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NCT00784589 · CITED IN SOURCE DOCUMENTS

Comparison Between Rituximab Treatment and General Corticotherapy Treatment in Patients With Pemphigus

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
PHASE3
Status at capture
COMPLETED
Registry last update
2017-06-14

Pemphigus is a severe and sometimes life-threatening disease with a mortality rate between 5 and 10 percent depending on the severity of disease and age of patients. The standard of care is high doses of corticosteroids (CS) (usually, prednisone, 1 to 1.5. mg/kg/day, which are often associated with immunosuppressive drugs i.e., azathioprine, mycophenolate mofetil, cyclophosphamide, cyclosporine, although only one randomised study has demonstrated the superiority of the combination of corticosteroids and immunosuppressive drugs as compared to corticosteroids alone (9). Because of the low frequency of the disease, control trials are difficult to conduct. Evaluation of the different treatment regimens proposed, i;e; corticosteroids alone, CS and immunosuppressive drugs, IV immunoglobulins or new therapeutic regimens such as rituximab (an anti-CD20 monoclonal antibody directed against B- lymphocytes) or immunoadsorbtion.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

No posted result sections were captured. Registered plans do not establish that a treatment works.

Who could take part
eligibility Criteria
Inclusion Criteria: * age \>=18 and \<= 80years * consent obtained from patient * effective female contraceptive method for women in procreate age * new case of pemphigus vulgaris (PV) or pemphigus foliaceus (PF) Exclusion Criteria: * pemphigus vulgaris (PV) or pemphigus foliaceus (PF) treated * pregnant woman or nursing mother * woman able to have a baby and without contraception during the clinical trial period * age \< 18 or \> 80 * karnovsky \< 50% * serious antecedents of allergy or anaphylactic reaction with human monoclonal antibody * patient with depletion lymphocytic treatment in the next month * unstable angina or ischemic heart disease * cardiac insufficiency * cardiac rhythm trouble uncontrolled * positive HIV serology * positive hepatitis B and / or C serology * no consent
healthy Volunteers
false
maximum Age
80 Years
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. intervention Names
    1. Drug: Rituximab
    label
    Rituximab
    type
    EXPERIMENTAL
  2. description
    General Corticotherapy
    intervention Names
    1. Drug: General Corticotherapy
    label
    Corticotherapy
    type
    ACTIVE_COMPARATOR
interventions
  1. arm Group Labels
    1. Corticotherapy
    description
    for patients with severe pemphigus : general corticotherapy Arm : 1.5mg/kg/d for 1 month, then 1.25mg/kg/d for 1 month, then 1mg/kg/d for 1 month, then 0.75 mg/kg/d for 1 month, then 0.5 mg/kg/d for 3 months, then 0.3 mg/kg/d for 3 months, then 0.2 mg/kg/d for 4 months, then 0.1 mg/kg/d for 4 months, for patients with moderated pemphigus : general corticotherapy Arm : 1mg/kg/d for 1 month, then 0.75mg/kg/d for 1 month, then 0.5mg/kg/d for 2 months, then 0.3 mg/kg/d for 2 months, then 0.2 mg/kg/d for 3 months, then 0.1 mg/kg/d for 3 months,
    name
    General Corticotherapy
    type
    DRUG
  2. arm Group Labels
    1. Rituximab
    description
    for patients with severe pemphigus : rituximab Arm : 1mg/kg/d for 1 month, then 0.75 mg/kg/d for 1 month, then 0.5 mg/kg/d for 1 month, then 0.3 mg/kg/d for 1 month, then 0.2 mg/kg/d for 1 month, then 0.1 mg/kg/d for 1 month for patients with moderated pemphigus : rituximab Arm : 0.5mg/kg/d for 1 month, then 0.30 mg/kg/d for 1 month, then 0.2 mg/kg/d for 1 month,
    name
    Rituximab
    type
    DRUG
Study design
allocation
RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
90
type
ESTIMATED
Registered outcome plans (not posted results)
primary Outcomes
  1. measure
    Number of patients with pemphigus controlled 24 months after the start of Rituximab treatment
    time Frame
    2 years
secondary Outcomes
  1. measure
    Number of patients with pemphigus with both cutaneous and mucosal lesions healing after 6 months of rituximab treatment
    time Frame
    6 months
Full study description
brief Summary
