Actively Recruiting
A Phase 1 Study of Rebecsinib, a Spliceosome Modulator That Inhibits ADAR1, in Patients With Relapsed or Refractory Secondary Acute Myeloid Leukemia or Higher-Risk Myelofibrosis
Led by Aspera Biomedicines, Inc. · Updated on 2026-06-04
28
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
Sponsors
A
Aspera Biomedicines, Inc.
Lead Sponsor
T
Tempus AI
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating rebecsinib, a new investigational drug, for patients with relapsed or refractory secondary Acute Myeloid Leukemia (sAML) or higher-risk Myelofibrosis (MF). The study is a Phase 1 trial focused on finding the safest and most effective dose of rebecsinib and assessing its safety and tolerability by monitoring side effects and adverse events. This research explores important outcomes such as response rates, progression-free survival, and overall survival in these patient groups. Participants will receive intravenous infusions of rebecsinib on Days 1, 4, 8, and 11 of each 28-day cycle. The doses tested range from 0.2 mg/kg to 8.0 mg/kg based on ideal body weight. The study involves up to six treatment cycles. Researchers will observe the maximum tolerated dose and biological activity of the drug during treatment. During the study, participants will undergo regular assessments including monitoring for side effects, blood tests, and disease progression evaluations using established criteria. Safety is closely monitored from enrollment to the start of any new therapy. The total duration for dose finding assessments is approximately two years, with safety and disease response monitored over about four months. These evaluations help researchers understand how rebecsinib affects the disease and patient health over time.
CONDITIONS
Brief Title
A Study of Rebecsinib for Patients With Relapsed/Refractory Secondary Acute Myeloid Leukemia or High Risk Myelofibrosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ability to understand and willing to sign written informed consent
- Diagnosis of refractory secondary AML evolved from MDS, MPN, or related conditions, or higher-risk myelofibrosis defined by DIPSS Plus or MIPSS70+ version 2.0
- Excess bone marrow blasts greater than 5%
- Ineligible for bone marrow transplant at enrollment
- Relapsed, refractory, or intolerant to available therapies including anthracycline chemotherapy, epigenetic modifiers, or venetoclax
- If no prior intensive induction chemotherapy, must have had at least one line of other therapy; if prior intensive induction, at least two lines of therapy
- Prior therapy must include mutation-specific inhibitors if relevant (e.g., FLT3 or IDH inhibitors)
- Creatinine clearance above 60 mL/min
- Recovered to clinical baseline from prior therapy toxic effects
- Age 18 years or older
- Both men and women of all races and ethnic groups eligible
- Women of childbearing potential must agree to pregnancy prevention and have negative pregnancy test
- ECOG performance status of 0 to 2
- Hemoglobin 8.0 g/dL or higher
- Total bilirubin 1.5 times upper limit of normal or less
- ALT and AST 1.5 times upper limit of normal or less
- Prothrombin time INR 2 or less; partial thromboplastin time 1.66 times upper limit of normal or less
You will not qualify if you...
- Pregnant or breastfeeding females
- Previous hematopoietic cell transplant
- Currently receiving another investigational agent
- Chemotherapy or investigational drug treatment within 2 weeks or 5 half-lives before study start or during study
- Current infection requiring systemic antibiotics
- Active infection with HIV, HBV, or HCV
- Concurrent or prior malignancy within 3 years except certain localized cancers
- Known central nervous system involvement by malignancy
- Untreated autoimmune conditions like autoimmune hemolytic anemia
- Uncompensated hypothyroidism not treated with replacement hormone
- Insufficient recovery from surgery-related wound healing
- Impaired cardiac function including recent myocardial infarction, significant ECG abnormalities, or other serious heart disease
- QT interval greater than 470 msec on ECG
- Use of medications affecting specific liver enzymes or transporters without appropriate management
- Inability to comply with safety monitoring requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive rebecsinib by intravenous infusion on Days 1, 4, 8, and 11 of each 28-day cycle to treat relapsed or refractory secondary acute myeloid leukemia or higher-risk myelofibrosis.
Visits on Days 1, 4, 8, and 11 of each cycle
Duration - Up to approximately 2 years
Participants are monitored for safety and clinical outcomes including adverse events, response rates, and survival after treatment ends.
Periodic visits as scheduled for safety and disease assessments
Trial Site Locations
Total: 1 location
1
UC San Diego Moores Cancer Center
San Diego, California, United States, 92093
Actively Recruiting
Research Team
C
Curis L Scribner, MD
K
Karla Mack
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
0
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