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Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
Phase 2 trial testing DFMO in Neuroblastoma in 140 participants. Completed in 24 August 2023.
| Lead sponsor | Giselle Sholler |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | prevention |
| Enrollment | 140 |
| Start date | 5 March 2015 |
| Primary completion | 27 March 2018 |
| Estimated completion | 24 August 2023 |
| Sites | 22 locations across United States |
Giselle Sholler — full company profile →
Under 21, any sex, with Neuroblastoma. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)
| Group | Value | 95% CI |
|---|---|---|
| Stratum 1 | 84 | 80 – 88 |
| Stratum 2 | 51 | 43 – 59 |
To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)
| Group | Value | 95% CI |
|---|---|---|
| Stratum 1 | 97 | 95 – 99 |
| Stratum 2 | 84 | 78 – 90 |
To continue to determine the safety and tolerability of DFMO as a single agent and in pediatric and young adult patients with high risk neuroblastoma that is in remission.
| Group | Value | 95% CI |
|---|---|---|
| DFMO Twice Daily | 57 |
Tests (p-value) of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype. Blood: microRNA analysis as predictor of DFMO effect, ornithine decarboxylase (ODC) single nucleotide polymorphism (SNP) analysis in DNA isolated from nucleated cells
| Group | Value | 95% CI |
|---|---|---|
| GG, GT, TT | 0.96 | |
| GG or GT, TT | 0.58 | |
| GG, GT or TT | 0.67 |
Pharmacokinetic assay Cmax/D Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days.
| Group | Value | 95% CI |
|---|---|---|
| Study Subjects Consented to PK Collection | 11958 | 5123 – 18793 |
Pharmacokinetic assay AUC(0-6 hr)/D Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days
| Group | Value | 95% CI |
|---|---|---|
| Study Subjects Consented to PK Collection | 47024 | 23868 – 70180 |
Pharmacokinetic assay- tmax, hr Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days
| Group | Value | 95% CI |
|---|---|---|
| Study Subjects Consented to PK Collection | 3.3 | 1.9 – 4.7 |
Time frame: Through study completion plus 30 days, an average of 2 years.. Reporting threshold: 4%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | All Study Subjects |
|---|---|---|
| Infection | Infections and infestations | — |
| Vomiting | Gastrointestinal disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Pain | General disorders | — |
| Fever | General disorders | — |
| Hyponatremia | Investigations | — |
| Benign vascular lesion | Vascular disorders | — |
| Swelling of Eye | Eye disorders | — |
| Hypoglycemia | Metabolism and nutrition disorders | — |
| Urticaria | Skin and subcutaneous tissue disorders | — |
| Constipation | Gastrointestinal disorders | — |
| Respiratory Disorder | Respiratory, thoracic and mediastinal disorders | — |
| Hypotension | Cardiac disorders | — |
| hearing loss | Ear and labyrinth disorders | — |
| Fracture | Musculoskeletal and connective tissue disorders | — |
| Influenza | Infections and infestations | — |
| Bacteremia | Infections and infestations | — |
| Obstruction | Gastrointestinal disorders | — |
| Reaction | System | All Study Subjects |
|---|---|---|
| ALT elevation | Hepatobiliary disorders | — |
| Neutrophil count decrease | Blood and lymphatic system disorders | — |
| AST elevation | Hepatobiliary disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Hearing Loss | Ear and labyrinth disorders | — |
| Anemia | Blood and lymphatic system disorders | — |
| Otitis Media | Infections and infestations | — |
Most-reported serious reactions: Infection, Vomiting, Diarrhea, Pain, Fever, Hyponatremia, Benign vascular lesion, Swelling of Eye.
Data from ClinicalTrials.gov NCT02395666 adverse events section.
The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO. The safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit. The procedures involved in this study include Medical history, Physical exam, Vital signs (blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the tumor(s), meta-iodobenzylguanidine (MIBG) scans, and Bone marrow aspirations. All of these tests and procedures are considered standard of care for this population. Drug administration is also part of this protocol, including an investigational new drug called DFMO. The proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day while on study. There will be 27 cycles. Each cycle will be 28 days in length.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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