OncologyPhase 1NCT03426605
A Study of LAM-003 in Patients With Acute Myeloid Leukemia
A Phase 1 study testing Open Label LAM-003 in people with Oncology and Acute Myeloid Leukemia, run by IRulya Therapeutics Inc. at 6 sites in United States.
Trial summary
A Phase 1 Dose-Escalation Study of LAM-003 in Patients with Acute Myeloid Leukemia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oncology, Acute Myeloid Leukemia
- What is being tested?
- Open Label LAM-003
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 6 sitesUnited States
- Study dates
- 16 Jan 2018to 5 Oct 2020
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Men and women of age ≥18 years.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
- Presence of measurable AML that has progressed during or relapsed after prior therapy
- All acute toxic effects of any prior antitumor therapy resolved to Grade 1.
- Adequate hepatic profile.
- Adequate renal function.
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- Adequate coagulation profile.
- Negative antiviral serology for human immunodeficiency virus (HIV), hepatitis B, and hepatitis C.
- For female subjects of childbearing potential, a negative serum pregnancy test.
- For both male and female subjects, willingness to use adequate contraception.
- Willingness and ability of the subject to comply with study activities.
- Evidence of a personally signed informed consent document.
Who cannot join
- Leukemic blast cell count >50 × 10^9/L before the start of study therapy and despite the use hydroxyurea, cytarabine, and/or cyclophosphamide.
- Presence of known central nervous system (CNS) leukemia.
- Presence of another major cancer.
- Ongoing Grade >1 proliferative or nonproliferative retinopathy.
- Significant cardiovascular disease or ECG abnormalities.
- Significant gastrointestinal disease
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- Uncontrolled ongoing infection.
- Pregnancy or breastfeeding.
- Major surgery within 4 weeks before the start of study therapy.
- Subject was a candidate for hematopoietic stem cell transplantation (HSCT).
- Ongoing severe graft-versus-house disease (GVHD) with Grade ≥2 serum bilirubin, Grade ≥3 skin involvement, or Grade ≥3 diarrhea at the start of study therapy.
- Prior solid organ transplantation.
- Ongoing immunosuppressive therapy other than corticosteroids.
- Use of a strong inhibitor or inducer of cytochrome P450 (CYP) 3A4.
- Use of a drug known to prolong the cardiac QT interval.
- Concurrent participation in another therapeutic or imaging clinical trial.
- Presence of a concomitant medical condition that (in the judgement of the investigator) interferes with the ability of the subject to participate in the study.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
University of Maryland
Baltimore, Maryland, 21201
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Virginia Cancer Specialists
Fairfax, Virginia, 22031
Hackensack Meridien Health
Hackensack, New Jersey, 07601
Yale University
New Haven, Connecticut, 06511
Weill Cornell Medical College
New York, New York, 10021
About this study
Official title: A Phase 1 Dose-Escalation Study of LAM-003 in Patients With Acute Myeloid Leukemia
A Phase 1 Dose-Escalation Study of LAM-003 in Patients with Acute Myeloid Leukemia
Read the detailed description
This clinical trial is a Phase 1 study evaluating the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of LAM-003 across a range of LAM-003 dose levels when administered to subjects with previously treated relapsed or refractory cute Myeloid Leukemia (AML).
Subjects will self-administer oral LAM-003 either once or twice per day as long as they are safely benefitting from therapy. Cohorts of 3 to 6 subjects will be sequentially enrolled at progressively higher dose levels of LAM-003 using a standard 3+3 dose-escalation design. Based on the pattern of dose-limiting toxicities observed in the first 4 weeks of therapy, escalation will proceed to define a recommended LAM-003 dosing regimen.
- Conditions
- Oncology, Acute Myeloid Leukemia
- Being tested
- Open Label LAM-003
- Medical subject headings
- Neoplasms, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Open Label LAM-003Drug
LAM-003
| Study group | Role | What they receive |
|---|---|---|
| LAM-003 | Experimental | Open label LAM-003 at three sequentially increasing starting dose levels of 200, 300 and 450 mg. Receives: Drug: Open Label LAM-003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD)
A primary objective was to determine the LAM-003 MTD and/or recommended dosing regimen (RDR) based on the pattern of dose-limiting toxicities (DLTs) in Cycle 1 of therapy. MTD as determined by DLTs.
Measured: At the end of the 28-day observation period for Cycle 1.
Secondary outcomes (6)
Adverse Event Assessment
Number and percentage of participants with an adverse event (AE).
Measured: Weekly during the first 4 weeks and then every 4 weeks for up to 48 weeks.
Maximum Plasma Concentration (Cmax)
The pharmacokinetic parameter Cmax was determined in plasma for the active metabolite of LAM-003A and LAM-003 (prodrug)
Measured: Cycle 1 Days 1, 2 and 8 (1 cycle = 28 days)
Time of Maximum Concentration [Tmax]
The pharmacokinetic parameter Tmax was determined in plasma for the active metabolite of LAM-003A and LAM-003 (prodrug)
Measured: Cycle 1 Days 1, 2 and 8 (1 cycle = 28 days)
Area Under the Curve [AUC]
The pharmacokinetic parameter area under the concentration-time curve was determined in plasma for the active metabolite of LAM-003A and LAM-003 (prodrug). AUClast is the area under the concentration-time curve from time-zero to the time of the last quantifiable concentration. AUCtau is the area under the concentration-time curve during the dosing interval where tau=24hours for once daily (QD) dosing. AUCtau was not calculated for LAM-003.
