Falcon: Study to Evaluate the Efficacy and Safety of Tilpisertib in Adults With Moderately to Severely Active Ulcerative Colitis

• Sponsor: Gilead Sciences (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT04130919
• Collaborator: (none)
• Enrollment: 19
• Locations: 49
• Arms: 4
• Actual Duration (Months): 23.8
• Patients Per Site: 0.4
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

The primary objective of this study is to demonstrate the efficacy of tilpisertib (formerly GS-4875) compared with placebo control in achieving clinical remission per modified Mayo Clinic Score (MCS) in adults with moderately to severely active ulcerative colitis (UC).

Condition or Disease	Intervention/Treatment	Phase
Ulcerative Colitis	Drug: Tilpisertib; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Blinded, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of GS-4875 in Subjects With Moderately to Severely Active Ulcerative Colitis
• Actual Study Start Date: Dec 20, 2019
• Actual Primary Completion Date: Feb 25, 2021
• Actual Study Completion Date: Dec 14, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Tilpisertib 300 mg Participants will receive blinded tilpisertib 300 mg for up to 10 weeks. An efficacy assessment will be performed at Week 10. Participants who achieve MCS response will continue on the blinded treatment for up to 50 weeks.	Drug: Tilpisertib; Tablets administered orally once daily; Other Names: GS-4875
Experimental: Tilpisertib 100 mg Participants will receive blinded tilpisertib 100 mg for up to 10 weeks. An efficacy assessment will be performed at Week 10. Participants who achieve MCS response will continue on the blinded treatment for up to 50 weeks.	Drug: Tilpisertib; Tablets administered orally once daily; Other Names: GS-4875
Placebo Comparator: Placebo Participants will receive blinded tilpisertib matching placebo for up to 10 weeks. An efficacy assessment will be performed at Week 10. Participants who achieve MCS response will continue on the blinded treatment for up to 50 weeks.	Drug: Placebo; Tablets administered orally once daily
Experimental: Open-label Tilpisertib 300 mg Based on the efficacy assessment results at Week 10, participants who do not achieve MCS response will have the option to receive open-label tilpisertib 300 mg for up to 50 weeks.	Drug: Tilpisertib; Tablets administered orally once daily; Other Names: GS-4875

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants Who Achieved Clinical Remission Per Modified Mayo Clinic Score (MCS) at Week 10 [Week 10]

   The modified MCS is a scoring system for assessment of ulcerative colitis (UC) activity and is composed of subscores from endoscopy (range: 0 to 3, where 0 = normal or inactive disease and 3 = severe disease [spontaneous bleeding, ulceration]), rectal bleeding (range: 0 to 3, where 0 = no blood seen and 3 = blood alone passes), stool frequency (range: 0 to 3, where 0 = normal number of stools and 3 = at least 5 or more stools more than normal), and physician's global assessment (PGA) (range: 0 to 3, where 0 = normal and 3 = severe disease). Total score for MCS ranges from 0 to 12 (sum of all subscores), with higher scores indicating higher disease activity. Clinical remission per modified MCS is defined as stool frequency subscore ≤ 1 and not greater than baseline, rectal bleeding subscore of 0, and endoscopic subscore ≤ 1 at Week 10.

Secondary Outcome Measures

1. Percentage of Participants Who Achieved Endoscopic Response at Week 10 [Week 10]

   Endoscopic response was defined as an endoscopic subscore of ≤ 1 at Week 10. Endoscopic subscore is a part of the modified MCS which is a scoring system for assessment of UC activity. Endoscopic subscore range: 0 to 3, where 0 = normal or inactive disease, 1 = mild disease (erythema, decreased vascular pattern), 2 = moderate disease (marked erythema, lack of vascular pattern, friability, erosions), and 3 = severe disease (spontaneous bleeding, ulceration).

