Venetoclax Solution for Blood Cancers

Recruiting at 4 trial locations

Overseen By Site Public Contact

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Children's Hospital Medical Center, Cincinnati

Must be taking: Venetoclax

Disqualifiers: Pregnancy, Breastfeeding, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 5

Duration: 4 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand how venetoclax, a cancer medication, behaves in the body when its tablets are crushed and dissolved in a liquid for easier consumption. The focus is on children and young adults with blood cancers who already use this liquid form of venetoclax as part of their treatment. Participants must have a blood cancer diagnosis, weigh at least 12 pounds, and have a central line (a type of long-term IV) for blood samples. The study will assess how the drug works and its safety when taken by mouth or through feeding tubes. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to contribute to early-stage research.

Do I have to stop taking my current medications for the trial?

The trial does not specify that you need to stop taking your current medications. You can take venetoclax with other chemotherapy drugs, so it's likely you can continue your current treatment, but you should confirm with the trial team.

Is there any evidence suggesting that this trial's treatment is likely to be safe?

Research shows that venetoclax is approved for use in adults, but its safety and effectiveness in children remain unknown. No studies have examined the effects of crushing venetoclax tablets and mixing them with liquid. This method is often used for individuals who cannot swallow pills, yet its impact on adults or children is unclear.

While venetoclax is typically prescribed for adults with certain blood cancers, its use in liquid form has not been studied. The body may absorb and react to the medicine differently in this form. Without specific research, predicting how well children or young adults might tolerate it as a liquid is challenging.

In summary, although venetoclax is used in adults, more research is needed to understand its safety and effectiveness when the tablets are crushed and dissolved, particularly for younger patients. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the venetoclax treatment because it offers a unique delivery method for patients who have difficulty swallowing pills. Unlike the traditional tablet form, this treatment involves crushing the tablets and dissolving them into a solution, making it easier for children and young adults to take. This novel approach could improve adherence to the treatment regimen and potentially enhance the effectiveness of the medication in these patient groups.

What evidence suggests that this trial's venetoclax solution could be effective for pediatric malignancies?

Research has shown that venetoclax can benefit children and young adults with certain cancers, particularly if their cancer has returned or worsened after treatment. In this trial, children and young adults will receive venetoclax from crushed tablets as part of their clinical care. Studies indicate that venetoclax, often combined with chemotherapy, shows promise in treating acute lymphoblastic leukemia (ALL) and other blood cancers. In some cases, combining venetoclax with other treatments has improved outcomes for young patients with advanced blood cancers. Although specific data on crushed venetoclax tablets is lacking, the drug itself has proven effective in similar situations, suggesting it may also work well as a liquid solution. ^{2 6 7 8 9}

Who Is on the Research Team?

Lauren Pommert, MD

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Are You a Good Fit for This Trial?

Inclusion Criteria

You must be younger than 39 years old to take part in the study.

You have been diagnosed with any type of blood cancer.

You need to have a central line for blood draws during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive venetoclax solution made from crushed tablets as part of their oncology treatment

4 weeks

Multiple visits for PK sampling

Pharmacokinetic Sampling

Peripheral blood and optional cerebrospinal fluid samples are collected to evaluate venetoclax pharmacokinetics

4 weeks

PK day between Days 5-12, optional CSF sampling around Days 8, 15, 22, and/or 28

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Venetoclax

How Is the Trial Designed?

Group I: Children and Young Adults Experimental Treatment 1 Intervention

Children and Young Adults who are prescribed venetoclax made from crushed tablets as part of their clinical care.

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials

844

Recruited

6,566,000+

Citations

1.

cancerresearchuk.org

cancerresearchuk.org/about-cancer/find-a-clinical-trial/a-study-looking-at-venetoclax-for-children-and-young-adults-with-cancer

A study looking at venetoclax for children and young adults ...

Researchers want to find out if it can help children and young adults with cancer when their cancer comes back or has continued to get worse during treatment.

2.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40062648/

Venetoclax Combined With Chemotherapy in Pediatric and ...

Venetoclax has promising preclinical and clinical activity in ALL. Here, we report the safety and preliminary efficacy of venetoclax combined with chemotherapy ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT03873857?rank=1

A Study to Assess Effectiveness of Venclexta (Venetoclax) ...

This study seeks to assess the effectiveness and safety of venetoclax in patients with relapsed and refractory chronic lymphocytic leukemia (CLL) in a ...

4.

connect.careboxhealth.com

connect.careboxhealth.com/en-US/trial/listing/451482

Pharmacokinetic Study of Venetoclax Tablets Crushed and ...

A Pharmacokinetic Study of Venetoclax Tablets Crushed and Dissolved Into a Solution in Children and Young Adults With Hematologic Malignancies. Estimated trial ...

5.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10133180/

Venetoclax in combination with chemotherapy as treatment for ...

Venetoclax in combination with hypomethylating agents or cytotoxic chemotherapy was effective in a subset of pediatric/AYA patients with advanced hematologic ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06131801

Pharmacokinetic Study of Venetoclax Tablets Crushed and ...

However, no PK data exists in adults or children using crushed tablets dissolved in liquid in this manner, and as a result, the venetoclax exposure with this ...

7.

venclexta.com

venclexta.com/important-safety-information

Important Safety Information

It is not known if VENCLEXTA is safe and effective in children. Important Safety Information. What is the most important information I should know about ...

8.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2019/208573s013lbl.pdf

VENCLEXTA® (venetoclax - accessdata.fda.gov

VENCLEXTA tablets should be swallowed whole and not chewed, crushed, or ... It is not known if VENCLEXTA is safe and effective in children. Who should not ...

9.

mayoclinic.org

mayoclinic.org/drugs-supplements/venetoclax-oral-route/description/drg-20311676

Venetoclax (oral route) - Side effects & dosage

Appropriate studies have not been performed on the relationship of age to the effects of venetoclax in the pediatric population. Safety and efficacy have not ...