Celiac DiseasePhase 1NCT02528799
Safety and Tolerability of Nexvax2 in Subjects With Celiac Disease
A Phase 1 study testing Nexvax2 in people with Celiac Disease, run by ImmusanT, Inc. at 4 sites in 2 countries.
Trial summary
A randomized, double-blind, placebo-controlled, dose titration trial, stratified by Human Leukocyte Antigen (HLA)-DQ2.5 genotype in subjects with celiac disease. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Celiac Disease
- What is being tested?
- Nexvax2
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 38 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Aug 2015to 6 Jan 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject has signed and understands the informed consent form before initiation of any study specific procedures.
- Subject is between 18 and 70 years old (inclusive) on the date of the Screening Visit.
- Subject has been diagnosed with celiac disease on the basis of intestinal histology showing villous atrophy according to expert guidelines current at the time of diagnosis.
- Subject has HLA DQ2.5 genotype (HLA-DQA1*05 and HLA-DQB1*02).
Who cannot join
- Subject has not been maintained on a gluten-free diet for at least 1 year.
- Celiac Dietary Adherence Test at screening indicates non-compliance to gluten-free diet (score >12).
- Serum levels of both recombinant human transglutaminase (tTG)-specific immunoglobulin-A (IgA) and deamidated gliadin peptide (DGP)-specific immunoglobulin-G (IgG) are elevated above the manufacturer's upper limit of normal. The elevation of one or other of the serology test for tTG IgA and DGP IgG is not an exclusion.
- Subject has uncontrolled complications of celiac disease or a medical condition which, in the opinion of the investigator, would impact the immune response or pose an increased risk to the subject.
- Subject is or has been using an immuno-modulatory or immune suppressing medical treatment during the 2 months prior to Screening, for example azathioprine, methotrexate, or biological
- Subject is female and premenopausal or perimenopausal and has a male partner who is not sterile, unless she is sterile, or she practices true abstinence, or unless throughout the entire study period and for 30 days after study drug discontinuation she is using a medically acceptable method of contraception.
Show all 24Show fewer
- Subject is male with a premenopausal or perimenopausal female partner who is not sterile, unless he is sterile, or he practices true abstinence, or unless throughout the entire study period and for 30 days after study drug discontinuation he is using a medically acceptable method of contraception, or unless his female partner is using a medically acceptable method of contraception.
- Subject is unable and/or unwilling to comply with study requirements.
- Subject has taken oral or parenteral corticosteroids within the previous six weeks prior to Screening. Topical or inhaled corticosteroids are acceptable.
- Subject has received an experimental therapy within 30 days prior to Screening.
- Subject has previously been enrolled and dosed in a clinical trial with Nevax2.
- Subject has any of the following laboratory abnormalities at Screening:
- Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) ≥ 2 × the upper limit of normal (ULN)
- Hemoglobin <10 g/dL
- Platelet count <100 × 109/L
- White blood cell count (WBC) outside the normal range and judged clinically significant by the investigator
- Direct bilirubin outside the normal range
- Any other clinically significant abnormal laboratory values, as determined by the investigator
- Subject is lactating, is known to be pregnant, has a positive pregnancy test at Screening or Treatment Day, intends to become pregnant, or is nursing.
- Subject has a history or presence of any medically significant condition considered by the investigator to have the potential to adversely affect participation in the study and/or interpretation of the study results.
- Subject has a history of severe allergic reactions (e.g., swelling of the mouth and throat, difficulty breathing, hypotension, or shock) that require medical intervention.
- Subject has donated blood ≤ 56 days prior to Screening and plans to donate blood within 5 weeks after study completion.
- Subject has a clinically relevant abnormality on electrocardiogram (ECG), as determined by the investigator.
- Other unspecified reasons that in the opinion of the investigator or the sponsor make the subject unsuitable for enrollment.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Australia3 sites
CMAX, A Division of IDT Australia Ltd
Adelaide, South Australia, 5000
Q-Pharm Pty Ltd.
Herston, Queensland, 4006
Linear Clinical Research
Nedlands, WA 6009
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Tolerability of Nexvax2 Preceded by a Dose Titration Period in Subjects With Celiac Disease Currently on a Gluten-Free Diet
A randomized, double-blind, placebo-controlled, dose titration trial, stratified by Human Leukocyte Antigen (HLA)-DQ2.5 genotype in subjects with celiac disease.
Read the detailed description
This is a randomized, double-blind, placebo-controlled, study to evaluate the safety and tolerability of Nexvax2 preceded by dose titration period in patients with celiac disease currently on a gluten-free diet. The study will consist of a Screening Period, a Treatment Period, and a Follow-up Period. Eligible subjects will be enrolled in one of three cohorts according to whether they are homozygous or not homozygous for both genes coding for HLA-DQ2.5.
