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Pemphigoid, BullousEarly Phase 1NCT04465292

The Effects of Tildrakizumab in Treatment of Bullous Pemphigoid

A Early Phase 1 study testing Tildrakizumab Prefilled Syringe in people with Pemphigoid, Bullous, run by Brigham and Women's Hospital.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

This is an open-label, pilot study evaluating the efficacy of tildrakizumab on the treatment of bullous pemphigoid (BP) in eligible patients (see detailed study protocol). Three total doses of tildrakizumab 100mg will be administered at Weeks 0, 4, 16 a total of 16 weeks of treatment by the study staff to patients… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pemphigoid, Bullous
What is being tested?
Tildrakizumab Prefilled Syringe
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
No sites listed yet
Study dates
1 Sep 2020to 1 May 2023
Phase
Early 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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • At least 18 years of age
  • Diagnosis of diffuse bullous pemphigoid clinically and confirmed on H\&E, DIF +/- IIF/ELISA
  • Able and willing to provide informed consent, participate in study visits, and undergo visit procedures

Who cannot join

  • Unusual and localized variant of bullous pemphigoid will be excluded, these include but not limited to, BP sine rash, dyshidrosiform pemphigoid, pemphigoid vegetans, pemphigoid nodularis, lichen planus pemphigoid, pretibial BP, vulva BP, peristomal BP, umbilical BP, Brunsting-Perry, BP localized only to sites of injury, radiation, amputation, paralysis, or other underlying dermatosis.
  • Prior tildrakizumab use.
  • Treatment with a systemic immune-regulating nonsteroidal medication(s) within 6 weeks of the baseline visit, or use of systemic steroid within 2 weeks of baseline visit. Examples of systemic immune-regulating nonsteroidal medications include azathioprine, methotrexate, mycophenolate mofetil, cyclosporine, intravenous immunoglobulins, dapsone, colchicine, sulfasalazine, or omalizumab.
  • Treatment with other biologic agents, such as TNF inhibitors, anti-IL17 agents, anti-IL12/23 agents, anti-IL4/13, anti-IL5, anti-IL23 agents or anti-eotaxin, within 4 months of baseline visit.
  • Use of rituximab within at 6 months (or until lymphocyte counts by CD19 and CD20 have normalized if longer than 6 months) of the baseline visit.
  • Concurrent use of any medication that may affect the efficacy of tildrakizumab.
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  • Use of belimumab within the past year (given tildrakizumab may enhance the adverse effect of belimumab)
  • Other active conditions, such as psoriasis or contact dermatitis, that may confound clinical evaluations of dermatitis and patient-reported symptoms.
  • Increased risk of infection or reactivated infection, including history of human immunodeficiency virus, hepatitis B, hepatitis C, endoparasitic infections, receipt of a live attenuated vaccine within 3 months of the baseline visit, chronic or acute infection requiring treatment within 4 weeks of the baseline visit, baseline immunodeficiency status otherwise nonspecified (i.e. history of recurrent or resistant infections)
  • History of malignancy excluding local cutaneous squamous cell carcinoma, basal cell carcinoma or cervical carcinoma in situ that has been fully treated.
  • Women who are or plan to become pregnant or breastfeed during study participation or are unable or not willing to use birth control during the study and for 4 months after the last dose of tildrakizumab. Options for birth control include abstinence, double barrier (i.e. male condom and female diaphragm), vasectomy, intrauterine device, and hormonal contraception. Females who have not had menses within 1 year of the baseline, bilateral tubal ligation, hysterectomy, and/or bilateral oophorectomy visit do not require additional methods contraception during study participation.
  • Unstable condition or status, as per study investigator's judgment, that may lead to more likely discontinuation from the study including but not limited to major, recurrent medical illnesses that may require hospital admission and/or discontinuation of tildrakizumab, surgery that would require discontinuation of tildrakizumab and/or major rehabilitation, inability to participate in all study visits.

Trial locations and contacts

No sites are listed yet.

Liset Chacin contact

617-264-5926

lchacin@bwh.harvard.edu

Alex Gionfriddo contact

agiofriddo@bwh.harvard.edu

About this study

This is an open-label, pilot study evaluating the efficacy of tildrakizumab on the treatment of bullous pemphigoid (BP) in eligible patients (see detailed study protocol). Three total doses of tildrakizumab 100mg will be administered at Weeks 0, 4, 16 a total of 16 weeks of treatment by the study staff to patients with bullous pemphigoid. The patients will be followed for a total of 24 weeks.

Read the detailed description

The investigators will recruit 16 patients with bullous pemphigoid 18 years of age and older with confirmed diagnosis of bullous pemphigoid on biopsy (both H\&E and Direct Immunofluorescence (DIF) +/- IIF and ELISA) At the initial screening visit, demographic information will be obtained, inclusion and exclusion criteria, and informed consent obtained for those deemed eligible for enrollment. Three total doses of Tildrakizumab 100mg will be administered at Weeks 0, 4, 16 a total of 16 weeks of treatment by the study staff. Subjects will be evaluated for improvement in primary and secondary endpoints using clinical examination and questionnaires during initial and follow-up visits at screening, Weeks 0, 4, 16, and 24, a total of 24 week followup period.

Being tested
Tildrakizumab Prefilled Syringe
Medical subject headings
Pemphigoid, Bullous

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Tildrakizumab Prefilled SyringeDrug

    Patients will receive Tildrakizumab 100 mg subcutaneously at Weeks 0, 4, and 16. The study staff will administer each dose.

Study groups in NCT04465292
Study groupRoleWhat they receive
InterventionExperimentalParticipants will receive Tildrakizumab 100mg at Weeks 0, 4, 16; three doses; a 16-week treatment course and 24-week followup
Receives: Drug: Tildrakizumab Prefilled Syringe

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in Disease Severity

    Disease severity category (mild: 1-9 lesion(s), moderate: 10-30 lesions, and severe: > 30

    Measured: Weeks 0, 4, 16, and 24

Secondary outcomes (6)

  1. Change in Bullous Pemphigoid Disease Activity Index (BPDAI)

    The BPDAI tool (9) computes 2 scores: total BPDAI activity and total BPDAI damage. The total BPDAI activity score is the arithmetic sum of the 3 subcomponents - cutaneous blisters/ erosions, cutaneous urticaria/erythema, and mucosal blisters/ erosions. The total BPDAI damage score is the arithmetic sum of the items rated regionally for damage caused by more permanent features such as post-inflammatory hyperpigmentation, scarring and other. BPDAI quantifies lesion number and size thresholds. Lesions are rated based on the regions affected. BPDAI gives additional weighting to areas of the skin primarily affected in BP, such as the limbs, and less emphasis to scalp and face, to better differentiate clinical response in BP. Scores can range from 0 to 360 for BPDAI total activity (maximum 240 for total skin activity and 120 for mucosal activity), and 0 to 12 for BPDAI damage, with higher scores indicating greater disease activity or damage.

    Measured: Weeks 0, 4, 16, and 24

  2. Change in BPDAI-Pruritus

    No evidence of itch (no excoriations) 0 Mild itch (isolated excoriation up to two body sites) 10 Moderate itch (excoriation on ≥ 3 body sites, impairment of daily activity 20 Severe itch (Generalized excoriation, sleep impairment) 30

    Measured: Weeks 0, 4, 16, and 24

  3. Change in Dermatology Life Quality Index (DLQI)

    Each question is scored on a four-point Likert scale:

    Very much = 3 A lot = 2 A little = 1 Not at all = 0 Not relevant = 0 Question unanswered = 0 The DLQI is calculated by adding the score of each question, resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. A score higher than 10 indicates that the patient's life is being severely affected by their skin disease.

    Meaning of scores 0-1 = no effect at all on patient's life 2-5 = small effect on patient's life 6-10 = moderate effect on patient's life 11-20 = very large effect on patient's life 21-30 = extremely large effect on patient's life

    Measured: Weeks 0, 4, 16, and 24

  4. anti-BP180, BP230 IgG

    Measured: Weeks 0, 4, 16, and 24

  5. Total IgE

    Measured: Weeks 0, 4, 16, and 24

  6. Cytokine level

    Measured: Weeks 0, 4, 16, and 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Jul 2020
  2. Study start1 Sep 2020 (estimated)
  3. Primary completion1 Dec 2022 (estimated)
  4. Study completion1 May 2023 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04465292
Sponsor's protocol ID
2020P000025
Status last verified
Jul 2020
First submitted
14 May 2020
Last updated
10 Jul 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Tildrakizumab Prefilled Syringe in people with Pemphigoid, Bullous, run by Brigham and Women's Hospital.

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 3 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 10 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. 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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Liset Chacin (617-264-5926, lchacin@bwh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Disease Severity (time frame: Weeks 0, 4, 16, and 24). 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 NCT04465292.

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 10 Jul 2020.

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