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New oral drug cocktail shows promise for kids with resistant neuroblastoma

NCT ID NCT00093353

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tested a combination of three oral drugs (irinotecan, temozolomide, and cefixime) in 30 children whose high-risk neuroblastoma had come back or not responded to standard treatment. The goal was to find the safest dose of irinotecan when given with the other two drugs. Cefixime was included to help prevent diarrhea, a common side effect of irinotecan. The study focused on side effects and dosing, not on curing the disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Start date

May 2004

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 30 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

DISEASE CHARACTERISTICS: * Histologically confirmed neuroblastoma AND/OR demonstration of tumor cells in the bone marrow with increased urinary catecholamines * High-risk disease meeting 1 of the following criteria: * Recurrent or progressive disease * Resistant or refractory disease (i.e., never achieved a complete response to therapy AND never had new sites of disease or progression of initial sites) * Measurable disease meeting at least 1 of the following criteria: * Unidimensionally measurable tumor ≥ 20 mm by MRI, CT scan, or x-ray OR ≥ 10 mm by spiral CT scan\* * At least 1 site with positive uptake by metaiodobenzylguanidine (MIBG) scan\* * Bone marrow with tumor cells seen on routine morphology (not by NSE staining only) of bilateral aspirate AND/OR biopsy on 1 bone marrow sample NOTE: \*Patients who never experienced disease recurrence or progression must demonstrate viable neuroblastoma in a biopsy of either bone marrow or bone and/or soft tissue site (biopsy must be performed ≥ 4 weeks after completion of prior radiotherapy if lesion was irradiated) PATIENT CHARACTERISTICS: Age * 1 to 30 at diagnosis Performance status * ECOG 0-2 Life expectancy * At least 2 months Hematopoietic * Absolute neutrophil count ≥ 750/mm\^3 * Platelet count ≥ 75,000/mm\^3 (without transfusion) * Hemoglobin ≥ 8.0 g/dL (transfusion allowed) Hepatic * SGPT and SGOT \< 5 times normal * Bilirubin ≤ 1.5 times normal Renal * Creatinine ≤ 1.5 times normal for age * No greater than 0.8 mg/dL (≤ 5 years of age) * No greater than 1.0 mg/dL (6 to 10 years of age) * No greater than 1.2 mg/dL (11 to 15 years of age) * No greater than 1.5 mg/dL (\> 15 years of age) Other * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * No allergy to cephalosporins * No active diarrhea * No uncontrolled infection PRIOR CONCURRENT THERAPY: Biologic therapy * See Chemotherapy * Recovered from prior immunotherapy * More than 3 weeks since prior biologic therapy and recovered * More than 2 days since prior hematopoietic growth factors * No concurrent epoetin alfa * No concurrent prophylactic hematopoietic growth factors during the first treatment course * No concurrent immunomodulating agents except steroids to control intracranial pressure Chemotherapy * Prior myeloablative therapy and autologous stem cell transplantation allowed * No prior allogeneic stem cell transplantation * More than 3 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered * Prior temozolomide, irinotecan, or topotecan allowed * No prior temozolomide and irinotecan as combination therapy * No other concurrent chemotherapy Endocrine therapy * See Biologic therapy Radiotherapy * At least 6 weeks since prior large field radiotherapy (e.g., total body irradiation, craniospinal therapy, whole abdomen, total lung, or \> 50% bone marrow space) and recovered * At least 4 weeks since prior radiotherapy to biopsied lesions (for study entry) and recovered * At least 6 weeks since prior MIBG therapy * Concurrent radiotherapy to painful lesions allowed provided the lesions are not used to assess treatment response Surgery * Not specified Other * No concurrent enzyme-inducing anticonvulsants (e.g., phenobarbital, phenytoin, or carbamazepine) * No other concurrent anticancer agents

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Conditions

The condition(s) this trial relates to.

diarrheal disease Drug-Related Side Effects and Adverse Reactions neuroblastoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Boston

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027-0700, United States

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Memorial Hospital - Chicago

    Chicago, Illinois, 60614, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

    Boston, Massachusetts, 02115, United States

  • Indiana University Cancer Center

    Indianapolis, Indiana, 46202-5289, United States

  • Lucile Packard Children's Hospital at Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital

    Houston, Texas, 77030-2399, United States

  • UCSF Comprehensive Cancer Center

    San Francisco, California, 94143, United States

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109-0718, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.