RecruitingPhase 3

Randomized Clinical Trial Comparing the Safety and Efficacy of Rituximab Versus Oral Cyclophosphamide in Severe Forms of Mucous Membrane Pemphigoid

France130 participantsStarted 2019-07-11

Plain-language summary

Mucous membrane pemphigoid (MMP) describes a group of chronic auto-immune bullous diseases (AIBD) of the basement membrane zone (BMZ), characterized by predominant or exclusive mucosal involvement, including oral, naso-pharyngeal, laryngo-tracheal, genital, oesophageal, anal and ocular mucous membranes. Circulating autoantibodies are directed against various antigens of the BMZ: BP180, laminin 332 and type 7 collagen. MMP is a rare disease with an incidence of 1.8 new cases/million inhabitants/year in France. Scar formation which is secondary to initial inflammation, is a characteristic feature of MMP, leading to major disability (e.g blindness and oesophageal, anal, vaginal stenosis) and life-threatening situations (e.g. laryngeal stenosis leading to respiratory failure). Dapsone is the first line treatment of mild/moderate forms of MMP. Dapsone is used both as initial treatment, and as maintenance therapy. However, severe forms of MMP can rapidly worsen leading to blindness, aphagia due to esophageal stenosis, respiratory failure due to tracheal or laryngeal stenosis, and urinary and sexual dysfunctions due to genital involvement. These patients are usually treated using immunosuppressive drugs. Indeed, corticosteroids (CS) are not recommended in MMP. Cyclophosphamide was considered as the most effective immunosuppressant in severe forms of MMP, before the use of rituximab, an anti-CD20 monoclonal antibody (MAb). Two series from our group have assessed the advantages and disadvantages of IV pulse and oral administration of cyclophosphamide in MMP. Oral administration seems more rapidly effective with 54% of complete remission (CR) after a median time of 24 weeks (16-52 weeks). The results of 41 patients with severe types of MMP (including a French series of 20 patients) treated with rituximab have been published. Rate of CR after one and two cycles were 66% and 90%, respectively. Clinical improvement was rapid, since a decrease in disease activity was observed after 4 weeks of treatment in 64% of patients. Our results and those of the literature suggest that rituximab might be more effective than cyclophosphamide, which has been considered as the gold standard of treatment in severe forms of disease, up to now.

Who can participate

Age range18 Years – 80 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female patients aged ≥18 years old and ≤ 80 years old with a newly diagnosed or previously diagnosed severe MMP diagnosed according to the International MMP Consensus (Chan 2002) on the following criteria:
✓. MMP is defined as "severe" in patients with:
✓. Patient having read and understood the information letter and signed the Informed Consent Form
✓. Patient with updated vaccinations. It is recommended that a patient's vaccination record and the need for immunization prior to study entry be carefully investigated.
✓. For women who are not postmenopausal (≥12 months of non-therapy-induced amenorrhoea) or surgically sterile (absence of ovaries and/or uterus) and who do not plan on having children anymore: agreement to remain abstinent or use two adequate methods of contraception, including at least one method with a failure rate of \<1% per year, during the treatment period and for at least 12 months after the last dose of study treatment.
✓. Patient agreement to avoid excessive exposure to sunlight during study participation
✓. Patient able to comply with the study protocol, in the investigator's judgment
✓. Patient affiliated with, or beneficiary of a social security category

What they're measuring

Proportion of patients achieving CR or Partial Remission (PR)

Timeframe: Month 12

Trial details

NCT IDNCT03295383

SponsorUniversity Hospital, Rouen

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-03

Contact for this trial

Pascal JOLY, Pr

+3323288 pascal.joly@chu-rouen.fr

View on ClinicalTrials.gov More Severe Forms of Mucous Membrane Pemphigoid trials

Plain-language summary

Who can participate

Age range18 Years – 80 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Male or female patients aged ≥18 years old and ≤ 80 years old with a newly diagnosed or previously diagnosed severe MMP diagnosed according to the International MMP Consensus (Chan 2002) on the following criteria:
✓. MMP is defined as "severe" in patients with:
✓. Patient having read and understood the information letter and signed the Informed Consent Form
✓. Patient with updated vaccinations. It is recommended that a patient's vaccination record and the need for immunization prior to study entry be carefully investigated.
✓. For women who are not postmenopausal (≥12 months of non-therapy-induced amenorrhoea) or surgically sterile (absence of ovaries and/or uterus) and who do not plan on having children anymore: agreement to remain abstinent or use two adequate methods of contraception, including at least one method with a failure rate of \<1% per year, during the treatment period and for at least 12 months after the last dose of study treatment.
✓. Patient agreement to avoid excessive exposure to sunlight during study participation
✓. Patient able to comply with the study protocol, in the investigator's judgment
✓. Patient affiliated with, or beneficiary of a social security category

Randomized Clinical Trial Comparing the Safety and Efficacy of Rituximab Versus Oral Cyclophosphamide in Severe Forms of Mucous Membrane Pemphigoid

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Contact for this trial

Randomized Clinical Trial Comparing the Safety and Efficacy of Rituximab Versus Oral Cyclophosphamide in Severe Forms of Mucous Membrane Pemphigoid

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Contact for this trial

Exclusion criteria