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Basal Cell CarcinomaPhase 2NCT04416516

Safety and Efficacy of ASN-002 Combined With a Hedgehog Pathway Inhibitor

A Phase 2 study testing ASN-002 in people with Basal Cell Carcinoma and Basal Cell Nevus Syndrome, run by Ascend Biopharmaceuticals Ltd at 7 sites in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The primary objectives are to: 1. Evaluate the safety and tolerability of intralesional ASN-002 when administered in combination with oral vismodegib in patients with Basal Cell Carcinomas (BCC)s; 2. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
ASN-002
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
7 sitesAustralia
Study dates
16 Jul 2020to 14 Feb 2024
Phase
Phase 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

  • Histologically confirmed previously untreated, BCC (nodular and superficial), 5-20 mm in maximum diameter, per the selection criteria for study BCC tumours. A punch biopsy (refer to study procedure manual for biopsy size selection) from the thickest part of all the target tumours is required for histological confirmation of BCC and to exclude BCC non-eligible subtypes.
  • Note: If a patient has mix of nodular and superficial BCC tumours, at least one target tumour should be a nodular BCC.
  • Removal of < 25% of the area of each biopsied tumour by initial biopsy performed 1-12 weeks before Day 1. If the initial biopsy was performed >8 weeks prior to screening, the investigator may re-biopsy the tumour, provided not > 25% of the area of the original tumour is removed. Non-study tumours may be resected or treated at the discretion of the Investigator prior to study entry or if they develop during the study.
  • Hedgehog pathway inhibitor treatment naïve.
  • Acceptable general health as determined by the investigator, i.e. no serious or active medical or psychiatric illness or recreational or therapeutic drug or alcohol use that, in the opinion of the Investigator, would interfere with treatment, assessment or compliance with the protocol, ability to provide informed consent, or patient safety.
  • 18 years of age or older.
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  • Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.
  • Screening laboratory values as follows:
  • Neutrophil count > 1500/mm3
  • Haemoglobin > 9 g/dL
  • Platelet count >100,000/mm3
  • Prothrombin INR < 1.5
  • Total bilirubin < 1.5 X upper limit of normal (ULN), except in the case of known Gilbert's syndrome
  • Aspartate transaminase (AST), alanine transaminase (ALT) or alkaline phosphatase (ALP) < 2X ULN
  • Creatinine < 1.5 X upper limit of normal (ULN)
  • Female patients who are documented infertile, postmenopausal for at least 1 year, surgically sterile or using acceptable and highly effective birth control for the duration of the study and for at least 3 months after last administration of the study treatments.
  • Male patients with female partners of child bearing potential, agreement to use two adequate contraception methods while being on vismodegib and for 3 months of completion. agreement not to donate semen for 3 months after completion of vismodegib.
  • Written informed consent prior to initiation of study-specified procedures.
  • Able and willing to comply with all study requirements, including surgical removal of tumour/tumour site at completion of study.
  • Baseline tissue sample adequate for determination of histological or other biomarkers.

Who cannot join

  • Known or suspected metastatic disease or other active, invasive malignancy.
  • Female patients of childbearing potential who are lactating or pregnant (negative serum pregnancy test needed prior to dosing).
  • Clinically active or uncontrolled skin disease or tattoos that would interfere with evaluation of the area surrounding the target tumour (e.g. eczema, unstable psoriasis, xeroderma pigmentosa).
  • Known history of sensitivity to any of the ingredients in ASN-002 and any Hh pathway inhibitors.
  • Immunocompromised (e.g. known Hepatitis B or C infection, HIV infection) or receiving or expected to receive an immunomodulating agent (including immunosuppressive agents, cytotoxic drugs, biological agents, immunoglobulins, interferon or other immune or cytokine-based therapies. Use of inhaled or oral corticosteroids at doses higher than physiological replacement doses is an exclusion criterion).
  • Has received or is expected to receive treatment with psoralen plus UVA or UVB therapy within 6 months of the Screening visit.
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  • Any prior systemic anti-tumour therapy or local treatment for target tumours prior to first dose.
  • History of immunological disorder, severe allergic reaction, moderate or severe asthma or known history of anaphylaxis or any other serious adverse reactions to any medication.
  • Any experimental or investigational agents within one month of first ASN-002 injection.
  • Any prior exposure to TG1041, TG1042 (ASN-002), any other adenoviral-based experimental agent, or any form of gene therapy within 6 months of first dose of vismodegib in the study.
  • Any prior exposure to vismodegib or any other Hh inhibitor within 6 months of first dose in the study.
  • Current therapy with any medications recognized to cause rhabdomyolysis or a prior history of rhabdomyolysis.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

Australia7 sites

  • Central Brisbane Dermatology

    Brisbane, Queensland, 4000

  • Princess Alexandra Hospital

    Brisbane, Queensland, 4102

  • Veracity Clinical Research

    Brisbane, Queensland, 4102

  • Sinclair Dermatology

    Melbourne, Victoria, 3000

  • Royal Melbourne Hospital

    Melbourne, Victoria, 3050

  • Burswood Dermatology

    Perth, Western Australia, 6100

  • Sunshine Coast University Hospital

    Sunshine Coast, Queensland

Other trials in Australia

About this study

Official title: A Phase 2A Study to Assess the Safety and Efficacy of ASN-002 Combined With a Hedgehog Pathway Inhibitor in the Treatment of Multiple Low Risk Basal Cell Carcinomas in Sporadic or Basal Cell Nevus Syndrome Patients

The primary objectives are to:

  1. Evaluate the safety and tolerability of intralesional ASN-002 when administered in combination with oral vismodegib in patients with Basal Cell Carcinomas (BCC)s;
  2. Evaluate the efficacy of intralesional ASN-002 in target tumours when administered in combination with oral vismodegib in patients with BCCs.

The secondary objective is to:

  1. Evaluate the efficacy of intralesional ASN-002 in non-target tumours when administered in combination with oral vismodegib in patients with BCCs.

The exploratory objective is to:

  1. Evaluate immunological biomarkers during the course of treatment.
Read the detailed description

Methodology:

This study will evaluate ASN-002 (in the dose range 0.5 to 1.5x10(11) vp/mL) with the Hh inhibitor vismodegib (Erivedge®). The study will initially evaluate two Arms receiving 1.0 x 10(11) vp/injection, and following a safety review, may implement further arms in an adaptive study design.

Following screening and baseline biopsies for target and non-target tumours, eligible subjects will be enrolled in the study.

Cycle 1: Treatment with vismodegib (daily dose of 150 mg) for 4 weeks and ASN-002 for 3 weeks (i.e., three ASN-002 injections in total):

  • Day 1 to Day 14 - vismodegib alone
  • Day 15 - vismodegib and ASN-002
  • Day 16 to Day 21 - vismodegib alone
  • Day 22 - vismodegib and ASN-002
  • Day 23 to Day 28 - vismodegib alone
  • Day 29 - ASN-002 alone

Clinical response will be assessed at Week 17, following which, the investigator may where clinically indicated, initiate Cycle 2.

Cycle 2: Treatment with vismodegib (daily dose of 150 mg) for 4 weeks, and one further injection with ASN-002:

  • Day 1 to Day 7 - vismodegib alone
  • Day 8 - vismodegib and ASN-002
  • Day 9 to Day 28 - vismodegib alone

Surgical excision for all patients will occur between Week 25 and Week 33 at the investigators discretion, and dependent on when patient completed study treatment (1 or 2 treatment cycles). Up to 10 BCCs to be excised including 3 target tumours. Excisions can be conducted over 2 visits as per Investigator's discretion.

The Investigator may enrol eligible patients parallel into either Arm 1 or Arm 2, based on the number of tumours present. Up to 10 study BCC tumours (up to 3 target and up to 7 non-target) will be selected per patient.

