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Chronic Myeloid LeukemiaNCT05611216

Clinical Outcomes of 3L+ Therapies Among Patients With Chronic Myeloid Leukemia and Those With T315I Mutation

An observational study of Chronic Myeloid Leukemia, run by Novartis Pharmaceuticals at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The study was a retrospective, non-interventional patient chart review and used a panel of oncologists/hematologists from the US to collect real-world clinical outcomes of patients with CML-CP in 3L+ and those with the T315I mutation. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Chronic Myeloid Leukemia
How many people
164 participants
Where
1 siteUnited States
Study dates
1 Dec 2020to 23 Dec 2021
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: Patients with Chronic Myeloid Leukemia and those with T315I Mutation in the United States

Who may be able to join

  • Physician selection
  • Physicians were eligible to participate in the study if they fulfilled all of the following criteria:
  • Completed medical subspecialty training
  • Reported hematology, medical oncology, or any other oncology subspecialties as the primary medical subspecialty
  • Were responsible for treatment decisions and follow-up for ≥ 1 adult patient with Ph+ CML-CP who received a 3L or those with the T315I mutation since January 2013 (the date from which molecular monitoring response on the International Scale (IS) became a more standard procedure/commonly available
  • Had access to molecular monitoring results reported on the IS, and with a sensitivity level of precision for molecular response of MR3 (BCR ABL1/ABL1 ≤ 0.1% or 3-log reduction) or better
Show all 18Show fewer
  • Patient selection Participating physicians were directed to provide information on patients who were included into the following separate cohorts. Each participating physician contributed up to 5 patient medical charts from each cohort.
  • For the 3L cohort:
  • Adult patients diagnosed with Ph+ CML-CP who initiated a 1L therapy, switched to a 2L therapy, and initiated a 3L therapy for CML-CP
  • All lines of therapy (TKIs or other CML treatments) received outside of an interventional clinical trial setting
  • 3L therapy was initiated on or after January 1st, 2013 (when molecular monitoring became a common practice in CML monitoring) and no later than November 30th, 2018, to have a minimum of 2 years of follow-up after therapy initiation, except if the patient died before
  • For the T315I cohort:
  • Adult patients diagnosed with Ph+ CML-CP who initiated ≥ 1 line of therapy for Ph+ CML-CP and T315I mutation was identified
  • All lines of therapy (TKIs or other CML treatments) received outside of an interventional clinical trial setting
  • Line of therapy identified as the T315I line of interest was initiated on or after January 1st, 2013, and no later than November 30th, 2018, to have a minimum of 2 years of follow-up after therapy initiation, except if the patient died before
  • For both cohorts:
  • Patients with Ph+ CML-CP for whom the physician had complete information on the CML related care from CML diagnosis and for ≥ 2 years after the initiation of line of therapy of interest (i.e., 3L or line of therapy identified as the T315I line of interest), unless the patient died before. Complete information included: CML treatments, treatment duration, routine laboratory (e.g., complete blood count (CBC), BCR-ABL), CML status (e.g., SOKAL risk score, CP/accelerated phase (AP)/ blast crisis (BC)), medications, and clinical status (e.g., history, physical exam)
  • The physician had access to molecular monitoring results reported on the IS from initiation of the line of therapy of interest and with a sensitivity level of precision for molecular response of MR3 (BCR-ABL1/ABL1≤0.1% or 3-log reduction) or better Of note, the cohorts were not mutually exclusive such that patients included in the 3L cohort with T315I mutation was included in the T315I cohort. Thereafter, there was an oversampling of patients with T315I mutation. Patients from the T315I cohort from the oversampling with a 3L were not included in the 3L cohort.

Who cannot join

  • Physicians and patients who did not meet study inclusion criteria detailed above were excluded.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Novartis Investigative Site

    East Hanover, New Jersey, 07936

Other trials in United States

About this study

Official title: Assessing Real-world Clinical Outcomes of 3L+ Therapies Among Patients With Chronic Myeloid Leukemia and Those With T315I Mutation in the United States (ARC Study)

The study was a retrospective, non-interventional patient chart review and used a panel of oncologists/hematologists from the US to collect real-world clinical outcomes of patients with CML-CP in 3L+ and those with the T315I mutation.

Read the detailed description

This study included two distinct cohorts of CML-CP patients; that is, patients with CML-CP who initiated 3L for CML-CP (i.e., 3L cohort) and patients with CML-CP with T315I mutation (i.e., T315I cohort).

