Finding an Effective Dose of GM1 to Reduce or Prevent Neuropathy (Numbness or Weakness) Due to Treatment With Paclitaxel (Phase II)

NCT ID: NCT05751668

Last Updated: 2025-08-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

98 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-02

Study Completion Date

2032-03-15

Brief Summary

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This phase II trial tests the safety, side effects, and best dose of monosialotetrahexosylganglioside (GM1) and whether it works in reducing or preventing chemotherapy-induced peripheral neuropathy (CIPN) in patients with breast cancer that has spread from where it first started (primary site) to other places in the body (metastatic) who are receiving treatment with paclitaxel. Chemotherapy drugs, such as paclitaxel, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Exposure to chemotherapy drugs like paclitaxel may cause a side effect called CIPN, which is a condition of weakness, numbness, and pain from nerve damage (usually in the hands and feet). GM1 is a part of the body's natural system that insulates nerves and helps to protect nerves from damage. Giving GM1 may help reduce or prevent CIPN in breast cancer patients receiving treatment with paclitaxel.

Detailed Description

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PRIMARY OBJECTIVES:

I. To obtain data to further support the safety of increasing monosialotetrahexosylganglioside (GM1) doses when given on day 1, concomitantly with paclitaxel. (Early phase) II. To evaluate the preliminary efficacy of GM1 compared with placebo at preventing paclitaxel-induced peripheral neuropathy sensory symptoms as measured by the six individual Quality of Life Questionnaire-Chemotherapy-Induced Peripheral Neuropathy 20 (QLQ-CIPN20) questions that quantify numbness (N), tingling (T), and pain in the fingers/hands and toes/feet. (Phase II)

SECONDARY OBJECTIVES:

I. To obtain additional data to support the safety of GM1 in the treated population. (Phase II) II. To obtain data to support that GM1 looks promising for preventing/decreasing acute paclitaxel pain syndrome as measured by the Acute Pain Syndrome Questionnaire. (Phase II)

EXPLORATORY OBJECTIVES:

I. Conduct of this clinical trial provides the opportunity to facilitate the better understanding of the natural history of paclitaxel-induced neuropathy, akin to what is being examined in a currently active trial, S1714. (Phase II) II. This clinical trial also provides an opportunity to conduct correlative studies to understand the mechanism of CIPN and/or identify biomarkers of CIPN or GM1 efficacy. (Phase II) III. To obtain efficacy data to assess the impact of GM1 on the anti-tumor activity of paclitaxel as evaluated by progression-free survival and overall survival. (Phase II)

OUTLINE: This is an early phase dose-escalation study of GM1 followed by a phase II study.

EARLY PHASE: Patients receive GM1 intravenously (IV) over 1 hour either once every 7 days, or once every 7 days for 3 doses followed by one week off, prior to paclitaxel administration.

PHASE II: Patients are randomized to 1 of 2 arms.

ARM I: Patients receive GM1 IV 1 hour prior to paclitaxel administration and paclitaxel IV weekly for 12 weeks or 3 weeks on/1 week off for 12 doses.

ARM II: Patients receive placebo IV 1 hour prior to paclitaxel administration and paclitaxel IV weekly for 12 weeks or 3 weeks on/1 week off for 12 doses.

Conditions

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Anatomic Stage IV Breast Cancer AJCC v8 Chemotherapy-Induced Peripheral Neuropathy Metastatic Breast Carcinoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Arm I (paclitaxel, GM1)

Patients receive GM1 IV 1 hour prior to paclitaxel administration and paclitaxel IV weekly for 12 weeks or 3 weeks on/1 week off for 12 doses.

Group Type EXPERIMENTAL

Paclitaxel

Intervention Type DRUG

Given IV

Monosialotetrahexosylganglioside

Intervention Type DRUG

Given IV

Questionnaire Administration

Intervention Type OTHER

Ancillary Studies

Quality-of-life assessment

Intervention Type OTHER

Ancillary Studies

Arm II (paclitaxel, placebo)

Patients receive placebo IV 1 hour prior to paclitaxel administration and paclitaxel IV weekly for 12 weeks or 3 weeks on/1 week off for 12 doses.