2. Percentage of Participants Who Achieved MCS Response at Week 10 [Week 10]

   The modified MCS is a scoring system for assessment of UC activity and is composed of subscores from endoscopy (range: 0 to 3, where 0 = normal or inactive disease and 3 = severe disease [spontaneous bleeding, ulceration]), rectal bleeding (range: 0 to 3, where 0 = no blood seen and 3 = blood alone passes), stool frequency (range: 0 to 3, where 0 = normal number of stools and 3 = at least 5 or more stools more than normal), and PGA (range: 0 to 3, where 0 = normal and 3 = severe disease). Total score for MCS ranges from 0 to 12 (sum of all subscores), with higher scores indicating higher disease activity. MCS response is defined as a decrease from baseline of ≥ 3 points and at least 30% in MCS, in addition to a ≥ 1 point decrease from baseline in the rectal bleeding subscore or a rectal bleeding subscore ≤ 1 at Week 10.

3. Percentage of Participants Who Achieved MCS Remission at Week 10 [Week 10]

   The modified MCS is a scoring system for assessment of UC activity and is composed of subscores from endoscopy (range: 0 to 3, where 0 = normal or inactive disease and 3 = severe disease [spontaneous bleeding, ulceration]), rectal bleeding (range: 0 to 3, where 0 = no blood seen and 3 = blood alone passes), stool frequency (range: 0 to 3, where 0 = normal number of stools and 3 = at least 5 or more stools more than normal), and PGA (range: 0 to 3, where 0 = normal and 3 = severe disease). Total score for MCS ranges from 0 to 12 (sum of all subscores), with higher scores indicating higher disease activity. MCS remission is defined as a MCS score of ≤ 2 and no individual subscore > 1 at Week 10.

4. Percentage of Participants Who Achieved Histologic Remission Based Upon the Geboes Scale at Week 10 [Week 10]

   Geboes histologic remission was assessed using the Geboes histologic scores to identify histologic changes in ulcerative colitis. Possible scores are Grade 0:Architectural changes(0.0=No abnormality to 0.3=Severe diffuse or multifocal abnormalities); Grade 1:Chronic inflammatory infiltrate(1.0=No increase to 1.3=Marked increase);Grade 2A:Eosinophils in lamina propria(2A.0=No increase to 2A.3-=Marked increase; Grade 2B:Neutrophils in lamina propria(2B.0= No increase to 2B.3=Marked increase);Grade 3:Neutrophils in epithelium (3.0=None to 3.3=>50% crypts involved);Grade 4:Crypt destruction(4.0=none to 4.3=Unequivocal crypt destruction),and Grade 5:Erosions and ulcerations:(5.0=No erosion, ulceration or granulation to 5.4=Ulcer or granulation tissue). Histologic remission defined as having Grade 0 of ≤ 0.3, Grade 1 of ≤ 1.1, Grade 2a of ≤ 2A.3, Grade 2b of 2B.0, Grade 3 of 3.0, Grade 4 of 4.0, and Grade 5 of 5.0. Geboes score ranges from 0 to 5.4. Lower values indicate better outcome.

5. Percentage of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs) [Blinded Treatment phase: First dose date up to 50.6 weeks plus 30 days; Open-label phase: First dose date up to 50.7 weeks plus 30 days]

   Treatment-emergent adverse events (TEAEs) for the Blinded Treatment phase are either defined as AEs with an onset date on or after the Blinded Treatment phase study drug start date and no later than 30 days after permanent discontinuation of the Blinded Treatment phase study drug if no Open-label Treatment phase study drug was taken, or any AEs with an onset date on or after the Blinded Treatment phase study drug start date and before the Open-label Treatment phase study drug start date if Open-label Treatment phase study drug was taken and/or any AEs leading to premature discontinuation of Blinded Treatment phase study drug. TEAEs for the Open-label Treatment phase are either defined as AEs with an onset date on or after the Open-label Treatment phase study drug start date and no later than 30 days after permanent discontinuation of the Open-lab Treatment phase study drug and/or any AEs leading to premature discontinuation of Open-label Treatment phase study drug.