Study design for the analyses of the 3L cohort:

  • The index date: date of initiation of 3L therapy for CML-CP
  • The study period: period of ≥ 24 months following the index date unless the patient died before
  • Patient characteristics were measured at CML diagnosis and at the index date
  • The clinical outcomes of interest were measured during the study period

Study design for the analyses of the T315I cohort:

  • The index date: date of initiation of a line of therapy identified as the T315I line of interest (i.e., identification of T315I mutation before initiation or over the course of line of therapy)
  • The study period: period of ≥ 24 months following the index date unless the patient died before
  • Patient characteristics were measured at CML diagnosis and at the index date.
  • The clinical outcomes of interest were measured during the study period
Keywords
CML,, CP,, case report from,, lines of therapy,, T315I mutation
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT05611216
Study groupRoleWhat they receive
Third-line (3L) Cohortpatients with Chronic Myeloid Leukemia - Chronic Phase (CML-CP) who initiated 3L for CML-CP
T315I Cohortpatients with CML-CP with T315I mutation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of lines of therapy

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  2. Number of patients with total number of lines

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  3. Number of patients: Treatment received

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  4. Number of patients: Calendar year of line of therapy initiation

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  5. Duration of line of therapy

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  6. Number of patients with most frequent treatment sequences from first- to third-line of therapy

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  7. Number of patients who died after initiation of third-line therapy

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  8. Number of patients who progressed to AP/BC after initiation of third-line therapy

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  9. Number of patients who underwent HSCT after initiation of third-line therapy

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  10. Number of patients developed graft versus host disease after undergoing HSCT3

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  11. Number of patients who were still on the third-line therapy as of the data collection date

    To evaluate treatment patterns in patients with CML-CP who were previously treated with TKI or other CML treatments and were relapsed/refractory to/were intolerant/had other reasons for switching of CML therapy

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

Secondary outcomes (20)
  1. Number of patients who achieved molecular response during third-line therapy

    To evaluate the effectiveness of TKIs and other CML treatments used in 3L+ settings in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  2. Number of patients who achieved cytogenic response during third-line therapy

    To evaluate the effectiveness of TKIs and other CML treatments used in 3L+ settings in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  3. Number of patients who achieved complete hematologic response during third-line therapy

    To evaluate the effectiveness of TKIs and other CML treatments used in 3L+ settings in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  4. Number of patients who achieved molecular response during the line of therapy identified as the T315I line of interest

    To evaluate the effectiveness of TKIs and other treatments used in a line of therapy identified as the T315I line of interest in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  5. Number of patients who achieved cytogenic response during the line of therapy identified as the T315I line of interest

    To evaluate the effectiveness of TKIs and other treatments used in a line of therapy identified as the T315I line of interest in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  6. Number of patients who achieved complete hematologic response during the line of therapy identified as the T315I line of interest

    To evaluate the effectiveness of TKIs and other treatments used in a line of therapy identified as the T315I line of interest in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  7. Number of lines of therapy in T315I cohort

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  8. Number of patients: Total number of lines in T315I cohort

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  9. Number pf patients: Line identified as the T315I line of interest

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  10. Number of patients: Treatment received in T315I Cohort

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  11. Number of patients: Calendar year of line of therapy initiation in T315I Cohort

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  12. Duration of line of therapy in T315I Cohort

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  13. Most frequent treatment sequences in T315I Cohort

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  14. Number of patients who died after initiation of the line identified as the T315I line of interest

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  15. Number of patients who progressed to AP/BC after initiation of the line identified as the T315I line of interest

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  16. Number of patients who underwent HSCT after initiation of the line identified as the T315I line of interest

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  17. Number of patients developed graft versus host disease after undergoing HSCT

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  18. Number of patients who were still on the line identified as the T315I line of interest as of the data collection date

    To evaluate treatment patterns in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  19. Number of patients with BCR-ABL testing in 3L cohort

    To evaluate real-world BCR-ABL testing frequency per the latest National Comprehensive Cancer Network (NCCN) guidelines in 3L settings in patients with CML-CP

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

  20. Number of patients with BCR-ABL testing in T315I cohort

    To evaluate real-world BCR-ABL testing frequency per the latest NCCN guidelines in patients with CML-CP with T315I mutation

    Measured: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Nov 2022
  2. Study start1 Dec 2020
  3. Primary completion23 Dec 2021
  4. Study completion23 Dec 2021

Resources

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
Retrospective
Enrolment
164 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05611216
Sponsor's protocol ID
CABL001AUS09
Status last verified
Apr 2023
First submitted
4 Nov 2022
Last updated
6 Apr 2023
Results posted
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Chronic Myeloid Leukemia, run by Novartis Pharmaceuticals at 1 site in United States.

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 18 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: finished, as of the sponsor's last update on 6 Apr 2023. 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 United States. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of lines of therapy (time frame: throughout the study period, approximately 5 years (On or after January 1st, 2013 and no later than November 30th, 2018)). The study lists 11 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 6 Apr 2023.

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