Efficacy and Safety of BBT-401-1S in Ulcerative Colitis | NCT03800420

NCT03800420

Status Module

Sponsor/Collaborators Module

Name	Class
KCRN Research, LLC	INDUSTRY

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
BBT-401-1S	Experimental	BBT-401-1S, Oral capsule, QD	Drug: BBT-401-1S first and then Placebo
Placebo	Placebo Comparator	Placebo, Oral capsule, QD	Drug: Placebo first and then BBT-401-1S

Interventions

Name	Type	Description	Arm Group Labels	Other Names
BBT-401-1S first and then Placebo	Drug	Total 16 week treatment period: The subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.	BBT-401-1S

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change From Baseline in Total Mayo Score	The Total Mayo score is an instrument designed to measure disease activity of ulcerative colitis, with scores ranging from 0 to 12 points. The Total Mayo score consists of 4 subscores( Stool frequency, Rectal bleeding, Findings of endoscopy, Physician global assessment), each graded from 0 to 3 with higher scores indicating more severe disease.	Week 8

Secondary Outcomes

Measure	Description	Time Frame
Change From Baseline in Partial Mayo Score	The Partial Mayo score is an instrument designed to measure disease activity of ulcerative colitis, with scores ranging from 0 to 9 points. The Partial Mayo score consists of 3 subscores( Stool frequency, Rectal bleeding, Physician global assessment), each graded from 0 to 3 with higher scores indicating more severe disease.	Week 8

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Provision of signed and dated informed consent form (ICF)
Stated willingness to comply with all study procedures and availability for the duration of the study
Male or female, aged >=18 years
Diagnosed with active UC for at least 3 months prior to screening
Total Mayo score >=5 and Endoscopic sub-score >=1
Stable dosing regimens of oral drugs (if currently administered) as follows: 5-ASA or sulfasalazine at a stable dose for at least 4 weeks, purine analogues (azathioprine, mercaptopurine, thiopurines) or methotrexate at a stable dose for at least 12 weeks, and low-dose oral corticosteroid (up to 20 mg prednisone/day or equivalent) for at least 4 weeks prior to the first dose of study treatment. Doses of oral drugs must remain stable until the end of study treatment (with possible exception for tapering steroid dose after 8 weeks)
For females of reproductive potential: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 3 months after the last dose
For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner during study participation and for an additional 3 months after the last dose

Exclusion Criteria:

Use of anti-TNF-a biologics or any other biologics for treatment of UC within 60 days prior to randomization.
Any rectal therapy for treatment of UC or intravenous corticosteroids within 2 weeks prior to randomization.
Presence of Crohn's disease, indeterminate colitis, ischemic colitis, fulminant colitis, ulcerative proctitis, or toxic mega-colon
Previous extensive colonic resection (subtotal or total colectomy)
Ileostomy, colostomy, or known fixed symptomatic stenosis of the intestine
Evidence of or treatment for Clostridium difficile infection or other pathogenic bowel infection within 60 days or for another intestinal pathogen within 30 days prior to randomization
Active infection with the HIV or Hepatitis B or C viruses
Clinically significant active extra-intestinal infection (e.g., pneumonia, pyelonephritis)
Clinically significant abnormal vital signs, physical examination or 12-lead electrocardiogram (ECG) at screening or baseline
Clinically significant abnormal results of liver function tests (ALT/AST, bilirubin and alkaline phosphatase) > 2X the upper limit of normal (ULN) at screening
Other clinically significant abnormal laboratory results at screening in the investigator's opinion
History of any clinically significant medical condition that, in the investigator's opinion, would preclude participation in the study
Pregnancy or lactation
Treatment with another investigational drug or other intervention within 30 days prior to screening

Contacts/Locations Module

Facility	Status	City	State	ZIP	Country	Contacts
Site 03		Sacramento	California	95821	United States
Site 01

References Module

No data available

No data is available for this block.

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
FG001	Placebo

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Jul 19, 2019	Aug 2, 2021

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

No data available

No data is available for this block.

Masking Info

subjects who were randomized

Arm/Group Information

ID	Title	Description
BG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
BG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG00012 BG0014 BG00216

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Categorical

Count of Participants

Participants

Title

Denominators

Categories

Race

Title	Measurements
White	BG00011 BG0014 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Change From Baseline in Total Mayo Score

The Total Mayo score is an instrument designed to measure disease activity of ulcerative colitis, with scores ranging from 0 to 12 points. The Total Mayo score consists of 4 subscores( Stool frequency, Rectal bleeding, Findings of endoscopy, Physician global assessment), each graded from 0 to 3 with higher scores indicating more severe disease.

