Promyelocytic Leukemia, Adult AcuteNCT04897490
RWE of 1st Line Treatment With ATO/ATRA for Adult APL
An observational study of Promyelocytic Leukemia, Adult Acute, run by Grupo Argentino de Tratamiento de la Leucemia Aguda at 6 sites in Argentina.
Trial summary
This is a multicenter, observational real world clinical trial with prospective follow up that will evaluate the treatment outcome of acute promyelocytic leukemia (APL) patients in the first line with arsenic trioxide and all trans retinoic acid (ATO/ATRA) based regimens in Argentina. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Promyelocytic Leukemia, Adult Acute
- What is being tested?
- Evaluation of first line treatment with ATO/ATRA outcome
- How many people
- 50 participants
- Where
- 6 sitesArgentina
- Study dates
- 1 Mar 2021to Dec 2026
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients with APL in first line treatment
Who may be able to join
- Patients 18 years or older.
- Signature of the form consent for participation in the study.
- Diagnosis of APL (either primary or secondary) according to the criteria of the World Health Organization (WHO), without prior treatment.
- Identification of the specific genetic alteration of APL by conventional karyotype, fluorescent in situ hybridization (FISH), reverse transcriptase polymerase chain reaction (RT-PCR or RQ-PCR). Identification of the transcript is recommended at the time of diagnosis isoforms: bcr1, bcr2, bcr3 essential to document the therapeutic response: Molecular remission
Who cannot join
- Presence of other concomitant active malignant tumors that require simultaneous treatment.
- Having received prior treatment for APL.
- Electrocardiogram abnormalities:
- Patients with a pre-existing diagnosis of Long QT Syndrome
- Patients with a baseline QTc of> 450msec. The Bazett formula should be used to measure the corrected QT interval (QT interval in msec divided by the square root of the RR interval in msec).
- Patients with a history or presence of significant ventricular or atrial tachyarrhythmia (Grade 3-4, CTCAE v5.2017).
Show all 13Show fewer
- Patients with right bundle branch block plus left anterior hemiblock. Bifascicular blocks are excluded.
- ECOG score 4.
- Stage III-IV heart failure.
- Serum creatinine ≥ 2.5 mg / dL (≥ 250 μmol / L) unless due to APL.
- Bilirubin ≥ 2.5 mg / dL, alkaline phosphatase, GPT or GOT> 3 times the normal limit unless it is for APL.
- Severe psychiatric illness.
- Women who are pregnant or who have decided to continue breastfeeding.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
Argentina6 sites, 6 recruiting
- Recruiting
CEMIC
CABA
Nicolás Cazap, Dr.
- Recruiting
FUNDALEU
CABA
Federico Sackmann, Dr.
- Recruiting
Hospital Italiano de La Plata
La Plata, Buenos Aires
Hernán Dick, Dr.
- Recruiting
Instituto Privado de Hematologia y Hemoterapia
Paraná, Entre Ríos Province
Pedro Negri Aranguren, Dr.
Florencia Negri Aranguren, Dr.
- Recruiting
Hospital Escuela de Agudos Dr. Ramón Madariaga
Posadas, Misiones Province
Diego Wolhein, Dr.
- Recruiting
Hospital Descentralizado Dr. Guillermo Rawson
Rawson, San Juan Province
María Celina Vanina, Dr.
About this study
Official title: Real World Evidence of First Line Treatment With Arsenic Trioxide Plus All Trans Retinoic Acid in Adult Patients With Acute Promyelocytic Leukemia
This is a multicenter, observational real world clinical trial with prospective follow up that will evaluate the treatment outcome of acute promyelocytic leukemia (APL) patients in the first line with arsenic trioxide and all trans retinoic acid (ATO/ATRA) based regimens in Argentina.
Read the detailed description
The purpose of this trial is to gather real world evidence of the characteristics of APL patients in Argentina who receive ATO/ATRA based treatment in first line following our national guidelines. The study primary endpoint is to evaluate event free survival and overall survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA) depending on risk category. Secondary endpoints are complete molecular response (CMR) rate, toxicity, early mortality and prognostic significance of FLT3.
Every APL patient diagnosed in our institutions will follow our guidelines with respect to diagnosis procedures. Risk category will depend on white blood cell counts (WBC), where WBC >10000 will be considered high risk (HR) and <10000 WBC, low risk (LR).
Patients will receive induction with ATO plus ATRA daily until hematologic remission or for a maximum of 60 days, followed by ATO 5 days/week, 4 weeks on 4 weeks off, for a total of 4 courses and ATRA 2 weeks on and 2 weeks off for a total of 7 courses.
HR patients will receive 2-3 doses of IDA at the beginning of induction. Central nervous system prophylaxis is contemplated for HR pts or those who have SNC bleeding.
Molecular response will be evaluated at the end of consolidation by RQ-PCR. LR patients who achieve CMR will not need to repeat molecular evaluations but HR patients will need RQ-PCR evaluation every 3 months during the first year and every 6 months during the second year.
- Conditions
- Promyelocytic Leukemia, Adult Acute
- Being tested
- Evaluation of first line treatment with ATO/ATRA outcome
- Keywords
- Acute Promyelocytic Leukemia, ATRA, ATO, IDA
- Medical subject headings
- Leukemia, Promyelocytic, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Evaluation of first line treatment with ATO/ATRA outcomeOther
Evaluation of first line treatment with ATO/ATRA outcome (o event free survival and overall survival and toxicity) in adult patients with APL.
| Study group | Role | What they receive |
|---|---|---|
| Adult APL in first line | Patients >/= 18 years old with recent diagnosis of acute promyelocytic leukemia who receive treatment with ATO/ATRA according to our local guidelines. HR patients will receive 2-3 additional doses of idarubicin. Receives: Other: Evaluation of first line treatment with ATO/ATRA outcome |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate overall survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA).
Evaluate overall survival of patients >/= 18 years old diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA) depending on risk category.
Measured: 36 months
Evaluate event free survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA).
Evaluate event free survival of patients >/= 18 years old diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA) depending on risk category.
Measured: 36 months
Secondary outcomes (4)
Evaluate complete molecular remission rate at the end of the consolidation treatment.
Measured: 36 months
Evaluate toxicity of the ATO/ATRA scheme (+/- IDA), measured according to type, frequency, severity and relation with the treatment of adverse events.
Measured: 36 months
Record early mortality (within 30 days of admission).
Measured: Within 30 days of admission
Evaluate whether the presence of mutated FLT3 has prognostic value in patients treated with ATRA/ATO.
The prognostic value will be analyzed by comparing rates of complete remission (CR), hematological relapse, molecular relapse, PFS and OS.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2021
- Study start1 Mar 2021
- Primary completion1 Jul 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Promyelocytic Leukemia, Adult AcuteStudies 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
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Grupo Argentino de Tratamiento de la Leucemia Aguda
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- María José Mela Osorio, Dr., Principal investigator, Grupo Argentino de Tratamiento de la Leucemia AgudaIsolda Fernández, Dr., Study chair, Grupo Argentino de Tratamiento de la Leucemia AgudaFederico Sackmann, Dr., Principal investigator, Grupo Argentino de Tratamiento de la Leucemia Aguda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04897490
- Sponsor's protocol ID
- GATLA 11-LPA-20
- Status last verified
- Jan 2024
- First submitted
- 18 May 2021
- Last updated
- 9 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Share study protocol
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real World Evidence of First Line Treatment With Arsenic Trioxide Plus All Trans Retinoic Acid in Adult Patients With Acute Promyelocytic Leukemia
- ClinicalTrials.gov ID
- NCT04897490
- Acronym / sponsor ID
- GATLA 11-LPA-20
- Conditions
- Promyelocytic Leukemia, Adult Acute
- Keywords
- Acute Promyelocytic Leukemia, ATRA, ATO, IDA
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Grupo Argentino de Tratamiento de la Leucemia AgudaUniversity, hospital or other
- Study start
- 1 Mar 2021 (actual)
- Primary completion
- 1 Jul 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 21 May 2021 (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 multicenter, observational real world clinical trial with prospective follow up that will evaluate the treatment outcome of acute promyelocytic leukemia (APL) patients in the first line with arsenic trioxide and all trans retinoic acid (ATO/ATRA) based regimens in Argentina.
Detailed description
The purpose of this trial is to gather real world evidence of the characteristics of APL patients in Argentina who receive ATO/ATRA based treatment in first line following our national guidelines. The study primary endpoint is to evaluate event free survival and overall survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA) depending on risk category. Secondary endpoints are complete molecular response (CMR) rate, toxicity, early mortality and prognostic significance of FLT3.
Every APL patient diagnosed in our institutions will follow our guidelines with respect to diagnosis procedures. Risk category will depend on white blood cell counts (WBC), where WBC >10000 will be considered high risk (HR) and <10000 WBC, low risk (LR).
Patients will receive induction with ATO plus ATRA daily until hematologic remission or for a maximum of 60 days, followed by ATO 5 days/week, 4 weeks on 4 weeks off, for a total of 4 courses and ATRA 2 weeks on and 2 weeks off for a total of 7 courses.
HR patients will receive 2-3 doses of IDA at the beginning of induction. Central nervous system prophylaxis is contemplated for HR pts or those who have SNC bleeding.
Molecular response will be evaluated at the end of consolidation by RQ-PCR. LR patients who achieve CMR will not need to repeat molecular evaluations but HR patients will need RQ-PCR evaluation every 3 months during the first year and every 6 months during the second year.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adult APL in first line | – | Other: Evaluation of first line treatment with ATO/ATRA outcomePatients >/= 18 years old with recent diagnosis of acute promyelocytic leukemia who receive treatment with ATO/ATRA according to our local guidelines. HR patients will receive 2-3 additional doses of idarubicin. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients with APL in first line treatment
Key inclusion criteria (4 listed)
- Patients 18 years or older.
- Signature of the form consent for participation in the study.
- Diagnosis of APL (either primary or secondary) according to the criteria of the World Health Organization (WHO), without prior treatment.
- Identification of the specific genetic alteration of APL by conventional karyotype, fluorescent in situ hybridization (FISH), reverse transcriptase polymerase chain reaction (RT-PCR or RQ-PCR). Identification of the transcript is recommended at the time of diagnosis isoforms: bcr1, bcr2, bcr3 essential to document the therapeutic response: Molecular remission
Key exclusion criteria (13 listed)
- Presence of other concomitant active malignant tumors that require simultaneous treatment.
- Having received prior treatment for APL.
- Electrocardiogram abnormalities:
- ECOG score 4.
- Stage III-IV heart failure.
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 |
|---|---|---|
| Evaluation of first line treatment with ATO/ATRA outcomeEvaluation of first line treatment with ATO/ATRA outcome (o event free survival and overall survival and toxicity) in adult patients with APL. | Other | Adult APL in first line |
Primary outcome
Evaluate overall survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA).
Time frame: 36 months
Evaluate overall survival of patients >/= 18 years old diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA) depending on risk category.
Evaluate event free survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA).
Time frame: 36 months
Evaluate event free survival of patients >/= 18 years old diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA) depending on risk category.
4 secondary outcomes
Evaluate complete molecular remission rate at the end of the consolidation treatment.
Time frame: 36 months
Evaluate toxicity of the ATO/ATRA scheme (+/- IDA), measured according to type, frequency, severity and relation with the treatment of adverse events.
Time frame: 36 months
Record early mortality (within 30 days of admission).
Time frame: Within 30 days of admission
Evaluate whether the presence of mutated FLT3 has prognostic value in patients treated with ATRA/ATO.
Time frame: 36 months
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 9 Jan 2024.
- Individual participant dataSharing planned: Yes. Share study protocol
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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.
María José Mela Osorio, Dr. principal investigator
Grupo Argentino de Tratamiento de la Leucemia Aguda
Isolda Fernández, Dr. study chair
Grupo Argentino de Tratamiento de la Leucemia Aguda
Federico Sackmann, Dr. principal investigator
Grupo Argentino de Tratamiento de la Leucemia Aguda
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Promyelocytic Leukemia, Adult Acute, run by Grupo Argentino de Tratamiento de la Leucemia Aguda at 6 sites in Argentina.
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 4 inclusion and 13 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 9 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Argentina. 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 Federico Sackmann, Dr. (+5491149720765, fsackmann@fundaleu.org.ar), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate overall survival of patients diagnosed with APL and treated in first line with ATO/ATRA or ATO/ATRA/Idarubicin (IDA). (time frame: 36 months). The study lists 2 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 9 Jan 2024.