- Conditions
- Celiac Disease
- Being tested
- Nexvax2, Nexvax2 placebo
- Medical subject headings
- Celiac Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nexvax2Biological
Nexvax2 intra-dermal injections twice weekly
Nexvax2 placeboBiological
Sodium chloride 0.9% intra-dermal injections twice weekly
| Study group | Role | What they receive |
|---|---|---|
| Nexvax2 DQ2.5 Homozygotes (Cohort 1) | Experimental | Nexvax2 by ID injections for a total of 14 doses over 46 days. Receives: Biological: Nexvax2 |
| Nexvax2 Placebo DQ2.5 Homozygotes (Cohort 1) | Placebo comparator | Nexvax2 Placebo by ID injections for a total of 14 doses over 46 days. Receives: Biological: Nexvax2 placebo |
| Nexvax2 DQ2.5 Non-homozygotes (Cohort 2) | Experimental | Nexvax2 by ID injections for a total of 14 doses over 46 days. Receives: Biological: Nexvax2 |
| Nexvax2 Placebo DQ2.5 Non-homozygotes (Cohort 2) | Placebo comparator | Nexvax2 Placebo by ID injections for a total of 14 doses over 46 days. Receives: Biological: Nexvax2 placebo |
| Nexvax2 DQ2.5 Non-homozygotes (Cohort 3) | Experimental | Nexvax2 by ID injections for 18 doses (up to 27 doses) over 60 days (maximum of 91 days). Receives: Biological: Nexvax2 |
| Nexvax2 Placebo DQ2.5 Non-homozygotes (Cohort 3) | Placebo comparator | Nexvax2 Placebo by ID injections for 18 doses (up to 27 doses) over 60 days (maximum of 91 days). Receives: Biological: Nexvax2 placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of toxicity and safety of Nexvax2 according to the "Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0"
Number and Percentage of Participants with Treatment-related Adverse Events assessed by the "Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0 will be tabulated using counts and proportions detailing frequently occurring, serious and severe events. Adverse events will be summarized using all adverse events experienced, although a sub-analysis may be conducted including only those adverse events in which the treating physician deems possibly, probably or definitely attributable to study treatments.
Measured: Treatment Period (~7 to 9 weeks)
Secondary outcomes (2)
Weekly Gastrointestinal Symptom Rating Scale (GSRS)
The GSRS score is the average weekly scores for 15 symptoms rated on a 7-point severity scale. GSRS scores over the 6-week Treatment Period will be compared.
Measured: Treatment Period (~7 to 9 weeks)
Plasma Cytokine Levels
The relative change from pre-dose levels up to 10 hours after dosing in the concentration of plasma cytokines.
Measured: Treatment Period (~7 to 9 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2015 (estimated)
- Study startAug 2015
- Primary completion6 Jan 2017
- Study completion6 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Celiac DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- ImmusanT, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert P. Anderson, MB ChB, PhD, Study chair, ImmusanT, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02528799
- Sponsor's protocol ID
- Nexvax2-1004
- Other IDs
- U1111-1173-7522
- Status last verified
- Apr 2017
- First submitted
- 17 Aug 2015
- Last updated
- 24 Dec 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Daveson AJM, Ee HC, Andrews JM, King T, Goldstein KE, Dzuris JL, MacDougall JA, Williams LJ, Treohan A, Cooreman MP, Anderson RP. Epitope-Specific Immunotherapy Targeting CD4-Positive T Cells in Celiac Disease: Safety, Pharmacokinetics, and Effects on Intestinal Histology and Plasma Cytokines with Escalating Dose Regimens of Nexvax2 in a Randomized, Double-Blind, Placebo-Controlled Phase 1 Study. EBioMedicine. 2017 Dec;26:78-90. doi: 10.1016/j.ebiom.2017.11.018. Epub 2017 Nov 22.
PubMed 29191561
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Tolerability of Nexvax2 Preceded by a Dose Titration Period in Subjects With Celiac Disease Currently on a Gluten-Free Diet
- ClinicalTrials.gov ID
- NCT02528799
- Acronym / sponsor ID
- Nexvax2-1004
- Conditions
- Celiac Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- ImmusanT, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2015
- Primary completion
- 6 Jan 2017 (actual)
- Study completion
- 6 Jan 2017 (actual)
- First posted
- 19 Aug 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A randomized, double-blind, placebo-controlled, dose titration trial, stratified by Human Leukocyte Antigen (HLA)-DQ2.5 genotype in subjects with celiac disease.