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S First and Then Placebo	Total 16 weeks First 12 weeks, BBT-401-1S, Oral capsule, QD. Followed by 4 weeks Placebo, Oral capsule, QD.
OG001	Placebo First and Then BBT-401-1S	Total 16 weeks First Placebo, 8 weeks Oral capsule, QD Followed by 8 weeks BBT-401-1S, Oral capsule, QD

Units	Counts
Participants	OG0000 OG0010

Secondary

Change From Baseline in Partial Mayo Score

The Partial Mayo score is an instrument designed to measure disease activity of ulcerative colitis, with scores ranging from 0 to 9 points. The Partial Mayo score consists of 3 subscores( Stool frequency, Rectal bleeding, Physician global assessment), each graded from 0 to 3 with higher scores indicating more severe disease.

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: The subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: The subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Histologic Assessment of Endoscopic Biopsy

Histological assessment of endoscopic biopsy is an appropriate method for evaluating mucosal healing (Carbonnel et al, 1994).

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Ulcerative Colitis Endoscopic Index of Severity (0-8)

The Ulcerative Colitis Endoscopic Index of Severity (UCEIS) is the first validated index for the assessment of overall endoscopic activity. The model incorporates the vascular pattern score(0-2), the presence of bleeding score(0-3) and the presence of erosions and ulcers score (0-3). Higher score of the sum of these subscore indicates more severe disease.

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants

Secondary

Number and Severity of TEAEs

Treatment Emergent Adverse Event (TEAE) is defined as an adverse event that occurs or worsens on or after the first dose of study drug.

Posted

Number

Number of events

up to 8 weeks after the last dose

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Secondary

Plasma Concentration of BBT- 401-1S

Analysis of Plasma Pharmacokinetics for BBT- 401 -1S

All samples were below LLOQ and therefore plasma concentrations could not be calculated, (Plasma PK samples of one active patient were not collected.)

Posted

Mean

95% Confidence Interval

ng/ml

Day 1, Week 4, Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Concentration of Serum CRP

Widely used serum indicator of inflammation in Ulcerative Colitis(UC).

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Concentration of Fecal Calprotectin

Reliable surrogate marker for disease activity in Ulcerative Colitis (UC).

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Concentration of Fecal Lactoferrin

A stool (fecal) test that is used to detect inflammation in the intestines.

No data was collected for this outcome measure due to early termination of study.

Posted

Week 8

ID	Title	Description
OG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.
OG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.

Units	Counts
Participants	OG000

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	BBT-401-1S	BBT-401-1S, Oral capsule, QD BBT-401-1S first and then Placebo: Total 16 week treatment period: Subject takes BBT-401-1S and Placebo. If the subject takes BBT-401-1S, QD for the first 12 weeks, then placebo for the last 4 weeks.	0	12	0	12	8	12
EG001	Placebo	Placebo, Oral capsule, QD Placebo first and then BBT-401-1S: Total 16 week treatment period: Subject take BBT-401-1S and Placebo. If the subject takes the placebo, QD for the first 8 weeks, then BBT-401-1S for the last 8 weeks.	0	4	0	4	1	4

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Colitis ulcerative	Gastrointestinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0002 events2 affected12 at risk EG0010 events0 affected4 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Flatulence	Gastrointestinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Gastrointestinal tract mucosal pigmentation	Gastrointestinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Haemorrhoids	Gastrointestinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Melaena	Gastrointestinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Nasopharyngitis	Infections and infestations	MedDRA 23.0	Non-systematic Assessment	EG0002 events2 affected12 at risk EG0011 events1 affected4 at risk
Onychomycosis	Infections and infestations	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Condition aggravated	General disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Faecal calprotectin increased	Investigations	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Stool analysis abnormal	Investigations	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 23.0	Non-systematic Assessment	EG0001 events1 affected12 at risk EG0010 events0 affected4 at risk

First MCP Posted Date

ID	Term
D003093	Colitis, Ulcerative

Ancestor Terms

ID	Term
D003092	Colitis
D005759	Gastroenteritis
D005767	Gastrointestinal Diseases
D004066	Digestive System Diseases
D015212	Inflammatory Bowel Diseases
D003108	Colonic Diseases
D007410	Intestinal Diseases