6. Percentage of Participants Who Experienced Laboratory Abnormalities [Blinded Treatment phase: First dose date up to 50.6 weeks plus 30 days; Open-label phase: First dose date up to 50.7 weeks plus 30 days]

   Treatment-emergent laboratory abnormalities for Blinded Treatment phase are defined as values that increase at least 1 toxicity grade from baseline at any postbaseline time point, up to and including the date of last dose of Blinded Treatment phase study drug plus 30 days for participants who permanently discontinued Blinded Treatment phase study drug or before the first dose of Open-label Treatment phase study drug. For the Open-label Treatment phase, treatment-emergent laboratory abnormalities are defined as values that increase at least 1 toxicity grade from Open-label baseline at any postbaseline time point, up to and including the date of last dose of Open-label Treatment phase study drug plus 30 days for participants who permanently discontinued Open-label phase study drug. For maximum postbaseline toxicity grade, the most severe graded abnormality from all tests was counted for each patient. Grade 1: mild; Grade 2: moderate; Grade 3: severe; Grade 4: life-threatening.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Key Inclusion Criteria:

• Males, or non-pregnant, non-lactating females, at least 18 years of age based on the date of the screening visit.

• UC of at least 3 months duration before randomization confirmed by endoscopy and histology at any time in the past AND a minimum disease extent of 15 centimeter (cm) from the anal verge. Documentation of endoscopy and histology consistent with the diagnosis of UC must be available in the source documents prior to the initiation of screening.

• Moderately to severely active UC as determined during screening by a centrally read endoscopy score ≥ 2, a Rectal Bleeding subscore ≥ 1, a Stool Frequency subscore ≥ 1 and Physicians Global Assessment (PGA) of ≥ 2 as defined by the Mayo Clinic Score; total MCS must be between 6 and 12, inclusive.

• Previously demonstrated an inadequate response (primary non-response) or loss of response (secondary non-response) to a tumor necrosis factor-alpha (TNFα) inhibitor (ie, infliximab, adalimumab, golimumab, or biosimilars). The induction treatment regimen resulting in inadequate response or loss of response should have been in accordance with local prescribing information/guidelines or as outlined below.

• Infliximab: 5 mg/kg at Weeks 0, 2, and 6

• Adalimumab: 160 mg on Day 1 (given in 1 day or split over consecutive days), followed by 80 mg 2 weeks later (Day 15), 40 mg 2 weeks later (Day 29) and every 2 weeks thereafter until Day 57

• Golimumab: 200 mg on Day 1 followed by 100 mg at Week 2

• May be receiving concomitant therapy for UC at the time of enrollment as specified in the protocol, provided the dose prescribed has been stable as indicated prior to randomization.

• Meet the following Tuberculosis (TB) screening criteria:

• No evidence of active TB, latent TB, or inadequately treated TB as evidenced by 1 of the following:

• A negative QuantiFERON test or equivalent assay reported by the central lab at screening or within 90 days prior to randomization date. OR

• A history of fully treated active or latent TB according to local standard of care. Investigator must verify adequate previous anti-TB treatment and provide documentation; these individuals do not require QuantiFERON testing and eligibility must be approved by the sponsor prior to enrollment in the study. AND

• A chest radiograph (views as per local guidelines with the report or films available for investigator review) taken at screening or within the 4 months prior to randomization without evidence of active or latent TB infection.

• Laboratory assessments at screening within the following parameters:

• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) and total bilirubin ≤ 2 X upper limit of normal (ULN)

• Estimated glomerular filtration rate (eGFR) ≥ 60 ml/min (1.0 mL/sec) as calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Cystatin C formula as described in protocol.

• Hemoglobin ≥ 8 g/dL (≥ 80 g/L)

• Absolute neutrophil count (ANC) ≥ 1.5 × 10^3/μL (≥ 1.5 GI/L)

• Platelets ≥ 100 × 10^3/μL (≥ 100 GI/L)

• White blood cells (WBC) ≥ 3 × 10^3/μL (≥ 3 GI/L)

• Absolute lymphocyte count ≥ 0.75 × 10^3/μL (≥ 0.75 GI/L)

Key Exclusion Criteria:

