VTP-1000 for Celiac Disease

Recruiting at 16 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Barinthus Biotherapeutics

Disqualifiers: Refractory celiac, IgA deficiency, others

Compensation: Varies

Number of Visits: 6

45 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and effectiveness of a new treatment called VTP-1000 for individuals with celiac disease. The researchers aim to assess how VTP-1000 affects the immune system when exposed to gluten, comparing different doses to a placebo. Individuals with celiac disease who follow a strict gluten-free diet and have specific blood markers (HLA-DQ2.5 genotype) may be suitable candidates for this study. As a Phase 1 trial, this research focuses on understanding how VTP-1000 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that VTP-1000 is likely to be safe for humans?

Research has shown that VTP-1000 is being tested for safety in people with celiac disease. Since this is the first human trial of VTP-1000, the researchers do not yet know how people will react to it. The main goal of this early trial is to assess how well participants tolerate the treatment and to identify any side effects. Participants receive different doses to determine which ones might be safe and effective. Because the testing process is in its early stages, researchers closely monitor all participants for any reactions. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about VTP-1000 for celiac disease because it offers a novel approach compared to existing treatments. Unlike the current standard of care, which primarily involves strict adherence to a gluten-free diet, VTP-1000 may target the underlying immune response that causes the symptoms. This new mechanism of action could potentially provide relief to patients who struggle with dietary restrictions. VTP-1000 is also being tested in multiple dose levels to optimize its effectiveness and safety, which could lead to more personalized treatment options for individuals with celiac disease.

What evidence suggests that VTP-1000 might be an effective treatment for celiac disease?

Research has shown that VTP-1000 could be a promising treatment for celiac disease. It employs antigen-specific immunotherapy, which helps the immune system adapt to gluten. Gluten poses the main problem for people with celiac disease. Early studies suggest that treatments like VTP-1000 might reduce the immune system's adverse reactions to gluten. However, as this is a new treatment, further research is needed to confirm its effectiveness. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for adults with celiac disease who have a specific genetic marker (HLA-DQ2.5), confirmed diagnosis through tests and biopsy, are on a controlled gluten-free diet, and not pregnant or breastfeeding. They should also have low levels of certain antibodies related to celiac disease.

Inclusion Criteria

I have the HLA-DQ2.5 genotype.

Participants who are on a well controlled gluten restricted diet

I am not pregnant or breastfeeding.

Exclusion Criteria

My celiac disease does not improve with a gluten-free diet.

I have a condition where my body doesn't produce enough IgA antibodies.

I am positive for HLA-DQ8.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Single Ascending Dose (SAD)

Participants receive a single dose of VTP-1000 at escalating dose levels to assess safety and tolerability

3 weeks

Includes a 3-day domicile period following administration

Multiple Ascending Dose (MAD)

Participants receive multiple doses of VTP-1000 at escalating dose levels, followed by a gluten challenge

8 weeks

3 doses every 2 weeks, followed by gluten challenge

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VTP-1000

Trial Overview The trial is testing VTP-1000's safety and how well it's tolerated in different doses compared to a placebo. It includes single dose tests followed by multiple doses where participants will eat gluten to see if the drug reduces their immune response.

How Is the Trial Designed?

Group I: VTP-1000 Dose 3 (SAD) Experimental Treatment 1 Intervention

3 dose levels in SAD and MAD parts of trial

Group II: VTP-1000 Dose 3 (MAD) Experimental Treatment 1 Intervention

3 dose levels in SAD and MAD parts of trial

Group III: VTP-1000 Dose 2 (SAD) Experimental Treatment 1 Intervention

3 dose levels in SAD and MAD parts of trial

Group IV: VTP-1000 Dose 2 (MAD) Experimental Treatment 1 Intervention

3 dose levels in SAD and MAD parts of trial

Group V: VTP-1000 Dose 1 (SAD) Experimental Treatment 1 Intervention

3 dose levels in SAD and MAD parts of trial

Group VI: VTP-1000 Dose 1 (MAD) Experimental Treatment 1 Intervention

3 dose levels in SAD and MAD parts of trial

Group VII: Matched Placebo (SAD) Placebo Group 1 Intervention

2 placebo comparators; 1 for each part of the study

Group VIII: Matched Placebo (MAD) Placebo Group 1 Intervention

2 placebo comparators; 1 for each part of the study

Who Is Running the Clinical Trial?

Barinthus Biotherapeutics

Lead Sponsor

Trials

12

Recruited

3,800+

Published Research Related to This Trial

The QUANTA Lite (™) h-tTG/DGP Screen assay demonstrated 100% sensitivity and 91.12% specificity in detecting antibodies related to celiac disease in a study involving 41 celiac patients and 169 controls.

This new assay is considered superior to conventional tests and may serve as the best initial screening tool for celiac disease, potentially reducing the need for invasive duodenal biopsies in certain populations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of a combination screening assay for celiac disease. Porcelli, B., Ferretti, F., Vindigni, C., et al.[2020]

In a study involving 20 patients with celiac disease, pre-treatment with ALV003, a glutenase mixture, effectively prevented immune responses to gluten, as shown by a significant reduction in T-cell responses compared to the placebo group (p=0.011).