Measured: Cycle 1 Days 1, 2 and 8 (1 cycle = 28 days)
Objective Response Rate
Tumor response by AML response criteria (Cheson 2003).
Measured: Every 8 to 12 weeks for up to 48 weeks.
Event-Free Survival (EFS) and Overall Survival (OS)
Event-free survival (EFS), defined as the interval from the start of study therapy to the earliest of the first documentation of disease relapse, disease progression, treatment failure (TF), or death from any cause. Overall survival (OS), defined as the interval from the start of study therapy to death from any cause.
Measured: Every 8 to 12 weeks for up to 48 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2018
- Study start16 Jan 2018
- Primary completion20 Feb 2020
- Study completion5 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for OncologyStudies 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
- Model description
- Open Label, Dose-Escalation
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- IRulya Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Langdon Miller, M.D., Study director, AI Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03426605
- Sponsor's protocol ID
- LAM-003-HEM-CLN02
- Status last verified
- May 2024
- First submitted
- 5 Jan 2018
- Last updated
- 3 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Dose-Escalation Study of LAM-003 in Patients With Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT03426605
- Acronym / sponsor ID
- LAM-003-HEM-CLN02
- Conditions
- Oncology, Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- IRulya Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Jan 2018 (actual)
- Primary completion
- 20 Feb 2020 (actual)
- Study completion
- 5 Oct 2020 (actual)
- First posted
- 8 Feb 2018 (actual)
- Results
- Posted 3 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 1 Dose-Escalation Study of LAM-003 in Patients with Acute Myeloid Leukemia
Detailed description
This clinical trial is a Phase 1 study evaluating the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of LAM-003 across a range of LAM-003 dose levels when administered to subjects with previously treated relapsed or refractory cute Myeloid Leukemia (AML).
Subjects will self-administer oral LAM-003 either once or twice per day as long as they are safely benefitting from therapy. Cohorts of 3 to 6 subjects will be sequentially enrolled at progressively higher dose levels of LAM-003 using a standard 3+3 dose-escalation design. Based on the pattern of dose-limiting toxicities observed in the first 4 weeks of therapy, escalation will proceed to define a recommended LAM-003 dosing regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open Label, Dose-Escalation
| Arm | Type | Receives |
|---|---|---|
| LAM-003 | Experimental | Drug: Open Label LAM-003Open label LAM-003 at three sequentially increasing starting dose levels of 200, 300 and 450 mg. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Men and women of age ≥18 years.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
- Presence of measurable AML that has progressed during or relapsed after prior therapy
- All acute toxic effects of any prior antitumor therapy resolved to Grade 1.
- Adequate hepatic profile.
Key exclusion criteria (17 listed)
- Leukemic blast cell count >50 × 10^9/L before the start of study therapy and despite the use hydroxyurea, cytarabine, and/or cyclophosphamide.
- Presence of known central nervous system (CNS) leukemia.
- Presence of another major cancer.
- Ongoing Grade >1 proliferative or nonproliferative retinopathy.
- Significant cardiovascular disease or ECG abnormalities.
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 |
|---|---|---|
| Open Label LAM-003LAM-003 | Drug | LAM-003 |
Primary outcome
Maximum Tolerated Dose (MTD)
Time frame: At the end of the 28-day observation period for Cycle 1.
A primary objective was to determine the LAM-003 MTD and/or recommended dosing regimen (RDR) based on the pattern of dose-limiting toxicities (DLTs) in Cycle 1 of therapy. MTD as determined by DLTs.
6 secondary outcomes
Adverse Event Assessment
Time frame: Weekly during the first 4 weeks and then every 4 weeks for up to 48 weeks.
Maximum Plasma Concentration (Cmax)
Time frame: Cycle 1 Days 1, 2 and 8 (1 cycle = 28 days)
Time of Maximum Concentration [Tmax]
Time frame: Cycle 1 Days 1, 2 and 8 (1 cycle = 28 days)
Area Under the Curve [AUC]
Time frame: Cycle 1 Days 1, 2 and 8 (1 cycle = 28 days)
Objective Response Rate
Time frame: Every 8 to 12 weeks for up to 48 weeks.
Event-Free Survival (EFS) and Overall Survival (OS)
Time frame: Every 8 to 12 weeks for up to 48 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Trial information can be found on AI Therapeutics websiteLink provided by the sponsor.
- 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.
Langdon Miller, M.D. study director
AI Therapeutics
6 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 Open Label LAM-003 in people with Oncology and Acute Myeloid Leukemia, run by IRulya Therapeutics Inc. at 6 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 12 inclusion and 17 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 3 May 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 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?
Yes. Results are posted in the registry (first posted 3 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Maximum Tolerated Dose (MTD) (time frame: At the end of the 28-day observation period for Cycle 1.). 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 3 May 2024.