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Recurrent Cutaneous MelanomaPhase 1NCT03420963

Donor Natural Killer Cells, Cyclophosphamide, and Etoposide in Treating Children and Young Adults With Relapsed or Refractory Solid Tumors

A Phase 1 study testing Cord Blood-derived Expanded Allogeneic Natural Killer Cells, Cyclophosphamide and Etoposide in people with Recurrent Cutaneous Melanoma and Recurrent Lip and Oral Cavity Carcinoma, run by M.D. Anderson Cancer Center at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This phase I trial studies the side effects and best dose of cord blood-derived expanded allogeneic natural killer cells (donor natural killer [NK] cells) and how well they work when given together with cyclophosphamide and etoposide in treating children and young adults with solid tumors that have come back… Read more about this study

Who is it for?
Ages 12 months to 40 years
What is being tested?
Cord Blood-derived Expanded Allogeneic Natural Killer Cells, Cyclophosphamide and Etoposide
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteUnited States
Study dates
31 Aug 2018to 26 Feb 2026
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 months to 40 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • SCREENING: Patients with relapsed or refractory solid tumors and without known curative therapy or therapy proven to proven to prolong survival with acceptable quality of life.
  • SCREENING: Patients older than 21 years must have a solid tumor considered by study doctor to be of the childhood cancer type.
  • SCREENING: Performance level as measured by Karnofsky >= 60% for patients > 16 years of age or Lansky >= 60% for patients =< 16 years of age.
  • SCREENING: Documentation of measurable or evaluable non-measurable disease.
  • SCREENING: At least one documented histological verification of solid tumor diagnosis. Can be from original diagnosis or more recent.
  • ENROLLMENT: Patient must have fully recovered (i.e. returned to baseline) from the clinically significant acute treatment-related toxicities of all prior treatments prior to beginning treatment on this protocol with exceptions of cytopenias resulting from persistent disease, hearing loss and alopecia.
Show all 25Show fewer
  • ENROLLMENT: Performance level as measured by Karnofsky >= 60% for patients > 16 years of age or Lansky >= 60% for patients =< 16 years of age.
  • ENROLLMENT: Creatinine clearance >= 60 mL/min/1.73m^2 (calculated by 24 hour [h] urine collection or nuclear glomerular filtration rate [GFR] scan if 24 h collection is not possible) or a serum creatinine based on age and gender as follows:
  • Age, maximum serum creatinine (mg/dL):
  • 1 month to < 6 months, male 0.4, female 0.4;
  • 6 months to < 1 year, male 0.5, female 0.5;
  • 1 to < 2 years, male 0.6, female 0.6;
  • 2 to < 6 years, male 0.8, female 0.8;
  • 6 to < 10 years, male 1, female 1;
  • 10 to < 13 years, male 1.2, female 1.2;
  • 13 to < 16 years, male 1.5, female 1.4;
  • >= 16 years, male 1.7, female 1.4.
  • ENROLLMENT: Adequate liver function, defined as: total bilirubin =< 2 mg/dl
  • ENROLLMENT: Adequate liver function, as defined as serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 2.5 x upper limit of normal (ULN) for age (unless Gilbert's disease or abnormal liver function due to primary disease).
  • ENROLLMENT: Evidence of adequate bone marrow function (defined by absolute neutrophil count >= 750), unless patient has documented tumor metastasis to the bone marrow or other condition that results in cytopenia without abnormal marrow function.
  • ENROLLMENT: Evidence of adequate bone marrow function (defined by platelets >= 50,000), unless patient has documented tumor metastasis to the bone marrow or other condition that results in cytopenia without abnormal marrow function.
  • ENROLLMENT: Pulmonary symptoms controlled by medication and pulse oximetry >= 92% on room air.
  • ENROLLMENT: Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the investigator. (Non-childbearing potential defined as pre-menarche, greater than one year post-menopausal or surgically sterilized).
  • ENROLLMENT: Confirmation that a cord blood donor which is matched with the recipient at a 4, 5, or 6/6 human leukocyte antigen (HLA) class I (serological) and HLA class II (molecular) antigens.
  • ENROLLMENT: Signed informed consent and if applicable pediatric assent.

Who cannot join

  • SCREENING: Primary tumors of the central nervous system.
  • SCREENING: Chronic corticosteroid dependence that is unable to be weaned to discontinue.
  • SCREENING: Determined by study doctor that patient is unlikely to meet inclusion criteria after screening.
  • ENROLLMENT: Uncontrolled arrhythmias or uncontrolled symptoms of cardiac disease noted by screening history and physical. Patients with known cardiac dysfunction should have an ejection fraction (EF) > 40% documented by echocardiogram (ECHO).
  • ENROLLMENT: Patients where the burden of pulmonary metastasis, location, or bulkiness of disease may cause high morbidity if localized swelling such as causing uncontrolled symptoms, oxygen dependence, or location near a major bronchi as determined by investigator.
  • ENROLLMENT: Pregnant females.
  • ENROLLMENT: Any uncontrolled systemic infection.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M D Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: Ex-Vivo Expanded Allogeneic NK Cells for the Treatment of Solid Tumors of Pediatric Origin in Children and Young Adults

This phase I trial studies the side effects and best dose of cord blood-derived expanded allogeneic natural killer cells (donor natural killer [NK] cells) and how well they work when given together with cyclophosphamide and etoposide in treating children and young adults with solid tumors that have come back (relapsed) or that do not respond to treatment (refractory). NK cells, white blood cells important to the immune system, are donated/collected from cord blood collected at birth from healthy babies and grown in the lab. Drugs used in chemotherapy, such as cyclophosphamide and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving NK cells together with cyclophosphamide and etoposide may work better in treating children and young adults with solid tumors.