Pemphigus is a severe and sometimes life-threatening disease with a mortality rate between 5 and 10 percent depending on the severity of disease and age of patients. The standard of care is high doses of corticosteroids (CS) (usually, prednisone, 1 to 1.5. mg/kg/day, which are often associated with immunosuppressive drugs i.e., azathioprine, mycophenolate mofetil, cyclophosphamide, cyclosporine, although only one randomised study has demonstrated the superiority of the combination of corticosteroids and immunosuppressive drugs as compared to corticosteroids alone (9). Because of the low frequency of the disease, control trials are difficult to conduct. Evaluation of the different treatment regimens proposed, i;e; corticosteroids alone, CS and immunosuppressive drugs, IV immunoglobulins or new therapeutic regimens such as rituximab (an anti-CD20 monoclonal antibody directed against B- lymphocytes) or immunoadsorbtion.
Source references
references
  1. citation
    Mignard C, Maho-Vaillant M, Golinski ML, Balaye P, Prost-Squarcioni C, Houivet E, Calbo SB, Labeille B, Picard-Dahan C, Konstantinou MP, Chaby G, Richard MA, Bouaziz JD, Duvert-Lehembre S, Delaporte E, Bernard P, Caux F, Alexandre M, Ingen-Housz-Oro S, Vabres P, Quereux G, Dupuy A, Debarbieux S, Avenel-Audran M, D'Incan M, Bedane C, Beneton N, Jullien D, Dupin N, Misery L, Machet L, Beylot-Barry M, Dereure O, Sassolas B, Benichou J, Joly P, Hebert V; French Study Group on Autoimmune Bullous Skin Diseases. Factors Associated With Short-term Relapse in Patients With Pemphigus Who Receive Rituximab as First-line Therapy: A Post Hoc Analysis of a Randomized Clinical Trial. JAMA Dermatol. 2020 May 1;156(5):545-552. doi: 10.1001/jamadermatol.2020.0290.
    pmid
    32186656
    type
    DERIVED
  2. citation
    Joly P, Maho-Vaillant M, Prost-Squarcioni C, Hebert V, Houivet E, Calbo S, Caillot F, Golinski ML, Labeille B, Picard-Dahan C, Paul C, Richard MA, Bouaziz JD, Duvert-Lehembre S, Bernard P, Caux F, Alexandre M, Ingen-Housz-Oro S, Vabres P, Delaporte E, Quereux G, Dupuy A, Debarbieux S, Avenel-Audran M, D'Incan M, Bedane C, Beneton N, Jullien D, Dupin N, Misery L, Machet L, Beylot-Barry M, Dereure O, Sassolas B, Vermeulin T, Benichou J, Musette P; French study group on autoimmune bullous skin diseases. First-line rituximab combined with short-term prednisone versus prednisone alone for the treatment of pemphigus (Ritux 3): a prospective, multicentre, parallel-group, open-label randomised trial. Lancet. 2017 May 20;389(10083):2031-2040. doi: 10.1016/S0140-6736(17)30070-3. Epub 2017 Mar 22.
    pmid
    28342637
    type
    DERIVED
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        if they had no event. Figure 3 Cumulative Incidence Over Time of First Major Relapse in Patients with GPA/MPA Image 3 14.8 Pemphigus Vulgaris (PV) PV Study 1 (NCT00784589) Non-U.S.-licensed rituximab in combination with short-term prednisone was compared to prednisone monotherapy as first-line treatment in 90 newly diagnosed adu
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        jor Relapse in Patients with GPA/MPA Patients were censored at the last follow-up dates if they had no event Figure 3 14.8 Pemphigus Vulgaris (PV) PV Study 1 ( NCT00784589 ) Non-U.S.-licensed rituximab in combination with short-term prednisone was compared to prednisone monotherapy as first-line treatment in 90 newly diagnosed ad
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        jor Relapse in Patients with GPA/MPA Patients were censored at the last follow-up dates if they had no event. Figure 3 14.8 Pemphigus Vulgaris (PV) PV Study 1 (NCT00784589) Non-U.S.-licensed rituximab in combination with short-term prednisone was compared to prednisone monotherapy as first-line treatment in 90 newly diagnosed adu
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        y every 6 months. The safety profile was consistent with the safety profile for rituximab in RA and GPA and MPA. Pemphigus Vulgaris (PV) PV Study 1 PV Study 1 (NCT00784589), a randomized, controlled, multicenter open-label study, evaluated the efficacy and safety of non-U.S.-licensed rituximab in combination with short-term predn
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        g 15 patients who also had prolonged low IgM. Three patients received treatment with intravenous immunoglobulin. Pemphigus Vulgaris (PV) PV Study 1 PV Study 1 (NCT00784589), a randomized, controlled, multicenter open-label study, evaluated the efficacy and safety of non-U.S.-licensed rituximab in combination with short-term predn
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        e was consistent with the safety profile for rituximab in RA and GPA and MPA. 6.4 Clinical Trials Experience in Pemphigus Vulgaris (PV) PV Study 1 PV Study 1 ( NCT00784589 ), a randomized, controlled, multicenter open-label study, evaluated the efficacy and safety of non-U.S.-licensed rituximab in combination with short-term pred
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      source url