Detailed description
This is a randomized, double-blind, placebo-controlled, study to evaluate the safety and tolerability of Nexvax2 preceded by dose titration period in patients with celiac disease currently on a gluten-free diet. The study will consist of a Screening Period, a Treatment Period, and a Follow-up Period. Eligible subjects will be enrolled in one of three cohorts according to whether they are homozygous or not homozygous for both genes coding for HLA-DQ2.5.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Nexvax2 DQ2.5 Homozygotes (Cohort 1) | Experimental | Biological: Nexvax2Nexvax2 by ID injections for a total of 14 doses over 46 days. |
| Nexvax2 Placebo DQ2.5 Homozygotes (Cohort 1) | Placebo comparator | Biological: Nexvax2 placeboNexvax2 Placebo by ID injections for a total of 14 doses over 46 days. |
| Nexvax2 DQ2.5 Non-homozygotes (Cohort 2) | Experimental | Biological: Nexvax2Nexvax2 by ID injections for a total of 14 doses over 46 days. |
| Nexvax2 Placebo DQ2.5 Non-homozygotes (Cohort 2) | Placebo comparator | Biological: Nexvax2 placeboNexvax2 Placebo by ID injections for a total of 14 doses over 46 days. |
| Nexvax2 DQ2.5 Non-homozygotes (Cohort 3) | Experimental | Biological: Nexvax2Nexvax2 by ID injections for 18 doses (up to 27 doses) over 60 days (maximum of 91 days). |
| Nexvax2 Placebo DQ2.5 Non-homozygotes (Cohort 3) | Placebo comparator | Biological: Nexvax2 placeboNexvax2 Placebo by ID injections for 18 doses (up to 27 doses) over 60 days (maximum of 91 days). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Subject has signed and understands the informed consent form before initiation of any study specific procedures.
- Subject is between 18 and 70 years old (inclusive) on the date of the Screening Visit.
- Subject has been diagnosed with celiac disease on the basis of intestinal histology showing villous atrophy according to expert guidelines current at the time of diagnosis.
- Subject has HLA DQ2.5 genotype (HLA-DQA1*05 and HLA-DQB1*02).
Key exclusion criteria (24 listed)
- Subject has not been maintained on a gluten-free diet for at least 1 year.
- Celiac Dietary Adherence Test at screening indicates non-compliance to gluten-free diet (score >12).
- Serum levels of both recombinant human transglutaminase (tTG)-specific immunoglobulin-A (IgA) and deamidated gliadin peptide (DGP)-specific immunoglobulin-G (IgG) are elevated above the manufacturer's upper limit of normal. The elevation of one or other of the serology test for tTG IgA and DGP IgG is not an exclusion.
- Subject has uncontrolled complications of celiac disease or a medical condition which, in the opinion of the investigator, would impact the immune response or pose an increased risk to the subject.
- Subject is or has been using an immuno-modulatory or immune suppressing medical treatment during the 2 months prior to Screening, for example azathioprine, methotrexate, or biological
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nexvax2Nexvax2 intra-dermal injections twice weekly | Biological | Nexvax2 DQ2.5 Homozygotes (Cohort 1), Nexvax2 DQ2.5 Non-homozygotes (Cohort 2), Nexvax2 DQ2.5 Non-homozygotes (Cohort 3) |
| Nexvax2 placeboSodium chloride 0.9% intra-dermal injections twice weekly | Biological | Nexvax2 Placebo DQ2.5 Homozygotes (Cohort 1), Nexvax2 Placebo DQ2.5 Non-homozygotes (Cohort 2), Nexvax2 Placebo DQ2.5 Non-homozygotes (Cohort 3) |
Primary outcome
Incidence of toxicity and safety of Nexvax2 according to the "Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0"
Time frame: Treatment Period (~7 to 9 weeks)
Number and Percentage of Participants with Treatment-related Adverse Events assessed by the "Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0 will be tabulated using counts and proportions detailing frequently occurring, serious and severe events. Adverse events will be summarized using all adverse events experienced, although a sub-analysis may be conducted including only those adverse events in which the treating physician deems possibly, probably or definitely attributable to study treatments.
2 secondary outcomes
Weekly Gastrointestinal Symptom Rating Scale (GSRS)
Time frame: Treatment Period (~7 to 9 weeks)
Plasma Cytokine Levels
Time frame: Treatment Period (~7 to 9 weeks)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2018.
- Derived from this study: PubMed 29191561Daveson AJM, Ee HC, Andrews JM, King T, Goldstein KE, Dzuris JL, MacDougall JA, Williams LJ, Treohan A, Cooreman MP, Anderson RP. Epitope-Specific Immunotherapy Targeting CD4-Positive T Cells in Celiac Disease: Safety, Pharmacokinetics, and Effects on Intestinal Histology and Plasma Cytokines with Escalating Dose Regimens of Nexvax2 in a Randomized, Double-Blind, Placebo-Controlled Phase 1 Study. EBioMedicine. 2017 Dec;26:78-90. doi: 10.1016/j.ebiom.2017.11.018. Epub 2017 Nov 22.
- MedlinePlusLink provided by the sponsor.
- ImmusanT, Inc.Link provided by the sponsor.
- Publication of ResultsLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert P. Anderson, MB ChB, PhD study chair
ImmusanT, Inc.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nexvax2 in people with Celiac Disease, run by ImmusanT, Inc. at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of toxicity and safety of Nexvax2 according to the "Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0" (time frame: Treatment Period (~7 to 9 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02528799, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Dec 2018.