• Currently displaying clinical signs of acute severe colitis, fulminant colitis, or toxic megacolon.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Gut P.C., dba Digestive Health Specialists of the Southeast	Dothan	Alabama	United States	36305
2	Om Research LLC	Lancaster	California	United States	93534
3	United Medical Doctors	Murrieta	California	United States	92563
4	Alliance Clinical Research	Poway	California	United States	92064
5	Alliance Medical Research	Coral Springs	Florida	United States	33071
6	Encore Borland-Groover Clinical Research	Jacksonville	Florida	United States	32256
7	A Plus Research, Inc	Miami	Florida	United States	33144
8	BRCR Medical Center Inc.	Plantation	Florida	United States	33322
9	Advanced Medical Research Center	Port Orange	Florida	United States	32127
10	Gastrointestinal Specialists of Georgia	Marietta	Georgia	United States	30060
11	Atlanta Gastroenterology Specialists, PC	Suwanee	Georgia	United States	30024
12	Louisiana Research Center, LLC	Shreveport	Louisiana	United States	71105
13	Kansas City Research Institute	Kansas City	Missouri	United States	64131
14	Advanced Biomedical Research of America	Las Vegas	Nevada	United States	89123
15	Consultants for Clinical Research	Cincinnati	Ohio	United States	45219
16	Gastroenterology Associates of Orangeburg	Orangeburg	South Carolina	United States	29118
17	Vanderbilt University Medical Center - IBD Clinic	Nashville	Tennessee	United States	37212-1375
18	Allied Digestive Disease Center	Cypress	Texas	United States	77429
19	Southwest Clinical Trials	Houston	Texas	United States	77074
20	Clinical Associates in Research Therapeutics of America, LLC	San Antonio	Texas	United States	78212
21	Texas Digestive Disease Consultants	San Marcos	Texas	United States	78666
22	Texas Digestive Disease Consultants	Southlake	Texas	United States	76092
23	Allegiance Research Specialists, LLC	Wauwatosa	Wisconsin	United States	53225
24	Coastal Digestive Health	Maroochydore	Queensland	Australia	4558
25	The Queen Elizabeth Hospital	Woodville	South Australia	Australia	5011
26	St Vincent's Hospital Melbourne	Fitzroy	Victoria	Australia	3065
27	Emeritus Research	Melbourne	Victoria	Australia	3124
28	Medizinische Universität Innsbruck, Universitätsklinik für Innere Medizin I	Innsbruck		Austria	6020
29	Medizinische Universitat Wien Klinik fur Innere Medizin III/Abt. fur Gastroenterologie and Hepatologie	Vienna		Austria	1090
30	Vancouver General Hospital - The Gordon and Leslie Diamond Health Care Centre	Vancouver	British Columbia	Canada	V5Z 1M9
31	Hopital Beaujon	Clichy		France	92110
32	CHU de Dijon Bourgogne	Dijon		France	21079
33	Centre Hospitalier Universitaire de Grenoble Alpes	Grenoble		France	38043
34	CHRU de Lille - Hôpital Claude Huriez	Lille		France	59000
35	CHU de Lyon Sud	Pierre-Benite		France	69495
36	CHRU Pontchaillou	Rennes		France	35033 Cedex 9
37	CHU de Saint Etienne	Saint-Etienne		France	42055
38	Hopital Rangueil	Toulouse		France	31059 cedex 9
39	CHRU de Nancy	Vandoeuvre-les-Nancy Cedex		France	54511
40	Universitätsklinikum Schleswig-Holstein, Campus Kiel, Klinik fur Innere Medizin I, Haus C, Haus K3	Kiel		Germany	24105
41	Eugastro GmbH	Leipzig		Germany	04103
42	Gastroenterologische Gemeinschaftspraxis Minden	Minden		Germany	32423
43	Istituto Clinico Humanitas	Rozzano		Italy	20089
44	Twoja Przychodnia - Szczecinskie Centrum Medyczne	Szczecin		Poland	71-434
45	"GASTROMED" Kopon, Zmudzinski i Wsp. Sp. J. Spec. Centrum Gastrologii i Endoskopii, Spec. Gabinety Lekarskie	Torun		Poland	87-100
46	Centrum Medyczne Melita Medical	Wroclaw		Poland	50-449
47	Gastroenterologische Praxis Balsiger, Seibold & Partner/Crohn-Colitis-Zentrum	Bern		Switzerland	3012
48	Inselspital Bern/Klinik fur Viszerale Chirurgie und Medizin/Bauchzentrum	Bern		Switzerland	CH-3010
49	Universitätsspital Zürich/Klinik für Gastroenterologie und Hepatologie	Zurich		Switzerland	8091

Sponsors and Collaborators

• Gilead Sciences

Investigators

• Study Director: Gilead Study Director, Gilead Sciences

Study Documents (Full-Text)

• Study Protocol - Jun 19, 2020
• Statistical Analysis Plan - Apr 5, 2021

More Information

Publications

None provided.

