Ignite Creation Date:
2025-12-25 @ 2:02 AM
Ignite Modification Date:
2025-12-26 @ 3:09 AM
Study NCT ID:
NCT06261060
Status:
RECRUITING
Last Update Posted:
2025-10-24
First Post:
2024-02-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder
Sponsor:
M.D. Anderson Cancer Center
Organization:
Study Overview
Official Title:
Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder
Status:
RECRUITING
Status Verified Date:
2025-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
Detailed Description:
Primary Objective:
• Evaluate the safety and tolerability of low-dose sirolimus in participants with RUNX1 familial platelet disorder (RUNX1-FPD)
Secondary Objectives:
* Evaluate increases in platelet counts during and after treatment with low-dose sirolimus
* Evaluate changes in somatic mutation variant allele frequency (VAF)
* Monitor the rate of somatic mutation acquisition (ie, mutation burden)
* Assess change in platelet aggregation score
* Measure the change from baseline in bleeding score (ISTH-BAT)
* Evaluate change in mTORC1 downstream signaling (pS6/EBP)
Exploratory Objectives:
* Measure rescue of elevated cytokine profiles
* Evaluate reversal of myeloid skewing using flow cytometry
* Determine changes in bone marrow (eg, megakaryocytic atypia and cellularity)
* Assess changes in patient-reported outcomes measures (eg, EORTC and PRO-CTCAE)
* Describe the pharmacokinetics of sirolimus in patients with RUNX1-FPD
* Determine the correlation between sirolimus trough levels and each endpoint
• Evaluate the safety and tolerability of low-dose sirolimus in participants with RUNX1 familial platelet disorder (RUNX1-FPD)
Secondary Objectives:
* Evaluate increases in platelet counts during and after treatment with low-dose sirolimus
* Evaluate changes in somatic mutation variant allele frequency (VAF)
* Monitor the rate of somatic mutation acquisition (ie, mutation burden)
* Assess change in platelet aggregation score
* Measure the change from baseline in bleeding score (ISTH-BAT)
* Evaluate change in mTORC1 downstream signaling (pS6/EBP)
Exploratory Objectives:
* Measure rescue of elevated cytokine profiles
* Evaluate reversal of myeloid skewing using flow cytometry
* Determine changes in bone marrow (eg, megakaryocytic atypia and cellularity)
* Assess changes in patient-reported outcomes measures (eg, EORTC and PRO-CTCAE)
* Describe the pharmacokinetics of sirolimus in patients with RUNX1-FPD
* Determine the correlation between sirolimus trough levels and each endpoint
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| NCI-2024-01333 | OTHER | NCI-CTRP Clinical Registry | View |