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Juvenile DermatomyositisPhase 3PRINTOJDMTRNCT00323960

Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis

A Phase 3 study testing 3 MPDN pulse + PDN, 3 MPDN pulse + PDN + CSA and 3 MPDN pulse + PDN + MTX in people with Juvenile Dermatomyositis, run by Istituto Giannina Gaslini at 1 site in Italy.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a 5-year project, involving 185 partners from 46 countries ((110 in 21 European Union (EU) States and 75 in 25 extra-EU States)), with a randomised clinical trials (RCT) in juvenile dermatomyositis (JDM): 5-year phase III single-blind, RCT in children with newly diagnosed JDM: prednisone (PDN) versus PDN plus… Read more about this study

Who is it for?
Ages 1 year to 18 years
Conditions the study is for
Juvenile Dermatomyositis
What is being tested?
3 MPDN pulse + PDN, 3 MPDN pulse + PDN + CSA and 3 MPDN pulse + PDN + MTX
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
139 participants
Where
1 siteItaly
Study dates
31 May 2006to 29 Nov 2015
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 1 year to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Inclusion Criteria. Each patient must meet all the following criteria in order to participate in this trial:

  1. Newly diagnosed and untreated children (only treatment with 1 NSAID is allowed and/or prednisone >1 mg/kg/day for no more than 1 month from diagnosis) with probable or definite diagnosis of JDM according to published (12;13). If a muscle biopsy will be performed (optional) it will be read by the pathologists of the participating centres (light and immunofluorescence). Slides of paraffin-embedded sections from all patients will be re-viewed by a blinded myopathologist at PRINTO.
  2. Age at enrolment ≤ 18 years.
  3. Female of child-bearing potential must have a negative pregnancy test at the beginning of the trial, and then every 3 months. If sexually active, they must agree to use adequate contraception, throughout study participation, and must have no intention of conceiving during the course of the study. Post-pubertal males must have no plans to father a child during the study and agree to use adequate birth control methods if sexually active.
  4. Ability to comply with the entire study procedures, ability to communicate meaningfully with the investigational staff, competence to give written informed consent; to be applied to the parents and/or patients, as appropriate
  5. Duly executed, written, informed consent obtained from the parents/patient.

Exclusion Criteria. Any of the following will exclude a patient from this trial:

  1. Neutrophil count <1,500/mm3 and/or platelet count <50,000/mm3
  2. Demonstration of cutaneous or gastrointestinal ulceration of JDM related pulmonary disease or cardiomyopathy at the time of diagnosis.
  3. History of poor compliance.
  4. Evidence of current use of alcohol or illicit drugs abuse.
  5. Live vaccines not allowed during the entire duration of the trial.

Dropout Criteria. Patients will be considered "treatment failures", and dropped from the trial but included in efficacy analysis, if any of the following will occur during the active period of the trial.

  1. Non compliance with study medication administration
  2. Enrolment in other therapeutic trials.

Trial locations and contacts

1 site in 1 country.

Italy1 site

  • Istituto Giannina Gaslini

    Genoa, 16147

Other trials in Italy

About this study

Official title: Five-year Single-blind, Phase III Effectiveness Randomised Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis: Prednisone Versus Prednisone Plus Cyclosporine a Versus Prednisone Plus Methotrexate

This is a 5-year project, involving 185 partners from 46 countries ((110 in 21 European Union (EU) States and 75 in 25 extra-EU States)), with a randomised clinical trials (RCT) in juvenile dermatomyositis (JDM): 5-year phase III single-blind, RCT in children with newly diagnosed JDM: prednisone (PDN) versus PDN plus methotrexate (MTX) versus PDN plus Cyclosporine A. The trial is aimed to find out the treatment regimen associated with the lowest occurrence of flare and the lowest drug related toxicity

Read the detailed description

Scientific objectives: The proposed project is aimed to improve treatment approaches for rare, severe and disabling paediatric rheumatic diseases (PRD). This goal will be achieved by the Paediatric Rheumatology International Trials Organisation (PRINTO) an international network whose main function is to provide a scientific base for current PRD treatments for which no evidence based data exist in the literature, and for drugs for which there is no support from industries.

This is a 5-year project, involving 46 countries (110 in 21 EU States and 75 in 25 extra-EU States), with a randomised clinical trials (RCT) in juvenile dermatomyositis (JDM): 5-year phase III single-blind, RCT in children with newly diagnosed JDM: prednisone (PDN) versus PDN plus methotrexate (MTX) versus PDN plus Cyclosporine A (CsA). The trial is aimed to find out the treatment regimen associated with the lowest occurrence of flare and the lowest drug related toxicity. The retention on treatment will be used as main measure of effectiveness.