Group Type PLACEBO_COMPARATOR

Paclitaxel

Intervention Type DRUG

Given IV

Questionnaire Administration

Intervention Type OTHER

Ancillary Studies

Quality-of-life assessment

Intervention Type OTHER

Ancillary Studies

Placebo Administration

Intervention Type DRUG

Given IV

Interventions

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Paclitaxel

Given IV

Intervention Type DRUG

Monosialotetrahexosylganglioside

Given IV

Intervention Type DRUG

Questionnaire Administration

Ancillary Studies

Intervention Type OTHER

Quality-of-life assessment

Ancillary Studies

Intervention Type OTHER

Placebo Administration

Given IV

Intervention Type DRUG

Other Intervention Names

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Anzatax Asotax Bristaxol Ganglioside GM-1 GM-1 GM1 Monosialoanglioside GM1

Eligibility Criteria

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Inclusion Criteria

* Documentation of disease: Histologic diagnosis of metastatic breast cancer in women or men
* Prior treatment- No previous exposure to GM1
* Planned administration of paclitaxel, either given weekly, or weekly 3 weeks on/1 week off, to patients with metastatic cancer at a dose of 80 mg/m\^2
* No planned treatment with concurrent immunotherapy
* Score of 1 (none) and/or 2 (a little) on the six individual European Organization for Research and Treatment of Cancer (EORTC) Quality of Life questionnaire (QLQ)- chemotherapy-induced peripheral neuropathy (CIPN)20 questions that quantify numbness (N), tingling (T), and pain in the fingers/hands and toes/feet (Items #31-36)
* No diagnosis of fibromyalgia
* No history of significant respiratory tract infection and/or infectious diarrhea within 14 days before registration
* No history of stroke or cerebrovascular accident in the past 6 months prior to registration
* No history of diagnosed neurologic or psychiatric disorders, including epilepsy or dementia
* For women of childbearing potential, not pregnant and not nursing, because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown.

Therefore, for women of childbearing potential, a negative pregnancy test done =\< 7 days prior to registration is required. Of note, a female of childbearing potential is a sexually mature female who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months)

* Ability to complete questionnaires by themselves or with assistance
* In order to complete the mandatory patient-completed measures, participants must be able to speak and/or read English and/or Spanish
* Persons with impaired decision making such that they cannot understand the benefits or risks of trial participation, per the judgement of the consenting clinician, will not be eligible
* Age \>= 18 years
* Eastern Cooperative Oncology Group (ECOG) performance status 0-2
* Absolute neutrophil count (ANC) \>= 1,000/mm\^3
* Platelet count \>= 100,000/mm\^3
* Creatinine =\< 1.5 x upper limit of normal (ULN)
* Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =\< 2.5 x upper limit of normal (ULN)
* Total bilirubin =\< 1.5 x ULN
* No planned use of duloxetine
* No planned use of cryotherapy, compression therapy, or cryocompression therapy at study entry

Exclusion Criteria

* N/A
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Alliance for Clinical Trials in Oncology