• Responsible Party: Gilead Sciences
• ClinicalTrials.gov Identifier: NCT04130919
• Other Study ID Numbers: GS-US-365-4237; 2019-001430-33
• First Posted: Oct 18, 2019
• Last Update Posted: Aug 24, 2022
• Last Verified: Jul 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Colitis; Colitis, Ulcerative; Ulcer

Study Results

Participant Flow

Recruitment Details	Participants were enrolled at study sites in Australia, Europe and the United States.
Pre-assignment Detail	32 participants were screened.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)	Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)	Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)	Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per Modified Mayo Clinic score (MCS) at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Participants who received tilpisertib 300 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.	Participants who received tilpisertib 100 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.	Participants who received placebo and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.
Period Title: Blinded Treatment Phase
STARTED	7	6	6	0	0	0
COMPLETED	5	4	3	0	0	0
NOT COMPLETED	2	2	3	0	0	0
Period Title: Blinded Treatment Phase
STARTED	0	0	0	4	3	3
COMPLETED	0	0	0	1	1	0
NOT COMPLETED	0	0	0	3	2	3

Baseline Characteristics

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)	Total
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Total of all reporting groups
Overall Participants	7	6	6	19
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	51 (11.8)	49 (22.6)	43 (18.4)	48 (17.2)
Sex: Female, Male (Count of Participants)
Female	2 28.6%	1 16.7%	3 50%	6 31.6%
Male	5 71.4%	5 83.3%	3 50%	13 68.4%
Race/Ethnicity, Customized (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	2 33.3%	2 10.5%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%	0 0%
White	7 100%	6 100%	3 50%	16 84.2%
Other	0 0%	0 0%	1 16.7%	1 5.3%
Not Permitted	0 0%	0 0%	0 0%	0 0%
Race/Ethnicity, Customized (Count of Participants)
Not Hispanic or Latino	6 85.7%	4 66.7%	6 100%	16 84.2%
Hispanic or Latino	1 14.3%	2 33.3%	0 0%	3 15.8%
Not Permitted	0 0%	0 0%	0 0%	0 0%
Region of Enrollment (Count of Participants)
Austria	1 14.3%	0 0%	1 16.7%	2 10.5%
United States	3 42.9%	3 50%	4 66.7%	10 52.6%
Australia	1 14.3%	1 16.7%	1 16.7%	3 15.8%
Switzerland	2 28.6%	1 16.7%	0 0%	3 15.8%
Germany	0 0%	1 16.7%	0 0%	1 5.3%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Who Achieved Clinical Remission Per Modified Mayo Clinic Score (MCS) at Week 10
Description	The modified MCS is a scoring system for assessment of ulcerative colitis (UC) activity and is composed of subscores from endoscopy (range: 0 to 3, where 0 = normal or inactive disease and 3 = severe disease [spontaneous bleeding, ulceration]), rectal bleeding (range: 0 to 3, where 0 = no blood seen and 3 = blood alone passes), stool frequency (range: 0 to 3, where 0 = normal number of stools and 3 = at least 5 or more stools more than normal), and physician's global assessment (PGA) (range: 0 to 3, where 0 = normal and 3 = severe disease). Total score for MCS ranges from 0 to 12 (sum of all subscores), with higher scores indicating higher disease activity. Clinical remission per modified MCS is defined as stool frequency subscore ≤ 1 and not greater than baseline, rectal bleeding subscore of 0, and endoscopic subscore ≤ 1 at Week 10.
Time Frame	Week 10

Outcome Measure Data

Analysis Population Description

Full analysis set included all randomized participants who received at least 1 dose of study drug.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.
Measure Participants	7	6	6
Number [percentage of participants]	0 0%	0 0%	0 0%

2. Secondary Outcome

Title	Percentage of Participants Who Achieved Endoscopic Response at Week 10
Description	Endoscopic response was defined as an endoscopic subscore of ≤ 1 at Week 10. Endoscopic subscore is a part of the modified MCS which is a scoring system for assessment of UC activity. Endoscopic subscore range: 0 to 3, where 0 = normal or inactive disease, 1 = mild disease (erythema, decreased vascular pattern), 2 = moderate disease (marked erythema, lack of vascular pattern, friability, erosions), and 3 = severe disease (spontaneous bleeding, ulceration).
Time Frame	Week 10

