT HUMAN INTERLEUKIN-2 (RHIL-2) IN ADULTS WITH PRIMARY OR SECONDARY ACUTE MYELOID LEUKEMIA (AML) IN MORPHOLOGIC COMPLETE REMISSION WITH MINIMAL RESIDUAL DISEASE (MRD) OR RELAPSED/REFRACTORY AML
Allogeneic Stem Cell Transplant to Induce Mixed Donor Chimerism in Patients with Sickle Cell Disease & Thalassemia NYMC 556
Eligibility
- Subject is 30 years of age or younger
- Diagnosis of Homozygous Hemoglobin S Disease or Heterozygous Hemoglobin Sickle Cell (SC) or S 0/+ thalassemia, or Sickle/variant resulting in Chronic Hemolytic Anemia with hemoglobin (HgB) ≤10 mg/dL
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT00408447
Principal Investigator
Mitchell S. Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
Cancer and Blood & Marrow Transplantation Wellness & Survivorship Research Registry
Eligibility
- Subject is one year old or older
- Malignant tumor diagnosed and/or treated at Westchester Medical Center and/or Maria Fareri Children’s Hospital
- Blood and marrow transplantation recipients transplanted at Westchester Medical Center and/or Maria Fareri Children’s Hospital
- More than one year from diagnosis and/or last treatment
Enrollment Status: Enrolling
Principal Investigator
Jessica Hochberg, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents and Young Adults with Sickle Cell Disease Following Myeloablative Conditioning (MAC) & Haploidentical Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback
Eligibility
- Subject is between six months and 35 years of age
- Homozygous Hemoglobin S Disease, or Hemoglobin S B0/+ thalassemia, or Hemoglobin SC Disease, or Beta thalassemia intermedia/majora
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT02675959
Principal Investigator
Mitchell S. Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
Pilot Study in the Treatment of Refractory Adenovirus (ADV) Infections with Related Donor ADV Specific Cytotoxic T-Cells (CTLs) in Children, Adolescents and Young Adult Recipients- NYMC 579
Eligibility
- Subject is 30 years of age or younger
- Adenovirus infections post allogeneic HSCT, with primary immunodeficiencies or post solid organ transplant with:
- Increasing or persistent quantitative ADV RT-PCR DNA copies despite two weeks of appropriate anti-viral therapy and/or
- Clinical symptoms attributed to adenovirus, including pneumonitis, hemorrhagic cystitis, colitis, hepatitis and/or
- Medical intolerance to anti-viral therapies including:
- Grade 2 renal insufficiency secondary to cidofovir
- Related donor available with a T-cell response to the viral MACS® GMP PepTivator antigen(s) of adenovirus.
- Third Party Related Allogeneic Donor: If original donor is not available or does not have a T-cell response: third party allogeneic donor (family donor > 3 HLA A, B, DR match to recipient) with a T-cell response at least to the viral MACS® GMP PepTivator antigen(s) of adenovirus.
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT03266627
Principal Investigator
Mitchell S. Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A Pilot Study in the Treatment of Refractory Cytomegalovirus (CMV) Infections with Related Donor CMV Specific Cytotoxic T-Cells (CTLs) in Children, Adolescents and Young Adult Recipients- NYMC 580
Eligibility
- Subject is 30 years of age or younger
- Refractory CMV infection post allogeneic HSCT, with primary immunodeficiencies or post solid organ transplant with either:
- Increasing or persistent quantitative qRT-PCR DNA copies despite two weeks of appropriate anti-viral therapy and/or
- Medical intolerance to anti-viral therapies including:
- ANC less than 500/mm2 secondary to ganciclovir
- 2 renal toxicity with foscarnet and/or
- Known resistance to ganciclovir and/or foscarnet; Related donor available with a T-cell response to the CMV MACS® GMP PepTivator antigen(s)
- Third Party Allogeneic Donor: If original donor is not available or does not have a T-cell response: third party related allogeneic donor (family donor > 3 HLA A, B, DR match to recipient) with IgG positive to CMV and/or a T-cell response to the CMV MACS® GMP PepTivator.
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT03266640
Principal Investigator
Mitchell S. Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A Pilot Study in the Treatment of Refractory Epstein-Barr (EBV) Infections with Related Donor EBV Specific Cytotoxic T-Cells (CTLs) in Children, Adolescents and Young Adult Recipients- NYMC 581
Eligibility
- Subject is 30 years of age or younger
- Epstein-Barr virus infections post allogeneic HSCT, primary immunodeficiencies or post solid organ transplant with:
- Increasing or persistent quantitative EBV RT-PCR DNA copies despite two weeks of appropriate anti-viral therapy and/or
- Progressive clinical symptoms attributable to EBV, including biopsy proven colitis, lymphadenopathy, hepatomegaly, splenomegaly and/or
- Medical intolerance to anti-viral therapies
- Related donor available with a T-cell response to the EBV MACS® GMP PepTivator antigen(s) causing the therapy-refractory EBV infection
- Third Party Related Allogeneic Donor: If original donor is not available or does not have a T-cell response: third party related allogeneic donor (family donor > 3 HLA A, B, DR match to recipient) with IgG positive to EBV and/or a T-cell response at least to the viral MACS® GMP PepTivator EBV Select (containing among other antigens, NA-1, LMP2A and BZLF-1).
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT03266653
Principal Investigator
Mitchell S. Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A phase II pilot trial to estimate survival after a non-total body irradiation (TBI) based conditioning regimen in patients diagnosed with B-acute lymphoblastic leukemia (ALL) who are pre-allogeneic hematopoietic cell transplantation (HCT) next-generation-sequence (NGS) minimal residual disease (MRD) negative
Eligibility
- Subject is between one year and 25 years of age
- Disease status: B-ALL in first (CR1) or second remission (CR2)
- No prior allogeneic hematopoietic stem cell transplant.
- Patients in CR1 or CR2 after blinatumomab treatment
- Patients in CR1 or CR2 after CAR-T cellular therapy
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT03509961
Principal Investigator
Jordan Brittni Milner, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing alpha/beta T cell and CD19+ B Cell Depletion: NYMC 588
Eligibility
- Subject is less than 30 years of age
- High-risk ALL in first remission, ALL in second remission or greater
- AML induction failure, AML with MRD or poor cytogenetics, AML in CR2 or greater; JMML
- Bone Marrow Failure syndromes
- Sickle Cell Disease, Beta-Thalassemia, Solid Tumors
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04099966
Principal Investigator
Jordan Brittni Milner, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A Pilot Study in the Treatment of Refractory BK Infections with Related Donor BK Specific Cytotoxic T-cells (CTLs) in Children, Adolescents and Young Adult Recipients – NYMC 590
Eligibility
- Subject is between one month and 30 years of age
- Patients with refractory BK infection post allogeneic HSCT, post solid organ transplantation or with primary immunodeficiencies with either increasing urine and/or plasma BK RT-PCR DNA (by 1 log) after 7 days or persistent quantitative qRT-PCR DNA copies after 14 days despite two weeks of appropriate anti-viral therapy and/or
- Medical intolerance to anti-viral therapies including 2 renal toxicity with cidofovir or other greater than grade 2 toxicities secondary to cidofovir and/or known resistance to cidofovir
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04197596
Principal Investigator
Mitchell Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC)
Eligibility
- Subject is no more than two years of age
- Infants with SCID, either typical or leaky or Omenn syndrome
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT03619551
Principal Investigator
Jordan Brittni Milner, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
J12106 A Phase II Trial of Reduced Intensity Conditioning of Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplant for High-risk Solid Tumors
Eligibility
- Subject is between one and 50 years of age
- Neuroblastoma or ganglioneuroblastoma either: Failure to achieve at least a PR after induction therapy with COG ANBL0532 or standard chemotherapy
- Refractory to induction chemotherapy with COG ANBL0532 or standard chemotherapy, or
- Patients with high risk disease
- Metastatic Ewing Sarcoma
- Osteosarcoma with metastatic disease beyond the lungs and/or with lung metastases not amenable to resection
- Desmoplastic small round cell tumor
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT01804634
Principal Investigator
