Trial Outcomes & Findings for Treatment of Anal High-grade Squamous Intraepithelial Lesions (HSIL) Through Use of a Chinese Herbal Topical Cream (NCT NCT00622440)

NCT ID: NCT00622440

Last Updated: 2020-04-24

Results Overview

Response assessed 12 weeks after treatment. Late Clinical Response (LCR): HSIL present at week 48 but none at week 60, with two independent reviews in agreement that HSIL absent at week 60. Complete response (CR): No HSIL on histology or cytology at weeks 48 or 60 (caveat: if HSIL at week 60, blinded chart notes and photographs were reviewed by two clinicians, and decision was reached by agreement or consensus whether HSIL had been missed at week 48. Cases that reviewers independently agreed had not been missed at week 48 were considered true recurrences) Partial Clinical Response (PCR): HSIL on cytology with no HSIL histology, or improvement \>50% in the number of lesions with HSIL, or an improvement \>50% in lesion size, area, or clinical characteristics (e.g. acetowhite staining, Lugol's staining, or vascular changes were improved) No Response (NR): HSIL present at weeks 48 \& 60 on histology, or improvement ≤ 50% in number, size, area or characteristics.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

70 participants

Primary outcome timeframe

Baseline, Week 48, and Week 60; up to 60 weeks

Results posted on

2020-04-24

Participant Flow

Participant milestones

Participant milestones
Measure
AIJP
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Baseline - Week 12 (Treatment)
STARTED
35
35
Baseline - Week 12 (Treatment)
COMPLETED
31
34
Baseline - Week 12 (Treatment)
NOT COMPLETED
4
1
Week 12 - Week 48 (Treatment)
STARTED
31
34
Week 12 - Week 48 (Treatment)
COMPLETED
28
28
Week 12 - Week 48 (Treatment)
NOT COMPLETED
3
6
Week 60 (Post-treatment Follow-up Visit)
STARTED
28
28
Week 60 (Post-treatment Follow-up Visit)
COMPLETED
28
27
Week 60 (Post-treatment Follow-up Visit)
NOT COMPLETED
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
AIJP
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Baseline - Week 12 (Treatment)
Received cream but did not return
4
1
Week 12 - Week 48 (Treatment)
Lost to Follow-up
3
5
Week 12 - Week 48 (Treatment)
Not evaluable - censored from dataset
0
1
Week 60 (Post-treatment Follow-up Visit)
Lost to Follow-up
0
1

Baseline Characteristics

Treatment of Anal High-grade Squamous Intraepithelial Lesions (HSIL) Through Use of a Chinese Herbal Topical Cream

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
AIJP
n=35 Participants
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
n=35 Participants
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Total
n=70 Participants
Total of all reporting groups
Age, Customized
24-39 years
5 participants
n=5 Participants
10 participants
n=7 Participants
15 participants
n=5 Participants
Age, Customized
40-59 years
25 participants
n=5 Participants
22 participants
n=7 Participants
47 participants
n=5 Participants
Age, Customized
60-74 years
5 participants
n=5 Participants
3 participants
n=7 Participants
8 participants
n=5 Participants
Sex/Gender, Customized
Men
34 participants
n=5 Participants
33 participants
n=7 Participants
67 participants
n=5 Participants
Sex/Gender, Customized
Women
1 participants
n=5 Participants
1 participants
n=7 Participants
2 participants
n=5 Participants
Sex/Gender, Customized
MTF (transgender)
0 participants
n=5 Participants
1 participants
n=7 Participants
1 participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
6 Participants
n=5 Participants
3 Participants
n=7 Participants
9 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
29 Participants
n=5 Participants
32 Participants
n=7 Participants
61 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
4 Participants
n=7 Participants
4 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
2 Participants
n=5 Participants
1 Participants
n=7 Participants
3 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=5 Participants
4 Participants
n=7 Participants
5 Participants
n=5 Participants
Race (NIH/OMB)
White
32 Participants
n=5 Participants
24 Participants
n=7 Participants
56 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
2 Participants
n=7 Participants
2 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
United States
35 participants
n=5 Participants
35 participants
n=7 Participants
70 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 48, and Week 60; up to 60 weeks

Response assessed 12 weeks after treatment. Late Clinical Response (LCR): HSIL present at week 48 but none at week 60, with two independent reviews in agreement that HSIL absent at week 60. Complete response (CR): No HSIL on histology or cytology at weeks 48 or 60 (caveat: if HSIL at week 60, blinded chart notes and photographs were reviewed by two clinicians, and decision was reached by agreement or consensus whether HSIL had been missed at week 48. Cases that reviewers independently agreed had not been missed at week 48 were considered true recurrences) Partial Clinical Response (PCR): HSIL on cytology with no HSIL histology, or improvement \>50% in the number of lesions with HSIL, or an improvement \>50% in lesion size, area, or clinical characteristics (e.g. acetowhite staining, Lugol's staining, or vascular changes were improved) No Response (NR): HSIL present at weeks 48 \& 60 on histology, or improvement ≤ 50% in number, size, area or characteristics.

Outcome measures

Outcome measures
Measure
AIJP
n=28 Participants
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
n=28 Participants
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Final Response of Anal High-grade Squamous Intraepithelial Lesions (HSIL)
LCR
3 participants
0 participants
Final Response of Anal High-grade Squamous Intraepithelial Lesions (HSIL)
CR
7 participants
3 participants
Final Response of Anal High-grade Squamous Intraepithelial Lesions (HSIL)
PCR
5 participants
6 participants
Final Response of Anal High-grade Squamous Intraepithelial Lesions (HSIL)
NR
13 participants
19 participants

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: Excludes 7 AIJP participants and 6 Placebo participants who dropped out before week 48. Excludes 1 Placebo participant deemed non-evaluable.

Percent of recommended applications of cream reported in participant diary. \>75% = Excellent \>50%-75% = Good \>25%-50% = Poor \<25% = Non-adherent

Outcome measures

Outcome measures
Measure
AIJP
n=28 Participants
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
n=28 Participants
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Treatment Adherence
Excellent
17 participants
15 participants
Treatment Adherence
Good
9 participants
6 participants
Treatment Adherence
Poor
1 participants
4 participants
Treatment Adherence
Non-adherent
1 participants
3 participants

SECONDARY outcome

Timeframe: Baseline, Week 48, and Week 60; up to 60 weeks

Population: Evaluable participants who finished 48 weeks of treatment and reported \>50% adherence to treatment

Response assessed 12 weeks after treatment (week 60), by treatment adherence assessed at 48 weeks. Late Clinical Response (LCR): HSIL present at week 48 but none at week 60; two independent reviews agree HSIL absent at week 60. Complete response (CR): No HSIL on histology or cytology at weeks 48 or 60 (caveat: if HSIL at week 60, blinded chart notes and photographs reviewed by two clinicians, with decision by agreement or consensus whether HSIL had been missed at week 48. Cases that reviewers independently agreed had not been missed at week 48 were considered true recurrences) Partial Clinical Response (PCR): HSIL on cytology with no HSIL histology, or improvement \>50% in number of lesions with HSIL, or improvement \>50% in lesion size, area, or clinical characteristics (e.g. acetowhite staining, Lugol's staining, or vascular changes improved) No Response (NR): HSIL present on histology, or improvement ≤ 50% in number, size, area or characteristics.

Outcome measures

Outcome measures
Measure
AIJP
n=26 Participants
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
n=21 Participants
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Response With >50% Adherence
LCR
3 participants
0 participants
Response With >50% Adherence
NR
12 participants
13 participants
Response With >50% Adherence
CR
7 participants
3 participants
Response With >50% Adherence
PCR
4 participants
5 participants

SECONDARY outcome

Timeframe: Baseline, Week 48, and Week 60; up to 60 weeks

Population: Evaluable participants who finished 48 weeks of treatement and reported \>75% adherence to treatment

Response assessed 12 weeks after treatment (week 60), by treatment adherence assessed at 48 weeks. Late Clinical Response (LCR): HSIL present at week 48 but none at week 60; two independent reviews agree that HSIL absent at week 60. Complete response (CR): No HSIL on histology or cytology (caveat: if HSIL at week 60, blinded chart notes and photographs were reviewed by two clinicians, and decision was reached by agreement or consensus whether HSIL had been missed at week 48. Cases that reviewers independently agreed had not been missed at week 48 were considered true recurrences) Partial Clinical Response (PCR): HSIL on cytology with no HSIL histology, or improvement \>50% in the number of lesions with HSIL, or an improvement \>50% in lesion size, area, or clinical characteristics (e.g. acetowhite staining, Lugol's staining, or vascular changes were improved) No Response (NR): HSIL present on histology, or improvement ≤ 50% in number, size, area or characteristics.

Outcome measures

Outcome measures
Measure
AIJP
n=17 Participants
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
n=15 Participants
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Response With >75% Adherence
LCR
3 participants
0 participants
Response With >75% Adherence
PCR
2 participants
5 participants
Response With >75% Adherence
CR
5 participants
1 participants
Response With >75% Adherence
NR
7 participants
9 participants

Adverse Events

AIJP

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
AIJP
n=35 participants at risk
AIJP (Arnebia Indigo Jade Pearl): Participants administer 1/4 teaspoon of AIJP cream twice daily for 48 weeks.
Placebo
n=35 participants at risk
Placebo: Participants administer 1/4 teaspoon of placebo cream twice daily for 48 weeks.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Perianal cancer
0.00%
0/35 • 60 Weeks
2.9%
1/35 • 60 Weeks

Other adverse events

Adverse event data not reported

Additional Information

Naomi Jay, PhD

University of California, San Francisco

Phone: 415-353-7443

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place