Assessment of Disease Burden in Hairy Cell Leukemia (BRAF)

Drug-free, single-center, prospective observational pilot study in hairy Cell Leukemia patients

Study Overview

• Status: Recruiting
• Conditions: Hairy Cell Leukemia
• Intervention / Treatment: Other: Peripheral and BM blood sample

Detailed Description

The V600E gene lesion of B-raf, specific and almost always present in patients with hairy cell leukemia, correlates with the presence of neoplastic cells, therefore of active disease. The measurement of the fractional abundance of the mutated gene, by ddPCR, could therefore constitute a method of molecular assessment of the minimal residual disease. In addition, the values of fractional abundance (FA) of the mutated allele obtained can be integrated coherently in patients' clinical context, along with their PB counts and BM findings.

Primary objective Verify whether the absence of mutation at the end of treatment, indicative of a state of complete molecular response to therapy, can represent a predictor of long treatment-free survival.

Secondary objectives Verify the association between the absence of mutation and the duration of response in patients who do not need treatment for at least 5 years after only one treatment with purine analogues (cladribine and pentostatin) and judged in CR according to current criteria.

• Study Type: Interventional
• Enrollment (Estimated): 45
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Pier Luigi Zinzani, MD; Phone Number: +390512143680; Email: pierluigi.zinzani@unibo.it
• Study Contact Backup: Name: Alessandro Broccoli, MD; Phone Number: +39 0512143680; Email: Alessandro.broccoli@studio.unibo.it

Study Locations

• Italy
  • Bologna, Italy, 40138
    • Recruiting
    • IRCCS Azienda Ospedaliero - Universitaria di Bologna
    • Contact: Pier Luigi Zinzani, MD
    • Contact: Pier Luigi Zinzani, MD; Phone Number: +390512143680; Email: pierluigi.zinzani@unibo.it
    • Contact: Alessandro Broccoli, MD; Phone Number: +39 0512143680; Email: Alessandro.broccoli@studio.unibo.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Histologically confirmed diagnosis of HCL patients:

   1. newly diagnosed and candidates for first-line cytoreductive treatment with analogues purines or
   2. in relapse after a previous line of treatment, with indication for rescue therapy (repetition of a purine analogue; use of targeted or innovative drugs), except splenectomy or
   3. in CR for at least 5 years after a first line of treatment, in the absence of clinical alterations indicative of a state of hematological relapse, or in any case in the absence of an indication for a new line of cytoreductive therapy (time-to-next treatment exceeding 5 years).
2. Age ≥ 18 years at enrollment
3. Signature of written informed consent

Exclusion Criteria:

1. Concomitant second malignancy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: HCL, B-raf V600E-mutated patients; For each patient only pheripheral and medullary blood sample and medullary biospy will be collected	Other: Peripheral and BM blood sample; Peripheral and BM blood samples will be analyzed with the ddPCR method

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression Free Survival (PFS)	Progression Free Survival (PFS)	through study completion, an average of 4 years
Time to next treatment	Time to next treatment	through study completion, an average of 4 years
Correlation between the share of mutated allele (fractional abundance) with the response to the treatment.Correlation between the share of mutated allele (fractional abundance) with the response to the treatment.	Correlation between the share of mutated allele (fractional abundance) with the response	through study completion, an average of 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
mutational pattern of B-raf i	Evaluation of the mutational pattern of B-raf in patients with HCL in long hematological response	through study completion, an average of 4 years

Collaborators and Investigators

• Sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna
• Investigators: Principal Investigator: Pier Luigi Zinzani, MD, IRCCS Azienda Ospedaliero-Universitaria di Bologna

Study record dates

Study Major Dates

• Study Start (Estimated): January 1, 2025
• Primary Completion (Estimated): November 1, 2025
• Study Completion (Estimated): November 1, 2025

Study Registration Dates

• First Submitted: December 3, 2024
• First Submitted That Met QC Criteria: January 13, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 13, 2025
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia; Leukemia, Hairy Cell
• Other Study ID Numbers: BRAF

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No