      https://api.fda.gov/drug/label.json?search=%28indications_and_usage%3Acancer+OR+indications_and_usage%3Acarcinoma+OR+indications_and_usage%3Acarcinomas+OR+indications_and_usage%3Alymphoma+OR+indications_and_usage%3Alymphomas+OR+indications_and_usage%3Aleukemia+OR+indications_and_usage%3Aleukaemia+OR+indications_and_usage%3Asarcoma+OR+indications_and_usage%3Asarcomas+OR+indications_and_usage%3Amelanoma+OR+indications_and_usage%3Amyeloma+OR+indications_and_usage%3Atumor+OR+indications_and_usage%3Atumors+OR+indications_and_usage%3Atumour+OR+indications_and_usage%3Atumours+OR+indications_and_usage%3Aneoplasm+OR+indications_and_usage%3Aneoplasms+OR+indications_and_usage%3Amalignant+OR+indications_and_usage%3Amalignancy+OR+indications_and_usage%3Amyelodysplastic+OR+indications_and_usage%3Amyelofibrosis+OR+indications_and_usage%3Amastocytosis+OR+indications_and_usage%3Aglioma+OR+indications_and_usage%3Aglioblastoma+OR+indications_and_usage%3Aneuroblastoma+OR+indications_and_usage%3Aretinoblastoma+OR+indications_and_usage%3Apheochromocytoma+OR+indications_and_usage%3Aparaganglioma+OR+indications_and_usage%3Amyeloproliferative+OR+indications_and_usage%3Awaldenstrom+OR+indications_and_usage%3Apolycythemia+OR+indications_and_usage%3Athrombocythemia+OR+indications_and_usage%3Adesmoid+OR+indications_and_usage%3Aneurofibromatosis+OR+indications_and_usage%3Ahistiocytosis%29+AND+%28openfda.application_number%3ANDA%2A+OR+openfda.application_number%3ABLA%2A%29&limit=100&skip=400&sort=id%3Aasc
      source record id
      7e0a4bc5-9a5f-4b66-ab41-a4417988ccc2
      source file sha256
      a4bef6778baed183e5bcf581938b0b23d3576dfd9eb6150f2904934a750c5fef
      payload sha256
      a00948cbfb640f7a3d0b62acd47afe2626b1e7dbd9fd71c7556375298a9ff119
      payload
      application numbers
      1. BLA761140
      clinical indication matching status
      not_inferred
      end exclusive
      true
      id
      nct-edge-f23e8c6610fc7edaba6d
      link basis
      explicit_NCT_identifier_in_acquired_source
      nct id
      NCT00784589
      occurrences
      1. context exact
        tely every 6 months. The safety profile was consistent with the safety profile for rituximab in RA, GPA and MPA. Pemphigus Vulgaris (PV) PV Study 1 PV Study 1 (NCT00784589), a randomized, controlled, multicenter open-label study, evaluated the efficacy and safety of non-U.S.-licensed rituximab in combination with short-term predn
        end
        27861
        exact text
        NCT00784589
        start
        27850
      offset unit
      unicode_code_points
      source file
      /tmp/cancer-full-research/indications/raw/labels-00400.json
      source file sha256
      a4bef6778baed183e5bcf581938b0b23d3576dfd9eb6150f2904934a750c5fef
      source json pointer
      /results/83/adverse_reactions/0
      source kind
      fda_spl_label
      source record id
      7e0a4bc5-9a5f-4b66-ab41-a4417988ccc2
      source string basis
      parsed_JSON_string_unmodified
      source string sha256
      f1260d729d603b1d2ba2b7fd5d0659e74e2d05ef2ce3cf5923d1955445a55bfd
      source url
      https://api.fda.gov/drug/label.json?search=%28indications_and_usage%3Acancer+OR+indications_and_usage%3Acarcinoma+OR+indications_and_usage%3Acarcinomas+OR+indications_and_usage%3Alymphoma+OR+indications_and_usage%3Alymphomas+OR+indications_and_usage%3Aleukemia+OR+indications_and_usage%3Aleukaemia+OR+indications_and_usage%3Asarcoma+OR+indications_and_usage%3Asarcomas+OR+indications_and_usage%3Amelanoma+OR+indications_and_usage%3Amyeloma+OR+indications_and_usage%3Atumor+OR+indications_and_usage%3Atumors+OR+indications_and_usage%3Atumour+OR+indications_and_usage%3Atumours+OR+indications_and_usage%3Aneoplasm+OR+indications_and_usage%3Aneoplasms+OR+indications_and_usage%3Amalignant+OR+indications_and_usage%3Amalignancy+OR+indications_and_usage%3Amyelodysplastic+OR+indications_and_usage%3Amyelofibrosis+OR+indications_and_usage%3Amastocytosis+OR+indications_and_usage%3Aglioma+OR+indications_and_usage%3Aglioblastoma+OR+indications_and_usage%3Aneuroblastoma+OR+indications_and_usage%3Aretinoblastoma+OR+indications_and_usage%3Apheochromocytoma+OR+indications_and_usage%3Aparaganglioma+OR+indications_and_usage%3Amyeloproliferative+OR+indications_and_usage%3Awaldenstrom+OR+indications_and_usage%3Apolycythemia+OR+indications_and_usage%3Athrombocythemia+OR+indications_and_usage%3Adesmoid+OR+indications_and_usage%3Aneurofibromatosis+OR+indications_and_usage%3Ahistiocytosis%29+AND+%28openfda.application_number%3ANDA%2A+OR+openfda.application_number%3ABLA%2A%29&limit=100&skip=400&sort=id%3Aasc
      spl effective time
      20260722
      spl set id
      da1c4de7-0e5b-4f72-97ec-7a8d368f085f

    Preserved source evidence · Not medical advice