Acute Myeloid LeukemiaPhase 1NCT03333005
Safety and Pharmacokinetics of Intravenous and Oral APX001 in Patients With Acute Myeloid Leukemia (AML) and Neutropenia
A Phase 1 study of Acute Myeloid Leukemia, run by Basilea Pharmaceutica at 3 sites in Germany.
Trial summary
An open-label, multi-center, Phase Ib study to determine the safety and pharmacokinetics of intravenous and oral APX001 in patients undergoing chemotherapy for Acute Myeloid Leukemia with neutropenia. A total of 20 patients will be enrolled in this study. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesGermany
- Study dates
- 27 Nov 2017to 26 Oct 2018
- Phase
- Phase 1
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provision of written consent
- Ages 18-75 inclusive, male or female
- Diagnosis of Acute Myeloid Leukemia
- Patients entering first induction treatment chemotherapy
- Expected to be neutropenic (<500 ANC/ul) for >/= 10 days
Who cannot join
- Patients who received systemic antifungal therapy for proven or probable fungal infections within the last 12 months
- Current fever (> 38 degrees Celsius)
- Concomitant use of rifampin, rifabutin, ergot alkaloids, terfenadine, astemizole, cisapride, pimozide, quinidine, long acting barbiturates, neostigmine and carbamazepine
Trial locations and contacts
3 sites in 1 country.
Germany3 sites
University of Cologne, Center for Integrated Oncology (CIO)
Cologne, 50931
Johannes Gutenberg, University of Mainz
Mainz, 55131
University of Munich, Grosshadern Campus
Munich, 81377
About this study
Official title: An Open Label, Multi-Center Study to Determine the Safety and Pharmacokinetics of Intravenous and Oral APX001 in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia With Neutropenia
An open-label, multi-center, Phase Ib study to determine the safety and pharmacokinetics of intravenous and oral APX001 in patients undergoing chemotherapy for Acute Myeloid Leukemia with neutropenia.
A total of 20 patients will be enrolled in this study. 10 patients in Cohort I, intravenous drug dosing and 10 patients will be enrolled in Cohort II, oral drug dosing.
All patients will receive chemotherapy for their AML according to local clinical standard of care as well as antifungal prophylaxis.
APX001 will be administered for 14 consecutive days, beginning on Study Day 3 after onset of chemotherapy
- Conditions
- Acute Myeloid Leukemia
- Being tested
- APX001 with Standard of Care anti-fungal agent
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APX001 with Standard of Care anti-fungal agentDrug
safety assessment
| Study group | Role | What they receive |
|---|---|---|
| APX001 with Standard of Care Anti-fungal agent | Experimental | Not described Receives: Drug: APX001 with Standard of Care anti-fungal agent |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with treatment-related adverse events as assessed and reported by CTCAE v4.0
Measured: One to forty-four days
Secondary outcomes (2)
Area under the plasma concentration versus time curve (AUC)
Measured: One to forty-four days
Peak Plasma Concentration (Cmax)
Measured: One to forty-four days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2017
- Study start27 Nov 2017
- Primary completion26 Oct 2018
- Study completion26 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies 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
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- The first 10 patients enrolled will be administered IV APX001. The second 10 patients enrolled will be administered oral APX001
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Basilea Pharmaceutica
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Clinical Trials Centre Cologne
- Responsible party
- Sponsor
- Investigators
- Marc Engelhardt, Study director, Basilea Pharmaceutica International Ltd, Allschwil
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03333005
- Sponsor's protocol ID
- APX001-103
- Status last verified
- May 2024
- First submitted
- 23 Oct 2017
- Last updated
- 17 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Multi-Center Study to Determine the Safety and Pharmacokinetics of Intravenous and Oral APX001 in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia With Neutropenia
- ClinicalTrials.gov ID
- NCT03333005
- Acronym / sponsor ID
- APX001-103
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Basilea PharmaceuticaIndustry (pharma, biotech, devices)
- Collaborators
- The Clinical Trials Centre Cologne
- Study start
- 27 Nov 2017 (actual)
- Primary completion
- 26 Oct 2018 (actual)
- Study completion
- 26 Oct 2018 (actual)
- First posted
- 6 Nov 2017 (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.
An open-label, multi-center, Phase Ib study to determine the safety and pharmacokinetics of intravenous and oral APX001 in patients undergoing chemotherapy for Acute Myeloid Leukemia with neutropenia.
A total of 20 patients will be enrolled in this study. 10 patients in Cohort I, intravenous drug dosing and 10 patients will be enrolled in Cohort II, oral drug dosing.
All patients will receive chemotherapy for their AML according to local clinical standard of care as well as antifungal prophylaxis.
APX001 will be administered for 14 consecutive days, beginning on Study Day 3 after onset of chemotherapy
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The first 10 patients enrolled will be administered IV APX001. The second 10 patients enrolled will be administered oral APX001
| Arm | Type | Receives |
|---|---|---|
| APX001 with Standard of Care Anti-fungal agent | Experimental | Drug: APX001 with Standard of Care anti-fungal agent |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Provision of written consent
- Ages 18-75 inclusive, male or female
- Diagnosis of Acute Myeloid Leukemia
- Patients entering first induction treatment chemotherapy
- Expected to be neutropenic (<500 ANC/ul) for >/= 10 days
Key exclusion criteria (3 listed)
- Patients who received systemic antifungal therapy for proven or probable fungal infections within the last 12 months
- Current fever (> 38 degrees Celsius)
- Concomitant use of rifampin, rifabutin, ergot alkaloids, terfenadine, astemizole, cisapride, pimozide, quinidine, long acting barbiturates, neostigmine and carbamazepine
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 |
|---|---|---|
| APX001 with Standard of Care anti-fungal agentsafety assessment | Drug | APX001 with Standard of Care Anti-fungal agent |
Primary outcome
Number of patients with treatment-related adverse events as assessed and reported by CTCAE v4.0
Time frame: One to forty-four days
2 secondary outcomes
Area under the plasma concentration versus time curve (AUC)
Time frame: One to forty-four days
Peak Plasma Concentration (Cmax)
Time frame: One to forty-four days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2024.
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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.
Marc Engelhardt study director
Basilea Pharmaceutica International Ltd, Allschwil
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study of Acute Myeloid Leukemia, run by Basilea Pharmaceutica at 3 sites in Germany.
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 5 inclusion and 3 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: finished, as of the sponsor's last update on 17 May 2024. 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 Germany. 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 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.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of patients with treatment-related adverse events as assessed and reported by CTCAE v4.0 (time frame: One to forty-four days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 May 2024.