Acute Myeloid LeukemiaPhase 1NCT03564288
Study to Find a Safe and Effective Dose of SKI-G-801 in the Treatment of Patients With Acute Myeloid Leukemia (AML)
A Phase 1 study testing SKI-G-801 in people with Acute Myeloid Leukemia, run by Oscotec Inc. at 2 sites in United States.
Trial summary
This Phase I study is designed to assess the safety, tolerability, pharmacokinetics and anti-tumor effect of increasing doses of study drug SKI-G-801 in patients with relapsed or refractory Acute Myeloid Leukemia (AML) who are unresponsive to currently available therapies. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- SKI-G-801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 2 sitesUnited States
- Study dates
- 23 Feb 2018to 2 Sep 2021
- 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
- Willing and able to provide written informed consent for participation, prior to completing any study-related procedures.
- Diagnosis of Acute Myeloid Leukemia (AML)
- Patients must have been off previous antileukemia therapy for at least 2 weeks or 5 half-lives, whichever is longer if the immediate prior regimen included only weekly chemotherapy; or 4 weeks or 5 half-lives, whichever is longer, from any therapy with therapeutic biologics and from any type of investigational therapy. Daily hydroxyurea for up to 2 weeks to keep the absolute blast count below 50 x 10⁹/L will be allowed, but must be discontinued 24 hours prior to administration of study drug. Hydroxyurea will be permitted during the first cycle of treatment if necessary.
- At least one prior induction regimen (with or without consolidation) which may have included hematopoietic stem cell transplantation (HSCT).
- Have adequate liver function.
- Have adequate renal (kidney) function.
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- Female patients must either be of non-childbearing potential, or, if of childbearing potential, have a negative urine pregnancy test at screening and agree not to try to become pregnant during the study and for 45 days after the final study drug administration. Women of childbearing potential, if heterosexually active, must agree to use 2 forms of highly effective birth control as determined by the protocol, starting at screening, throughout the study period and for 45 days after the final study drug administration.
- Female patients must agree not to breastfeed at screening, throughout the study period and for 45 days after the final study drug administration.
- Male patients with female spouse/partner of childbearing potential, must agree to use 2 forms of highly effective birth control as determined by the protocol, starting at screening, throughout the study period and for 45 days after the final study drug administration.
Who cannot join
- Patient has a diagnosis of Acute Promyelocytic Leukemia (APL) or chronic myelogenous leukemia in blast crisis.
- If patient is post allogenic transplant and requires therapy for graft vs host disease (GVHD) within 14 days prior to date of screening.
- Requires treatment with concomitant drugs that prolong QT/QTc interval.
- Recent history of cardiac ischemic disease (acute myocardial infarction within 6 months; uncontrolled angina); severe uncontrolled ventricular arrhythmia; recent transient ischemic attack or stroke within 6 months of screening; poorly controlled hypertension (systolic blood pressure >140 mm Hg or diastolic blood pressure >90 mm Hg).
- Patient has active, untreated central nervous system (CNS) disease.
