MelanomaPhase 1/2NCT03391050
A Study of APR-246 in Combination With Dabrafenib in Resistant Patients With BRAF V600 Mutant Melanoma
A Phase 1/2 study testing APR-246 and Dabrafenib in people with Melanoma, run by Aprea Therapeutics at 3 sites in Belgium.
Trial summary
The purpose of this study is to make a preliminary assessment of the efficacy of a combined APR-246 and dabrafenib therapy regimen in patients with BRAFV600 mutant unresectable and/or metastatic cutaneous melanoma resistant to the dabrafenib/trametinib combination. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Melanoma
- What is being tested?
- APR-246 and Dabrafenib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 3 sitesBelgium
- Study dates
- 18 Jan 2018to 8 Aug 2018
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with confirmed BRAF V600 mutation-positive unresectable and/or metastatic malignant cutaneous melanoma, as determined locally by a validated test and treated with dabrafenib/trametinib first line combination therapy or second line after first line immunotherapy.
- Patients that have progressed according to RECIST 1.1 after at least 4 weeks of treatment with dabrafenib/trametinib and remained on dabrafenib full dose (150mg bid) treatment for the study.
- Measurable disease according to RECIST 1.1 criteria. For phase II only, metabolic measurable disease (according to PERCIST).
- Availability of tissue from a metastatic lesion. A new biopsy is required unless inaccessible. An archival sample is accepted in that case after discussion with the sponsor.
- ECOG Performance Status of 0 or 1.
- Patients able to swallow and retain oral medication.
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- Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
- For female patients of childbearing potential, a pregnancy test (serum) will be performed within 7 days before inclusion. Woman of childbearing potential must be willing to use one highly effective form of contraception during anticancer treatment and for at least six months thereafter. Men must agree to use condom during the course of this study and at least six months after the last administration of the study treatment and contraception should be considered for partner of childbearing potential.
- Adequate organ system function.
- Signed informed consent before any study specific procedure and/or treatment happens.
Who cannot join
- Presence of uveal melanoma and/or other non-cutaneous melanomas.
- Current use of a prohibited medication or need for any of these medications during treatment with study drug and within 28 days before the first administration of APR-246. I.e., no anti-cancer other than that given in this clinical trial, no immunotherapy, no hormonal cancer therapy, no radiation therapy (except palliative) and no experimental medications are permitted during the trial. All alternative therapies must first be approved by the sponsor. Supportive care therapies are allowed.
- Unresolved toxicity greater than NCI-CTCAE(v4) Grade 1 from previous anti-cancer therapy except alopecia.
- Presence of active gastrointestinal disease or other condition that will interfere significantly with the absorption, distribution, metabolism, or excretion of drugs.
- Known HIV, active hepatitis B or hepatitis C infection.
- Primary malignancy of the central nervous system.
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- History of familial long QT, serious ventricular arrhythmia (no VT > 130 bpm and > 5 extra beats per minute), no QTc ≥ 480 msec calculated from a single ECG reading or a mean of 3 ECG readings using Fridericia's correction (QTcF = QT/RR0.33) or bradycardia (< 45 bpm).
- Untreated or symptomatic brain metastasis, leptomeningeal disease or spinal cord compression. Patients who are on a stable dose of corticosteroids > 1 month or off corticosteroids for 2 weeks can be enrolled.
- History of acute coronary syndromes (including unstable angina), coronary angioplasty, or stenting, or thrombo-embolic event within the past 24 weeks from signature of ICF.
- Class II, III, or IV heart failure as defined by the New York Heart Association (NYHA) functional classification system.
- Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to the study drugs, or excipients.
- Uncontrolled diabetes, hypertension or other medical conditions that may interfere with assessment of toxicity.
- Pregnant or lactating woman.
Trial locations and contacts
3 sites in 1 country.
Belgium3 sites
Institut Jules Bordet
Brussels, 1000
Universitair Ziekenhuis Antwerpen
Edegem, 2650
CHU UCL Namur - site Sainte-Elisabeth
Namur, 5000
About this study
Official title: A Phase Ib/II Study to Investigate the Safety and Clinical Activity of APR-246 in Combination With Dabrafenib in Patients With BRAF V600 Mutant Unresectable and/or mEtastatic Cutaneous MElanoma Resistant to Dabrafenib/Trametinib Combination
The purpose of this study is to make a preliminary assessment of the efficacy of a combined APR-246 and dabrafenib therapy regimen in patients with BRAFV600 mutant unresectable and/or metastatic cutaneous melanoma resistant to the dabrafenib/trametinib combination. In addition, the study aims to assess the safety profile of the combined APR-246 and dabrafenib therapy regimen, to explore potential biomarkers, and to further describe the anti-tumour activity of the combination of APR-246 and dabrafenib. The trial will enroll up to 31 evaluable patients.
- Conditions
- Melanoma
- Being tested
- APR-246, Dabrafenib
- Keywords
- Melanoma, Malignant melanoma, Skin cancer, Resistant cancer, Unresectable cancer, Metastatic cancer, BRAF mutant
- Medical subject headings
- Melanoma, Skin Neoplasms, Neoplasm Metastasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APR-246Drug
Intravenous infusion
DabrafenibDrug
Oral administration
| Study group | Role | What they receive |
|---|---|---|
| APR-246 + Dabrafenib | Experimental | Not described Receives: Drug: APR-246; Drug: Dabrafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase Ib: Adverse Events (AEs)
Clinical and laboratory adverse events (AEs) and serious adverse events (SAEs) will be reported and graded
Measured: Up to 30 days after last study treatment day, or at end of study visit due to progression, whichever occurs later (treatment cycles are stopped due to progression, toxicity or patient's decision)
Phase Ib: Dose Limiting Toxicities (DLTs)
Measured: Until end of cycle 1 (cycle length is 28 days)
Phase II: Objective response rate by RECIST1.1
Measured: Until progression (assessed up to 12 months)
Secondary outcomes (8)
Clinical benefit rate
Proportion of patients with a CR, PR or Stable Disease (SD) ≥ 4 months
Measured: Until progression (assessed up to 12 months)
Duration of response
Measured: Until progression (assessed up to 12 months)
Progression free survival (PFS)
Measured: Until progression (assessed up to 12 months)
Area under the plasma concentration versus time curve (AUC) for APR-246
Measured: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Plasma drug concentration at a specified time t (Ct) for APR-246
Measured: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Maximum observed plasma concentration (Cmax) of APR-246
Measured: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Time to reach maximum plasma concentration following drug administration (tmax) for APR-246
Measured: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Assessment of metabolic response
According to classical PERCIST criteria (30%) modified PERCIST criteria (15%) and the consistency classification
Measured: Until Cycle 2 Day 1 (cycle length is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2018
- Study start18 Jan 2018
- Primary completion8 Aug 2018
- Study completion8 Aug 2018
Why it stopped: Target patient population - difficult to find patientes
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for MelanomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aprea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Jules Bordet Institute
- Responsible party
- Sponsor
- Investigators
- Ahmad Awada, PhD, Study chair, Jules Bordet Institute, Brussels, BelgiumJoseph Kerger, MD, Principal investigator, Jules Bordet Institute, Brussels, Belgium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03391050
- Sponsor's protocol ID
- APR-633
- Status last verified
- Jul 2019
- First submitted
- 19 Dec 2017
- Last updated
- 31 Jul 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Lehmann S, Bykov VJ, Ali D, Andren O, Cherif H, Tidefelt U, Uggla B, Yachnin J, Juliusson G, Moshfegh A, Paul C, Wiman KG, Andersson PO. Targeting p53 in vivo: a first-in-human study with p53-targeting compound APR-246 in refractory hematologic malignancies and prostate cancer. J Clin Oncol. 2012 Oct 10;30(29):3633-9. doi: 10.1200/JCO.2011.40.7783. Epub 2012 Sep 10.
PubMed 22965953Deneberg S, Cherif H, Lazarevic V, Andersson PO, von Euler M, Juliusson G, Lehmann S. An open-label phase I dose-finding study of APR-246 in hematological malignancies. Blood Cancer J. 2016 Jul 15;6(7):e447. doi: 10.1038/bcj.2016.60. No abstract available.
PubMed 27421096
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib/II Study to Investigate the Safety and Clinical Activity of APR-246 in Combination With Dabrafenib in Patients With BRAF V600 Mutant Unresectable and/or mEtastatic Cutaneous MElanoma Resistant to Dabrafenib/Trametinib Combination
- ClinicalTrials.gov ID
- NCT03391050
- Acronym / sponsor ID
- APR-633
- Conditions
- Melanoma
- Keywords
- Melanoma, Malignant melanoma, Skin cancer, Resistant cancer, Unresectable cancer, Metastatic cancer, BRAF mutant
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Target patient population - difficult to find patientes
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Aprea TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Jules Bordet Institute
- Study start
- 18 Jan 2018 (actual)
- Primary completion
- 8 Aug 2018 (actual)
- Study completion
- 8 Aug 2018 (actual)
- First posted
- 5 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to make a preliminary assessment of the efficacy of a combined APR-246 and dabrafenib therapy regimen in patients with BRAFV600 mutant unresectable and/or metastatic cutaneous melanoma resistant to the dabrafenib/trametinib combination. In addition, the study aims to assess the safety profile of the combined APR-246 and dabrafenib therapy regimen, to explore potential biomarkers, and to further describe the anti-tumour activity of the combination of APR-246 and dabrafenib. The trial will enroll up to 31 evaluable patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| APR-246 + Dabrafenib | Experimental | Drug: APR-246, Drug: Dabrafenib |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients with confirmed BRAF V600 mutation-positive unresectable and/or metastatic malignant cutaneous melanoma, as determined locally by a validated test and treated with dabrafenib/trametinib first line combination therapy or second line after first line immunotherapy.
