A Phase I Trial of AZD3965 in Patients With Advanced Cancer

NCT ID: NCT01791595

Last Updated: 2022-04-11

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-04-23

Study Completion Date

2020-11-17

Brief Summary

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The main aims of this clinical study are to find out the maximum dose that can be given safely to patients, the potential side effects of the drug and how they can be managed and what happens to AZD3965 inside the body.

AZD3965 is a type of drug called a monocarboxylate transporter 1 inhibitor which is being used to stop the growth of cancer cells and kill cancer cells by blocking the action of one of the proteins involved in moving chemical compounds in and out of the cells of the body. This will be the first time that this type of drug has been given to patients.

The drug is a capsule and is taken daily. The study is in two parts. In Part 1 of the study, small groups of patients are treated at increasing doses to find the highest safe dose and best dose to give to patients in Part 2 of the study. It is planned that 40 patients will be entered into Part 1 of the trial.

In Part 2, the dose found to be safe in Part 1 is given to patients with diffuse large B-cell lymphoma (DLBCL) and Burkitt's lymphoma (BL). It is planned that 20 patients will be entered into Part 2 of the trial.

Patients will need to visit the hospital weekly for two months and then every fortnight. Patients will have regular blood and urine tests, scans, heart traces and eye tests amongst other clinical tests. Research blood samples will also be taken to look at what happens to the drug inside the body. Treatment is planned to be given for up to 6 months, but patients benefiting from treatment will be able to keep having it for as long as they continue to benefit. It is important to explain that this is the first study of this drug and patients will have advanced cancer so it is unlikely that patients will benefit directly from taking part but the study may help improve future treatment of cancer.

Detailed Description

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Part 1 follows a rolling six dose escalation schedule of AZD3965 given once daily (OD) or twice daily (BD) until the maximum tolerated dose (MTD) is defined.

The recommended Phase II dose (RP2D) is based on the safety and pharmacokinetic (PK) results from Part 1.

All patients in Part 2 are treated at this RP2D to further explore the tolerability of this dose and schedule and to explore proof of principle of MCT1 inhibition in tumour types that were shown to express MCT1 and in which AZD3965 showed some effect pre-clinically (DLBCL and BL).

Conditions

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Adult Solid Tumor Diffuse Large B Cell Lymphoma Burkitt Lymphoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AZD3965 Cohort 1 (5 mg OD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 5 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 5 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

AZD3965 Cohort 2 (10 mg OD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 10 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 10 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

AZD3965 Cohort 3 (20 mg OD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 20 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 20 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

AZD3965 Cohort 4 (30 mg OD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 30 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 30 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

AZD3965 Cohort 5 (15 mg BD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 30 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 15 mg AZD3965 BD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

AZD3965 Cohort 6 (10 mg BD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 20 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 10 mg AZD3965 BD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

AZD3965 Expansion Cohort (10 mg BD)

Group Type EXPERIMENTAL

AZD3965

Intervention Type DRUG

Day -7: single dose of 20 mg AZD3965 orally prior to start of continuous treatment (first 3 trial participants in the Expansion Cohort only; subsequent patients started treatment at Cycle 1, Day 1).

Cycle 1, Day 1: commenced dosing of 10 mg AZD3965 BD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Interventions

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AZD3965

Day -7: single dose of 5 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 5 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

AZD3965

Day -7: single dose of 10 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 10 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

AZD3965

Day -7: single dose of 20 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 20 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

AZD3965

Day -7: single dose of 30 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 30 mg AZD3965 OD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

AZD3965

Day -7: single dose of 30 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 15 mg AZD3965 BD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

AZD3965

Day -7: single dose of 20 mg AZD3965 orally prior to start of continuous treatment.

Cycle 1, Day 1: commenced dosing of 10 mg AZD3965 BD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

AZD3965

Day -7: single dose of 20 mg AZD3965 orally prior to start of continuous treatment (first 3 trial participants in the Expansion Cohort only; subsequent patients started treatment at Cycle 1, Day 1).

Cycle 1, Day 1: commenced dosing of 10 mg AZD3965 BD orally for up to 6 28-day cycles.

Trial participants benefitting from treatment could continue beyond 6 cycles for as long as they continued to benefit on agreement between the Investigator and the Sponsor.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Part 1:

* Histologically or cytologically proven advanced solid tumour or lymphoma, refractory to conventional treatment or for which no conventional therapy exists.
* Available archived tumour samples.