Methodology: The present protocol is the natural follow up of previous work conducted by PRINTO. In particular the RCT foreseen in this protocol is modelled after the successful completion of an early phase trial with MTX in juvenile idiopathic arthritis, and will use validated JDM outcome measures for the evaluation of response to therapy.

It is the basic premise of this protocol that, without i) the involvement of the international paediatric rheumatology community, ii) the innovative type of mechanism described herein, these studies would never be conducted.

Objectives. The goals of the current protocol is therefore the natural follow-up of the objectives achieved with the previous grants and, in particular, of projects designed to discern new models for the successful conduct of clinical trials in children with rare diseases, and to develop standardized and validated measures for the evaluation of response to therapy in JDM.

The proposed trial in JDM (prednisone [PDN] versus PDN plus methotrexate [MTX] versus PDN plus cyclosporine [CsA]), should serve as a model for the successful running of early phase clinical trials for severe and disabling rare diseases of childhood.

The ultimate aim of these trials is to provide evidence-based information about the clinical utility of drugs in the management of rare paediatric conditions.

Being tested
3 MPDN pulse + PDN, 3 MPDN pulse + PDN + CSA, 3 MPDN pulse + PDN + MTX
Keywords
Juvenile dermatomyositis, randomised actively controlled clinical trial, prednisone, cyclosporine, methotrexate, effectiveness
Medical subject headings
Dermatomyositis

What participants receive

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

  • 3 MPDN pulse + PDNDrug

    3 methylprednisolone pulses followed (30 mg/kg/pulse max 1 gram) followed by prednisone 2 mg/Kg/day to be tapered to 0.2 mg/kg/day in 6 months and then discontinued in 2 years.

  • 3 MPDN pulse + PDN + CSADrug

    3 methylprednisolone pulses followed (30 mg/kg/pulse max 1 gram) followed by prednisone 2 mg/Kg/day to be tapered to 0.2 mg/kg/day in 6 months and then discontinued in 2 years; Cyclosporine 5 mg/Kg/day in 2 oral doses

  • 3 MPDN pulse + PDN + MTXDrug

    3 methylprednisolone pulses followed (30 mg/kg/pulse max 1 gram) followed by prednisone 2 mg/Kg/day to be tapered to 0.2 mg/kg/day in 6 months and then discontinued in 2 years; Methotrexate 15-20 mg/m2 once per week. Patients treated with MTX will receive concomitant folic or folinic acid according to the attending physician decision.

Study groups in NCT00323960
Study groupRoleWhat they receive
Methylprednisolone pulse (MPDN)+PDN+CSAActive comparatorMPDN= methylprednisolone pulse PDN= prednisone or equivalent CSA= cyclosporine A
Receives: Drug: 3 MPDN pulse + PDN + CSA
MPDN+PDN+MTXActive comparatorMPDN= methylprednisolone pulse PDN= prednisone or equivalent MTX= methotrexate
Receives: Drug: 3 MPDN pulse + PDN + MTX
MPDN+PDNActive comparatorMPDN= methylprednisolone PDN= prednisone or equivalent
Receives: Drug: 3 MPDN pulse + PDN

What is being measured

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

  1. Responder Status Defined as 20% Improvement in at Least 3 Core Set Variables With no More Than 1 of the Remaining Variables, (Muscle Strength Excluded), Worsened by > 30%.

    The PRINTO Juvenile Dermatomyositis (JDM) core set variables are:

    1. muscle strength by the mean of the Childhood Myositis Assessment Scale (CMAS);
    2. physician's global assessment of disease activity on a 10 cm Visual Analogue Scale (VAS);
    3. global disease activity assessment by the mean of the Disease Activity Index (DAS);
    4. parent's/patient's global assessment of overall well-being on a 10 cm VAS;
    5. functional ability assessment by the mean of the Childhood Health Assessment Questionnaire (CHAQ)
    6. health-related quality of life assessment.

    Measured: 6 months

  2. Time to Clinical Remission

    Clinical remission is defined as the status of inactive disease for at least 6 continuous months defined as normal muscle strength (CMAS equal to 52) and physician global assessment of disease activity equal to 0.

