Recommended Phase 2 Dose (RP2D) (Phase I)
Primary
· 21 days
| Group | Value | 95% CI |
|---|---|---|
| ONC201 125 mg | 125 |
Last reviewed · How we verify
NCT02420795
Akt/ERK Inhibitor ONC201 in Treating Patients With Relapsed or Refractory Non-Hodgkin's Lymphoma
Terminated
Phase 1, PHASE2
Results posted
Last updated 4 April 2022
What this trial tests
Phase 1, PHASE2 trial testing Akt/ERK Inhibitor ONC201 in Central Nervous System Lymphoma in 16 participants. Terminated before completion.
Timeline
3 November 2015
Primary endpoint
16 November 2020
16 November 2020
16 November 2020
Quick facts
| Lead sponsor | M.D. Anderson Cancer Center |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 16 |
| Start date | 3 November 2015 |
| Primary completion | 16 November 2020 |
| Estimated completion | 16 November 2020 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Akt/ERK Inhibitor ONC201 — full drug profile →
- Laboratory Biomarker Analysis — full drug profile →
- Pharmacological Study — full drug profile →
Conditions studied
- Central Nervous System Lymphoma — all drugs for Central Nervous System Lymphoma →
- Gastric Mantle Cell Lymphoma — all drugs for Gastric Mantle Cell Lymphoma →
- Recurrent Mantle Cell Lymphoma — all drugs for Recurrent Mantle Cell Lymphoma →
- Recurrent Non-Hodgkin Lymphoma — all drugs for Recurrent Non-Hodgkin Lymphoma →
Sponsor
M.D. Anderson Cancer Center — full company profile →
Who can join
18 and older, any sex, with Central Nervous System Lymphoma or Gastric Mantle Cell Lymphoma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Overall Response Rate (Phase 1 and 2)
Primary
· Up to 63 days (first 3 courses)
Defined as either progressive disease or stable disease observed assessed by the Revised International Workshop Standardization Response Criteria for non-Hodgkin lymphoma.
Progressive Disease
| Group | Value | 95% CI |
|---|---|---|
| ONC201 125 mg | 3 | |
| ONC201 250 mg | 1 | |
| ONC201 625 mg | 3 |
Stable Disease
| Group | Value | 95% CI |
|---|---|---|
| ONC201 125 mg | 1 | |
| ONC201 250 mg | 1 | |
| ONC201 625 mg | 1 |
Overall Survival (OS)
Secondary
· every 3 months for 1 year, then every 6 months, up to 3 years
Overall survival is the time in months from start of study treatment to date of death due to any cause.
| Group | Value | 95% CI |
|---|---|---|
| ONC201 125 mg | 29 | 1 – 30 |
| ONC201 250 mg | 15 | 1 – 30 |
| ONC201 625 mg | 24 | 1 – 48 |
Progression-free Survival (PFS)- (Phase 2)
Secondary
· every 3 months for 1 year, then every 6 months, up to 6 years
Progression free survival is defined as time in weeks from start of study treatment to first documentation of objective tumor progression or up to death due to any cause, whichever occurs first.
| Group | Value | 95% CI |
|---|---|---|
| ONC201 625 mg | 5 | 1 – 10 |
Adverse events — posted to ClinicalTrials.gov
Time frame: From the first dose through every 3 months after the last dose of study medication, up to 1 year.. Reporting threshold: 1%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
ONC201 125 mg
Serious: 0/4 (0%)
Deaths: 0/4
ONC201 250 mg
Serious: 1/2 (50%)
Deaths: 0/2
ONC201 625 mg
Serious: 1/5 (20%)
Deaths: 1/5
Serious adverse events (5 terms)
| Reaction | System | ONC201 125 mg | ONC201 250 mg | ONC201 625 mg |
|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | — | — | — |
| Dehydration | General disorders | — | — | — |
| Blood and lymphatic system disorders- Other | Blood and lymphatic system disorders | — | — | — |
| Myocardial Infarction | Infections and infestations | — | — | — |
| Edema Limbs | General disorders | — | — | — |
Other adverse events (83 terms — click to expand)
| Reaction | System | ONC201 125 mg | ONC201 250 mg | ONC201 625 mg |
|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | — | — | — |
| Fatigue | General disorders | — | — | — |
| Anemia | Blood and lymphatic system disorders | — | — | — |
| Edema limbs | General disorders | — | — | — |
| Anxiety | Nervous system disorders | — | — | — |
| AST increased | General disorders | — | — | — |
| Dizziness | Nervous system disorders | — | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — |
| Platelet count decreased | Blood and lymphatic system disorders | — | — | — |
| Weakness | General disorders | — | — | — |
| Weight loss | Investigations | — | — | — |
| Right Hip Pain | General disorders | — | — | — |
| Numbness/Tingling | Nervous system disorders | — | — | — |
