L-Annamycin + Cytarabine for AML

Recruiting at 15 trial locations

Overseen By Erikson Wasyl, MS

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Moleculin Biotech, Inc.

Must not be taking: QT prolonging drugs

Disqualifiers: APL, MDS/AML, Cardiac issues, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

312 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new combination of treatments, L-Annamycin (a liposomal formulation of the chemotherapy drug annamycin) and Cytarabine, for individuals with acute myeloid leukemia (AML) that has recurred or did not respond to initial treatment. The goal is to determine if this combination can achieve remission, meaning the cancer becomes undetectable. Participants will receive varying doses to identify the most effective one. This trial may suit adults who have previously tried one treatment for AML without success and have no major health issues, such as heart problems or active infections. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you should not have had chemotherapy, radiation, or major surgery within 2 weeks before starting the study drug, and you should not be on medications that significantly prolong the QT/QTc interval. It's best to discuss your specific medications with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that liposomal annamycin, when combined with cytarabine, is generally well-tolerated by patients with acute myeloid leukemia (AML). Early results from previous studies indicated that liposomal annamycin is safe, with no heart-related side effects reported, even in heavily pre-treated patients. This is encouraging for those considering joining a trial, as it suggests the treatment has a good safety record so far.

Cytarabine, the other drug in the combination, is a well-known and widely used treatment for AML. While it can cause some common side effects, such as nausea or low blood cell counts, these are usually manageable with medical care.

Overall, these findings provide a strong basis for the safety of this treatment combination in the ongoing study for AML. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about L-Annamycin combined with Cytarabine for treating acute myeloid leukemia (AML) because L-Annamycin is a unique liposomal formulation of an anthracycline antibiotic, which may allow for better delivery to cancer cells with potentially reduced heart-related side effects. Unlike standard AML treatments such as standard anthracyclines and Cytarabine alone, L-Annamycin's liposomal delivery could enhance its ability to penetrate and remain in cancer cells for more effective treatment. This combination aims to improve treatment outcomes for patients by targeting cancer cells more precisely while minimizing damage to healthy cells, setting it apart from existing therapies.

What evidence suggests that this trial's treatments could be effective for AML?

Research has shown that Liposomal Annamycin may help treat acute myeloid leukemia (AML), particularly in patients whose cancer hasn't responded to other treatments. In this trial, some participants will receive Liposomal Annamycin combined with Cytarabine. One study found that this combination extended patient survival. On average, patients survived 9 months, while those with no signs of disease lived 15 months. This combination is generally safe and doesn't cause serious side effects for most people, particularly avoiding heart problems. Overall, these findings suggest that this treatment could be effective for people with AML that has returned or hasn't responded to treatment. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

Adults with Acute Myeloid Leukemia (AML) that didn't respond to their first treatment can join. They should have only had one prior therapy, no recent chemo or major surgery, and be in decent physical shape (ECOG 0-2). Their liver and kidneys must work well enough to handle the trial drugs.

Inclusion Criteria

Has a life expectancy of greater than six weeks at screening

For WCBP: Must agree to not donate ova and use a highly effective method of birth control from the time of informed consent through 6 months after their last randomized dose of study drug

Has received no investigational therapy within 4 weeks prior to the first randomized dose of study drug

Exclusion Criteria

Has clinically relevant serious comorbid medical conditions

Has any condition that, in the opinion of the PI, places the subject at unacceptable risk if he/she were to participate in the study

Pregnant or breastfeeding

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment - Part A

Randomized, double-blind, placebo-controlled study to determine the optimal dosage regimen of L-Annamycin in combination with Cytarabine

5 weeks

Multiple visits for treatment and assessments

Interim Analysis

Interim analysis to evaluate efficacy and safety, and determine continuation of treatment arms

2 weeks

Treatment - Part B

Expansion study at the optimal dosage regimen determined in Part A

5 weeks

Multiple visits for treatment and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

• Cytarabine
• Liposomal Annamycin

Trial Overview The study is testing L-Annamycin combined with Cytarabine as a second attempt at inducing remission in AML patients whose disease came back or didn't respond initially. It's a phase 2/3 trial comparing this combo against a placebo plus Cytarabine.

