G-csf for Juvenile Myelomonocytic Leukaemias (JMML)

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
St. Jude Children's Research Hospital, Memphis, TN
Juvenile Myelomonocytic Leukaemias (JMML)+18 More
G-csf - Biological
Eligibility
< 65
All Sexes
Eligible conditions
Select

Study Summary

TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies

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Eligible Conditions

  • Juvenile Myelomonocytic Leukaemias (JMML)
  • Acute Lymphoblastic Leukemia (ALL)
  • Non-Hodgkin's Lymphoma (NHL)
  • Acute Myeloid Leukemia (AML)
  • NK-Cell Leukemia
  • Lymphoma, Hodgkins
  • Leukemia Chronic Myelogenous Leukemia (CML)
  • Myelodysplastic Syndromes (MDS)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Juvenile Myelomonocytic Leukaemias (JMML)

Study Objectives

This trial is evaluating whether G-csf will improve 2 primary outcomes and 4 secondary outcomes in patients with Juvenile Myelomonocytic Leukaemias (JMML). Measurement will happen over the course of One year after the transplant date of the last enrolled patient.

Day 100
The cumulative incidence of transplant related mortality
Day 120
The number of patients experiencing Blinatumomab permanent discontinuation due to toxicity
Day 90
Maximum effective dose for prophylactic CD45RA-depleted DLI
One year after the transplant date of the last enrolled patient
One-year Event Free Survival (EFS) after completion of the protocol
The cumulative incidence of acute and chronic Graft-Versus-Host Disease (GVHD)
The estimate of cumulative incidence of relapse

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Juvenile Myelomonocytic Leukaemias (JMML)

Trial Design

1 Treatment Group

Transplant participants
1 of 1
Experimental Treatment

This trial requires 140 total participants across 1 different treatment group

This trial involves a single treatment. G-csf is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Transplant participantsParticipants receive a conditioning regimen of ATG (rabbit),Cyclophosphamide 60 mg/kg intravenous once daily, mesna, fludarabine, thiotepa, melphalan, followed by HPC,A Infusion(TCRα/β+ and CD19+ depleted),HPC, A infusion (if needed to achieve goal CD34+ cell dose.CD45RA-depleted DLI will be given at least two weeks after engraftment. Blinatumomab will be given at least one week post-DLI, and only to patients with CD19+ malignancies. G-csf 5mcg/kg subcutaneous or intravenous daily until ANC >2000 for 2 consecutive days. Cells for infusion are prepared using the CliniMACS system.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Filgrastim
FDA approved
Melphalan
FDA approved
Blinatumomab
FDA approved
Coenzyme M
FDA approved
CliniMACS
2005
Completed Phase 3
~880
Fludarabine
FDA approved
Thiotepa
FDA approved
Cyclophosphamide
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 90 days after the transplant date of the last enrolled patient.
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 90 days after the transplant date of the last enrolled patient. for reporting.

Closest Location

St. Jude Children's Research Hospital - Memphis, TN

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. You must have received 1 prior treatment for Juvenile Myelomonocytic Leukaemias (JMML) or one of the other 18 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Inclusion Criteria for Transplant Recipient
Age less than or equal to 21 years.
Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.
Has a suitable single haplotype matched (≥ 3 of 6) family member donor.
High risk hematologic malignancy. High risk ALL in CR1. Examples include, but not limited to: t(9;22) with persistent or recurrent transcript, hypodiploid cytogenetics, MRD >1% at the end of induction, M2 or greater marrow at the end of induction, recurrent or rising MRD after induction, Infants with MLL fusion or t(4;11), relapse after prior CART therapy.
ALL in High risk CR2. Examples include, but not limited to t(9;22), BM relapse <36 mo CR1 or <6mo after completion of therapy, any T-ALL, very early (< 6mo CR1) isolated CNS relapse, late BM relapse with poor response to standard reinduction therapy(e.g. MRD positive or recurrence after two blocks), relapse after prior CART therapy.
ALL in CR3 or subsequent.
AML in high risk CR1 (diagnosis of AML includes myeloid sarcoma). Examples include but not limited to: preceding MDS or MDS-related AML, FAB M0, FAB M6, FAB M7 with high risk genetics such as ML not t(1;22), MRD > 0.1% after two cycles of induction, MRD > 1% after one cycle of induction, FLT3-ITD in combination with NUP98-NSD1 fusion or WT1 mutation, any high risk cytogenetics such as: DEK-NUP214 [t(6;9)], KAT6A-CREBBP [t(8;16)], RUNX1-CBFA2T3 [t(16;21)], -7, -5, 5q-, KMT2A-MLLT10 [t(6;11)], KMT2A-MLLT4 [t(10;11)], inv(3)(q21q26.2), CBFA2T3-GLIS2 [inv(16)(p13.3q24.3)], NUP98-KDM5A [t(11;12)(p15;p13)], ETV6-HLXB [t(7;12)(q36;p13)], NUP98-HOXA9 [t(7;11)(p15.4;p15)], NUP98-NSD1.
AML in CR2 or subsequent.
Therapy related AML, with prior malignancy in CR > 12mo

Patient Q&A Section

What are common treatments for leukemia, myeloid?