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care

Irvine, California, United States

Site Status RECRUITING

UC Irvine Health/Chao Family Comprehensive Cancer Center

Orange, California, United States

Site Status RECRUITING

Morton Plant Hospital

Clearwater, Florida, United States

Site Status RECRUITING

Mount Sinai Medical Center

Miami Beach, Florida, United States

Site Status RECRUITING

Saint Anthony's Hospital Cancer Care Center

St. Petersburg, Florida, United States

Site Status RECRUITING

UI Health Care Mission Cancer and Blood - Ankeny Clinic

Ankeny, Iowa, United States

Site Status RECRUITING

Mercy Hospital

Cedar Rapids, Iowa, United States

Site Status RECRUITING

Oncology Associates at Mercy Medical Center

Cedar Rapids, Iowa, United States

Site Status RECRUITING

UI Health Care Mission Cancer and Blood - West Des Moines Clinic

Clive, Iowa, United States

Site Status RECRUITING

Iowa Methodist Medical Center

Des Moines, Iowa, United States

Site Status RECRUITING

UI Health Care Mission Cancer and Blood - Des Moines Clinic

Des Moines, Iowa, United States

Site Status RECRUITING

UI Health Care Mission Cancer and Blood - Laurel Clinic

Des Moines, Iowa, United States

Site Status RECRUITING

University of Maryland/Greenebaum Cancer Center

Baltimore, Maryland, United States

Site Status RECRUITING

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Site Status RECRUITING

Coborn Cancer Center at Saint Cloud Hospital

Saint Cloud, Minnesota, United States

Site Status RECRUITING

Saint Luke's Hospital

Chesterfield, Missouri, United States

Site Status RECRUITING

Southeastern Medical Oncology Center-Clinton

Clinton, North Carolina, United States

Site Status RECRUITING

Southeastern Medical Oncology Center-Goldsboro

Goldsboro, North Carolina, United States

Site Status RECRUITING

Southeastern Medical Oncology Center-Jacksonville

Jacksonville, North Carolina, United States

Site Status RECRUITING

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States

Site Status RECRUITING

Toledo Clinic Cancer Centers-Toledo

Toledo, Ohio, United States

Site Status SUSPENDED

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

Legacy Mount Hood Medical Center

Gresham, Oregon, United States

Site Status NOT_YET_RECRUITING

Legacy Good Samaritan Hospital and Medical Center

Portland, Oregon, United States

Site Status NOT_YET_RECRUITING

Legacy Meridian Park Hospital

Tualatin, Oregon, United States

Site Status NOT_YET_RECRUITING

Legacy Cancer Institute Medical Oncology and Day Treatment

Vancouver, Washington, United States

Site Status NOT_YET_RECRUITING

Legacy Salmon Creek Hospital

Vancouver, Washington, United States

Site Status NOT_YET_RECRUITING

Marshfield Medical Center-EC Cancer Center

Eau Claire, Wisconsin, United States

Site Status RECRUITING

Marshfield Medical Center-Marshfield

Marshfield, Wisconsin, United States

Site Status RECRUITING

Marshfield Medical Center - Minocqua

Minocqua, Wisconsin, United States

Site Status RECRUITING

Marshfield Medical Center-Rice Lake

Rice Lake, Wisconsin, United States

Site Status RECRUITING

Marshfield Medical Center-River Region at Stevens Point

Stevens Point, Wisconsin, United States

Site Status RECRUITING

Marshfield Medical Center - Weston

Weston, Wisconsin, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Elizabeth Cathcart-Rake, MD

Role: CONTACT

507-768-4411

Aishwarya Vijendran

Role: CONTACT

773-834-9613

Facility Contacts

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Site Public Contact

Role: primary

877-827-8839

Site Public Contact

Role: primary

877-827-8839

Site Public Contact

Role: primary

855-314-8646

Site Public Contact

Role: primary

305-674-2625

Site Public Contact

Role: primary

Site Public Contact

Role: primary

515-241-3305

Site Public Contact

Role: primary

319-365-4673

Site Public Contact

Role: primary

319-363-2690

Site Public Contact

Role: primary

515-241-3305

Site Public Contact

Role: primary

515-241-6727

Site Public Contact

Role: primary

515-241-3305

Site Public Contact

Role: primary

515-241-3305

Site Public Contact

Role: primary

800-888-8823

Site Public Contact

Role: primary

855-776-0015

Site Public Contact

Role: primary

877-229-4907

Site Public Contact

Role: primary

314-205-6936

Site Public Contact

Role: primary

919-587-9084

Site Public Contact

Role: primary

919-587-9084

Site Public Contact

Role: primary

910-587-9084

Site Public Contact

Role: primary

800-293-5066

Site Public Contact

Role: primary

405-271-8777

Site Public Contact

Role: primary

503-413-2150

Site Public Contact

Role: primary

800-220-4937

Site Public Contact

Role: primary

503-413-1742

Site Public Contact

Role: primary

Site Public Contact

Role: primary

503-413-2150

Site Public Contact

Role: primary

800-782-8581

Site Public Contact

Role: primary

800-782-8581

Site Public Contact

Role: primary

800-782-8581

Site Public Contact

Role: primary

800-782-8581

Site Public Contact

Role: primary

800-782-8581

Site Public Contact

Role: primary

800-782-8581

Other Identifiers

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NCI-2022-04929

Identifier Type: OTHER

Identifier Source: secondary_id

A222101

Identifier Type: -

Identifier Source: org_study_id

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