4 Participants Needed

Stem Cell Collection for Familial Platelet Disorder

CH
Overseen ByChitra Hosing, MD
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: M.D. Anderson Cancer Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you have been treated with an investigational drug within 30 days of screening, you may not be eligible to participate.

Is stem cell mobilization safe for humans?

Stem cell mobilization using drugs like G-CSF and plerixafor has been generally well-tolerated in humans, with side effects similar to those of the individual drugs. Studies in normal volunteers and patients with various conditions have shown that these treatments are safe, although some patients may not respond well due to previous treatments or other factors.12345

How is the treatment for Familial Platelet Disorder using Autologous CD34+ Hematopoietic Stem Cells Mobilization different from other treatments?

This treatment is unique because it involves mobilizing the patient's own stem cells (autologous) using a combination of chemotherapy and growth factors like granulocyte colony-stimulating factor (G-CSF), which increases the number of stem cells available for collection. This approach is different from traditional methods that may rely solely on bone marrow or do not use the combination of chemotherapy and growth factors to enhance stem cell yield.56789

What data supports the effectiveness of the treatment Autologous CD34+ Hematopoietic Stem Cells Mobilization for Familial Platelet Disorder?

Research shows that using growth factors like G-CSF and drugs like plerixafor can significantly increase the number of stem cells collected, which is crucial for successful transplantation. This approach has been effective in patients with blood-related conditions, suggesting it may also be beneficial for Familial Platelet Disorder.710111213

Who Is on the Research Team?

CH

Chitra Hosing

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with RUNX1 Familial Platelet Disorder, which affects blood clotting. Participants must be eligible to undergo procedures to collect stem cells from their own blood.

Inclusion Criteria

Participants with systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg
I am willing to follow the study's birth control rules.
Participants willing and able to provide informed consent, as appropriate
See 6 more

Exclusion Criteria

I have been diagnosed with MDS or a blood cancer.
I haven't taken any experimental drugs recently.
Participants unable to comply with the study procedures
See 19 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Mobilization and Apheresis

Participants receive G-CSF injections on Days 1-5, with optional plerixafor on Day 5 or 6 for stem cell mobilization and collection

1 week
Daily visits for injections

Follow-up

Participants are monitored for safety and effectiveness after stem cell collection

1 year

What Are the Treatments Tested in This Trial?

Interventions

  • Autologous CD34+ Hematopoietic Stem Cells Mobilization
Trial Overview The study tests the safety of using drugs Plerixafor and G-CSF (filgrastim or biosimilar) to mobilize stem cells in the bloodstream and then collecting these cells through a process called Apheresis.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Autologous CD34+ Hematopoietic Stem Cells Mobilization and ApheresisExperimental Treatment3 Interventions

Autologous CD34+ Hematopoietic Stem Cells Mobilization is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Autologous CD34+ Hematopoietic Stem Cells Mobilization for:
🇪🇺
Approved in European Union as Autologous CD34+ Hematopoietic Stem Cells Mobilization for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials
3,107
Recruited
1,813,000+

RUNX1 Foundation

Collaborator

Trials
1
Recruited
4+

Published Research Related to This Trial

A systematic review of 28 randomized controlled trials found that combining cyclophosphamide with growth factors like molgramostim or filgrastim significantly improves the yield of hematopoietic stem cells (HSCs), essential for successful autologous transplantation.
While some mobilization strategies increase CD34+ cell yield, they may also lead to higher toxicity, indicating that treatment plans should be tailored to individual patient needs and safety considerations.
Systematic review of randomized controlled trials of hematopoietic stem cell mobilization strategies for autologous transplantation for hematologic malignancies.Sheppard, D., Bredeson, C., Allan, D., et al.[2022]
In a study of 74 patients with hematologic malignancies, 72% successfully collected enough stem cells during the first mobilization attempt, allowing for prompt engraftment within 10-12 days.
For patients who initially failed to collect adequate stem cells, administering high-dose G-CSF immediately led to an 88% success rate in achieving the target stem cell count, significantly increasing the collection rate from 0.16 to 0.61 CD34+ cells/kg/day.
Successful PBSC mobilization with high-dose G-CSF for patients failing a first round of mobilization.Gazitt, Y., Freytes, CO., Callander, N., et al.[2004]
High-dose chemotherapy combined with stem cell support has significantly improved the procedures for harvesting hematopoietic stem and progenitor cells, leading to better outcomes in autologous transplantation.
The development of hematopoietic growth factors and new mobilizing drugs, like plerixafor, has optimized stem cell collection, reduced hospitalization times, and made the process more cost-effective for patients, especially those with poor mobilization.
[Hematopoietic stem cells mobilization: state of the art in 2011 and perspectives].Bijou, F., Ivanovic, Z., Boiron, JM., et al.[2021]

Citations

Systematic review of randomized controlled trials of hematopoietic stem cell mobilization strategies for autologous transplantation for hematologic malignancies. [2022]
Successful PBSC mobilization with high-dose G-CSF for patients failing a first round of mobilization. [2004]
[Hematopoietic stem cells mobilization: state of the art in 2011 and perspectives]. [2021]
[Modalities of mobilization and hematopoietic stem cells objectives in autologous transplantation: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)]. [2021]
Hematopoietic stem cell mobilization: a clinical protocol. [2021]
Management strategies for the hard-to-mobilize patient. [2005]
A risk-based approach to optimize autologous hematopoietic stem cell (HSC) collection with the use of plerixafor. [2022]
Recombinant human interleukin-3 (rhIL-3) enhances the mobilization of peripheral blood progenitor cells by recombinant human granulocyte colony-stimulating factor (rhG-CSF) in normal volunteers. [2017]
Mobilization of blood stem cells. [2015]
10.United Statespubmed.ncbi.nlm.nih.gov
Harvesting autologous stem cells from a patient with red blood cell abnormalities of β-thalassemia intermedia. [2014]
11.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Mobilization and harvesting of peripheral blood stem cells. [2019]
12.United Statespubmed.ncbi.nlm.nih.gov
Peripheral blood stem cell mobilization tactics. [2010]
13.United Statespubmed.ncbi.nlm.nih.gov
First experience of autologous peripheral blood stem cell mobilization with biosimilar granulocyte colony-stimulating factor. [2015]
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