"Common treatments for leukemia and myeloid include chemotherapy, targeted therapy drugs (such as imatinib and sunitinib), phototherapy, stem cell transplantation and more. Stem cells from cord blood is a promising source of hematopoietic stem cells for treating leukemia and myeloid. Antimalarial and antifungal drugs and pyrimethamine can be used to treat myeloid. More studies about these therapeutic modalities are needed." - Anonymous Online Contributor

Unverified Answer

What causes leukemia, myeloid?

"MDS/AML arise from hematopoietic stem cells (HSCs) getting errors in their DNA during normal development or through inherited or acquired genetic alterations before or during blood progenitor cell formation. MDS/AML are thought to occur from a malignant clone or myeloproliferative disorder. MDS/AML are characterized by monocytic or megaclonal malignancies often involving chromosome abnormalities. Targeted therapy can improve the overall outcome of patients with MDS/AML." - Anonymous Online Contributor

Unverified Answer

How many people get leukemia, myeloid a year in the United States?

"Almost 100,000 persons per year will develop leukemias, mainly acute myelogenous leukemia, but also acute lymphoblastic leukemia. The number of leukemias diagnosed during the first year of life is around 25,000 per year." - Anonymous Online Contributor

Unverified Answer

What is leukemia, myeloid?

"Hematological malignancies are a complex group of diseases, which present in different forms and with different symptoms. Hereditary causes of leukemia are also an important component in our concept of disease. They contribute greatly to the etiology of the disease, although the contribution is only partial." - Anonymous Online Contributor

Unverified Answer

Can leukemia, myeloid be cured?

"Chronic myelomonocytic leukemia can be reversed and myeloid leukemias can be cured to at least the extent that they can be prevented, thereby substantially improving the survival and quality of life in this group of patients. The cure can most often be achieved in patients with low to intermediate cytogenetic risk, with a survival of over three years. The disease can be reversed in patients of all ages who are high risk or in refractory cases. Patients are often discharged and may be followed-up at regular intervals, and with careful observation, the disease can be reversed and the patients can eventually be placed on a surveillance program to monitor them for potential relapse." - Anonymous Online Contributor

Unverified Answer

What are the signs of leukemia, myeloid?

"Symptoms of acute leukemia include fatigue, loss of appetite, loss of hair, swollen and painful lymph nodes, loss of weight, a fast heart rate and low blood cell counts. Symptoms of chronic leukemia include feeling tired, feeling unsteady on his feet, loss of hair, feeling hot and cold, shortness of breath with physical exertion, weight loss and loss of appetite.\n- What are the signs of myeloid leukemia?\n- What is pneumonia?\nAn pneumonia has four main components. "The first is often pain, often low-grade or none at all, in the chest and usually lasts several days." - Anonymous Online Contributor

Unverified Answer

What does tcrα/β+ usually treat?

"Tcrα-negative/β-negative acute myeloblastic leukemia is uncommon in the pediatric population, but has a very poor prognosis. The absence of Tcrα-positive T cells and/or TCRγ expression has not been defined in other pediatric acute myeloid leukemias. Therefore, the role of Tcrα+ T-cell responses in patients with these pediatric acute myeloid leukemias is unclear. The current standard regimen for treatment of acute myeloid leukemia in the pediatric age group is the induction of remission by the anthracyclines." - Anonymous Online Contributor

Unverified Answer

Does tcrα/β+ improve quality of life for those with leukemia, myeloid?

"In summary, patients treated with Tcrα/β+ experienced a significant improvement in their HRQOL with respect to baseline scores. Results from a recent paper support the continued investigation of this multimodality approach to myeloid patients suffering from B-CLL or MDS." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for leukemia, myeloid?

"There are many reasons that patients of all ages with leukemia might enroll in a clinical trial, and they need to be informed about the possibility of receiving either a curative or palliative procedure, as well as the risks of having side effects that might be undesirable either for the patient or for others. For each specific patient, the doctors decide how to manage their disease as they see fit." - Anonymous Online Contributor

Unverified Answer

How does tcrα/β+ work?

"We demonstrate that Tcrα/β+ is a distinct entity that is capable of ligation to various cell lineages, and we identify a variety of molecular and cellular targets involved with Tcrα/β+-mediated fusion events including those mediating cell-cell adhesion/migration, oncogene acquisition, and oncogenesis." - Anonymous Online Contributor

Unverified Answer

Does leukemia, myeloid run in families?

"Despite the large number of patients in this study, patients with AML were very unlikely to be a member of a family with a history of the condition. For these reasons, a gene for AML should not be on the list of candidate genes in the AMGL gene, because it would need to have an unusual mode of action that would explain its frequency in patients with AML but very low occurrence in AML-related families, even without the gene itself being a major susceptibility gene." - Anonymous Online Contributor

Unverified Answer

What is tcrα/β+?

"Tcrα/β+T cells are an important aspect of natural immunity of T cell receptors. TCRα/β+T cells can kill T cells in their own peripheral tissue. The TCRα/β+T cells are involved in the destruction of myeloid leukemia and some solid tumors. The TCRα/β+T cells can be used as cancer therapeutics. It was an important discovery concerning the use of TCRα/β+ T cells in cancer treatment." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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