Myelodysplastic SyndromeMDS & PRRTNCT06510868
Evaluating Myelodysplastic Syndrome Risks in NET Patients Planned for Peptide Radionuclide Therapy
An observational study of Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
This is a prospective observational study which aims to identify individuals predisposed to developing myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) could improve patient outcomes in different ways. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- What is being tested?
- Peptide receptor radionuclide therapy (PRRT) and Blood collection
- Type of treatment
- Surgery or procedure
- How many people
- 45 participants
- Where
- 1 siteCanada
- Study dates
- 1 Aug 2024to 30 Jun 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This research proposes to study the genetic changes that occur pre-PRRT and post-PRRT using blood samples obtained from a patient population at Princess Margaret Hospital. Cohort A will consist of 20 patients that have had PRRT within the past 4 years. Cohort B will consist of 20 patients planned for PRRT. Cohort C will consist of 1-5 patients post PRRT, diagnosed with t-MN. In other words, patients will already be receiving PRRT or have received PRRT within 4 years.
Who may be able to join
- ECOG 0-3
- Life expectancy > 6 months
- Informed consent and willingness to undergoing serial genetic panel CHIP testing.
- Cohort Specific criteria
- Cohort A: PRRT completed within 5 years of enrolment
- Cohort B: PRRT planned to commence within 4 months of enrolment
- Cohort C: diagnosis of MDS or AML following prior PRRT.
Who cannot join
- Unwillingness to provide blood sample and follow up as per protocol
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site, 1 recruiting
- Recruiting
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 2M9
Rebecca Wong, MD, 416-946-4501 ext. 5736, rebecca.wong@rmp.uhn.on.ca
About this study
This is a prospective observational study which aims to identify individuals predisposed to developing myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) could improve patient outcomes in different ways. First, it will enable improved patient selection for PRRT where alternative treatment options are available. Second, understanding the final pathway and how it is modulated by PRRT could allow the design of strategies to halt this process. Third, while it is unknown whether the development of MDS and AML is a late effect of radiopharmaceuticals in general or it is confined to cancer populations or specific radioisotopes will need to be confirmed. Finally, understanding this devastating complication is expected to be the cornerstone towards advancing radiopharmaceuticals' role in the adjuvant setting.
Read the detailed description
Radiopharmaceuticals is currently used for the treatment of metastatic cancer date. While radiopharmaceuticals are generally well tolerated, one of its most devastating long-term toxicities is the development of therapy related myeloid neoplasms (t-MN), an umbrella term for myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). PRRT (Receptor Radionuclide Therapy) is a targeted radiopharmaceutical therapy (RPT) used to treat neuroendocrine tumors. RPTs use drugs to attack cancer cells while reducing harm to healthy tissue. PRRT delivers high doses of radiation to tumors in the body to destroy or slow their growth and reduce disease side effects.
While PRRT is generally well tolerated, one of its long-term side effects is the development of therapy related myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). The identification of genetic changes that lead to the development of MDS and AML during PRRT is a growing area of research. It is now known that the genetic changes that lead to progression into AML typically occur through many years of pre-leukemic hematopoietic stem cell clonal evolution, before development of late mutations that lead to malignant disease. The short interval between exposure to PRRT and appearance of MDS and AML would suggest some patients are already at high risk of developing AML and are potentially detectable. The ability to identify individuals predisposed to developing MDS/AML could improve patient selection for PRRT and design strategies to mitigate the development of MDS/AML.
This research proposes to study the genetic changes that occur pre-PRRT and post-PRRT using blood samples obtained from a patient population at Princess Margaret Hospital. Cohort A will consist of 20 patients that have had PRRT within the past 4 years. Cohort B will consist of 20 patients planned for PRRT. Cohort C will consist of 1-5 patients post PRRT, diagnosed with t-MN.
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Being tested
- Peptide receptor radionuclide therapy (PRRT), Blood collection
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Peptide receptor radionuclide therapy (PRRT)Radiation
Specialized type of radionuclide therapy used to treat neuroendocrine tumors.