Outcome Measure Data

Analysis Population Description

Participants in the Full Analysis Set were analyzed.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.
Measure Participants	7	6	6
Number [percentage of participants]	14.3 204.3%	16.7 278.3%	0 0%

3. Secondary Outcome

Title	Percentage of Participants Who Achieved MCS Response at Week 10
Description	The modified MCS is a scoring system for assessment of UC activity and is composed of subscores from endoscopy (range: 0 to 3, where 0 = normal or inactive disease and 3 = severe disease [spontaneous bleeding, ulceration]), rectal bleeding (range: 0 to 3, where 0 = no blood seen and 3 = blood alone passes), stool frequency (range: 0 to 3, where 0 = normal number of stools and 3 = at least 5 or more stools more than normal), and PGA (range: 0 to 3, where 0 = normal and 3 = severe disease). Total score for MCS ranges from 0 to 12 (sum of all subscores), with higher scores indicating higher disease activity. MCS response is defined as a decrease from baseline of ≥ 3 points and at least 30% in MCS, in addition to a ≥ 1 point decrease from baseline in the rectal bleeding subscore or a rectal bleeding subscore ≤ 1 at Week 10.
Time Frame	Week 10

Outcome Measure Data

Analysis Population Description

Participants in the Full Analysis Set were analyzed.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.
Measure Participants	7	6	6
Number [percentage of participants]	28.6 408.6%	16.7 278.3%	16.7 278.3%

4. Secondary Outcome

Title	Percentage of Participants Who Achieved MCS Remission at Week 10
Description	The modified MCS is a scoring system for assessment of UC activity and is composed of subscores from endoscopy (range: 0 to 3, where 0 = normal or inactive disease and 3 = severe disease [spontaneous bleeding, ulceration]), rectal bleeding (range: 0 to 3, where 0 = no blood seen and 3 = blood alone passes), stool frequency (range: 0 to 3, where 0 = normal number of stools and 3 = at least 5 or more stools more than normal), and PGA (range: 0 to 3, where 0 = normal and 3 = severe disease). Total score for MCS ranges from 0 to 12 (sum of all subscores), with higher scores indicating higher disease activity. MCS remission is defined as a MCS score of ≤ 2 and no individual subscore > 1 at Week 10.
Time Frame	Week 10

Outcome Measure Data

Analysis Population Description

Participants in the Full Analysis Set were analyzed.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.
Measure Participants	7	6	6
Number [percentage of participants]	0 0%	0 0%	0 0%

5. Secondary Outcome

Title	Percentage of Participants Who Achieved Histologic Remission Based Upon the Geboes Scale at Week 10
Description	Geboes histologic remission was assessed using the Geboes histologic scores to identify histologic changes in ulcerative colitis. Possible scores are Grade 0:Architectural changes(0.0=No abnormality to 0.3=Severe diffuse or multifocal abnormalities); Grade 1:Chronic inflammatory infiltrate(1.0=No increase to 1.3=Marked increase);Grade 2A:Eosinophils in lamina propria(2A.0=No increase to 2A.3-=Marked increase; Grade 2B:Neutrophils in lamina propria(2B.0= No increase to 2B.3=Marked increase);Grade 3:Neutrophils in epithelium (3.0=None to 3.3=>50% crypts involved);Grade 4:Crypt destruction(4.0=none to 4.3=Unequivocal crypt destruction),and Grade 5:Erosions and ulcerations:(5.0=No erosion, ulceration or granulation to 5.4=Ulcer or granulation tissue). Histologic remission defined as having Grade 0 of ≤ 0.3, Grade 1 of ≤ 1.1, Grade 2a of ≤ 2A.3, Grade 2b of 2B.0, Grade 3 of 3.0, Grade 4 of 4.0, and Grade 5 of 5.0. Geboes score ranges from 0 to 5.4. Lower values indicate better outcome.
Time Frame	Week 10

