Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of VE202 in Patients with Mild-to-Moderate Ulcerative Colitis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vedanta Biosciences Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

11 May 2023 → 12 Aug 2025

Decision date (initial)

2024-10-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-512558-40-00

EudraCT number

2021-001280-24

ClinicalTrials.gov

NCT05370885

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

Double-Blind Portion (Parts 1, 2, and 3) Objectives:
• To evaluate the efficacy of 8 weeks of treatment with VE202 in terms of endoscopic response at Day 56
• To evaluate the safety of VE202 in Part 1 and Part 2 of the study

Open-Label Portion (Part 4) Objectives:
•Evaluate the safety of VE202 treatment in Part 4 of the study

Conditions and MedDRA coding

Mild-to-moderate ulcerative colitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. Able and willing to provide written informed consent prior to initiation of any study specific procedure or drug administration and understands the potential risks and benefits of study enrollment and study drug administration. When appropriate, informed consent may be provided by a legally authorized representative (LAR).
2. If a male patient (according to sex assignment at birth): ‒ If not vasectomized, agrees to wear a condom when engaging in sexual intercourse with any partner of childbearing potential from the time of enrollment until 3 months after the last dose of study drug ‒ Agrees not to donate sperm for purpose of reproduction from the time of enrollment until 3 months after the last dose of study drug
3. Able and willing to follow study procedures (eg, comply with study visits and procedures, provide blood and stool samples).
4. 18 to 75 years of age.
5. Documented clinical and endoscopic diagnosis of UC at least 3 months prior to randomization
6. Active mild to moderate UC, as defined by the following: a. Disease that extends at least 15 cm from the anal verge b. A modified Mayo score of 4 to 8 with: i. Mayo endoscopic subscore of ≥ 2 based on screening flexible sigmoidoscopy ii. Rectal bleeding score of ≥ 1
7. Is up to date with current local colorectal cancer surveillance recommendations (eg, has had a surveillance colonoscopy within 12 months if indicated based on extent and duration of UC); this may be performed during screening.
8. Has never received a biologic agent, Janus kinase inhibitor, or sphingosine-1-phosphate modulators for the treatment of UC
9. If receiving corticosteroids, dose must be stable for at least 4 weeks and be no higher than 10 mg QD prednisone (or prednisone equivalent) or budesonide 9 mg QD.
10. Doses of other allowable UC medications must be stable for at least 8 weeks before randomization.
11. If a female patient (according to sex assignment at birth): ‒ Is not of childbearing potential, defined as post-menopausal for at least 1 year or surgically sterile due to hysterectomy, bilateral oophorectomy, or bilateral tubal ligation ‒ If of childbearing potential, must have a negative pregnancy test and agree to either remain celibate or use a highly effective form of birth control (as defined in Appendix 1) from the time of enrollment until 3 months after the last dose of study drug ‒ Agrees not to donate eggs (ova, oocytes) for purposes of assisted reproduction from the time of enrollment until 3 months after the last dose of study drug ‒ Is not breastfeeding
12. Open-Label Portion (Part 4), Independent of Part 1 Eligibility: Able and willing to provide written informed consent prior to initiation of any study-specific procedure or drug administration in Part 4

Exclusion criteria 18

1. Known history of CD or indeterminate colitis
2. Receipt of FMT or other fecal-derived preparation within 6 months prior to randomization
3. Use of systemic or non-absorbable oral antibiotics within the prior 4 weeks before randomization or anticipated within the study period
4. Use of probiotics within the prior 2 weeks before randomization (consumption of food products such as yogurt, kombucha, kimchi, and kefir is permissible)
5. Receipt of herbal, botanical, or traditional medicinal preparations within the 2 weeks prior to randomization (consumption of mint, turmeric, or other herbal teas is permissible)
6. Active drug or alcohol abuse
7. Active colonic dysplasia
8. A known diagnosis of primary sclerosing cholangitis
9. Allergy to VE202 or any of its components
10. Allergy to vancomycin or any of its components
11. A diagnosis of any non-IBD diarrheal illness (eg, Clostridioides difficile, celiac disease, parasitic infection) within 3 months prior to randomization
12. Known or suspected toxic megacolon, abdominal abscess, and/or small bowel ileus at the time of screening
13. Any other anatomic or medical contraindication to flexible sigmoidoscopy
14. Evidence of an active infection
15. Recent fever, defined as > 38.0 °C (rectal equivalent) within 3 days prior to randomization
16. Open-Label Portion (Part 4), Independent of Part 1 Eligibility: Patient received or plans to receive another investigational drug or device before entry or before completion of Part 4
17. Open-Label Portion (Part 4), Independent of Part 1 Eligibility: Presence of an unresolved AE (except for an AE directly related to UC) or clinically significant finding on a physical examination or laboratory result that, in the Investigator’s opinion, would limit the patient’s ability to participate in or complete the remainder of the study
18. Open-Label Portion (Part 4), Independent of Part 1 Eligibility: Underwent colectomy, ostomy, or other intestinal surgery (excluding cholecystectomy or appendectomy) since enrolling into the double-blind portion of the study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Endoscopic response rate defined as a reduction of 1 point or more in Mayo endoscopic subscore on flexible sigmoidoscopy from baseline to Day 56
2. Grade ≥ 3 Treatment-emergent adverse event (TEAEs) that are related to VE202 or VE202 placebo
3. Serious adverse event (SAEs) that are related to VE202 or VE202 placebo
4. OPEN-LABEL PORTION (PART 4): TEAEs, SAEs, AESIs, MAAEs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vedanta Biosciences Inc.

Sponsor organisation

Vedanta Biosciences Inc.

Address

19 Blackstone Street

City

Cambridge

Postcode

02139-3709

Country

United States

Scientific contact point

Organisation

Vedanta Biosciences Inc.

Contact name

Dr. Steven Shiff

Public contact point

Organisation

Vedanta Biosciences Inc.

Contact name

Mary Garfield

Third parties 5

Locations

6 EU/EEA countries · 21 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 41 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications