Multiple MyelomaPhase 1NCT04543305
A Study of PRT1419 in Patients With Relapsed/Refractory Hematologic Malignancies
A Phase 1 study testing PRT1419 in people with Multiple Myeloma and Acute Myeloid Leukemia, run by Prelude Therapeutics Incorporated at 5 sites in United States.
Trial summary
This is a Phase 1 dose-escalation study of PRT1419, a myeloid cell leukemia 1 (MCL1) inhibitor, in patients with relapsed/refractory hematologic malignancies. The purpose of this study is to define the dosing schedule, maximally tolerated dose and/or estimate the optimal biological dose to be used in subsequent… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma, Acute Myeloid Leukemia, Non Hodgkin Lymphoma
- What is being tested?
- PRT1419
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 5 sitesUnited States
- Study dates
- 28 Sep 2020to 21 Mar 2022
- Phase
- Phase 1
- 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
Who may be able to join
- Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
- Adequate organ function (bone marrow, hepatic, renal, cardiovascular)
- Left ventricular ejection fraction of ≥50%
- Female patients of childbearing potential must have a negative pregnancy test within 7 days of the start of treatment and must agree to use a highly effective method of contraception during the trial
- Patients must have recovered from the effects of any prior cancer related therapy, radiotherapy or surgery (toxicity ≤ Grade 1)
- All patients on prior investigational agents must wait at least 5 half-lives of the agent in question, or 14 days, whichever is longer before study entry
Show all 15Show fewer
- AML patients only: Pathologically confirmed diagnosis of AML as defined by the WHO Classification and patients with targeted mutations must have been treated with appropriate therapy for their disease
- White blood cell count < 25 x 10^9/L. Hydrea or leukapheresis are permitted to meet this criterion.
- CMML patients only: intermediate-2 or high risk per CMML-specific prognostic scoring system (CPSS) or clinical/molecular CPSS (CPSS-mol) criteria. Must have failed prior therapy with a hypomethylating agent.
- MDS patients only: Intermediate, high, or very high risk by International Prognostic Scoring System-Revised [IPSS-R] criteria that is relapsed or refractory to approved therapies or MDS/MPN Overlap Syndrome (displaying both fibrosis and dysplastic features).
- NHL patients only: Histologically or cytologically confirmed NHL, including B- and T-cell lymphomas that is relapsed or refractory or intolerant to approved therapies. Must have one lesion that can be measured for response
- MM patients only: Measurable disease defined by one or more of the following: Serum M-protein ≥ 0.5 g/dL, Urine M-protein ≥ 200 mg/24 hours, Serum Free Light Chain (sFLC) > 10 mg/dL with normal serum FLC ratio. Presence of soft tissue plasmacytoma confirmed by imaging
- NHL and MM patients only: must have the following lab values within 14 days prior to study Day 1:
- ANC ≥1.0 x 10^3 μL
- Platelet count ≥50,000 μL
Who cannot join
- Known hypersensitivity to any of the components of PRT1419
- Female patients who are pregnant or lactating
- Mean QTcF interval of >480 msec
- History of heart failure, additional risk factors for arryhthmias or requiring concomitant medications that prolong the QT/QTc interval
- Hematopoietic stem-cell transplant < 90 days or have GVHD Grade >1 at study entry
- Uncontrolled intercurrent illnesses
Show all 15Show fewer
- Treatment with strong inhibitors of CYP2C8 and/or P-glycoprotein for which there are no therapeutic substitutions
- Inflammatory disorders of the gastrointestinal tract, or subjects with GI malabsorption
- HIV positive; known active hepatitis B or C
- Prior exposure to an MCL1 inhibitor
- History of another malignancy except:
- Malignancy treated with curative intent with no known active disease for >2 years at study entry
- Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease
- Adequately treated carcinoma in situ without evidence of disease
- Other concurrent low-grade malignancies (i.e chronic lymphocytic leukemia (Rai 0)) may be considered after consultation with Sponsor.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Colorado Blood Cancer Institute
Denver, Colorado, 80218
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Florida Cancer Specialists
Lake Mary, Florida, 32742
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Florida Cancer Specialists
Sarasota, Florida, 34232
About this study
Official title: A Phase 1, Open-Label, Multicenter, Dose-Escalation Study of PRT1419 in Patients With Relapsed/Refractory Hematologic Malignancies
This is a Phase 1 dose-escalation study of PRT1419, a myeloid cell leukemia 1 (MCL1) inhibitor, in patients with relapsed/refractory hematologic malignancies. The purpose of this study is to define the dosing schedule, maximally tolerated dose and/or estimate the optimal biological dose to be used in subsequent development of PRT1419.
Read the detailed description
This is a multicenter, open-label, dose-escalation Phase 1 study of PRT1419, a MCL1 inhibitor, evaluating patients in two cohorts as part of a 28-day treatment cycle in adult patients with multiple myeloma (MM), non-Hodgkin's lymphoma (NHL), acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), high-risk myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) overlap syndrome. Cohort A will evaluate PRT1419 administered as monotherapy in patients with either AML, CMML and/or high-risk MDS or MDS/MPN overlap. Cohort B will evaluate PRT1419 administered as monotherapy in patients with NHL or MM. The study will employ a "3+3" dose escalation design. The dose may be escalated until a dose limiting toxicity is identified.