Part 2:
* Histologically proven DLBCL or BL, which is relapsed or refractory to conventional treatment or for which no conventional therapy exists or has been refused by the patient.
* Confirmed available tumour samples which can be obtained and used for the study to confirm MCT1 and MCT4 expression as demonstrated by immunohistochemistry.
* Measurable disease according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or International Working Group (IWG) criteria for Lymphoma.
2. Life expectancy of at least 12 weeks.
3. World Health Organization (WHO) performance status of 0 or 1.
4. Haematological and biochemical indices within the ranges shown below.

Laboratory Test Value required:
* Haemoglobin (Hb) ≥9.0 g/dL (90 g/L) or ≥10.0 g/dL (100 g/L) if transfusion within last 4 weeks.
* Absolute neutrophil count (ANC) Part 1: ≥1.5 x 10\^9/L; Part 2: ≥1.0 x 10\^9/L.
* Platelet count Part 1: ≥100 x 10\^9/L; Part 2: ≥50 x 10\^9/L.
* Serum bilirubin ≤1.5 x upper limit of normal (ULN).
* Alanine aminotransferase (ALT), aspartate aminotransferase (AST) and alkaline phosphatase (ALP) ≤2.5 x ULN or ≤5 x ULN in presence of liver metastases (ALP ≤5 x ULN in presence of bone metastases).
* Glomerular filtration rate (GFR) either: Calculated creatinine clearance or: Isotope clearance measurement (uncorrected) ≥50 mL/min.
* Prothrombin time \<1.5 x ULN.
* Glucose (fasting) \<7.8 mmol/L;
* Lactate between 0.5 and 2.5 mmol/L inclusive and bicarbonate between 22 mmol/L and 1.5 x ULN inclusive.
5. Left ventricular ejection fraction (LVEF) \>50%.
6. 18 years or over.
7. Written (signed and dated) informed consent and be capable of co-operating with treatment and follow-up.

Exclusion Criteria

1. Radiotherapy (except for palliative reasons), endocrine therapy, immunotherapy or chemotherapy during the previous four weeks (six weeks for nitrosoureas, Mitomycin-C and 4 weeks for investigational medicinal products) before treatment.
2. Ongoing toxic manifestations of previous treatments greater than National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade 1. Exceptions to this are alopecia or certain Grade 1 toxicities, which in the opinion of the Investigator and the Cancer Research UK Centre for Drug Development should not exclude the patient.
3. Symptomatic brain or leptomeningeal metastases.
4. Patients with known retinal disease or macular degeneration affecting visual acuity as assessed by ophthalmologic tests.
5. Female patients who are able to become pregnant (or are already pregnant or lactating). However, those patients who have a negative serum or urine pregnancy test before enrolment and agree to use two forms of contraception (one highly effective form plus a barrier method) \[oral, injected or implanted hormonal contraception and condom; intra-uterine device and condom; diaphragm with spermicidal gel and condom\] or agree to sexual abstinence, effective from the first administration of AZD3965, throughout the trial and for six months afterwards are considered eligible.
6. Male patients with partners of child-bearing potential (unless they agree to take measures not to father children by using a barrier method of contraception \[condom plus spermicide\] or to sexual abstinence effective from the first administration of AZD3965, throughout the trial and for six months afterwards. Men with partners of child-bearing potential must also be willing to ensure that their partner uses an effective method of contraception for the same duration for example, hormonal contraception, intrauterine device, diaphragm with spermicidal gel or sexual abstinence). Men with pregnant or lactating partners must be advised to use barrier method contraception (for example, condom plus spermicidal gel) to prevent exposure of the foetus or neonate.
7. Any major surgery in the preceding eight weeks prior to the start of treatment or major thoracic or abdominal surgery from which the patient has not yet recovered.
8. Patients who are unable to swallow oral medication.
9. Alterations to corticosteroid dose within 2 weeks prior to first dose of AZD3965.
10. Gastrointestinal disorders likely to interfere with absorption of the study drug (e.g. partial bowel obstruction or malabsorption).
11. At high medical risk because of non-malignant systemic disease including active uncontrolled infection.
12. Known to be serologically positive for hepatitis B, hepatitis C or human immunodeficiency virus (HIV). (N.B. Mandatory testing not required).
13. History of serious allergy or auto-immune disease.
14. Diabetes mellitus (patients with diet controlled diabetes may be included with fasting glucose \<7.8 mmol/l and normal haemoglobin A1c \[HbA1c\]).
15. Cardiac conditions as follows:

* Clinically significant cardiovascular event within 6 months prior to study entry to include:

