Hematology: Clinical Studies Open For Enrollment
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) less than or equal to 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
A prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT01186913
Principal Investigator
Jordan Brittni Milner, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
The use of dose dense rituximab for high risk patients with newly diagnosed acute immune thrombocytopenic purpura
Enrollment Status: Enrolling
Principal Investigator
Jordan Brittni Milner, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
NYMC-598, A Phase II Trial of Targeted Immunotherapy with Daratumumab Following Myeloablative Total Body Irradiation (TBI)-Based Conditioning and Allogeneic Hematopoietic Cell Transplantation in Children, Adolescents and Young Adults with High-Risk T-Cell Acute Lymphoblastic Leukemia and Lymphoma (T ALL/T LLy) (ALLO-T-DART) (NCT04972942)
Eligibility
- Age: less than or equal to 39 years
- Diagnosis: T-cell acute lymphoblastic leukemia in second or subsequent remission (less than or equal to 5% blasts).
- Relapsed T-cell lymphoblastic lymphoma with complete response after reduction therapy.
- Donor Status: Planned allogeneic stem cell transplantation with donor identified.
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04972942
Principal Investigator
Mitchell S. Cairo, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
Eligibility
- Male or Female Age ≤ 25 years
- An available related HLA-haploidentical donor that is determined to be the most appropriate donor for the patient by the treating physician
- AML in CR1 (defined as <5% blasts in BM by morphology and flow cytometry) having at least one of these high-risk features
- Recovery from prior cycle of chemotherapy as defined by an absolute neutrophil count ≥ 500/mm3
- AML secondary to select germline marrow failure disorders (with exception of Fanconi Anemia) may be eligible but require approval from Protocol Chairs prior to enrollment
- Exclusion Criteria: for matched healthy controls; Active extramedullary disease, Unresolved/ongoing and serious viral, bacterial, or fungal infection despite appropriate treatment, Prior allogeneic transplant and Patients with Fanconi Anemia and Down syndrome
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT04836390
Principal Investigator
Aliza Gardenswartz, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu
Eligibility
- Part B: Adult and Adolescent Participant Inclusion Criteria
- Participant with SCD. Documentation of SCD genotype HbSS or HbSB, may be based on history of laboratory testing or must be confirmed by laboratory testing during Screening.
- Male or female, Age 12 years and older, inclusive at Screening. Participants age 12 to < 18 years will only be enrolled after evaluation of safety in this age cohort in Part C Cohort C1.
- More than or equal to 2 and ≤ 10 VOCs, within 12 months of Screening.
- Hb ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator.
- For participants taking HU and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator and no sign of hematological toxicity.
- Female participants of child-bearing potential must agree to use a highly effective method of contraception or practice abstinence from study start to 84 days after the last dose of study drug. Male participants are eligible to participate if they agree to the following requirements during the study intervention period and for 84 days after the last dose of study intervention, which corresponds to the time needed to eliminate reproductive safety risk of the study intervention:
- Refrain from donating sperm
- Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent.
- Must agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person.
- Female participants of child-bearing potential must have a negative pregnancy test before administration of study drug.
- Participant has provided documented informed consent or written informed parental/guardian consent and participant assent has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines.
- Exclusion Criteria:
- Female participant who is breastfeeding or pregnant.
- Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or has received an RBC or exchange transfusion for any reason within 90 days of Day 1.
- Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF.
- Screening laboratory test of ALT > 4 × ULN for age.
- Participants known to have:
- active hepatitis A, B, or C (HepA Ab IgM positivity, HBsAg, HBVDNA positivity, or HCVRNA positivity)
- evidence of HIV infection with detectable viral load
- ADULTS: Estimated glomerular filtration rate <60 mL/min/1.73 m2 at the Screening Visit, calculated by the central laboratory (using 2021 CKD-EPI eGFR equations), or is on chronic dialysis; ADOLESCENTS: Estimated creatinine clearance <60 mL/min at the Screening Visit, calculated by the central laboratory (using Cockcroft-Gault formula), or is on chronic dialysis.
- History of malignancy within the past 2 years prior to treatment Day 1 requiring chemotherapy and/or radiation (with the exception of local therapy for non-melanoma skin malignancy).
- History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
- Acute coronary syndrome, myocardial infarction, or coronary revascularization,
- Congestive heart failure requiring hospitalization,
- Uncontrolled clinically significant arrhythmias, high grade atrioventricular block (ie, Mobitz II or third degree), placement of an implantable cardiac-defibrillator or history of ventricular fibrillation,
- Pulmonary hypertension.
- Has received any vaccine within 7 days prior to Day 1.
- Received EPO or other hematopoietic growth factor treatment within 28 days of signing ICF or is anticipated to require such agents during the study.
- Current or recent use of voxelotor. Recent use is defined as within 10 days prior to Day 1.
- Current or recent use of crizanlizumab. Recent use is defined as within 90 days prior to Day 1.
- Ongoing or recent use of strong or moderate inducers of CYP3A4/CYP3A5. Recent is defined as within 5 elimination half-lives or 14 days, whichever is longer prior to Day 1.
- Ongoing or recent use of strong or moderate inhibitors of CYP3A4/CYP3A5. Recent is defined as within 5 elimination half-lives prior to Day 1.
- Ongoing or recent use of the P-glycoprotein substrates digoxin or dabigatran. Recent is defined as within 5 elimination half-lives prior to Day 1.
- Use of prohibited prescription or nonprescription drugs and dietary supplements (including herbal and alternative medications). Marijuana and all forms of ingested or inhaled cannabidiol (CBD) and THC-containing products usage meeting DSM-V criteria for substance abuse disorder is prohibited. However, occasional marijuana use is allowed, except for 24 hours prior to neurocognitive assessments as outlined in the Schedule of Assessments.
- Known allergy to osivelotor or allergic reaction to other Hb polymerization inhibitors.
- Part C: Pediatric Participant Inclusion Criteria
- Participant with SCD. Documentation of SCD genotype HbSS or HbSB may be based on history of laboratory testing or must be confirmed by laboratory testing during Screening.
- Age by cohort at Screening:
- Cohort C1: 12 to < 18 years
- Cohort C2: 6 to < 12 years
- Cohort C3: 2 to < 6 years
- Cohort C4: 6 months to < 2 years
- Hb ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator.
- For participants taking HU and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator and no sign of hematological toxicity.
- Written informed parental/guardian consent and participant assent has been obtained per institutional review board (IRB)/Ethics Committee (EC) policy and requirements, consistent with ICH guidelines.
- Exclusion Criteria:
- Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or has received an RBC or exchange transfusion for any reason within 90 days of Day 1.
- Screening laboratory test of ALT > 4 × ULN for age.
- Acute illness or clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy, including acute bacterial infection requiring antibiotics within 14 days prior to the study drug administration.
- Participants known to have:
- active hepatitis A, B, or C or HIV (HepA Ab IgM positivity, HBsAg, HBVDNA positivity, or HCVRNA positivity)
- evidence of HIV infection with detectable viral load
- Has received any vaccine within 7 days prior to Day 1.
- History of severe allergic reaction (including anaphylaxis) to any substance, or previous status asthmaticus.
- Unlikely to comply with the study procedures.
Enrollment Status: Enrolling
Study Information
ClinicalTrials.gov | NCT05431088
Principal Investigator
Edo Schaefer, MD
Contact for Study Screening
Lauren_Harrison@nymc.edu