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Evaluating a new treatment for children with hard-to-treat neuroblastoma

A Phase I Clinical Study Evaluating the Safety of Peptide Receptor Radionuclide Therapy (PRRT) With 177Lu-DOTA0-Tyr3-Octreotate in Children With Refractory or Recurrent Neuroblastoma Expressing Somatostatin Receptors.

Phase 1 Interventional Institut Claudius Regaud · NCT03966651

This study is testing a new treatment for children with hard-to-treat neuroblastoma to see how much of the medicine they can safely receive.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment18 (estimated)
Ages1 Year to 18 Years
SexAll
SponsorInstitut Claudius Regaud Academic / other
Drugs / interventionsChemotherapy, Radiation
Locations13 sites (Besançon and 12 other locations)
Trial IDNCT03966651 on ClinicalTrials.gov

What this trial studies

This clinical trial is a multicenter, open-label phase I dose escalation study aimed at determining the Maximum Tolerated Dose (MTD) of 177Lu-DOTATATE, a peptide receptor radionuclide therapy, in children diagnosed with refractory or recurrent neuroblastoma. Participants will receive the treatment intravenously for two cycles, spaced six weeks apart, over a total study duration of five months. The trial focuses on children who have not responded to standard therapies and have confirmed somatostatin receptor expression.

Who should consider this trial

Good fit: Ideal candidates are children aged 1 to 18 years with histologically confirmed neuroblastoma that is recurrent or refractory after at least two prior treatments.

Not a fit: Patients with neuroblastoma that has not been confirmed histologically or those who have not undergone prior standard treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children with difficult-to-treat neuroblastoma, potentially improving survival rates.

How similar studies have performed: Other studies using peptide receptor radionuclide therapy have shown promise in treating neuroblastoma, suggesting potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Histologically confirmed diagnosis of neuroblastoma (patients can be included whatever the results of the 123ImIBG scan).
2. Recurrent or refractory neuroblastoma following at least two prior standard treatment regimen.
3. Positive 68Ga-DOTATOC PET within 6 weeks prior to day 1 dosing. Note: PET positivity is visually defined as follow: uptake should be equivalent or higher than the liver uptake for all lesions identified by conventional neuroblastoma imaging working.
4. Patient for whom no effective conventional therapy exists.
5. a) For dose levels 1 (80 MBq/kg) \& 2 (100 MBq/kg): Age \> 1 year and \< 18 years at the time of enrollment into the study. b) For dose level 3 (120 MBq/kg):

   - If at least one patient \< 2 years old has been enrolled in one of the previous dose levels (80 or 100 MBq/kg): Age \> 1 year and \< 18 years at the time of enrollment into the study.

   - If no patient \< 2 years old was enrolled in one of the previous dose levels (80 or 100 MBq/kg): Age ≥ 2 years and \< 18 years at the time of enrollment into the study.
6. Life expectancy greater than 3 months.
7. Adequate performance Status defined as: Karnofsky or Lansky Play Performance Scale ≥ 50% (depending on patient's age).
8. Adequate recovery from major surgery prior to receiving study treatment.
9. Patients must have recovered (to CTCAE grade 1 or baseline) from any acute toxicity resulting from any prior anti-cancer treatment (except alopecia and ototoxicity).
10. Patient must have adequate organ function as defined by the following values (within 6 weeks of first dose of study treatment):

    1. Bone marrow function:

       If no bone marrow disease:

       Platelets ≥ 100 x 109/L (unsupported for 72 hours) Absolute Neutrophil Count (ANC) ≥ 0.75 x 109/L Hemoglobin \> 7.5 g/dL (transfusions are allowed)

       In case of bone marrow disease:

       Platelets ≥ 75 x109/L (unsupported for 72 hours) ANC ≥0.5 x 109/L Hemoglobin \> 7.5 g/dL (transfusions are allowed)
    2. Renal function:

       Serum creatinine ≤1.5 ULN for age; if higher, a calculated Glomerular Filtration Rate (GFR) (2009 Schwartz formula\*) must be ≥ 60 ml/min/1.73 m2

       \* eGFR (mL/min/1,73 m²) = height (cm) x 36,5 / serum creatinine (µmol/L)
    3. Liver function:

       AST and ALT ≤2.5 ULN and total bilirubin ≤1.5 ULN. In case of liver metastases, AST and ALT ≤5 ULN and total bilirubin ≤2.5 ULN
    4. Cardiac function: Shortening fraction ≥ 28% or ejection fraction ≥ 55% by echocardiogram, with no clinical congestive heart failure associated. Normal pulmonary artery pressure.
11. Patient assent and patients/parent(s)/legal guardian(s) written informed consent that is consistent with French law and ICH-GCP guidelines.
12. Patients with reproductive potential (girls post menarche and males after 1st ejaculation) and sexually active must agree to practice effective contraceptive measures for the duration of study drug therapy and for at least 7 months (for females) or 4 months (for males) after completion of study drug therapy (in accordance with CTFG guidelines).
13. Patient affiliated to a Social Health Insurance in France.

Exclusion Criteria:

1. Children with negative 68Ga-DOTATOC PET.
2. Chemotherapy within 4 weeks prior to the start of study treatment, high dose chemotherapy with stem cell transplantation within 3 months prior to start study treatment, long acting somatostatin analogues within 30 days prior to start of study treatment, biological therapy or investigational agents within 4 weeks prior to the start of study treatment or prior to passing 5 half-lives, i.e. systemic clearance, whatever comes first.
3. Any previous molecular radiotherapy (PRRT, 131ImiBG or other)
4. External Beam Radiation (EBR) therapy within 30 days before starting study treatment.
5. Prior extensive EBR therapy:

   * to more than 25% of the bone marrow;
   * for two kidneys : D50% (Right+ left kidneys) ≥15Gy and D30% (Right+ left kidneys) ≥ 20Gy" ; In case of a single remaining kidney (D50% ≥ 15Gy)
6. Known brain metastases, unless these metastases have been treated and stabilized for at least 3 months prior to enrolment in the study. Patients with a history of brain metastases must have a head CT or MRI with contrast to document stable disease prior to enrolment in the study.
7. Other known co-existing malignancies.
8. Hypersensitivity to 177Lu-DOTATOC, amino acid solution or 68GaDOTATATE.
9. Pre-existing clinically significant hyperkalemia not adequately corrected.
10. Participation in another study with an experimental molecule and/or procedure within 1 month prior to the first dose of experimental treatment.
11. Patients with any other significant medical, psychiatric, or surgical condition, currently uncontrolled by treatment, which may interfere with completion of the study.
12. Childbearing or lactating patient.

Where this trial is running

Besançon and 12 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions NeuroblastomaPRRT177Lu-DOTATATE
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.