Imatinib + Fampridine for Gastrointestinal Stromal Tumor

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of California, San Diego

Must not be taking: OCT2 inhibitors, CYP3A4/5 inhibitors

Disqualifiers: Metastatic GIST, Brain metastases, Cardiac impairment, Chronic liver disease, others

Stay on Your Current Meds You can continue your current medications while participating

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

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine the safest effective dose of fampridine when combined with imatinib (Gleevec, a cancer treatment) for gastrointestinal stromal tumors (GIST) with a specific DNA mutation in exon 11 of the KIT gene. It also investigates whether this drug combination can effectively combat the tumor. Individuals with a recently diagnosed GIST with this mutation who have not yet begun treatment may be suitable candidates. Participants will take these drugs before surgery to assess their potential in shrinking the tumor. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new combination therapy.

Will I have to stop taking my current medications?

You may need to stop taking certain medications before joining the trial, especially if they interact with the study drugs. Specifically, you cannot take medications that inhibit certain enzymes or transporters, like cimetidine or quinidine, or those that affect liver enzymes like CYP3A4/5. It's best to discuss your current medications with the trial team to see if any changes are needed.

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

Previous studies have shown that imatinib is quite safe for treating gastrointestinal stromal tumors (GIST). Patients using imatinib often experience positive and lasting results. It is a well-researched drug with a strong history in GIST treatments.

Fampridine, however, is newer in this context. This trial tests how much fampridine can be safely given with imatinib. Although specific safety data for this combination is not yet available, imatinib’s safety record is reassuring. As this is an early-phase trial, researchers are determining the safest dose of fampridine when used with imatinib. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for gastrointestinal stromal tumors (GISTs), which typically rely heavily on just imatinib, this new combination therapy adds fampridine into the mix. Fampridine is being explored for its potential to enhance the effectiveness of imatinib by improving nerve function, which could lead to better tumor control. Researchers are excited because this duo might offer a more powerful approach by combining the well-known cancer-fighting capabilities of imatinib with the unique neurological benefits of fampridine, potentially leading to improved outcomes for patients.

What evidence suggests that this trial's treatments could be effective for gastrointestinal stromal tumor?

Research has shown that imatinib effectively treats gastrointestinal stromal tumors (GIST). Studies have found that imatinib helps many patients live longer and slows disease progression, with over 80% of patients experiencing benefits and the 2-year survival rate increasing from 26% to 76%. This trial tests fampridine alongside imatinib to determine if it can enhance these positive effects. The goal is to find a safe dose of fampridine that works well with imatinib to combat the tumors. ^{1 3 4 6 7}

Who Is on the Research Team?

Paul Fanta, MD

Principal Investigator

University of California, San Diego

Are You a Good Fit for This Trial?

This trial is for patients with a type of tumor in the digestive system called GIST, specifically those with a KIT gene mutation. Participants will take drugs before tumor surgery and must attend clinic visits for checkups.

Inclusion Criteria

Provision of written informed consent prior to any screening procedures

Has measurable or evaluable disease per Response Evaluation Criteria in Solid Tumors v1.1

Life expectancy of ≥ 5 years

Exclusion Criteria

Unwilling or unable to comply with the protocol

My gastrointestinal tumor has a specific mutation identified by advanced testing.

Of childbearing potential

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Run-in Period

Participants receive imatinib monotherapy for 7 days before starting combination treatment

1 week

Treatment

Participants receive imatinib in combination with fampridine at one of three dose levels for at least 2 months

8 weeks

Regular clinic visits for checkups and tests

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Fampridine
• Imatinib

Trial Overview The study tests how much fampridine can be safely given with imatinib to treat GIST. It aims to find the highest safe dose and see if this drug combo works against the tumor.

How Is the Trial Designed?

Group I: Imatinib plus Fampridine Experimental Treatment 2 Interventions

Imatinib in combination with fampridine at one of three dose levels (10 mg every other day, or 10 mg every day, or 10 mg twice a day) after a 7-day run-in period with imatinib monotherapy.

Who Is Running the Clinical Trial?

University of California, San Diego

Lead Sponsor

Trials

1,215

Recruited

1,593,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3837608/

Imatinib treatment for gastrointestinal stromal tumour (GIST) The use of imatinib has revolutionized the management of GIST and altered its natural history, substantially improving survival time and delaying disease ...

2. jamanetwork.com jamanetwork.com/journals/jamaoncology/fullarticle/2601220

Correlation of Long-term Results of Imatinib in Advanced ... Imatinib front-line treatment of advanced and/or metastatic gastrointestinal stromal tumors leads to long-term survival (≥10 years) in a substantial minority ...

3. withpower.com withpower.com/trial/phase-1-gastrointestinal-stromal-tumors-11-2025-db87f

Imatinib + Fampridine for Gastrointestinal Stromal Tumor Trial Overview The study tests how much fampridine can be safely given with imatinib to treat GIST. It aims to find the highest safe dose and see if this drug ...

4. touchoncology.com touchoncology.com/gastrointestinal-cancers/journal-articles/advanced-and-metastatic-gastrointestinal-stromal-tumours-review-of-current-knowledge-and-latest-advances/

Advanced and Metastatic Gastrointestinal Stromal Tumours Although imatinib significantly improves both PFS and OS in patients with advanced GISTs, most patients treated with imatinib will eventually ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC2932930/

Efficacy evaluation of imatinib treatment in patients with ... Clinical benefit was demonstrated in more than 80% of patients, resulting in a substantial improvement in the 2-year survival rate from 26% to 76%[5,6].

6. nejm.org nejm.org/doi/full/10.1056/NEJMoa020461

Efficacy and Safety of Imatinib Mesylate in Advanced ... Imatinib induced a sustained objective response in more than half of patients with an advanced unresectable or metastatic gastrointestinal stromal tumor.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8937014/

Phase II Trial of Imatinib Plus Binimetinib in Patients With ... Five of eight (62.5%) patients achieved significant pathologic response (SPR; ≥ 90% treatment effects; Fig 4), which appeared more superior comparing with ...