Blood collectionDiagnostic test
Patients will have approximately 5 ml of blood drawn 6,12,24,36,48, 60 months and at the time of MDS/AML diagnosis on follow up. Genomic DNA will be extracted from serum sample using the Qiagen QIAamp DNA Mini Kit. Single-molecule molecular inversion probes (smMIPs) will be used to detect mutations. Single nucleotide variants (SNVs), short insertions and deletions (indels), and mutated myeloid genes will be captured (e.g PPM1D, DNMT3A, TET2, TP53).
| Study group | Role | What they receive |
|---|---|---|
| Previous PRRT | Patients who have received PRRT within the last 4 years. There are no baseline levels available for cohort A patients. Sample size: 20. Receives: Diagnostic Test: Blood collection | |
| Planned for PRRT | Patients who are scheduled to start PRRT in the next 3 months. Pre-PRRT clonal expansion status will only be available form this cohort. These patients will provide a comprehensive record of development of CH from exposure to PRRT. Sample size: 20. Receives: Radiation: Peptide receptor radionuclide therapy (PRRT); Diagnostic Test: Blood collection | |
| Post PRRT Diagnosed with t-MN (MDS or AML) | Patients who have t-MN (MDS or AML). Sample size: 5. Receives: Diagnostic Test: Blood collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify individuals predisposed to developing MDS/AML to improve patient selection for PRRT where alternative treatment options are available.
Determining the proportion of patients who screen positive for "prodromal AML genetic panel" pre PRRT.
Measured: 5 years
Secondary outcomes (5)
Detection of Genetic Mutations in the Blood Post-PRRT
Enrolled patients will undergo serial genetic panel testing of blood samples annually for up to 5 years to detect genetic mutations.
Measured: 5 years
Assessment of Variant Allele Frequencies Post-PRRT
Annual genetic panel testing will be conducted to determine variant allele frequencies in the blood of patients post-PRRT for up to 5 years. The frequencies of specific gene mutations (e.g., PPM1D, TET2, DNMT3A, TP53) will be measured and analyzed in relation to clinical characteristics and treatment history.
Measured: 5 years
Incidence of Therapy-Related Myeloid Neoplasms (t-MN) Post-PRRT
The incidence of therapy-related myeloid neoplasms (MDS and AML) will be monitored in patients post-PRRT over a 5-year follow-up period. Data will include the time to t-MN development and any associated genetic mutations identified through annual blood genetic testing.
Measured: 5 years
Proportion of Patients Developing MDS/AML Post-PRRT
Enrolled patients will undergo annual genetic panel testing for up to 5 years to determine the proportion who develop myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) after receiving PRRT. Data collected will include baseline clinical characteristics, the amount of PRRT received, prior antineoplastic therapies, and PRRT-related adverse events to help identify factors associated with the development of these conditions.
Measured: 5 years
Identification of Clonal Mutations Conferring Increased Risk of MDS/AML Post-PRRT
Enrolled patients will undergo annual genetic panel testing for up to 5 years to identify clonal mutations associated with an increased risk of developing myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) after receiving PRRT.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2024
- Study start1 Aug 2024
- Primary completion1 Apr 2028 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- 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
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06510868
- Sponsor's protocol ID
- 22-5247
- Acronym
- MDS & PRRT
- Status last verified
- Mar 2026
- First submitted
- 12 Jul 2024
- Last updated
- 5 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating Myelodysplastic Syndrome Risks in NET Patients Planned for Peptide Radionuclide Therapy
- ClinicalTrials.gov ID
- NCT06510868
- Acronym / sponsor ID
- MDS & PRRT / 22-5247
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 1 Apr 2028 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 19 Jul 2024 (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.
This is a prospective observational study which aims to identify individuals predisposed to developing myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) could improve patient outcomes in different ways. First, it will enable improved patient selection for PRRT where alternative treatment options are available. Second, understanding the final pathway and how it is modulated by PRRT could allow the design of strategies to halt this process. Third, while it is unknown whether the development of MDS and AML is a late effect of radiopharmaceuticals in general or it is confined to cancer populations or specific radioisotopes will need to be confirmed. Finally, understanding this devastating complication is expected to be the cornerstone towards advancing radiopharmaceuticals' role in the adjuvant setting.
Detailed description
Radiopharmaceuticals is currently used for the treatment of metastatic cancer date. While radiopharmaceuticals are generally well tolerated, one of its most devastating long-term toxicities is the development of therapy related myeloid neoplasms (t-MN), an umbrella term for myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). PRRT (Receptor Radionuclide Therapy) is a targeted radiopharmaceutical therapy (RPT) used to treat neuroendocrine tumors. RPTs use drugs to attack cancer cells while reducing harm to healthy tissue. PRRT delivers high doses of radiation to tumors in the body to destroy or slow their growth and reduce disease side effects.
While PRRT is generally well tolerated, one of its long-term side effects is the development of therapy related myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). The identification of genetic changes that lead to the development of MDS and AML during PRRT is a growing area of research. It is now known that the genetic changes that lead to progression into AML typically occur through many years of pre-leukemic hematopoietic stem cell clonal evolution, before development of late mutations that lead to malignant disease. The short interval between exposure to PRRT and appearance of MDS and AML would suggest some patients are already at high risk of developing AML and are potentially detectable. The ability to identify individuals predisposed to developing MDS/AML could improve patient selection for PRRT and design strategies to mitigate the development of MDS/AML.
This research proposes to study the genetic changes that occur pre-PRRT and post-PRRT using blood samples obtained from a patient population at Princess Margaret Hospital. Cohort A will consist of 20 patients that have had PRRT within the past 4 years. Cohort B will consist of 20 patients planned for PRRT. Cohort C will consist of 1-5 patients post PRRT, diagnosed with t-MN.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Previous PRRT | – | Diagnostic Test: Blood collectionPatients who have received PRRT within the last 4 years. There are no baseline levels available for cohort A patients. Sample size: 20. |
| Planned for PRRT | – | Radiation: Peptide receptor radionuclide therapy (PRRT), Diagnostic Test: Blood collectionPatients who are scheduled to start PRRT in the next 3 months. Pre-PRRT clonal expansion status will only be available form this cohort. These patients will provide a comprehensive record of development of CH from exposure to PRRT. Sample size: 20. |
| Post PRRT Diagnosed with t-MN (MDS or AML) | – | Diagnostic Test: Blood collectionPatients who have t-MN (MDS or AML). Sample size: 5. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This research proposes to study the genetic changes that occur pre-PRRT and post-PRRT using blood samples obtained from a patient population at Princess Margaret Hospital. Cohort A will consist of 20 patients that have had PRRT within the past 4 years. Cohort B will consist of 20 patients planned for PRRT. Cohort C will consist of 1-5 patients post PRRT, diagnosed with t-MN. In other words, patients will already be receiving PRRT or have received PRRT within 4 years.
Key inclusion criteria (7 listed)
- ECOG 0-3
- Life expectancy > 6 months
- Informed consent and willingness to undergoing serial genetic panel CHIP testing.
- Cohort Specific criteria
Key exclusion criteria (1 listed)
- Unwillingness to provide blood sample and follow up as per protocol
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 |
|---|---|---|
| Peptide receptor radionuclide therapy (PRRT)Specialized type of radionuclide therapy used to treat neuroendocrine tumors. | Radiation | Planned for PRRT |
| Blood collectionPatients will have approximately 5 ml of blood drawn 6,12,24,36,48, 60 months and at the time of MDS/AML diagnosis on follow up. Genomic DNA will be extracted from serum sample using the Qiagen QIAamp DNA Mini Kit. Single-molecule molecular inversion probes (smMIPs) will be used to detect mutations. Single nucleotide variants (SNVs), short insertions and deletions (indels), and mutated myeloid genes will be captured (e.g PPM1D, DNMT3A, TET2, TP53). | Diagnostic test | Planned for PRRT, Post PRRT Diagnosed with t-MN (MDS or AML), Previous PRRT |
Primary outcome
Identify individuals predisposed to developing MDS/AML to improve patient selection for PRRT where alternative treatment options are available.
Time frame: 5 years
Determining the proportion of patients who screen positive for "prodromal AML genetic panel" pre PRRT.
5 secondary outcomes
Detection of Genetic Mutations in the Blood Post-PRRT
Time frame: 5 years
Assessment of Variant Allele Frequencies Post-PRRT
Time frame: 5 years
Incidence of Therapy-Related Myeloid Neoplasms (t-MN) Post-PRRT
Time frame: 5 years
Proportion of Patients Developing MDS/AML Post-PRRT
Time frame: 5 years
Identification of Clonal Mutations Conferring Increased Risk of MDS/AML Post-PRRT
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2026.
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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.
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 Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by University Health Network, Toronto at 1 site in Canada.
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 7 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 5 Mar 2026. 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 Canada. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rebecca Wong (416-946-4501 ext. 5736, rebecca.wong@uhn.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identify individuals predisposed to developing MDS/AML to improve patient selection for PRRT where alternative treatment options are available. (time frame: 5 years). 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 5 Mar 2026.