Aliza Gardenswartz, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
ALXN1210-TMA-314 A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to < 18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)
Eligibility
- Subject is no older than 17 years of age
- Received Hematopoietic Stem Cell Transplant within the past 12 months
- Diagnosis of Thrombotic Microangiopathy (TMA) that persists for at least 72 hours despite initial management
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04557735
Principal Investigator
Mitchell Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
ALXN1210-TMA-313 A PHASE 3, OPEN-LABEL, RANDOMIZED, MULTICENTER STUDY OF RAVULIZUMAB IN ADULT AND ADOLESCENT PARTICIPANTS WHO HAVE THROMBOTIC MICROANGIOPATHY (TMA) AFTER HEMATOPOIETIC STEM CELL TRANSPLANT (HSCT)
Eligibility
- Subject is at least 12 years of age
- Received HSCT within the past 12 months
- Diagnosis of TMA that persists for at least 72 hours despite initial management
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04543591
Principal Investigator
Mitchell Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A Phase II Pilot Study of Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults with High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant: A Multicenter Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) Study (EXCEL Trial)
Eligibility
- Subject is no older than 25 years of age
- High-risk AML, as defined by one of the following:
- AML in CR1 (defined as less than 5% blasts in BM by morphology and flow cytometry) having at least one of these high-risk features:
- Mutations associated with high risk disease (Appendix A) Other high-risk features not explicitly stated in Appendix A can be considered after discussion/approval with the protocol chair/team
- MRD-positive at the end of Induction I chemotherapy (defined as flow cytometry at least 0.1% blasts)
- AML in at least CR2 (defined by less than 5% blasts in BM by morphology and flow cytometry)
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04836390
Principal Investigator
Aliza Gardenswartz, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A Pilot Study of SARS-CoV-2 Specific Cytotoxic T Lymphocytes (SARS-CoV-2-CTLs) for Treatment of Mild to Moderate Coronavirus Disease 2019 (COVID-19)
Eligibility
- Subject is between 18 - 65 years of age
- Proven infection with SARS-CoV-2, and
- Hospitalized at the time of enrollment, and
- HLA Matched Family Related donor with recent SARS-CoV-2 infection is at least 10 days out from symptom onset, and
- In Stage I or II of disease (mild or moderate) at the time of enrollment, and
- One of the following high-risk conditions:
- Chronic lung disease not requiring oxygen at home prior to admission (including but not limited to COPD, cystic fibrosis, asthma and sickle cell disease)
- Underlying heart disease (including hypertension)
- Patients with an acute myocardial infarction within the last three months will require cardiology clearance prior to enrollment
- Diabetes mellitus (type I or II)
- Obesity (BMI at least 30)
- Immunosuppressed
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04896606
Principal Investigator
Mitchell Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
A PHASE I MULTI-DOSE STUDY OF HUMAN PLACENTAL HEMATOPOIETIC STEM CELL DERIVED NATURAL KILLER CELLS (CYNK-001) WITH OR WITHOUT RECOMBINANT HUMAN INTERLEUKIN-2 (RHIL-2) IN ADULTS WITH PRIMARY OR SECONDARY ACUTE MYELOID LEUKEMIA (AML) IN MORPHOLOGIC COMPLETE REMISSION WITH MINIMAL RESIDUAL DISEASE (MRD) OR RELAPSED/REFRACTORY AML
Eligibility
- Subject is at least 18 years of age
- Primary or Secondary acute myeloid leukemia (AML)
- Patients in first of second Morphological Complete Remission (CR), Morphological Complete Remission with incomplete hematologic recovery (CRi), or Morphologic Leukemia-free State (MLFS)
- Relapsed AML are defined as having relapsed after achieving at least 1 CR, including relapse after allogeneic stem cell transplantation (at least two months after transplant)
- Refractory AML, defined as not achieving CR, CRi, or MLFS after two or more cycles of induction therapy (primary refractory) or not achieving CR after treatment for relapsed AML
- Secondary AML (MDS transformation): Secondary AML patients are eligible to participate if they have received a minimum of one prior line of treatment for AML
- Treatment-related AML: Treatment-related AML patients are eligible to participate if they have received a minimum of one prior line of treatment for AML
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04310592
Principal Investigator
Mitchell Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu