RUNX1 Familial Platelet DisorderPhase 1NCT06414889
Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder
A Phase 1 study testing G-CSF (filgrastim or biosimilar), Apheresis and Plerixafor in people with RUNX1 Familial Platelet Disorder, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- RUNX1 Familial Platelet Disorder
- What is being tested?
- G-CSF (filgrastim or biosimilar), Apheresis and Plerixafor
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 20 May 2024to 30 Jun 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Participants who meet all of the following criteria are eligible to be included in the study:
- Are aged ≥ 18 to 75 years
- 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
Show all 13Show fewer
- 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
- If the apheresis team decides that a central venous catheter (CVC) is to be placed, platelet count should be ≥ 50,000 prior to catheter placement.
- Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure
Who cannot join
- 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 procedures.
- 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)
Show all 25Show fewer
- Have recent prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ Note: Cancer treated with curative intent < 5 years previously may be allowed following approval from the study investigator. 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Chitra Hosing, MD, 713-745-3219, cmhosing@mdanderson.org
Chitra Hosing, MD
About this study
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
Read the detailed description
Primary Objective:
- To evaluate the safety of harvesting HSCs in participants with RUNX1 FPD
Secondary Objective
- To evaluate the feasibility and other relevant information of collecting HSCs from participants with RUNX1 FPD
- Conditions
- RUNX1 Familial Platelet Disorder
- Being tested
- G-CSF (filgrastim or biosimilar), Apheresis, Plerixafor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
G-CSF (filgrastim or biosimilar)Drug
Also called FILGRASTIM SD/01
Given by IV or SC
ApheresisProcedure or surgery
Given by procedure
PlerixaforDrug
Given by IV
| Study group | Role | What they receive |
|---|---|---|
| Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis | Experimental | On Days 1-5, participants will receive a Granulocyte colony-stimulating factor (G-CSF), such as filgrastim, as an injection or by vein over about 5 minutes.
If the study doctor thinks it is needed, participants will also receive plerixafor as an injection under the skin on Day 5 (and 6, if you have 2 days of apheresis). Receives: Drug: G-CSF (filgrastim or biosimilar); Procedure: Apheresis; Drug: Plerixafor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and adverse events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2024
- Study start20 May 2024
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RUNX1 Familial Platelet DisorderStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- RUNX1 Foundation
- Responsible party
- Sponsor
- Investigators
- Chitra Hosing, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06414889
- Sponsor's protocol ID
- 2023-0799
- Other IDs
- NCI-2024-04300
- Status last verified
- Jun 2026
- First submitted
- 10 May 2024
- Last updated
- 11 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- Acronym / sponsor ID
- 2023-0799
- Conditions
- RUNX1 Familial Platelet Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 4 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- RUNX1 Foundation
- Study start
- 20 May 2024 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 16 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
Detailed description
Primary Objective:
- To evaluate the safety of harvesting HSCs in participants with RUNX1 FPD
Secondary Objective
- To evaluate the feasibility and other relevant information of collecting HSCs from participants with RUNX1 FPD
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis | Experimental | Drug: G-CSF (filgrastim or biosimilar), Procedure: Apheresis, Drug: PlerixaforOn Days 1-5, participants will receive a Granulocyte colony-stimulating factor (G-CSF), such as filgrastim, as an injection or by vein over about 5 minutes. If the study doctor thinks it is needed, participants will also receive plerixafor as an injection under the skin on Day 5 (and 6, if you have 2 days of apheresis). |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Participants who meet all of the following criteria are eligible to be included in the study:
- Are aged ≥ 18 to 75 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
Key exclusion criteria (25 listed)
- 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 procedures.
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| G-CSF (filgrastim or biosimilar)Also: FILGRASTIM SD/01Given by IV or SC | Drug | Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis |
| ApheresisGiven by procedure | Procedure or surgery | Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis |
| PlerixaforGiven by IV | Drug | Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis |
Primary outcome
Safety and adverse events (AEs)
Time frame: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
Related trials for RUNX1 Familial Platelet Disorder
RecruitingPhase 1
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
RecruitingPhase 1
Low-Dose Sirolimus for the Treatment of RUNX1 Familial Platelet Disorder
Familial Platelet Disorder With Associated Myeloid Malignancy
RecruitingObservational study
Longitudinal Studies of Patient With FPDMM
Inherited Hematological Diseases, Rare Diseases
RecruitingPhase 2
Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder
Familial Platelet Disorder, Hematopoietic
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chitra Hosing principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing G-CSF (filgrastim or biosimilar), Apheresis and Plerixafor in people with RUNX1 Familial Platelet Disorder, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chitra Hosing, MD ((713) 745-3219, cmhosing@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026.