- Being tested
- PRT1419
- Medical subject headings
- Multiple Myeloma, Leukemia, Myeloid, Acute, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRT1419Drug
PRT1419 will be administered orally
| Study group | Role | What they receive |
|---|---|---|
| PRT1419 | Experimental | PRT1419 will be administered orally Receives: Drug: PRT1419 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To describe dose limiting toxicities (DLT) of PRT1419
Dose limiting toxicities will be evaluated through the first cycle
Measured: Baseline through Day 28
To determine the maximally tolerated dose (MTD) and/or optimal biological dose (OBD)
The MTD and/or OBD will be established for further investigation in participants with multiple myeloma, Non-Hodgkin's Lymphoma, acute myeloid leukemia and myelodysplastic syndrome
Measured: Baseline through approximately 2 years
To determine the recommended phase 2 dose (RP2D) and schedule of PRT1419
The RP2D will be established for further investigation in participants with multiple myeloma, Non-Hodgkin's Lymphoma, acute myeloid leukemia and myelodysplastic syndrome
Measured: Baseline through approximately 2 years
Secondary outcomes (3)
To describe the adverse event profile and tolerability of PRT1419
Adverse events as characterized by type, frequency, severity, timing, seriousness and relationship to study therapy
Measured: Baseline through approximately 2 years
To describe the pharmacokinetic profile of PRT1419
PRT1419 pharmacokinetics will be calculated including the maximum observed plasma concentration
Measured: Baseline through approximately 2 years
To describe any anti-tumor activity of PRT1419
Anti-tumor activity of PRT1419 will be based on the measurement of objective responses
Measured: Baseline through approximately 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2020
- Study start28 Sep 2020
- Primary completion21 Mar 2022
- Study completion21 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple MyelomaStudies 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prelude Therapeutics Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04543305
- Sponsor's protocol ID
- PRT1419-01
- Status last verified
- Nov 2022
- First submitted
- 2 Sep 2020
- Last updated
- 15 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Multicenter, Dose-Escalation Study of PRT1419 in Patients With Relapsed/Refractory Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT04543305
- Acronym / sponsor ID
- PRT1419-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Prelude Therapeutics IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 28 Sep 2020 (actual)
- Primary completion
- 21 Mar 2022 (actual)
- Study completion
- 21 Mar 2022 (actual)
- First posted
- 10 Sep 2020 (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 Phase 1 dose-escalation study of PRT1419, a myeloid cell leukemia 1 (MCL1) inhibitor, in patients with relapsed/refractory hematologic malignancies. The purpose of this study is to define the dosing schedule, maximally tolerated dose and/or estimate the optimal biological dose to be used in subsequent development of PRT1419.
Detailed description
This is a multicenter, open-label, dose-escalation Phase 1 study of PRT1419, a MCL1 inhibitor, evaluating patients in two cohorts as part of a 28-day treatment cycle in adult patients with multiple myeloma (MM), non-Hodgkin's lymphoma (NHL), acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), high-risk myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) overlap syndrome. Cohort A will evaluate PRT1419 administered as monotherapy in patients with either AML, CMML and/or high-risk MDS or MDS/MPN overlap. Cohort B will evaluate PRT1419 administered as monotherapy in patients with NHL or MM. The study will employ a "3+3" dose escalation design. The dose may be escalated until a dose limiting toxicity is identified.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PRT1419 | Experimental | Drug: PRT1419PRT1419 will be administered orally |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
- Adequate organ function (bone marrow, hepatic, renal, cardiovascular)
- Left ventricular ejection fraction of ≥50%
- Female patients of childbearing potential must have a negative pregnancy test within 7 days of the start of treatment and must agree to use a highly effective method of contraception during the trial
- Patients must have recovered from the effects of any prior cancer related therapy, radiotherapy or surgery (toxicity ≤ Grade 1)
Key exclusion criteria (15 listed)
- Known hypersensitivity to any of the components of PRT1419
- Female patients who are pregnant or lactating
- Mean QTcF interval of >480 msec
- History of heart failure, additional risk factors for arryhthmias or requiring concomitant medications that prolong the QT/QTc interval
- Hematopoietic stem-cell transplant < 90 days or have GVHD Grade >1 at study entry
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 |
|---|---|---|
| PRT1419PRT1419 will be administered orally | Drug | PRT1419 |
Primary outcome
To describe dose limiting toxicities (DLT) of PRT1419
Time frame: Baseline through Day 28
Dose limiting toxicities will be evaluated through the first cycle
To determine the maximally tolerated dose (MTD) and/or optimal biological dose (OBD)
Time frame: Baseline through approximately 2 years
The MTD and/or OBD will be established for further investigation in participants with multiple myeloma, Non-Hodgkin's Lymphoma, acute myeloid leukemia and myelodysplastic syndrome
To determine the recommended phase 2 dose (RP2D) and schedule of PRT1419
Time frame: Baseline through approximately 2 years
The RP2D will be established for further investigation in participants with multiple myeloma, Non-Hodgkin's Lymphoma, acute myeloid leukemia and myelodysplastic syndrome
3 secondary outcomes
To describe the adverse event profile and tolerability of PRT1419
Time frame: Baseline through approximately 2 years
To describe the pharmacokinetic profile of PRT1419
Time frame: Baseline through approximately 2 years
To describe any anti-tumor activity of PRT1419
Time frame: Baseline through approximately 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Nov 2022.
Related trials for Multiple Myeloma
Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
Multiple Myeloma
Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
Multiple Myeloma
Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PRT1419 in people with Multiple Myeloma and Acute Myeloid Leukemia, run by Prelude Therapeutics Incorporated at 5 sites 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 15 inclusion and 15 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 15 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
To describe dose limiting toxicities (DLT) of PRT1419 (time frame: Baseline through Day 28). 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 15 Nov 2022.