- Patients that have progressed according to RECIST 1.1 after at least 4 weeks of treatment with dabrafenib/trametinib and remained on dabrafenib full dose (150mg bid) treatment for the study.
- Measurable disease according to RECIST 1.1 criteria. For phase II only, metabolic measurable disease (according to PERCIST).
- Availability of tissue from a metastatic lesion. A new biopsy is required unless inaccessible. An archival sample is accepted in that case after discussion with the sponsor.
- ECOG Performance Status of 0 or 1.
Key exclusion criteria (13 listed)
- Presence of uveal melanoma and/or other non-cutaneous melanomas.
- Current use of a prohibited medication or need for any of these medications during treatment with study drug and within 28 days before the first administration of APR-246. I.e., no anti-cancer other than that given in this clinical trial, no immunotherapy, no hormonal cancer therapy, no radiation therapy (except palliative) and no experimental medications are permitted during the trial. All alternative therapies must first be approved by the sponsor. Supportive care therapies are allowed.
- Unresolved toxicity greater than NCI-CTCAE(v4) Grade 1 from previous anti-cancer therapy except alopecia.
- Presence of active gastrointestinal disease or other condition that will interfere significantly with the absorption, distribution, metabolism, or excretion of drugs.
- Known HIV, active hepatitis B or hepatitis C infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| APR-246Intravenous infusion | Drug | APR-246 + Dabrafenib |
| DabrafenibOral administration | Drug | APR-246 + Dabrafenib |
Primary outcome
Phase Ib: Adverse Events (AEs)
Time frame: Up to 30 days after last study treatment day, or at end of study visit due to progression, whichever occurs later (treatment cycles are stopped due to progression, toxicity or patient's decision)
Clinical and laboratory adverse events (AEs) and serious adverse events (SAEs) will be reported and graded
Phase Ib: Dose Limiting Toxicities (DLTs)
Time frame: Until end of cycle 1 (cycle length is 28 days)
Phase II: Objective response rate by RECIST1.1
Time frame: Until progression (assessed up to 12 months)
8 secondary outcomes
Clinical benefit rate
Time frame: Until progression (assessed up to 12 months)
Duration of response
Time frame: Until progression (assessed up to 12 months)
Progression free survival (PFS)
Time frame: Until progression (assessed up to 12 months)
Area under the plasma concentration versus time curve (AUC) for APR-246
Time frame: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Plasma drug concentration at a specified time t (Ct) for APR-246
Time frame: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Maximum observed plasma concentration (Cmax) of APR-246
Time frame: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Time to reach maximum plasma concentration following drug administration (tmax) for APR-246
Time frame: Until Cycle 1 Day 8 (Phase Ib) or Cycle 1 Day 1 (Phase II)
Assessment of metabolic response
Time frame: Until Cycle 2 Day 1 (cycle length is 28 days)
Results and safety
The study was stopped early (reason given: Target patient population - difficult to find patientes). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2019.
- Background: PubMed 22965953Lehmann S, Bykov VJ, Ali D, Andren O, Cherif H, Tidefelt U, Uggla B, Yachnin J, Juliusson G, Moshfegh A, Paul C, Wiman KG, Andersson PO. Targeting p53 in vivo: a first-in-human study with p53-targeting compound APR-246 in refractory hematologic malignancies and prostate cancer. J Clin Oncol. 2012 Oct 10;30(29):3633-9. doi: 10.1200/JCO.2011.40.7783. Epub 2012 Sep 10.
- Background: PubMed 27421096Deneberg S, Cherif H, Lazarevic V, Andersson PO, von Euler M, Juliusson G, Lehmann S. An open-label phase I dose-finding study of APR-246 in hematological malignancies. Blood Cancer J. 2016 Jul 15;6(7):e447. doi: 10.1038/bcj.2016.60. No abstract available.
- Aprea Therapeutics AB's website (Sponsor)Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ahmad Awada, PhD study chair
Jules Bordet Institute, Brussels, Belgium
Joseph Kerger, MD principal investigator
Jules Bordet Institute, Brussels, Belgium
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing APR-246 and Dabrafenib in people with Melanoma, run by Aprea Therapeutics at 3 sites in Belgium.
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 10 inclusion and 13 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 31 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Belgium. 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 combined 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.
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?
The study was stopped early (reason given: Target patient population - difficult to find patientes). 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?
Phase Ib: Adverse Events (AEs) (time frame: Up to 30 days after last study treatment day, or at end of study visit due to progression, whichever occurs later (treatment cycles are stopped due to progression, toxicity or patient's decision)). The study lists 3 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 NCT03391050, which also lists 2 linked publications.
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 31 Jul 2019.