How Is the Trial Designed?

Group I: Part A / Treatment Arm 3 Active Control 1 Intervention

230 mg/m2/day L-Annamycin for Injection for three consecutive days in combination with 2.0 g/m2/day Cytarabine Injection for five consecutive days.

Group II: Part B / Treatment Arm X Active Control 1 Intervention

Optimal dosage regimen (as determined in Part A) of L Annamycin for Injection (190 mg/m2/day or 230 mg/m2/day) for three consecutive days in combination with 2.0 g/m2/day Cytarabine Injection for five consecutive days (i.e., the same as Treatment Arm 2 or 3, respectively, of Part A).

Group III: Part A / Treatment Arm 2 Active Control 1 Intervention

190 mg/m2/day L-Annamycin for Injection for three consecutive days in combination with 2.0 g/m2/day Cytarabine Injection for five consecutive days.

Group IV: Part B / Treatment Arm 1 Placebo Group 1 Intervention

Placebo (0.9% Sodium Chloride Injection) for three consecutive days in combination with 2.0 g/m2/day Cytarabine Injection for five consecutive days (i.e., the same as Treatment Arm 1 of Part A).

Group V: Part A / Treatment Arm 1 Placebo Group 1 Intervention

placebo (0.9% Sodium Chloride Injection, i.e., the diluent for L-Annamycin for Injection) for three consecutive days in combination with 2.0 g/m2/day Cytarabine Injection for five consecutive days

Who Is Running the Clinical Trial?

Moleculin Biotech, Inc.

Lead Sponsor

Trials

8

Recruited

550+

Citations

1. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/5948/505823/Preliminary-Results-from-a-Phase-1-2-Study-of

Preliminary Results from a Phase 1/2 Study of Liposomal ... Conclusion: Initial data suggested that L-ANN is safe and active in heavily pretreated r/r AML patients with no signs of cardiotoxicity. This ...

2. targetedonc.com targetedonc.com/view/annamycin-demonstrates-encouraging-overall-survival-in-refractory-aml

Annamycin Demonstrates Encouraging Overall Survival in ... New clinical trial results reveal annamycin's promise in treating relapsed AML, showing improved survival rates and a favorable safety profile.

3. onclive.com onclive.com/view/annamycin-plus-cytarabine-drive-potential-os-benefit-in-acute-myeloid-leukemia

Annamycin Plus Cytarabine Drive Potential OS Benefit in ... Annamycin and cytarabine combination showed a median overall survival of 9 months in AML patients, with 15 months in those achieving complete ...

4. moleculin.com moleculin.com/wp-content/uploads/2024/06/P537_Papayannidis.pdf

LIPOSOMAL ANNAMYCIN IN COMBINATION WITH ... Preliminary results from a phase ½ study of liposomal annamycin (L-ANN) in combination with cytarabine for the treatment of patients with acute myeloid leukemia ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03315039

Study of Liposomal Annamycin for the Treatment ... Determined by acute myeloid leukemia (AML) response rate based on the International Working Group (IWG) Response Criteria in AML (Cheson, 2003). Anti-leukemic ...

6. accessdata.fda.gov accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=550116

Search Orphan Drug Designations and Approvals - FDA liposomal annamycin. Date Designated: 03/20/2017. Orphan Designation: Treatment of acute myeloid leukemia. Orphan Designation Status: Designated. FDA Orphan ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S000649712312547X

Preliminary Results from a Phase 1/2 Study of Liposomal ... Conclusion: Initial data suggested that L-ANN is safe and active in heavily pretreated r/r AML patients with no signs of cardiotoxicity. This study is currently ...