| Platelet Count Decreased | Blood and lymphatic system disorders | — | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — | — |
| Appetite change | Metabolism and nutrition disorders | — | — | — |
| Back pain | General disorders | — | — | — |
| Blood and lymphatic system disorder | Blood and lymphatic system disorders | — | — | — |
| Blurred vision | Eye disorders | — | — | — |
| Bug bites | General disorders | — | — | — |
| BUN increased | Renal and urinary disorders | — | — | — |
| Constipation | Gastrointestinal disorders | — | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Creatinine increased | Investigations | — | — | — |
| Dehydration | Metabolism and nutrition disorders | — | — | — |
| Dry mouth | Gastrointestinal disorders | — | — | — |
| Dyspepsia | Gastrointestinal disorders | — | — | — |
| Dysphagia | Gastrointestinal disorders | — | — | — |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Ear Pain | Ear and labyrinth disorders | — | — | — |
| Edema trunk | General disorders | — | — | — |
| Elevated LDH | General disorders | — | — | — |
| Fall | Injury, poisoning and procedural complications | — | — | — |
| Fever | General disorders | — | — | — |
| GI bleeding | Gastrointestinal disorders | — | — | — |
| Hearing impaired | Ear and labyrinth disorders | — | — | — |
| Hematuria | Renal and urinary disorders | — | — | — |
| Hypercalcemia | Metabolism and nutrition disorders | — | — | — |
| Hyperglycemia | Metabolism and nutrition disorders | — | — | — |
Most-reported serious reactions: Anemia, Dehydration, Blood and lymphatic system disorders- Other, Myocardial Infarction, Edema Limbs.
Data from ClinicalTrials.gov NCT02420795 adverse events section.
Sponsor's own description
This phase I/II trial studies the side effects and the best dose of v-akt murine thymoma viral oncogene homolog (Akt)/mitogen-activated protein kinase 1(ERK) inhibitor ONC201 and to see how well it works in treating patients with non-Hodgkin's lymphoma that has returned after a period of improvement or does not respond to treatment. Akt/ERK inhibitor ONC201 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Targeting apoptosis in cancer therapy.
Carneiro BA, El-Deiry WS. · · 2020 · cited 1823× · PMID 32203277 · DOI 10.1038/s41571-020-0341-y -
Role of PI3K/AKT pathway in cancer: the framework of malignant behavior.
Jiang N, Dai Q, Su X, Fu J, et al · · 2020 · cited 419× · PMID 32333246 · DOI 10.1007/s11033-020-05435-1 -
Developing TRAIL/TRAIL death receptor-based cancer therapies.
Yuan X, Gajan A, Chu Q, Xiong H, et al · · 2018 · cited 200× · PMID 29541897 · DOI 10.1007/s10555-018-9728-y -
ONC201 and imipridones: Anti-cancer compounds with clinical efficacy.
Prabhu VV, Morrow S, Rahman Kawakibi A, Zhou L, et al · · 2020 · cited 137× · PMID 33142238 · DOI 10.1016/j.neo.2020.09.005 -
Discovery and clinical introduction of first-in-class imipridone ONC201.
Allen JE, Kline CL, Prabhu VV, Wagner J, et al · · 2016 · cited 133× · PMID 27602582 · DOI 10.18632/oncotarget.11814 -
Novel Apoptosis-Inducing Agents for the Treatment of Cancer, a New Arsenal in the Toolbox.
Lim B, Greer Y, Lipkowitz S, Takebe N. · · 2019 · cited 61× · PMID 31370269 · DOI 10.3390/cancers11081087 -
Dopamine Receptor D5 is a Modulator of Tumor Response to Dopamine Receptor D2 Antagonism.
Prabhu VV, Madhukar NS, Gilvary C, Kline CLB, et al · · 2019 · cited 51× · PMID 30559168 · DOI 10.1158/1078-0432.ccr-18-2572 -
Mechanisms of imipridones in targeting mitochondrial metabolism in cancer cells.
Bonner ER, Waszak SM, Grotzer MA, Mueller S, et al · · 2021 · cited 50× · PMID 33336683 · DOI 10.1093/neuonc/noaa283
Verify or expand the search:
- PubMed search for NCT02420795
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Other recruiting trials for Central Nervous System Lymphoma
Currently open trials in the same condition.
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Other M.D. Anderson Cancer Center trials
Trials by the same sponsor.
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- NCT07076498 — Engineered HSV-1 M032 for the Treatment of Children and Adults With Newly Diagnosed Diffuse Midline Glioma After Standar · Phase 1 · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
Data sources for this page
- Trial protocol + status: ClinicalTrials.gov NCT02420795 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by M.D. Anderson Cancer Center
- Last refreshed: 4 April 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02420795.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing