Memphis, TNNCT07585136Now EnrollingIRB Ready

Bone Marrow Failure Syndrome Clinical Trial in Memphis, TN

Access cutting-edge bone marrow failure syndrome treatment through this phase 1 clinical trial at a research site in Memphis. Study-provided care at no cost to qualified participants.

Sponsored by St. Jude Children's Research Hospital

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Expert Care in Memphis

Access bone marrow failure syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related bone marrow failure syndrome treatment provided free

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Check if you qualify for this bone marrow failure syndrome clinical trial in Memphis, TN

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Why Participate?

  • No-Cost Study Care

  • Local to Memphis

    Convenient for TN residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Memphis site if eligible
  4. 4Begin participation

About This Bone Marrow Failure Syndrome Study in Memphis

The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy. Primary objective: \- To characterize the safety of Filgrastim plus plerixafor in participants with life threatening blood disorders as determined by the incidence of adverse events (AEs). Secondary Objectives: * To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts. * To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts. * To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells/kg).

Sponsor: St. Jude Children's Research Hospital

Who Can Participate

Inclusion Criteria

Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy
Age ≥ 18 years - 25 years
The following hematological parameters need to be met (regardless of transfusion or growth factor support)
Hb \> 8 g/dL
ANC \> 500/mm3
Platelet \> 30,000/mm3
Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis
Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed
Karnofsky score \>80
Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II
Female participants of childbearing age should have a negative serum pregnancy test within one week of beginning Filgrastim and plerixafor administration

Exclusion Criteria

Participant with sickle cell disease
Participant who has had a prior autologous or allogeneic HSCT
Active viral, bacterial, fungal, or parasitic infection
Total bilirubin \>2.5x ULN or transaminases \>5x ULN
Moderate or severe renal failure defined as serum/plasma creatinine \>1.5 mg/dL and an estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73 m2 based on the CKD-Epi equation or the St. Jude equation
Diagnosis of MDS or other hematologic malignancy
History of malignancy
Known allergy to or contraindication for Filgrastim or plerixafor administration, or medications routinely administered during apheresis
Splenomegaly (size greater than upper limit of normal on examination)
Any disease or concomitant process that is not compatible with the study as per investigator opinion
Concomitant treatment with alternative investigational agent or participation in another clinical trial with an investigational drug within 5 half-lives of the investigational agent
Unwillingness to use a highly effective method of contraception for 1 month after plerixafor or GCSF
Inability or unwillingness of research participant to give written informed consent.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Memphis?

Yes, this clinical trial (NCT07585136) has an active research site in Memphis, TN that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Bone Marrow Failure Syndrome Treatment Options in Memphis, TN

If you're searching for bone marrow failure syndrome treatment options in Memphis, TN, this clinical trial (NCT07585136) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Memphis research site is actively enrolling participants for this phase 1 clinical trial. You'll receive care from experienced bone marrow failure syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all bone marrow failure syndrome clinical trials near you to find additional studies recruiting in your area.

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