- Other protocol defined inclusion/exclusion criteria could apply.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
USC Norris Comprehensive Cancer Center, Clinical Investigations Support Office (CISO), 1441 Eastlake Ave., Rm. 7327
Los Angeles, California, 90033
Innovative Clinical Research Institute
Whittier, California, 90603
About this study
Official title: A Phase 1 Dose Escalation Trial of SKI-G-801 in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)
This Phase I study is designed to assess the safety, tolerability, pharmacokinetics and anti-tumor effect of increasing doses of study drug SKI-G-801 in patients with relapsed or refractory Acute Myeloid Leukemia (AML) who are unresponsive to currently available therapies. Eligible participants will receive cycles of treatment involving IV infusion of SKI-G-801 daily for 14 days followed by 14 days off. Treatment cycles will be repeated until progressive disease or unacceptable toxicity.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- SKI-G-801
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SKI-G-801Drug
SKI-G-801 is administered as an IV infusion over 10 minutes
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation Cohort | Experimental | To identify the recommended phase 2 dose (RP2D) of SKI-G-801 in patients with relapsed or refractory AML (Acute Myeloid Leukemia) Receives: Drug: SKI-G-801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended phase 2 dose (RP2D)
RP2D of SKI-G-801 determined using Neuenschwander's continual reassessment method (N-CRM)
Measured: From Cycle 1, Day 1 until disease progression, unacceptable toxicity, patient withdrawal from study, or judged not to be in patient's interest to continue in study, assessed up to 36 months
Patients in complete remission or showing partial response (overall response rate [ORR])
Number of patients showing composite complete remission (complete remission [CR], complete remission with incomplete platelet recovery [CRp], and complete remission with incomplete hematologic recovery [CRi]) of SKI-G-801 according to the Response Criteria in AML
Measured: Up to 30 days following last dose of study drug
Patients in complete remission
Number of patients showing complete remission (CR)
Measured: Day 84 (± 3 days)
Duration of remission
Number of days between a patient's first reported status of complete remission (CR) and the earlier of disease relapse or death from any cause
Measured: From date of first reported status of CR to the date of disease relapse or death (+ 1 day); or to date of last available disease status report for patients who do not relapse, assessed up to 36 months
Duration of event free survival
Number of days between start of treatment to date of event
Measured: Day 1 to date of event (first documented treatment failure, relapse from CR or Cri [CR with incomplete hematologic recovery], or death due to any cause), assessed up to 36 months
Time to treatment response (TTR)
Number of days between the start of treatment to the date of first subsequent disease status of complete remission (CR)
Measured: Day 1 to date of first subsequent disease status of CR (+ 1 day), assessed up to 36 months
Dose limiting toxicity (DLT) Adverse Events (AEs)
Number of any DLT AEs within the first cycle of each patient's treatment with SKI-G-801
Measured: Up to Day 28
Secondary outcomes (4)
Incidence of Adverse Events (AEs)
Number, severity (as graded by National Cancer Institute Common Terminology Criteria for Adverse Events [NCI-CTCAE] v4.03), seriousness and relatedness to treatment of treatment-emergent AEs
Measured: Up to 30 days following last dose of study drug
Number of participants with clinical laboratory abnormalities
Measured: Up to 30 days following last dose of study drug
Number of participants with overall safety profiles
Measured: Up to 30 days following last dose of study drug
Number of participants with electrocardiogram (ECG) abnormalities
Measured: Up to 30 days following last dose of study drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2018
- Study start23 Feb 2018
- Primary completion2 Sep 2021
- Study completion2 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oscotec Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PPD Development, LP
- Responsible party
- Sponsor
- Investigators
- Eunice Wang, MD, Principal investigator, Roswell Park Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03564288
- Sponsor's protocol ID
- OSCO-P1301
- Status last verified
- May 2022
- First submitted
- 3 May 2018
- Last updated
- 21 Feb 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Dose Escalation Trial of SKI-G-801 in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT03564288
- Acronym / sponsor ID
- OSCO-P1301
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Oscotec Inc.Industry (pharma, biotech, devices)
- Collaborators
- PPD Development, LP
- Study start
- 23 Feb 2018 (actual)
- Primary completion
- 2 Sep 2021 (actual)
- Study completion
- 2 Sep 2021 (actual)
- First posted
- 20 Jun 2018 (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 Phase I study is designed to assess the safety, tolerability, pharmacokinetics and anti-tumor effect of increasing doses of study drug SKI-G-801 in patients with relapsed or refractory Acute Myeloid Leukemia (AML) who are unresponsive to currently available therapies. Eligible participants will receive cycles of treatment involving IV infusion of SKI-G-801 daily for 14 days followed by 14 days off. Treatment cycles will be repeated until progressive disease or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation Cohort | Experimental | Drug: SKI-G-801To identify the recommended phase 2 dose (RP2D) of SKI-G-801 in patients with relapsed or refractory AML (Acute Myeloid Leukemia) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Willing and able to provide written informed consent for participation, prior to completing any study-related procedures.
- Diagnosis of Acute Myeloid Leukemia (AML)
- Patients must have been off previous antileukemia therapy for at least 2 weeks or 5 half-lives, whichever is longer if the immediate prior regimen included only weekly chemotherapy; or 4 weeks or 5 half-lives, whichever is longer, from any therapy with therapeutic biologics and from any type of investigational therapy. Daily hydroxyurea for up to 2 weeks to keep the absolute blast count below 50 x 10⁹/L will be allowed, but must be discontinued 24 hours prior to administration of study drug. Hydroxyurea will be permitted during the first cycle of treatment if necessary.
- At least one prior induction regimen (with or without consolidation) which may have included hematopoietic stem cell transplantation (HSCT).
- Have adequate liver function.
Key exclusion criteria (6 listed)
- Patient has a diagnosis of Acute Promyelocytic Leukemia (APL) or chronic myelogenous leukemia in blast crisis.
- If patient is post allogenic transplant and requires therapy for graft vs host disease (GVHD) within 14 days prior to date of screening.
- Requires treatment with concomitant drugs that prolong QT/QTc interval.
- Recent history of cardiac ischemic disease (acute myocardial infarction within 6 months; uncontrolled angina); severe uncontrolled ventricular arrhythmia; recent transient ischemic attack or stroke within 6 months of screening; poorly controlled hypertension (systolic blood pressure >140 mm Hg or diastolic blood pressure >90 mm Hg).
- Patient has active, untreated central nervous system (CNS) disease.
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 |
|---|---|---|
| SKI-G-801SKI-G-801 is administered as an IV infusion over 10 minutes | Drug | Dose Escalation Cohort |
Primary outcome
Recommended phase 2 dose (RP2D)
Time frame: From Cycle 1, Day 1 until disease progression, unacceptable toxicity, patient withdrawal from study, or judged not to be in patient's interest to continue in study, assessed up to 36 months
RP2D of SKI-G-801 determined using Neuenschwander's continual reassessment method (N-CRM)
Patients in complete remission or showing partial response (overall response rate [ORR])
Time frame: Up to 30 days following last dose of study drug
Number of patients showing composite complete remission (complete remission [CR], complete remission with incomplete platelet recovery [CRp], and complete remission with incomplete hematologic recovery [CRi]) of SKI-G-801 according to the Response Criteria in AML
Patients in complete remission
Time frame: Day 84 (± 3 days)
Number of patients showing complete remission (CR)
Duration of remission
Time frame: From date of first reported status of CR to the date of disease relapse or death (+ 1 day); or to date of last available disease status report for patients who do not relapse, assessed up to 36 months
Number of days between a patient's first reported status of complete remission (CR) and the earlier of disease relapse or death from any cause
Duration of event free survival
Time frame: Day 1 to date of event (first documented treatment failure, relapse from CR or Cri [CR with incomplete hematologic recovery], or death due to any cause), assessed up to 36 months
Number of days between start of treatment to date of event
Time to treatment response (TTR)
Time frame: Day 1 to date of first subsequent disease status of CR (+ 1 day), assessed up to 36 months
Number of days between the start of treatment to the date of first subsequent disease status of complete remission (CR)
Dose limiting toxicity (DLT) Adverse Events (AEs)
Time frame: Up to Day 28
Number of any DLT AEs within the first cycle of each patient's treatment with SKI-G-801
4 secondary outcomes
Incidence of Adverse Events (AEs)
Time frame: Up to 30 days following last dose of study drug
Number of participants with clinical laboratory abnormalities
Time frame: Up to 30 days following last dose of study drug
Number of participants with overall safety profiles
Time frame: Up to 30 days following last dose of study drug
Number of participants with electrocardiogram (ECG) abnormalities
Time frame: Up to 30 days following last dose of study drug
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 21 Feb 2024.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
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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.
Eunice Wang, MD principal investigator
Roswell Park Cancer Institute
2 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 SKI-G-801 in people with Acute Myeloid Leukemia, run by Oscotec Inc. at 2 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 9 inclusion and 6 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 21 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
Recommended phase 2 dose (RP2D) (time frame: From Cycle 1, Day 1 until disease progression, unacceptable toxicity, patient withdrawal from study, or judged not to be in patient's interest to continue in study, assessed up to 36 months). The study lists 7 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 21 Feb 2024; AzTrials last refreshed its copy on 26 Oct 2026.