Study of ASN-002 to Treat Basal Cell Carcinomas (BCCs) in Individuals With Basal Cell Nevus Syndrome (BCNS)

NCT03208296 | Suspended Phase 1 FDA Drug

• 1 other identifier: ASN-002-002
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective is to confirm the safety of treating multiple BCCs once weekly x 3 weeks in individuals with Basal Cell Nevus Syndrome (BCNS). The secondary objectives of the study are to obtain preliminary data on the effectiveness of ASN-002 in the treatment of BCCs in individuals with Basal Cell Nevus Syndrome (BCNS) by

1. 1.evaluating the histological clearance of BCCs in patients with BCNS, and
2. 2.assessing the clinical changes of BCCs after treatment with ASN-002, and
3. 3.assessing the systemic effect of ASN-002 by determining response in non-injected lesions
4. 4.assess the safety and clinical changes after a second cycle of ASN-002 injections

Conditions

Basal Cell Carcinoma in Basal Cell Nevus Syndrome

Keywords

Basal Cell Nevus Syndrome; Basal Cell Carcinoma

Outcome Measures

Primary Outcomes (1)

• Safety of ASN-002 will be studied in terms of AEs reported in individuals with Basal Cell Nevus Syndrome (BCNS) receiving ASN-002 using CTCAE 4.03.

  Clinical safety will assessed in terms of changes in vital signs, AEs, serious AEs (SAEs), laboratory abnormalities and withdrawals from study. Local skin and injection site reactions will be assessed in detail scoring erythema, ulceration, pain and overall severity as none, mild, moderate or severe using protocol-specific modifications of the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) v4.03.

  The assessment will be conducted at every visit after first dose, week1, week2, week 3, Months1, 2, 3, 4 and at Month 6.

Secondary Outcomes (6)

• Treatment efficacy in term of percentage of histological cure of BCC lesions at 6 months

  6 months from baseline

• Clinical changes in BCCs after treatment with ASN-002 in- terms of change in lesion size (mm)

  6 months from baseline.

• The systemic effect of ASN-002 will be assessed for non-injected lesions by studying the change in lesion size from baseline to end of the study.

  6 months from baseline

• The safety in terms of the AEs reported (using CTCAE 4.03) after 6 months from first dose in retreatment cycle

  6 months

• The systemic effect of ASN-002 will be assessed for non-injected lesions by studying the rate of histological clearance after 6 months of first dose.

  6 months from baseline

Study Arms (3)

Cohort A

EXPERIMENTAL

Subjects with 4 or more lesions will receive 1.0 x 10\^11 vp/injection of ASN-002 into each of 4 BCCs, weekly x 3, i.e. weeks 1, 2, and 3

Biological: ASN-002

Cohort B 1.5

EXPERIMENTAL

Subjects with 4 or more lesions will receive 1.5 x 10\^11 vp/injection of ASN-002 into each of 3 BCCs, weekly x 3, i.e. weeks 1, 2, and 3

Biological: ASN-002

Cohort B 1.0

EXPERIMENTAL

Subjects with 4 or more lesions will receive 1.0 x 10\^11 vp/injection of ASN-002 into each of 3 BCCs, weekly x 3, i.e. weeks 1, 2, and 3

Biological: ASN-002

Interventions

ASN-002 BIOLOGICAL

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.

Cohort A; Cohort B 1.0; Cohort B 1.5

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must satisfy established criteria for the diagnosis of BCNS (Section 1.1.2, Table 1).
• Must have at least 4 target lesions, clinically consistent with BCC.
• Up to 6 target lesions, including each of the 3 or 4 to-be-injected lesions and 1 or 2 non-injected lesions must be biopsied.
• At least 3 target lesions, 2 to be injected and one to be non-injected, must be biopsy proven BCC per criteria in Synopsis Table 3: Modified Criteria for Low Risk BCC in BCNS Patients
• Removal of \< 25% of the area of the tumor by initial biopsy performed within 12 weeks before screening visit. A 2mm punch biopsy is recommended for histological confirmation of BCC.
• Screening laboratory values as follows:
• Neutrophil count \> 1500/mm3
• Hemoglobin \> 10 g/dL
• Platelet count \> 100,000/mm3
• 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) \< 1.5X ULN
• Creatinine \< 1.5 X upper limit of normal (ULN)
• years of age or older at Screening visit.
• Infertile, postmenopausal, surgically sterile or using acceptable and highly effective birth control methods for the duration of the study and for 3 months after last administration of ASN-002.
• Written informed consent prior to initiation of study-specified procedures.

You may not qualify if:

• Target tumor biopsy shows evidence of:
• micronodular features,
• squamous metaplasia,
• sclerosing BCC,
• morpheic BCC, or
• peri-neural involvement.
• cystic BCC
• Eastern Cooperative Oncology Group (ECOG) performance status \> 2.
• Known or suspected metastatic disease.
• Female participants must be non-lactating and non-pregnant.
• Clinically active or uncontrolled skin disease that would interfere with evaluation of the area surrounding the target tumor (e.g. eczema, unstable psoriasis, xeroderma pigmentosa).
• Known history of sensitivity to any of the ingredients in ASN-002.
• Treatment with psoralen plus UVA or UVB therapy within 3 months of screening and agrees not to receive such treatment until excision site is confirmed to be well healed at the post-surgery study visits
• Prior systemic or local treatment for target tumors.
• 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.

Sponsors & Collaborators

• Ascend Biopharmaceuticals Ltd: lead

Study Sites (1)

OHSU

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Basal Cell Nevus Syndrome; Carcinoma, Basal Cell

Interventions

gusacitinib

Condition Hierarchy (Ancestors)

Odontogenic Cysts; Jaw Cysts; Bone Cysts; Cysts; Neoplasms; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Basal Cell; Neoplastic Syndromes, Hereditary; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Jaw Diseases; Stomatognathic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Clement Leong

  Ascend Biopharmaceuticals Ltd

  STUDY CHAIR

• Anna Bar

  Oregon Health and Science University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a dose escalation study to determine the safety and efficacy of ASN-002 in treatment of BCCs in BCNS patients.

Study Record Dates

• First Submitted: June 29, 2017
• First Posted: July 5, 2017
• Study Start: December 1, 2017
• Primary Completion: December 31, 2024
• Study Completion: March 31, 2025
• Last Updated: February 6, 2023

Record last verified: 2023-01

Locations

US (1)