AzTrials
AzTrials

NeuroblastomaPhase 2NCT02605421

Myeloablative Consolidation Therapy and Tandem Autologous Stem Cell Rescue in Patients With High-Risk Neuroblastoma

A Phase 2 study testing Thiotepa, Cyclophosphamide and Melphalan in people with Neuroblastoma, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is a phase II single center study to administer two courses of myeloablative consolidation chemotherapy each followed by an autologous peripheral blood stem cell (PBSC) rescue in patients with high-risk neuroblastoma who have completed induction chemotherapy (independent of this study). Read more about this study

Who is it for?
Ages Up to 30 years
Conditions the study is for
Neuroblastoma
What is being tested?
Thiotepa, Cyclophosphamide and Melphalan
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
Jun 2016to Dec 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 Up to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult
  • Less than 30 years of age at diagnosis of neuroblastoma
  • End of Induction disease evaluation demonstrating CR, PR, MR or SD
  • Hematopoietic Recovery from last induction course of chemotherapy
  • No uncontrolled infection
  • Minimum frozen PBSCs of 2 x 10^6 CD34 cells/kg for each transplant are mandatory and a PBSC of 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of no less than 6 x 10^6 CD34 cells/kg is encouraged). These must all be collected prior to the initiation of consolidation.
  • Adequate organ function defined as:
  • Hepatic: AST and ALT < 3 x upper limit of institutional normal; ALT ≤ 3 x ULN for age; total bilirubin ≤ 1.5 x ULN for age, if baseline was normal, > 1.0 1.5 x baseline if baseline was abnormal
  • Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 45%, no clinical congestive heart failure
  • Pulmonary: no evidence of dyspnea at rest and norequirement for supplemental oxygen
  • Renal: Creatinine clearance or GFR > 60 mL/min/1.73m^2. If a creatinine clearance is performed at end induction and the result is < 100 ml/min/1.73m^2, a GFR must then be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m^2
  • Recovery from acute toxicities of last cycle of induction chemotherapy
  • Appropriate written consent - adult or parent/guardian if patient is < 18 years of age and minor information sheet if patient is > 8 years of age

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Lisa Burke contact

612-273-8482

lburke3@Fairview.org

Ashish Gupta, MBBS, MPH contact

612-626-2961

stef0030@umn.edu

United States1 site, 1 recruiting

  • Masonic Cancer Center, University of Minnesota

    Minneapolis, Minnesota, 55455

    Lisa Burke, RN, 612-273-8482, lburke3@Fairview.org

    Ashish Gupta, MBBS, MPH

    Recruiting

Other trials in United States

About this study

Official title: Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma

This is a phase II single center study to administer two courses of myeloablative consolidation chemotherapy each followed by an autologous peripheral blood stem cell (PBSC) rescue in patients with high-risk neuroblastoma who have completed induction chemotherapy (independent of this study). Ideally, patients should begin consolidation chemotherapy no later than 8 weeks after the start of Induction Cycle #5; however it is strongly recommended to begin consolidation within 4-6 weeks after the start of Induction Cycle #5.

Conditions
Neuroblastoma
Keywords
High-risk neuroblastoma, Neuroblastoma, Myeloablative chemotherapy, Myeloablative consolidation chemotherapy, Autologous peripheral blood stem cell rescue, PBSC, Autologous stem cell rescue
Medical subject headings
Neuroblastoma

What participants receive

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

  • ThiotepaDrug

    Thiotepa by IV once daily for 3 doses on Days -7, -6 and -5. Given as part of Consolidation Course #1 along with Cyclophosphamide.

  • CyclophosphamideDrug

    Cyclophosphamide by IV once daily for 4 doses on Days -5, -4, -3 and -2. Given as part of Consolidation Course #1 along with Thiotepa.

  • MelphalanDrug

    Melphalan by IV once daily for 3 doses on Days -8, -7, and -6. Given as part of Consolidation Course #2 along with Etoposide and Carboplatin.

  • EtoposideDrug

    Etoposide by IV once daily for 4 doses on Days -8, -7, -6 and -5. Given as part of Consolidation Course #2 along with Melphalan and Carboplatin.

  • CarboplatinDrug

    Carboplatin by IV once daily for 4 doses on Days -8, -7, -6 and -5. Given as part of Consolidation Course #2 along with Etoposide and Melphalan.

  • Autologous Stem Cell InfusionBiological

    On Day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.

  • Granulocyte colony stimulating factorBiological

    Also called G-CSF

    Beginning on day 0 after infusion of the PBSC, patients will receive G-CSF SQ or IV (SQ preferred) 5 micrograms/kg once daily and continuing once daily until post-nadir ANC > 2000/μL for 3 consecutive days.

Study groups in NCT02605421
Study groupRoleWhat they receive
Patients Treated for NeuroblastomaExperimentalConsolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Receives: Drug: Thiotepa; Drug: Cyclophosphamide; Drug: Melphalan; Drug: Etoposide; Drug: Carboplatin; Biological: Autologous Stem Cell Infusion; Biological: Granulocyte colony stimulating factor

What is being measured

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

  1. Progression Free Survival

    Percentage of patients with progression free survial. Kaplan-Meier curves and 95% confidence intervals will be used to estimate.

    Measured: 3 years from first PBSC infusion

Secondary outcomes (3)

  1. Time to Engraftment

    Defined as an absolute neutrophil count (ANC) greater than or equal to 0.5 x 109/L for three consecutive days by day 42 after first transplant.

    Measured: Day 42

  2. Relapse

    Percentage of patients with relapse. Relapse will be defined as any new lesion; increase of any measurable lesion by >25%; previous negative bone is positive.

    Measured: 3 years from first PBSC infusion

  3. Overall Survival

    Kaplan-Meier curves and 95% confidence intervals will be used to estimate overall survival.

    Measured: 3 years from first PBSC infusion

Timeline

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

  1. First posted16 Nov 2015 (estimated)
  2. Study startJun 2016
  3. Primary completion1 Oct 2027 (estimated)
  4. Study completionDec 2028 (estimated)

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02605421
Sponsor's protocol ID
2015LS108
Status last verified
Oct 2026
First submitted
12 Nov 2015
Last updated
27 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Thiotepa, Cyclophosphamide and Melphalan in people with Neuroblastoma, run by Masonic Cancer Center, University of Minnesota 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?

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: looking for participants now, as of the sponsor's last update on 27 Oct 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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

Contact Lisa Burke (612-273-8482, lburke3@Fairview.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression Free Survival (time frame: 3 years from first PBSC infusion). 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 NCT02605421.

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 27 Oct 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in