Following review of at least Week 5 data for N=6 patients in Arm 1 and Arm 2, further Arms with varying doses of ASN-002 (in the dose range 0.5x10(11) vp or 1.5x10(11) vp) may be explored at the discretion of the Safety Review Committee (SRC). Vismodegib or ASN-002 may be evaluated as monotherapies to provide control groups to allow comparison of treatment Arms.

Safety and clinical assessments will be performed at Weeks 1, 3, 4, 5, 7, 17, 25 to 33.

Histological response will be evaluated in all study tumours via excision between Week 25 and Week 33 (as per investigator's discretion).

Six patients will be recruited to each Arm of the study, each Arm may be expanded to 12 patients at the discretion of the SRC.

Being tested
ASN-002
Keywords
high frequency basal cell carcinoma
Medical subject headings
Carcinoma, Basal Cell, Basal Cell Nevus Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ASN-002Biological

    ASN-002 has been designed for clinical applications, especially for intratumoral administration in the treatment of various cancers. This rAd vector delivers the gene of interest, in the case of ASN-002 the human IFNγ gene, into target cells. The rAd vector in ASN-002 is replication deficient and although it infects cells, it is not able to replicate in the tumor or in normal human cells. The infected cells are able to transcribe and translate the IFNγ DNA leading to a sustained local concentration of IFNγ in the tumor mass that is designed to avoid high levels of systemic IFNγ that may be lead to unacceptable toxicity.

Study groups in NCT04416516
Study groupRoleWhat they receive
Arm 1, Patients with 1 TumourExperimentalParticipants with 1 Target Tumour will receive 3 x ASN-002 1.0x10(11) Injections + VISMODEGIB (150 mg) daily for 4 weeks.
Receives: Biological: ASN-002
Arm 2, Patients with 3 or more TumoursExperimentalParticipants with 3 or more Target Tumours will receive 3 x ASN-002 1.0x10(11) Injections (per tumour) + VISMODEGIB (150 mg) daily for 4 weeks.
Receives: Biological: ASN-002
Arm 6 Patients with 3 or more TumoursExperimentalParticipants with 3 or more Target Tumours will receive 3 x ASN-002 1.5x10(11) Injections (per tumour) + VISMODEGIB (150 mg) daily for 4 weeks
Receives: Biological: ASN-002

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of ASN-002 related Adverse Events in patients with previously untreated nBCC

    Incidence of Adverse Adverse events will be monitored

    Measured: Participants will be followed up to 6 months

  2. Microscopic clearance of the injected Target basal cell carcinoma.

    Histological clearance (HC) will be defined as the absence of detectable evidence of BCC tumor cell nests in serial histological samples as determined by central pathology review.

    Measured: Microscopic examinations of sample collected at weeks 25-33 after the first dose.

Secondary outcomes (1)

  1. Microscopic clearance of the injected Non-Target basal cell carcinoma.

    Histological clearance (HC) will be defined as the absence of detectable evidence of BCC tumor cell nests in serial histological samples as determined by central pathology review.

    Measured: Microscopic examinations of sample collected at weeks 25-33 after the first dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Jun 2020
  2. Study start16 Jul 2020
  3. Primary completion14 Feb 2024
  4. Study completion14 Feb 2024

Resources

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04416516
Sponsor's protocol ID
ASN-002-003
Status last verified
Apr 2024
First submitted
21 May 2020
Last updated
10 Apr 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ASN-002 in people with Basal Cell Carcinoma and Basal Cell Nevus Syndrome, run by Ascend Biopharmaceuticals Ltd at 7 sites in Australia.

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 20 inclusion and 12 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 10 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in Australia. 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 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 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?

Incidence of ASN-002 related Adverse Events in patients with previously untreated nBCC (time frame: Participants will be followed up to 6 months). The study lists 2 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 NCT04416516.

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 10 Apr 2024.

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