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Tests treatment safety and results for RUNX1 Familial Platelet Disorder
Official title Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder
ClinicalTrials.gov ID: NCT06414889
What this study is testing
What is G-CSF (filgrastim or biosimilar)?
G-CSF (filgrastim or biosimilar) is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for runx1 familial platelet disorder.
Also referred to as FILGRASTIM SD/01.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75. Healthy volunteers may be eligible.
You may be able to join if
- Participants who meet all of the following criteria are eligible to be included in the study:
- Are aged ≥ 18 to 75 years a. Once a favorable review of safety has been completed by the SMC in 3 participants aged ≥ 18 years, the study will be...
- Are willing and able to provide informed consent, as appropriate (either directly or through a legally authorized representative [LAR]), as described...
- Have a confirmed diagnosis of RUNX1 FPD, verified by a Clinical Laboratory Improvement Amendments (CLIA)-certified genetic sequencing report.
- Clearance by apheresis team to proceed
You likely can't join if
- Participants who meet any of the following criteria are excluded from the study:
- Participants with cognitive impairments and/or any serious unstable pre-existing medical condition or psychiatric disorder that can interfere with...
- Have uncontrolled bleeding
- Are using supplemental oxygen
- Have known severe splenomegaly (≥ 20 cm)
- Have a diagnosis of MDS or hematologic malignancies, as defined by WHO hematolymphoid tumor classification fifth edition (Khourey et al 2022)...
See the full eligibility criteria
- Participants who meet all of the following criteria are eligible to be included in the study:
- Are aged ≥ 18 to 75 years a. Once a favorable review of safety has been completed by the SMC in 3 participants aged ≥ 18 years, the study will be opened to participants aged ≥ 12 years.
- Are willing and able to provide informed consent, as appropriate (either directly or through a legally authorized representative [LAR]), as described in Appendix 1, Section 13.1
- Have a confirmed diagnosis of RUNX1 FPD, verified by a Clinical Laboratory Improvement Amendments (CLIA)-certified genetic sequencing report.
- Clearance by apheresis team to proceed
- Have systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg
- Are eligible for HSCT per institution requirements
- Have a Lansky (age \< 16 years)/Karnofsky performance status of ≥ 70 (see Appendix 2, Section 13.2).
- Are willing and able to comply with protocol-defined contraceptive requirements (see Appendix 3 Section 13.3)
- Have a platelet count ≥ 50,000/μL for initiation of apheresis, assessed within 24 hours prior to the procedure, or, if \< 50,000/μL are administered platelets on the day of the collection a. If the apheresis team...
- Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure
- Participants who meet any of the following criteria are excluded from the study:
- Participants with cognitive impairments and/or any serious unstable pre-existing medical condition or psychiatric disorder that can interfere with safety or with obtaining informed consent or compliance with study...
- Have uncontrolled bleeding
- Are using supplemental oxygen
- Have known severe splenomegaly (≥ 20 cm)
- Have a diagnosis of MDS or hematologic malignancies, as defined by WHO hematolymphoid tumor classification fifth edition (Khourey et al 2022) hematolymphoid tumor classification fifth edition (Khourey et al 2022)
- Have recent prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ Note: Cancer treated with curative intent \ 5 years previously is allowed.
- Have any prior or current myeloproliferative or a significant coagulation or immunodeficiency disorder
- Have advanced liver disease, defined as any of the following:
- Persistent aspartate transaminase, alanine transaminase, or direct bilirubin value \> 5× the upper limit of normal (ULN) at screening
- Screening prothrombin time (PT) or partial thromboplastin time (PTT) \> 1.5× ULN
- Have had prior HSCT or gene therapy
- Have history of concomitant sickle cell disease
- Have been treated with an investigational drug within 30 days of screening or 5 half-lives (whichever is longer)
- Have a positive test result for HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) at screening
- Participants with positive hepatitis B core antibody (HbcAb) and/or hepatitis B-e antibody (HbeAb) are eligible provided viral load is negative by quantitative polymerase chain reaction (qPCR).
- Participants who are positive for anti-hepatitis C antibody are eligible as long as they have a negative HCV viral load by qPCR.
- Have a positive infectious disease panel at screening for human T-lymphotropic virus 1 or 2 (HTLV-1 and HTLV-2), or syphilis (rapid plasma 24 reagin [RPR])
- Have clinically significant and active bacterial, viral, fungal, or parasitic infection at screening
- Have a white blood cell (WBC) count \< 2 × 109/L
- Have a left ventricular ejection fraction \< 45%
- Have a screening estimated glomerular filtration rate \< 60 mL/min/1.73 m2
- Have a diagnosis of a significant psychiatric disorder that could seriously impede the ability to participate in the study
- For women of childbearing potential: are pregnant or breastfeeding or lack adequate contraception
- Are unable to comply with the study procedures, as assessed by the investigator
The study team makes the final eligibility decision.
Where it's taking place
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Houston, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.