Outcome Measure Data

Analysis Population Description

Participants in the Full Analysis Set with at least 1 histological assessment were analyzed.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.
Measure Participants	6	6	4
Number [percentage of participants]	16.7 238.6%	16.7 278.3%	25.0 416.7%

6. Secondary Outcome

Title	Percentage of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs)
Description	Treatment-emergent adverse events (TEAEs) for the Blinded Treatment phase are either defined as AEs with an onset date on or after the Blinded Treatment phase study drug start date and no later than 30 days after permanent discontinuation of the Blinded Treatment phase study drug if no Open-label Treatment phase study drug was taken, or any AEs with an onset date on or after the Blinded Treatment phase study drug start date and before the Open-label Treatment phase study drug start date if Open-label Treatment phase study drug was taken and/or any AEs leading to premature discontinuation of Blinded Treatment phase study drug. TEAEs for the Open-label Treatment phase are either defined as AEs with an onset date on or after the Open-label Treatment phase study drug start date and no later than 30 days after permanent discontinuation of the Open-lab Treatment phase study drug and/or any AEs leading to premature discontinuation of Open-label Treatment phase study drug.
Time Frame	Blinded Treatment phase: First dose date up to 50.6 weeks plus 30 days; Open-label phase: First dose date up to 50.7 weeks plus 30 days

Outcome Measure Data

Analysis Population Description

Safety Analysis Set included all participants who received at least one dose of study drug.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)	Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)	Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)	Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Participants who received tilpisertib 300 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.	Participants who received tilpisertib 100 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.	Participants who received placebo and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.
Measure Participants	7	6	6	4	3	3
Number [percentage of participants]	57.1 815.7%	50.0 833.3%	50.0 833.3%	50.0 263.2%	66.7 NaN	66.7 NaN

7. Secondary Outcome

Title	Percentage of Participants Who Experienced Laboratory Abnormalities
Description	Treatment-emergent laboratory abnormalities for Blinded Treatment phase are defined as values that increase at least 1 toxicity grade from baseline at any postbaseline time point, up to and including the date of last dose of Blinded Treatment phase study drug plus 30 days for participants who permanently discontinued Blinded Treatment phase study drug or before the first dose of Open-label Treatment phase study drug. For the Open-label Treatment phase, treatment-emergent laboratory abnormalities are defined as values that increase at least 1 toxicity grade from Open-label baseline at any postbaseline time point, up to and including the date of last dose of Open-label Treatment phase study drug plus 30 days for participants who permanently discontinued Open-label phase study drug. For maximum postbaseline toxicity grade, the most severe graded abnormality from all tests was counted for each patient. Grade 1: mild; Grade 2: moderate; Grade 3: severe; Grade 4: life-threatening.
Time Frame	Blinded Treatment phase: First dose date up to 50.6 weeks plus 30 days; Open-label phase: First dose date up to 50.7 weeks plus 30 days

Outcome Measure Data

Analysis Population Description

Participants in the Safety Analysis Set were analyzed.

Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)	Tilpisertib 100 mg (Blinded Treatment Phase)	Placebo (Blinded Treatment Phase)	Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)	Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)	Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.	Participants who received tilpisertib 300 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.	Participants who received tilpisertib 100 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.	Participants who received placebo and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.
Measure Participants	7	6	6	4	3	3
Grade 1	42.9 612.9%	66.7 1111.7%	16.7 278.3%	25.0 131.6%	33.3 NaN	66.7 NaN
Grade 2	28.6 408.6%	33.3 555%	50.0 833.3%	25.0 131.6%	0 NaN	33.3 NaN
Grade 3	14.3 204.3%	0 0%	0 0%	25.0 131.6%	66.7 NaN	0 NaN
Grade 4	0 0%	0 0%	0 0%	0 0%	0 NaN	0 NaN