    Measured: 60 months

Secondary outcomes (2)

  1. Time to Major Therapeutic Changes

    Time to major therapeutic changes is defined as the addition of CSA or MTX or any other disease-modifying antirheumatic drug (DMARS) in any of the 3 groups or discontinuation of assigned therapy for any reason including adverse events. Retention on treatment was used as main measure of effectiveness.

    Measured: 60 months

  2. Time to Prednisone, or Equivalent, Discontinuation

    Prednisone or equivalent glucocorticoid discontinuation is defined as the complete discontinuation of glucocorticoids

    Measured: 60 months

Timeline

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

  1. First posted10 May 2006 (estimated)
  2. Study start31 May 2006
  3. Primary completion12 May 2011
  4. Study completion29 Nov 2015

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
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
139 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00323960
Sponsor's protocol ID
IGG-PRINTO-002
Other IDs
AIFA, Myositis Association
Acronym
PRINTOJDMTR
Status last verified
Mar 2023
First submitted
9 May 2006
Last updated
28 Mar 2023
Data monitoring committee
No
Individual data sharing
Yes: Data will be provided upon request to the Paediatric Rheumatology InterNational Trials Organisation (PRINTO) network as per its bylaws

Publications

  • Miller LC, Sisson BA, Tucker LB, DeNardo BA, Schaller JG. Methotrexate treatment of recalcitrant childhood dermatomyositis. Arthritis Rheum. 1992 Oct;35(10):1143-9. doi: 10.1002/art.1780351006.

    PubMed 1418005

  • Al-Mayouf S, Al-Mazyed A, Bahabri S. Efficacy of early treatment of severe juvenile dermatomyositis with intravenous methylprednisolone and methotrexate. Clin Rheumatol. 2000;19(2):138-41. doi: 10.1007/s100670050032.

    PubMed 10791626

  • Heckmatt J, Hasson N, Saunders C, Thompson N, Peters AM, Cambridge G, Rose M, Hyde SA, Dubowitz V. Cyclosporin in juvenile dermatomyositis. Lancet. 1989 May 13;1(8646):1063-6. doi: 10.1016/s0140-6736(89)92456-2.

    PubMed 2566009

  • Pistoia V, Buoncompagni A, Scribanis R, Fasce L, Alpigiani G, Cordone G, Ferrarini M, Borrone C, Cottafava F. Cyclosporin A in the treatment of juvenile chronic arthritis and childhood polymyositis-dermatomyositis. Results of a preliminary study. Clin Exp Rheumatol. 1993 Mar-Apr;11(2):203-8.

    PubMed 8508564

  • Ruperto N, Pistorio A, Ravelli A, Angioloni S, Martini A; Paediatric Rheumatology International Trials Organisation (PRINTO). The PRINTO juvenile dermatomyositis trial - Authors' reply. Lancet. 2016 Jun 25;387(10038):2601. doi: 10.1016/S0140-6736(16)30671-7. No abstract available.

    PubMed 27353819

  • Dale A, Milosevic I, Goldacre B; COMPare project team. The PRINTO juvenile dermatomyositis trial. Lancet. 2016 Jun 25;387(10038):2600-2601. doi: 10.1016/S0140-6736(16)30845-5. No abstract available.

    PubMed 27353816

  • Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, Fischbach M, Magnusson B, Sterba G, Avcin T, Brochard K, Corona F, Dressler F, Gerloni V, Apaz MT, Bracaglia C, Cespedes-Cruz A, Cimaz R, Couillault G, Joos R, Quartier P, Russo R, Tardieu M, Wulffraat N, Bica B, Dolezalova P, Ferriani V, Flato B, Bernard-Medina AG, Herlin T, Trachana M, Meini A, Allain-Launay E, Pilkington C, Vargova V, Wouters C, Angioloni S, Martini A; Paediatric Rheumatology International Trials Organisation (PRINTO). Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial. Lancet. 2016 Feb 13;387(10019):671-678. doi: 10.1016/S0140-6736(15)01021-1. Epub 2015 Nov 30.

    PubMed 26645190

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing 3 MPDN pulse + PDN, 3 MPDN pulse + PDN + CSA and 3 MPDN pulse + PDN + MTX in people with Juvenile Dermatomyositis, run by Istituto Giannina Gaslini at 1 site in Italy.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 28 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Italy. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Responder Status Defined as 20% Improvement in at Least 3 Core Set Variables With no More Than 1 of the Remaining Variables, (Muscle Strength Excluded), Worsened by > 30%. (time frame: 6 months). The study lists 2 primary outcomes in all. 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 NCT00323960, which also lists 7 linked publications.

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 28 Mar 2023.

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