Despite the immune response being abolished with ALV003, symptoms related to gluten ingestion were still reported in both groups, indicating that while ALV003 can block immune activation, it may not alleviate all symptoms associated with gluten consumption.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effects of ALV003 pre-digestion of gluten on immune response and symptoms in celiac disease in vivo. Tye-Din, JA., Anderson, RP., Ffrench, RA., et al.[2010]

The IgA anti-tissue transglutaminase (anti-tTG) antibody assay is as reliable as the conventional IgA anti-endomysium antibody (EMA) assay for screening celiac disease in IgA-sufficient children, with a sensitivity of 89% and a negative predictive value of 98%.

Both tests showed high specificity (94% for anti-tTG and 97% for EMA), but the positive predictive value was lower for anti-tTG (67%) compared to EMA (80%), indicating that while both tests are effective, EMA may be slightly more reliable in confirming positive results.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tissue transglutaminase enzyme-linked immunosorbent assay as a screening test for celiac disease in pediatric patients. Chan, AW., Butzner, JD., McKenna, R., et al.[2019]

Celiac disease affects about 1-2% of the population and requires a strict gluten-free diet (GFD) as the only effective treatment, but compliance is low at around 50%.

In children, 95% achieve normalization of duodenal mucosa within two years on a GFD, while adults may take 3-5 years for complete healing, highlighting the need for ongoing monitoring and support.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Descriptive Study of the Different Tools Used to Evaluate the Adherence to a Gluten-Free Diet in Celiac Disease Patients. Rodrigo, L., Pérez-Martinez, I., Lauret-Braña, E., et al.[2019]

In a phase 2 trial involving 41 adults with celiac disease, the glutenase ALV003 significantly protected against gluten-induced mucosal injury, as evidenced by stable villus height to crypt depth ratios and intraepithelial lymphocyte counts compared to the placebo group.

While ALV003 showed efficacy in preventing mucosal damage during a gluten challenge, there were no significant differences in patient symptoms between the ALV003 and placebo groups, indicating that while the treatment may protect the intestine, it does not necessarily alleviate symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Glutenase ALV003 attenuates gluten-induced mucosal injury in patients with celiac disease. Lähdeaho, ML., Kaukinen, K., Laurila, K., et al.[2015]

Citations

1. beyondceliac.org beyondceliac.org/research-news/phase-1-study-assessing-vtp-1000-in-patients-with-celiac-disease/

Phase 1 First-in-Human Study Assessing VTP-1000 ... AVALON is a Phase 1 clinical trial that aims to assess the safety and tolerability of VTP-1000, a potential treatment for celiac disease.

2. trialx.com trialx.com/5-ongoing-breakthrough-clinical-trials-advancing-celiac-disease-research/

5 Ongoing Breakthrough Clinical Trials Advancing Celiac ... The study is primarily assessing safety, tolerability, and immune response to VTP-1000 in adults who are already following a well-controlled ...

3. investors.barinthusbio.com investors.barinthusbio.com/news-releases/news-release-details/barinthus-bio-reports-first-quarter-2025-financial-results-and

Barinthus Bio Reports First Quarter 2025 Financial Results ... Looking ahead, we remain on track to announce Phase 1 single ascending dose data for the celiac program using VTP-1000 in the third quarter of ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06310291

NCT06310291 | VTP-1000 in Adults With Celiac Disease GLU001 is a first-in-human clinical trial to assess the safety and tolerability of VTP-1000 for adults with celiac disease. This trial will assess VTP-1000 ...

5. celiac.org celiac.org/about-celiac-disease/future-therapies-for-celiac-disease/

Future Therapies for Celiac Disease VTP-1000 is an antigen-specific immunotherapy utilizing Barinthus Bio's SNAP Tolerance Platform to promote immune tolerance to gluten. The Celiac Disease ...

6. investors.barinthusbio.com investors.barinthusbio.com/news-releases/news-release-details/barinthus-bio-initiates-phase-1-clinical-trial-vtp-1000

Barinthus Bio Initiates Phase 1 Clinical Trial of VTP-1000 ... The first-in-human Phase 1 trial aims to evaluate the safety and tolerability in adults with celiac disease. OXFORD, United Kingdom, Sept ...

7. celiac.org celiac.org/2024/08/27/investigating-immune-tolerance-in-celiac-disease-avalon-clinical-trial/

AVALON Clinical Trial AVALON is a phase 1 clinical trial that aims to assess the safety and tolerability of the investigational drug VTP-1000 in adults with celiac disease.

8. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/595937

VTP-1000 in Adults With Celiac Disease GLU001 is a first-in-human clinical trial to assess the safety and tolerability of VTP-1000 for adults with celiac disease.

9. trialx.com trialx.com/clinical-trials/listings/309024/vtp-1000-in-adults-with-celiac-disease/

VTP-1000 in Adults With Celiac Disease Overview. GLU001 is a first-in-human clinical trial to assess the safety and tolerability of VTP-1000 for adults with celiac disease.