Clinical trial NCT06781515: Assessment of Disease Burden in Hairy Cell Leukemia
Official title: Assessment of Disease Burden in Hairy Cell Leukemia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Hairy Cell Leukemia
Lead sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna
Sponsor class: OTHER
First posted: 2025-01-17
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Progression Free Survival (PFS)
timeFrame: through study completion, an average of 4 years
measure: Time to next treatment
timeFrame: through study completion, an average of 4 years
measure: 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.
timeFrame: through study completion, an average of 4 years
Secondary outcomes:
measure: mutational pattern of B-raf i
timeFrame: through study completion, an average of 4 years
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: IRCCS Azienda Ospedaliero - Universitaria di Bologna
Study officials:
affiliation: IRCCS Azienda Ospedaliero-Universitaria di Bologna
name: Pier Luigi Zinzani, MD
role: PRINCIPAL_INVESTIGATOR
References: