Study to Find a Safe and Effective Dose of SKI-G-801 in the Treatment of Patients With Acute Myeloid Leukemia (AML)

NCT03564288 | Completed Phase 1 FDA Drug

• 1 other identifier: OSCO-P1301
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 2

Brief 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. 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

Outcome Measures

Primary Outcomes (7)

• Recommended phase 2 dose (RP2D)

  RP2D of SKI-G-801 determined using Neuenschwander's continual reassessment method (N-CRM)

  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

  Up to 30 days following last dose of study drug

• Patients in complete remission

  Number of patients showing complete remission (CR)

  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

  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

  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)

  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

  Up to Day 28

Secondary Outcomes (4)

• Incidence of Adverse Events (AEs)

  Up to 30 days following last dose of study drug

• Number of participants with clinical laboratory abnormalities

  Up to 30 days following last dose of study drug

• Number of participants with overall safety profiles

  Up to 30 days following last dose of study drug

• Number of participants with electrocardiogram (ECG) abnormalities

  Up to 30 days following last dose of study drug

Study Arms (1)

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)

Drug: SKI-G-801

Interventions

SKI-G-801 DRUG

SKI-G-801 is administered as an IV infusion over 10 minutes

Dose Escalation Cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Oscotec Inc.: lead
• PPD Development, LP: collaborator

Study Sites (2)

USC Norris Comprehensive Cancer Center, Clinical Investigations Support Office (CISO), 1441 Eastlake Ave., Rm. 7327

Los Angeles, California, 90033, United States

Location

Innovative Clinical Research Institute

Whittier, California, 90603, United States

Location

Related Links

• FDA Safety Alerts and Recalls

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Eunice Wang, MD

  Roswell Park Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2018
• First Posted: June 20, 2018
• Study Start: February 23, 2018
• Primary Completion: September 2, 2021
• Study Completion: September 2, 2021
• Last Updated: February 21, 2024

Record last verified: 2022-05

Locations

US (2)