Read the detailed description

PRIMARY OBJECTIVE:

  1. Determine the safety, maximum tolerated dose and/or recommended phase II dose of cord blood-derived expanded allogeneic natural killer cells (expanded allogeneic cord donor natural killer [NK] cells) following chemotherapy.

SECONDARY OBJECTIVES:

  1. Determine the persistence of adoptively-transferred cord NK cells after solid tumor directed chemotherapy.

II. Preliminarily define the antitumor activity to adoptively transferred NK cells following the study preparative regimen in the confines of a phase I study.

III. Determine the immunophenotype and function of the infused NK cell product. IV. Preliminarily evaluate for any correlate of phenotype, killer cell immunoglobulin-like receptor (kir) haplotype, and function with overall response.

OUTLINE: This is a dose escalation study of cord blood derived allogeneic NK cells.

Patients receive cyclophosphamide intravenously (IV) once daily (QD) over 30 minutes and etoposide IV QD over 60 minutes on days 1-5 in the absence of unacceptable toxicity. Patients then receive cord blood derived allogeneic NK cells IV on day 8.

After completion of study treatment, patients are followed up at 3-4 days, and then every week for 30 days.

Being tested
Cord Blood-derived Expanded Allogeneic Natural Killer Cells, Cyclophosphamide, Etoposide
Medical subject headings
Melanoma, Mouth Neoplasms, Endocrine Gland Neoplasms, Mesothelioma, Malignant, Pharyngeal Neoplasms, Skin Neoplasms, Sarcoma, Thyroid Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cord Blood-derived Expanded Allogeneic Natural Killer CellsBiological

    Also called Allogeneic CB-derived Ex vivo-expanded NK Cells, CB-derived Expanded Allogeneic NK Cells, UCB-derived Expanded Allogeneic NK Cells, Umbilical Cord Blood-derived Expanded Allogeneic Natural Killer Cells

    Given IV

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida

    Given IV

  • EtoposideDrug

    Also called Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar

    Given IV

Study groups in NCT03420963
Study groupRoleWhat they receive
Treatment (cyclophosphamide, etoposide, NK cells)ExperimentalPatients receive cyclophosphamide IV QD over 30 minutes and etoposide IV QD over 60 minutes on days 1-5 in the absence of unacceptable toxicity. Patients then receive cord blood derived allogeneic NK cells IV on day 8.
Receives: Biological: Cord Blood-derived Expanded Allogeneic Natural Killer Cells; Drug: Cyclophosphamide; Drug: Etoposide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events

    Since toxicity is the primary outcome, patients with non-measurable disease (who are still evaluable) will be included in the analysis and in these cases standard methods for categorizing response of non-measurable disease will be used. Toxicity rate will be estimated separately by dose and cohort along with a 95% confidence interval. Adverse events will be tabulated for all the patients separately by dose levels.

    Measured: Up to 30 days after the NK cell infusion

  2. Maximum tolerated dose and/or recommended phase 2 dose of cord blood-derived expanded allogeneic natural killer (NK) cells following chemotherapy

    Primary analyses in this phase I trial are descriptive and exploratory. Toxicity rate will be estimated separately by dose and cohort along with a 95% confidence interval. Adverse events will be tabulated for all the patients separately by dose levels.

    Measured: Up to 30 days after the NK cell infusion

Secondary outcomes (4)

  1. Response rate per immune-related therapy trials Response Evaluation Criteria in Solid Tumors (irRECIST)

    Complete response and partial response will be estimated separately by dose and cohort along with a 95% confidence interval. Correlation of NK cell persistence, phenotype, and function with overall response will be estimated using two-sample t-test/Wilcoxon sum-rank test and analysis of variance (ANOVA)/Kruskal-Wallis test as appropriate.

    Measured: Up to 30 days after the NK cell infusion

  2. NK cell persistence, phenotype, and function

    Correlation of NK cell persistence, phenotype, and function with overall response will be estimated using two-sample t-test/Wilcoxon sum-rank test and ANOVA/Kruskal-Wallis test as appropriate.

    Measured: Up to 30 days after the NK cell infusion

  3. Overall survival (OS)

    The Kaplan-Meier method will be used to estimate the distribution of OS. The description statistics of rate of OS may be analyzed separately by different tumor types and response/stable patients. Cox proportional hazards regression analysis may also be conducted to model the association between OS and factors of interest.

    Measured: Up to 30 days after the NK cell infusion

  4. Time to progression (TTP)

    The Kaplan-Meier method will be used to estimate the distribution of TTP. The description statistics of rate of TTP may be analyzed separately by different tumor types and response/stable patients. Cox proportional hazards regression analysis may also be conducted to model the association between TTP and factors of interest.

    Measured: Up to 30 days after the NK cell infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Feb 2018
  2. Study start31 Aug 2018
  3. Primary completion26 Feb 2026
  4. Study completion26 Feb 2026

Why it stopped: \<75% participation

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03420963
Sponsor's protocol ID
2017-0085
Other IDs
NCI-2018-00909, 2017-0085, P30CA016672
Status last verified
Mar 2026
First submitted
29 Jan 2018
Last updated
20 Mar 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cord Blood-derived Expanded Allogeneic Natural Killer Cells, Cyclophosphamide and Etoposide in people with Recurrent Cutaneous Melanoma and Recurrent Lip and Oral Cavity Carcinoma, run by M.D. Anderson Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 20 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: \<75% participation). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (time frame: Up to 30 days after the NK cell infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03420963.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2026.

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