Clinical Trial Details
— Status: Suspended
Administrative data
| NCT number |
NCT03208296 |
| Other study ID # |
ASN-002-002 |
| Secondary ID |
|
| Status |
Suspended |
| Phase |
Phase 1/Phase 2
|
| First received |
|
| Last updated |
|
| Start date |
December 1, 2017 |
| Est. completion date |
March 31, 2025 |
Study information
| Verified date |
January 2023 |
| Source |
Ascend Biopharmaceuticals Ltd |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial 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. evaluating the histological clearance of BCCs in patients with BCNS, and
2. assessing the clinical changes of BCCs after treatment with ASN-002, and
3. assessing the systemic effect of ASN-002 by determining response in non-injected lesions
4. assess the safety and clinical changes after a second cycle of ASN-002 injections
Description:
Methodology:
Patients with BCNS who meet the Eligibility Requirements below will be studied. After
informed consent is obtained and baseline evaluation, each lesion to be injected will receive
ASN-002 once weekly x 3 weeks. Subjects will be premedicated with acetaminophen, 1000mg, 30
minutes prior to injection and continue 650mg, four times/day for two days after injection.
Evaluation for toxicity and safety will be performed at 1, 2, 3 and 4 months after the first
injection. All target lesions will be excised at 6 months and the subject evaluated for
healing one month later.
Subjects with 5 or more lesions will be entered into Cohort A and will receive 1.0 x 1011
vp/injection into each of 4 BCCs, weekly x 3, i.e. weeks 1, 2, and 3, i.e. a total dose of
4.0 x 1011 vp on each day of injections. If one subject in this cohort experiences Grade 3 or
greater local or systemic toxicities by week 4, three additional subjects will be accrued to
this cohort. If none of the first 3 subjects or no more than one of the six subjects in the
expanded cohort experiences Grade 3 or greater local or systemic toxicities by week 4, the
total dose of 4.0 x 1011 vp/day will be accepted for further clinical assessment. All four
injected lesions and 1 or 2 non-injected lesions will be excised after 6 months.
Cohort B will be open for accrual to patients with 4 or more lesions once Cohort A is
accepted for further clinical assessment or if Cohort A has >1/6 Grade 3 AEs. Subjects in
Cohort B will receive 1.5 or 1.0 x 1011 vp/injection into each of 3 BCCs, weekly x 3, i.e.
weeks 1, 2, and 3, i.e. a total dose of 4.5 or 3.0 x 1011 vp on each day of injections as
outlined in Synopsis Table 2 below. If one subject in this cohort experiences Grade 3 or
greater local or systemic toxicities by week 4, three additional subjects will be accrued to
this cohort. If none of the first 3 subjects or no more than one of the six subjects in the
expanded Cohort B1.5 or B1.0 experiences Grade 3 or greater local or systemic toxicities by
week 4, that total dose of 4.5 or 3.0 x 1011 vp/day will be accepted for further clinical
assessment. All three injected lesions and 1-2 non-injected lesions will be excised after 6
months.
Cohort C may be designed based on the results observed in Cohorts A and B with the agreement
of the Protocol Steering Committee. If a total dose >4.5 x 1011 vp/day of injection ASN-002
on any single day or more than 3 weekly injections are to be administered, a formal amendment
to the Protocol must be approved before accrual to Cohort C is permitted.
Each cohort will be assessed for safety, dose escalation and possible cohort expansion as
described above. The week 4 assessment of toxicity and apparent efficacy of the first 3
subjects in each cohort will be used to determine the need to increase the size of that
cohort to 6 subjects. Cohorts will be assessed clinically at 1, 2, 3 and 4 months. Treated
tumors will be resected at 6 months, based on observations in the ongoing Australia trial
study that inflammation persisting at 16 weeks could not be reliably distinguished from
residual tumor. The 1st and 3rd secondary endpoints, histological clearance of injected and
non-injected lesions, will be assessed by the Pathology Department of each clinical site.
Representative slides will be submitted for possible central review to be performed at the
discretion of Ascend. Each participant will also be seen one month after tumor resection to
assess healing and post-surgical results Each subject may be evaluated on the 7 month visit
for a second cycle of 3 weekly ASN-002 injections into new or previously untreated BCC
lesions at a dose accepted for further clinical assessment. Subjects who receive a second
cycle will be followed for 6 months as before, but lesion excision will be at the discretion
of the investigator.
In the event study subjects in any Cohort do not proceed to histological confirmation of
response per protocol, they may be replaced as long as the reason for not proceeding to
histological confirmation of response per protocol was not investigational product related
adverse events and so long as the Protocol Steering Committee concurs with the Sponsor that
no limiting safety issues have been identified.
Depending on results, and at the discretion of the Sponsor and the Protocol Steering
Committee, one Cohort may be expanded to a total of 24 subjects so long as no more than one
of six subjects already included at that dose and schedule of ASN-002 experienced Grade 3 or
greater local or systemic toxicities. The selection of the cohort to be expanded will be
based on favorable response data from the subjects already included at that dose and schedule
of ASN-002,