1. acute coronary syndrome (myocardial infarction or unstable angina),
2. congestive heart failure requiring therapy.
* Severe valvular heart disease (as defined by British Society of Echocardiography).
* Presence of an atrial or ventricular arrhythmia, other than atrial fibrillation with well controlled ventricular rate, for which treatment is indicated (anti-arrhythmic drugs or implantable cardioverter defibrillator).
* Second degree Mobitz type 1 (Wenckebach) heart block with symptoms, or second degree Mobitz type 2 or third degree heart block with or without symptoms unless functioning pacing system.
* QTc \>450 msec in adult male and \>460 msec in adult females (QTc to be verified manually \[Fridericia's Correction\]).
* History of congenital long QT syndrome.
* History of Torsade de Pointes (or any concurrent medication with a known risk of inducing QT prolongation).
* Uncontrolled hypertension (blood pressure ≥160/100 mmHg despite medical therapy).
16. Extensive radiotherapy to greater than 25% of bone marrow within 8 weeks. Prior autologous bone transplant will not exclude a patient.
17. Is a participant, or plans to participate in another interventional clinical trial, whilst taking part in this Phase I study of AZD3965. Participation in an observational or interventional clinical trial that does not involve administration of an IMP would be acceptable.
18. Any other condition which in the Investigator's opinion would not make the patient a good candidate for the clinical trial.
19. For Part 2 only: Current malignancies of other types, with the exception of adequately treated cone-biopsied in situ carcinoma of the cervix uteri; basal or squamous cell carcinoma of the skin; and patients with low risk prostate cancer on surveillance (with a Gleason score of ≤6 and a Prostate Specific Antigen of ≤10).
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AstraZeneca

INDUSTRY

Sponsor Role collaborator

Cancer Research UK

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ruth Plummer, Prof

Role: PRINCIPAL_INVESTIGATOR

Freeman Hospital, Newcastle

Locations

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Royal Marsden Hospital

Sutton, London, United Kingdom

Site Status

The Beatson West of Scotland, Glasgow

Glasgow, , United Kingdom

Site Status

Leicester Royal Infirmary

Leicester, , United Kingdom

Site Status

University College London Hospitals

London, , United Kingdom

Site Status

The Christie

Manchester, , United Kingdom

Site Status

Freeman Hospital

Newcastle upon Tyne, , United Kingdom

Site Status

Derriford Hospital

Plymouth, , United Kingdom

Site Status

Countries

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United Kingdom

References

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Noble RA, Bell N, Blair H, Sikka A, Thomas H, Phillips N, Nakjang S, Miwa S, Crossland R, Rand V, Televantou D, Long A, Keun HC, Bacon CM, Bomken S, Critchlow SE, Wedge SR. Inhibition of monocarboxyate transporter 1 by AZD3965 as a novel therapeutic approach for diffuse large B-cell lymphoma and Burkitt lymphoma. Haematologica. 2017 Jul;102(7):1247-1257. doi: 10.3324/haematol.2016.163030. Epub 2017 Apr 6.

Reference Type BACKGROUND
PMID: 28385782 (View on PubMed)

McNeillis R, Greystoke A, Walton J, Bacon C, Keun H, Siskos A, Petrides G, Leech N, Jenkinson F, Bowron A, Halford S, Plummer R. A case of malignant hyperlactaemic acidosis appearing upon treatment with the mono-carboxylase transporter 1 inhibitor AZD3965. Br J Cancer. 2020 Apr;122(8):1141-1145. doi: 10.1038/s41416-020-0727-8. Epub 2020 Feb 20.

Reference Type DERIVED
PMID: 32076124 (View on PubMed)

Kershaw S, Cummings J, Morris K, Tugwood J, Dive C. Optimisation of immunofluorescence methods to determine MCT1 and MCT4 expression in circulating tumour cells. BMC Cancer. 2015 May 10;15:387. doi: 10.1186/s12885-015-1382-y.

Reference Type DERIVED
PMID: 25957999 (View on PubMed)

Lamb R, Harrison H, Hulit J, Smith DL, Lisanti MP, Sotgia F. Mitochondria as new therapeutic targets for eradicating cancer stem cells: Quantitative proteomics and functional validation via MCT1/2 inhibition. Oncotarget. 2014 Nov 30;5(22):11029-37. doi: 10.18632/oncotarget.2789.

Reference Type DERIVED
PMID: 25415228 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Related Links

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Other Identifiers

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2010-024463-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CRUKD/12/004

Identifier Type: -

Identifier Source: org_study_id

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