Adverse Events

Time Frame	Adverse Events: Blinded Treatment phase: First dose date up to 50.6 weeks plus 30 days; Open-label Treatment phase: First dose date up to 50.7 weeks plus 30 days; All-Cause Mortality: Enrollment up to Week 70.3
Adverse Event Reporting Description	Adverse Events: Safety Analysis Set included all participants who received at least one dose of study drug; All-Cause Mortality: All Randomized Analysis Set included all participants who were randomized in the study.
Arm/Group Title	Tilpisertib 300 mg (Blinded Treatment Phase)		Tilpisertib 100 mg (Blinded Treatment Phase)		Placebo (Blinded Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
Arm/Group Description	Tilpisertib 300 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 300 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.		Tilpisertib 100 mg tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive tilpisertib 100 mg tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.		Placebo tablet orally once daily up to Week 10. Participants who achieved clinical response per modified MCS at Week 10, continued to receive placebo tablets orally once daily for a maximum of 50 weeks in the Blinded Treatment phase.		Participants who received tilpisertib 300 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.		Participants who received tilpisertib 100 mg and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.		Participants who received placebo and did not achieve clinical response per modified MCS at Week 10 in the Blinded Treatment phase, received tilpisertib 300 mg tablets orally once daily for up to 50 weeks in the Open-label Treatment phase.
All Cause Mortality
	Tilpisertib 300 mg (Blinded Treatment Phase)		Tilpisertib 100 mg (Blinded Treatment Phase)		Placebo (Blinded Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/7 (0%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Serious Adverse Events
	Tilpisertib 300 mg (Blinded Treatment Phase)		Tilpisertib 100 mg (Blinded Treatment Phase)		Placebo (Blinded Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/7 (14.3%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Gastrointestinal disorders
Proctalgia	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Infections and infestations
Campylobacter infection	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Other (Not Including Serious) Adverse Events
	Tilpisertib 300 mg (Blinded Treatment Phase)		Tilpisertib 100 mg (Blinded Treatment Phase)		Placebo (Blinded Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 300 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Tilpisertib 100 mg (Open-label Treatment Phase)		Tilpisertib 300 mg From Placebo (Open-label Treatment Phase)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	4/7 (57.1%)		3/6 (50%)		2/6 (33.3%)		2/4 (50%)		2/3 (66.7%)		2/3 (66.7%)
Blood and lymphatic system disorders
Anaemia	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Ear and labyrinth disorders
External ear inflammation	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Gastrointestinal disorders
Colitis ulcerative	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		1/4 (25%)		0/3 (0%)		1/3 (33.3%)
Dyspepsia	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Haemorrhoids thrombosed	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
General disorders
Pyrexia	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Immune system disorders
Seasonal allergy	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Infections and infestations
Asymptomatic COVID-19	0/7 (0%)		0/6 (0%)		0/6 (0%)		1/4 (25%)		0/3 (0%)		0/3 (0%)
Cystitis	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Hordeolum	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Mastitis	0/7 (0%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		1/3 (33.3%)
Sinusitis	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Viral infection	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Viral upper respiratory tract infection	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Metabolism and nutrition disorders
Hypophosphataemia	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Iron deficiency	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Vitamin B12 deficiency	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Vitamin D deficiency	0/7 (0%)		1/6 (16.7%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Musculoskeletal and connective tissue disorders
Arthralgia	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Back pain	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Intervertebral disc protrusion	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Osteonecrosis	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Pain in extremity	0/7 (0%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		1/3 (33.3%)		0/3 (0%)
Nervous system disorders
Headache	0/7 (0%)		1/6 (16.7%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		1/3 (33.3%)
Psychiatric disorders
Insomnia	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Reproductive system and breast disorders
Erectile dysfunction	0/7 (0%)		0/6 (0%)		0/6 (0%)		0/4 (0%)		1/3 (33.3%)		0/3 (0%)
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Skin and subcutaneous tissue disorders
Pruritus	1/7 (14.3%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)
Rash	1/7 (14.3%)		0/6 (0%)		0/6 (0%)		1/4 (25%)		0/3 (0%)		0/3 (0%)
Rash erythematous	0/7 (0%)		0/6 (0%)		1/6 (16.7%)		0/4 (0%)		0/3 (0%)		0/3 (0%)

Limitations/Caveats

Gilead made the decision to discontinue the development of tilpisertib since a new molecular entity was able to achieve greater target coverage. The decision was not due to any safety concerns. Since only 19 participants were enrolled, none of the planned statistical analyses were performed. During the coronavirus disease 2019 (COVID-19) pandemic, there were changes to protocol visits and procedures where necessary to mitigate the impact of the pandemic to the study.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met: The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or The study has been completed at all study sites for at least 2 years