- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06414889
Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Enrollment (Estimated)
Phase
- Early Phase 1
Contacts and Locations
Study Contact
- Name: Chitra Hosing, MD
- Phone Number: (713) 745-3219
- Email: cmhosing@mdanderson.org
Study Locations
-
-
Texas
-
Houston, Texas, United States, 77030
- Recruiting
- MD Anderson Cancer Center
-
Contact:
- Chitra Hosing, MD
- Phone Number: 713-745-3219
- Email: cmhosing@mdanderson.org
-
Principal Investigator:
- Chitra Hosing, MD
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion 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 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
Exclusion Criteria:
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)
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis
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). |
Given by IV or SC
Other Names:
Given by procedure
Given by IV
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
|
Through study completion; an average of 1 year.
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Chitra Hosing, M.D. Anderson Cancer Center
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2023-0799
- NCI-2024-04300 (Other Identifier: NCI-CTRP Clinical Registry)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on RUNX1 Familial Platelet Disorder
-
M.D. Anderson Cancer CenterNot yet recruitingFamilial Platelet Disorder | HematopoieticUnited States
-
Region StockholmKarolinska InstitutetRecruitingPlatelet Disorder | Thrombocytopathy | Platelet Function DisorderSweden
-
The First Affiliated Hospital of Soochow UniversityRecruiting
-
University Hospital, GenevaCompletedAcquired Platelet DisorderSwitzerland
-
Ayman SaadSobi, Inc.Withdrawn
-
Goethe UniversityCompletedAcquired Platelet Function DisorderGermany
-
Hôpital Necker-Enfants MaladesBambino Gesù Hospital and Research InstituteTerminatedAcquired Platelet Function DisorderFrance
-
Institut National de la Santé Et de la Recherche...Enrolling by invitationCharacterization of New Candidate Genes in Cases of Human Inherited Thrombocytopenia (CATCH) (CATCH)Thrombocytopenia | Inherited Platelet DisorderFrance
-
IRCCS Policlinico S. MatteoCompleted
-
Centre Hospitalier Universitaire, AmiensRecruitingPlatelet Aggregation | Heparin-induced Thrombocytopenia | Platelet Activation | Platelet DisorderFrance
Clinical Trials on G-CSF (filgrastim or biosimilar)
-
Seoul St. Mary's HospitalUnknownLeukemia, Myeloid, AcuteKorea, Republic of
-
Hanmi Pharmaceutical Company LimitedCompletedHealthyUnited States
-
MenoGeniX, Inc.National Institutes of Health (NIH); National Institute on Aging (NIA)CompletedPostmenopausal SymptomsUnited States
-
University of LeipzigCompletedAcute-On-Chronic Liver FailureGermany
-
Buddhist Tzu Chi General HospitalTerminated
-
Stanford UniversityRecruitingCervix Cancer | Endometrial CancerUnited States
-
University of FlorenceCompleted
-
Aalborg University HospitalTurku University Hospital; Oslo University Hospital; Helsinki University Central... and other collaboratorsTerminatedMalignant LymphomaDenmark, Sweden, Finland, Norway
-
University of Kansas Medical CenterMillennium Pharmaceuticals, Inc.CompletedAutologous Transplant | Malignant Lymphoma, Stem Cell TypeUnited States
-
Washington University School of MedicineCompletedLymphoma, Non-Hodgkin | Multiple MyelomaUnited States