## Protocol Section
### Identification Module
**NCT ID:** NCT06414889

**Brief Title:** Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With 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

#### Organization Study ID Info
**ID:** 2023-0799
#### Organization
**Class:** OTHER
**Full Name:** M.D. Anderson Cancer Center
#### Secondary ID Infos
**Domain:** NCI-CTRP Clinical Registry
**ID:** NCI-2024-04300
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2027-06-30

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-22

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-21

**Overall Status:** RECRUITING

#### Primary Completion Date
**Date:** 2025-06-30

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-05-20

**Type:** ACTUAL

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-16
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-10
**Study First Submit QC Date:** 2024-05-10
### Sponsor Collaborators Module

#### Collaborators

**Class:** UNKNOWN

**Name:** RUNX1 Foundation

#### Lead Sponsor

**Class:** OTHER

**Name:** M.D. Anderson Cancer Center

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Is US Export:** False

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** 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

### Conditions Module

**Conditions:**

- RUNX1 Familial Platelet Disorder

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** OTHER

#### Enrollment Info

**Count:** 4

**Type:** ESTIMATED

**Phases:**

- EARLY_PHASE1

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** 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).

**Intervention Names:**

- Drug: G-CSF (filgrastim or biosimilar)

- Procedure: Apheresis

- Drug: Plerixafor

**Label:** Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

**Description:** Given by IV or SC

**Name:** G-CSF (filgrastim or biosimilar)

**Other Names:**

- FILGRASTIM SD/01

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

**Description:** Given by procedure

**Name:** Apheresis

**Type:** PROCEDURE

#### Intervention 3

**Arm Group Labels:**

- Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

**Description:** Given by IV

**Name:** Plerixafor

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

**Measure:** Safety and adverse events (AEs)

**Time Frame:** Through study completion; an average of 1 year.

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

Participants who meet all of the following criteria are eligible to be included in the study:

1. 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.
2. 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
3. Have a confirmed diagnosis of RUNX1 FPD, verified by a Clinical Laboratory Improvement Amendments (CLIA)-certified genetic sequencing report.
4. Clearance by apheresis team to proceed
5. Have systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg
6. Are eligible for HSCT per institution requirements
7. Have a Lansky (age \< 16 years)/Karnofsky performance status of ≥ 70 (see Appendix 2, Section 13.2).
8. Are willing and able to comply with protocol-defined contraceptive requirements (see Appendix 3 Section 13.3)
9. 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 decides that a central venous catheter (CVC) is to be placed, platelet count should be ≥ 50,000 prior to catheter placement.
10. Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure

Exclusion Criteria:

Participants who meet any of the following criteria are excluded from the study:

1. 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.
2. Have uncontrolled bleeding
3. Are using supplemental oxygen
4. Have known severe splenomegaly (≥ 20 cm)
5. 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)
6. 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.
7. Have any prior or current myeloproliferative or a significant coagulation or immunodeficiency disorder
8. Have advanced liver disease, defined as any of the following:

   1. Persistent aspartate transaminase, alanine transaminase, or direct bilirubin value \> 5× the upper limit of normal (ULN) at screening
   2. Screening prothrombin time (PT) or partial thromboplastin time (PTT) \> 1.5× ULN
9. Have had prior HSCT or gene therapy
10. Have history of concomitant sickle cell disease
11. Have been treated with an investigational drug within 30 days of screening or 5 half-lives (whichever is longer)
12. Have a positive test result for HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) at screening

    1. 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).
    2. Participants who are positive for anti-hepatitis C antibody are eligible as long as they have a negative HCV viral load by qPCR.
13. 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 \[RPR\])
14. Have clinically significant and active bacterial, viral, fungal, or parasitic infection at screening
15. Have a white blood cell (WBC) count \< 2 × 109/L
16. Have a left ventricular ejection fraction \< 45%
17. Have a screening estimated glomerular filtration rate \< 60 mL/min/1.73 m2
18. Have a diagnosis of a significant psychiatric disorder that could seriously impede the ability to participate in the study
19. For women of childbearing potential: are pregnant or breastfeeding or lack adequate contraception
20. Are unable to comply with the study procedures, as assessed by the investigator

**Healthy Volunteers:** True

**Maximum Age:** 75 Years

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** cmhosing@mdanderson.org

**Name:** Chitra Hosing, MD

**Phone:** (713) 745-3219

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Houston

**Contacts:**

***Contact 1:***

- **Email:** cmhosing@mdanderson.org

- **Name:** Chitra Hosing, MD

- **Phone:** 713-745-3219

- **Role:** CONTACT

***Contact 2:***

- **Name:** Chitra Hosing, MD

- **Role:** PRINCIPAL_INVESTIGATOR

**Country:** United States

**Facility:** MD Anderson Cancer Center

**State:** Texas

**Status:** RECRUITING

**Zip:** 77030

#### Overall Officials

**Official 1:**

**Affiliation:** M.D. Anderson Cancer Center

**Name:** Chitra Hosing

**Role:** PRINCIPAL_INVESTIGATOR

### References Module

#### See Also Links

**Label:** MD Anderson Cancer Center

**URL:** http://www.mdanderson.org

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000006402

- Term: Hematologic Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC15

- Name: Blood and Lymph Conditions

- Abbrev: All

- Name: All Conditions

### Condition Browse Module - Browse Leaves

- ID: M5072

- Name: Blood Platelet Disorders

- Relevance: HIGH

- As Found: Platelet Disorder

- ID: M9490

- Name: Hematologic Diseases

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000001791

- Term: Blood Platelet Disorders

### Intervention Browse Module - Ancestors

- ID: D000000276

- Term: Adjuvants, Immunologic

- ID: D000007155

- Term: Immunologic Factors

- ID: D000045505

- Term: Physiological Effects of Drugs

- ID: D000019380

- Term: Anti-HIV Agents

- ID: D000044966

- Term: Anti-Retroviral Agents

- ID: D000000998

- Term: Antiviral Agents

- ID: D000000890

- Term: Anti-Infective Agents

### Intervention Browse Module - Browse Branches

- Abbrev: Infe

- Name: Anti-Infective Agents

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M340828

- Name: Plerixafor

- Relevance: HIGH

- As Found: Commitment

- ID: M1945

- Name: Lenograstim

- Relevance: HIGH

- As Found: Effectiveness

- ID: M3628

- Name: Adjuvants, Immunologic

- Relevance: LOW

- As Found: Unknown

- ID: M10201

- Name: Immunologic Factors

- Relevance: LOW

- As Found: Unknown

- ID: M21350

- Name: Anti-HIV Agents

- Relevance: LOW

- As Found: Unknown

- ID: M25428

- Name: Anti-Retroviral Agents

- Relevance: LOW

- As Found: Unknown

- ID: M4314

- Name: Antiviral Agents

- Relevance: LOW

- As Found: Unknown

- ID: M4214

- Name: Anti-Infective Agents

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: C000088327

- Term: Plerixafor

- ID: D000078224

- Term: Lenograstim

### Misc Info Module

- Version Holder: 2024-05-31
