Childhood Acute Lymphoblastic LeukemiaNCT05729178
Unravelling the Role of KCTD Protein Family in the Clinical Management of Childhood Acute Lymphoblastic Leukemias
An observational study of Childhood Acute Lymphoblastic Leukemia, run by IRCCS SYNLAB SDN at 1 site in Italy.
Trial summary
A transcriptomic analysis of bone marrow from B-ALL patients was performed by our research group for identifying novel protein/factor with a putative role of disease biomarker. Along with some already known B-ALL biomarkers, our analysis highlighted deregulation of some members of an emerging protein class denoted as… Read more about this study
- Who is it for?
- Ages 1 year to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Childhood Acute Lymphoblastic Leukemia
- What is being tested?
- observational study
- How many people
- 80 participants
- Where
- 1 siteItaly
- Study dates
- 2 Mar 2020to 31 Jul 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: The study foresees the possibility of recruiting approximately 60 patients with B-ALL and 15-20 patients with T-ALL. Furthermore, control subjects with age and sex comparable to those of the patients will be recruited. Finally, about 100 units of cord blood that do not meet the criteria for therapeutic cryopreservation and for which consent has been given for their use for scientific research will be used
Who may be able to join
- Patients aged 1-18 years of both sexes diagnosed with B- ALL and T-ALL;
- Patients who will have signed the informed consent
Who cannot join
- Patients who refuse to participate in the study;
- Patients who do not fall within the age range mentioned above
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
IRCCS Synlab SDN
Naples, 80143
Laura Pierri, MSC, 0812408470, direzionescientifica.irccssdn@synlab.it
About this study
A transcriptomic analysis of bone marrow from B-ALL patients was performed by our research group for identifying novel protein/factor with a putative role of disease biomarker. Along with some already known B-ALL biomarkers, our analysis highlighted deregulation of some members of an emerging protein class denoted as KCTD (Potassium ChannelTetramerization Domain-containing proteins). Starting from our preliminary observations, and considering that KCTDs havenever been studied in ALL, we decided to study these proteins in B- and T-ALL affected pediatric patients, enrolled by our research group in collaboration with AORN Santobono-Pausilipon pediatric oncological hospital.Indeed, the present research program aims at opening a new scenario for the study of KCTD proteins in childhood leukemias. The final goal of the project will be to evaluate the translational relevance of selected deregulated KCTDs as novel biomarkers useful for B-ALL and T-ALL diagnostics, and patient management.
- Conditions
- Childhood Acute Lymphoblastic Leukemia
- Being tested
- observational study
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
observational studyOther
analysis of biomarkers of interest (DNA-RNA and proteins) in MNC purified from the subjects' blood
| Study group | Role | What they receive |
|---|---|---|
| Pediatric acute lymphoblastic leukemia patients | pediatric patients of 1-16 years old affected by acute lymphoblastic leukemia Receives: Other: observational study | |
| healthy subjects | cord blood from healthy donors that cannot be used for clinical purposes Receives: Other: observational study |
What is being measured
The primary outcome is the main question the study is designed to answer.
KCTD expression levels, by RNAseq approach, in a cohort of B and T cell ALL affected patients compared to unaffected controls
We enroll during this project about 15-20 B-ALL, and 3-6 T-ALL patients.RNAseq experiments for transcriptome analysis will be performed on a study cohort of at least 8 B-ALL patients, 3-4 T-ALL patients, and naive B or T cells purified from cord blood as control for B- and T-ALL correspondingly. Raw counts will be used and an empirical Bayes approach applied (DESeq2, R package). The results will include, for each gene, log-fold change (log2), base mean, p-value and adjusted p-values (Benjamimi-Hochberg). DEgenes output will be exported into format suitable for IPA Ingenuity pathway AnalysisSoftware (Qiagen), for gene annotations, functional analysis and biomarker prediction. The KCTD proteins that will be dereguleted in B-ALL and T-ALL will be studied in detail.
Measured: 1-12 months
RT-PCR validation of deregulated KCTDs and identification of possible KCTDs interactors in leukemia by functional proteomic approach
After the interpretation of transcriptome data we will perform specific validation experiments by reat-time pcr, westernblotting, flow cytometry and microscopy. Briefly,total RNA will be purified from about 5e6 total MNC and used for RT-PCR experiments; BM smear will be collected for immunofluorescence analysis of deregulated KCTDs; protein total extract will be obtained from at least 1 e 6 total MNC and stored at -80°C until Western Blotting; finally, live mononuclear cells will be treted for permeabilization and cytoplasmic immunostaining of the KCTDs of interest. All experiments will be settled-up on B- and T-ALL in vitro human model systems and then performed on patients and controls cells. Moreover, in order to identify possible interactors of KCTDs proteins we will use B-ALL and T-ALL cell lines for functional proteomics analysis.
Measured: 13-24 months
Correlation of B-ALL patients clinical data with KCTDs expression level
Firstly, we will evaluated the usefulness of the deregulated KCTD proteins as novel markers of leukemia to be tested by multiparametric clinical flowcytometry. A specific protocol has been already setted-up by our research team, and it is able to stain cytoplasmicantigens (such as KCTD proteins) with surface antigens commonly used for to study hematoogical compartments by flowcytometry. This approach will be useful especially in monitoring patient response to treatment as well as detection of minimal residual disease. Moreover, being able to detect cytoplasmic KCTDs in combination with surface antigens, we planned to describe the KCTDs expression levels in multiple cellular population residing in marrow and peripheral blood.Secondly, the overall data obtained in this study will be also interpreted looking at the patient clinics with particular reference to the response to therapy, the risk of relapse and, when possible, resistance to therapy.
Measured: 25-36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2023
- Study start2 Mar 2020
- Primary completion1 Feb 2024
- Study completion31 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- IRCCS SYNLAB SDN
- Sponsor type
- University, hospital or other
- Collaborators
- Santobono-Pausilipon Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05729178
- Sponsor's protocol ID
- 5/19
- Status last verified
- Feb 2024
- First submitted
- 27 Jan 2023
- Last updated
- 25 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Unravelling the Role of KCTD Protein Family in the Clinical Management of Childhood Acute Lymphoblastic Leukemias
- ClinicalTrials.gov ID
- NCT05729178
- Acronym / sponsor ID
- 5/19
- Conditions
- Childhood Acute Lymphoblastic Leukemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- IRCCS SYNLAB SDNUniversity, hospital or other
- Collaborators
- Santobono-Pausilipon Hospital
- Study start
- 2 Mar 2020 (actual)
- Primary completion
- 1 Feb 2024 (actual)
- Study completion
- 31 Jul 2025 (estimated)
- First posted
- 15 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A transcriptomic analysis of bone marrow from B-ALL patients was performed by our research group for identifying novel protein/factor with a putative role of disease biomarker. Along with some already known B-ALL biomarkers, our analysis highlighted deregulation of some members of an emerging protein class denoted as KCTD (Potassium ChannelTetramerization Domain-containing proteins). Starting from our preliminary observations, and considering that KCTDs havenever been studied in ALL, we decided to study these proteins in B- and T-ALL affected pediatric patients, enrolled by our research group in collaboration with AORN Santobono-Pausilipon pediatric oncological hospital.Indeed, the present research program aims at opening a new scenario for the study of KCTD proteins in childhood leukemias. The final goal of the project will be to evaluate the translational relevance of selected deregulated KCTDs as novel biomarkers useful for B-ALL and T-ALL diagnostics, and patient management.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pediatric acute lymphoblastic leukemia patients | – | Other: observational studypediatric patients of 1-16 years old affected by acute lymphoblastic leukemia |
| healthy subjects | – | Other: observational studycord blood from healthy donors that cannot be used for clinical purposes |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- The study foresees the possibility of recruiting approximately 60 patients with B-ALL and 15-20 patients with T-ALL. Furthermore, control subjects with age and sex comparable to those of the patients will be recruited. Finally, about 100 units of cord blood that do not meet the criteria for therapeutic cryopreservation and for which consent has been given for their use for scientific research will be used
Key inclusion criteria (2 listed)
- Patients aged 1-18 years of both sexes diagnosed with B- ALL and T-ALL;
- Patients who will have signed the informed consent
Key exclusion criteria (2 listed)
- Patients who refuse to participate in the study;
- Patients who do not fall within the age range mentioned above
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| observational studyanalysis of biomarkers of interest (DNA-RNA and proteins) in MNC purified from the subjects' blood | Other | Pediatric acute lymphoblastic leukemia patients, healthy subjects |
Primary outcome
KCTD expression levels, by RNAseq approach, in a cohort of B and T cell ALL affected patients compared to unaffected controls
Time frame: 1-12 months
We enroll during this project about 15-20 B-ALL, and 3-6 T-ALL patients.RNAseq experiments for transcriptome analysis will be performed on a study cohort of at least 8 B-ALL patients, 3-4 T-ALL patients, and naive B or T cells purified from cord blood as control for B- and T-ALL correspondingly. Raw counts will be used and an empirical Bayes approach applied (DESeq2, R package). The results will include, for each gene, log-fold change (log2), base mean, p-value and adjusted p-values (Benjamimi-Hochberg). DEgenes output will be exported into format suitable for IPA Ingenuity pathway AnalysisSoftware (Qiagen), for gene annotations, functional analysis and biomarker prediction. The KCTD proteins that will be dereguleted in B-ALL and T-ALL will be studied in detail.
RT-PCR validation of deregulated KCTDs and identification of possible KCTDs interactors in leukemia by functional proteomic approach
Time frame: 13-24 months
After the interpretation of transcriptome data we will perform specific validation experiments by reat-time pcr, westernblotting, flow cytometry and microscopy. Briefly,total RNA will be purified from about 5e6 total MNC and used for RT-PCR experiments; BM smear will be collected for immunofluorescence analysis of deregulated KCTDs; protein total extract will be obtained from at least 1 e 6 total MNC and stored at -80°C until Western Blotting; finally, live mononuclear cells will be treted for permeabilization and cytoplasmic immunostaining of the KCTDs of interest. All experiments will be settled-up on B- and T-ALL in vitro human model systems and then performed on patients and controls cells. Moreover, in order to identify possible interactors of KCTDs proteins we will use B-ALL and T-ALL cell lines for functional proteomics analysis.
Correlation of B-ALL patients clinical data with KCTDs expression level
Time frame: 25-36 months
Firstly, we will evaluated the usefulness of the deregulated KCTD proteins as novel markers of leukemia to be tested by multiparametric clinical flowcytometry. A specific protocol has been already setted-up by our research team, and it is able to stain cytoplasmicantigens (such as KCTD proteins) with surface antigens commonly used for to study hematoogical compartments by flowcytometry. This approach will be useful especially in monitoring patient response to treatment as well as detection of minimal residual disease. Moreover, being able to detect cytoplasmic KCTDs in combination with surface antigens, we planned to describe the KCTDs expression levels in multiple cellular population residing in marrow and peripheral blood.Secondly, the overall data obtained in this study will be also interpreted looking at the patient clinics with particular reference to the response to therapy, the risk of relapse and, when possible, resistance to therapy.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Childhood Acute Lymphoblastic Leukemia, run by IRCCS SYNLAB SDN at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 25 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Laura Pierri, MSC (0812408470, direzionescientifica.irccssdn@synlab.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
KCTD expression levels, by RNAseq approach, in a cohort of B and T cell ALL affected patients compared to unaffected controls (time frame: 1-12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2024.