Clinical trial • Phase II • Immunology | Musculoskeletal

Hydroxychloroquine sulfate for Primary Sjögren's syndrome

Phase II trial of Hydroxychloroquine sulfate for Primary Sjögren's syndrome.

Overview

Trial Therapeutic Area
Immunology | Musculoskeletal
Trial Disease
Primary Sjögren's syndrome
Trial Stage
Phase II
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
12-01-2024
First CTIS Authorization Date
20-02-2024

Trial design

Randomised, placebo arms: placebo of hydroxychloroquine; placebo of leflunomide; placebo of mycophenolate mofetil. active investigational products/doses available in protocol: plaquenil 200 mg film-coated tablet (hydroxychloroquine sulfate) — max daily dose 400 mg; arava 20 mg film-coated tablets (leflunomide) — max daily dose 20 mg; mycophenolate mofetil teva 500 mg film-coated tablets — max daily dose 2000 mg.-controlled Phase II trial in Netherlands, Sweden, Italy and others.

Randomised
Yes
Comparator
Placebo arms: Placebo of hydroxychloroquine; Placebo of leflunomide; Placebo of mycophenolate mofetil. Active investigational products/doses available in protocol: PLAQUENIL 200 mg film-coated tablet (hydroxychloroquine sulfate) — max daily dose 400 mg; Arava 20 mg film-coated tablets (leflunomide) — max daily dose 20 mg; Mycophenolate mofetil Teva 500 mg film-coated tablets — max daily dose 2000 mg.
Target Sample Size
235
Trial Duration For Participant
168

Stratification factors

  • Cohort (symptom-based vs systemic activity): ESSPRI ≥5 (high symptoms, low systemic activity) versus ESSDAI ≥5 (moderate/high systemic disease activity)

Eligibility

Recruits 235 No vulnerable population selected. Participants must provide written informed consent prior to any study-related procedures (inclusion criteria). Age < 18 years are excluded. Informed consent documents for adults are available (e.g. L1_SIS and ICF adults - GR)..

Pregnancy Exclusion
Pregnant or breastfeeding women or women wanting to conceive either during or within two years after the end of the treatment period
Vulnerable Population
No vulnerable population selected. Participants must provide written informed consent prior to any study-related procedures (inclusion criteria). Age < 18 years are excluded. Informed consent documents for adults are available (e.g. L1_SIS and ICF adults - GR).

Inclusion criteria

  • {"criterion_text":"- Cohort 1 : Having given written informed consent prior to undertaking any study-related procedures"}
  • {"criterion_text":"- Cohort 2 : Use highly reliable contraception during study treatment from the screening and for two years after stopping treatment"}
  • {"criterion_text":"- Cohort 1 : Patients with pSS according to ACR/EULAR 2016 criteria or AECG 2002 criteria"}
  • {"criterion_text":"- Cohort 1 : With a high level of symptoms (ESSPRI ≥ 5) and low systemic disease activity"}
  • {"criterion_text":"- Cohort 1 : Negative pregnancy test (serum at screening)"}
  • {"criterion_text":"- Cohort 1 : Use highly reliable contraception during study treatment from the screening and for two years after stopping treatment"}
  • {"criterion_text":"- Cohort 2 : Having given written informed consent prior to undertaking any study-related procedures"}
  • {"criterion_text":"- Cohort 2 : Patients with pSS according to ACR/EULAR 2016 criteria or AECG 2002 criteria"}
  • {"criterion_text":"- Cohort 2 : With moderate/high systemic disease activity, as defined by ESSDAI ≥ 5"}
  • {"criterion_text":"- Cohort 2 : Negative pregnancy test (serum at screening)"}

Exclusion criteria

  • {"criterion_text":"- Age < 18 years"}
  • {"criterion_text":"- Previous treatment with HCQ, LEF, or MMF in the last 3 months"}
  • {"criterion_text":"- Previous treatment with rituximab, other B-cell targeted biologic therapy or cyclophosphamide in the last 6 months"}
  • {"criterion_text":"- Previous treatment with anti-TNF, abatacept, tocilizumab or belimumab or any other biologic in the setting of a past clinical trial in the last 3 months"}
  • {"criterion_text":"- Severe life-threatening systemic involvement requiring cyclophosphamide or high-dose corticosteroids, or any drug considered as an exclusion criteria"}
  • {"criterion_text":"- Impairment of other severe immunodeficiency states"}
  • {"criterion_text":"- Patients with active malignancy or history of malignancy within the last 5 years except non-melanoma skin cancer"}
  • {"criterion_text":"- Patients with active gastrointestinal tract ulceration, hemorrhage and perforation"}
  • {"criterion_text":"- Patients with history of cardiomyopathy"}
  • {"criterion_text":"- Patients with known hereditary deficiency of hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) such as Lesch-Nyhan and Kelley-Seegmiller syndrome"}
  • {"criterion_text":"- Serious infection in the past month"}
  • {"criterion_text":"- Pregnant or breastfeeding women or women wanting to conceive either during or within two years after the end of the treatment period"}
  • {"criterion_text":"- Evidence of active tuberculosis infection"}
  • {"criterion_text":"- Active HCV (positive PCR)"}
  • {"criterion_text":"- Active HBV infection (positivity for HBS antigen, or positivity for anti-HBC antibody without any HBS antigen)"}
  • {"criterion_text":"- HIV infection (positive serology)"}
  • {"criterion_text":"- Positive SARS-Cov2 PCR (if vaccinated for COVID-19, no PCR is required; if history of COVID-19 infection, positive serology is sufficient)"}
  • {"criterion_text":"- Cytopenia defined as neutrophils < 1.0 G/L, lymphocytes < 0.5 G/L, Hb < 10 g/dl or platelets < 100 G/L"}
  • {"criterion_text":"- Moderate to severe renal insufficiency (GFR < 30 ml/min)"}
  • {"criterion_text":"- Severe hypogammaglobulinemia defined as gamma globulins or IgG < 5 g/l"}
  • {"criterion_text":"- Reduced hepatic function: AST or ALT > 2x ULN"}
  • {"criterion_text":"- Prolonged ECG's corrected QT interval (>500 ms)"}
  • {"criterion_text":"- Women of childbearing potential not using highly effective methods of contraception"}
  • {"criterion_text":"- Known history of maculopathy"}
  • {"criterion_text":"- Patients not informed of the risk of alcohol consumption and of the recommendations to avoid alcohol during the entire study"}
  • {"criterion_text":"- Not affiliated to a social security regime (specific for France)"}
  • {"criterion_text":"- Participation in another interventional trial"}
  • {"criterion_text":"- Contra-indication to HCQ: pre-existing retinopathy, hypersensitivity to HCQ or to any of the excipients of the specialty used"}
  • {"criterion_text":"- Contra-indication to MMF: hypersensitivity to mycophenolate mofetil, acid mycophenolic, mycophenolate sodium or to any of the excipients of the specialty used"}
  • {"criterion_text":"- Contra-indication tor LEF: hypersensitivity to the active substance, the main active metabolite teriflunomide or to any excipients of the specialty used"}
  • {"criterion_text":"- Concomitant treatment with corticosteroids more than 10 mg/day of prednisone equivalent at screening or inclusion (randomisation)"}
  • {"criterion_text":"- Concomitant treatment with other immunomodulators including methotrexate, azathioprine, cyclophosphamide, cyclosporine and tacrolimus"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. • Cohort 2: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm.","definition_or_measurement_approach":"Response measured as proportion of patients achieving a response according to the preliminary STAR at week 24 (preliminary STAR assessment at Week 24)."}

Recruitment

Planned Sample Size
235
Recruitment Window Months
45
Consent Approach
Written informed consent is required from participants prior to undertaking any study-related procedures. Only adults (Age ≥ 18) are eligible (Age < 18 years excluded). An adult informed consent form is available (document: L1_SIS and ICF adults - GR). Study documents (protocol synopses) are provided in multiple languages (translations present: FR, ES, NL, GR, IT, NO, SE).

Geography

Total Number Of Sites
25
Total Number Of Participants
235

Netherlands

Earliest CTIS Part Ii Submission Date
08-02-2024
Latest Decision Or Authorization Date
02-08-2024
Processing Time Days
176
Number Of Sites
2
Number Of Participants
30

Sites

Site Name
Universitair Medisch Centrum Groningen
Department Name
Rheumatology and Clinical Immunology
Principal Investigator Name
Hendrika BOOTSMA
Principal Investigator Email
h.bootsma@int.umcg.nl
Contact Person Name
Hendrika BOOTSMA
Contact Person Email
h.bootsma@int.umcg.nl
Site Name
Universitair Medisch Centrum Utrecht
Department Name
Division of Internal Medicine and Dermatology, Rheumatology & Clinical Immunology
Principal Investigator Name
Helen LEAVIS
Principal Investigator Email
H.Leavis@umcutrecht.nl
Contact Person Name
Helen LEAVIS
Contact Person Email
H.Leavis@umcutrecht.nl

Sweden

Earliest CTIS Part Ii Submission Date
14-02-2024
Latest Decision Or Authorization Date
31-07-2024
Processing Time Days
168
Number Of Sites
3
Number Of Participants
25

Sites

Site Name
Lunds Universitet
Department Name
Rhumatology
Principal Investigator Name
Giovanni CAGNOTTO
Principal Investigator Email
giovanni.cagnotto@med.lu.se
Contact Person Name
Giovanni CAGNOTTO
Contact Person Email
giovanni.cagnotto@med.lu.se
Site Name
Karolinska University Hospital
Department Name
Rhumatology
Principal Investigator Name
Marika KVARNSTROM
Principal Investigator Email
marika.kvarnstrom@sll.fr
Contact Person Name
Marika KVARNSTROM
Contact Person Email
marika.kvarnstrom@sll.fr
Site Name
Uppsala University Hospital
Department Name
Rhumatology
Principal Investigator Name
Gunnel NORDMARK
Principal Investigator Email
gunnel.nordmark@medsci.uu.se
Contact Person Name
Gunnel NORDMARK
Contact Person Email
gunnel.nordmark@medsci.uu.se

Italy

Earliest CTIS Part Ii Submission Date
08-02-2024
Latest Decision Or Authorization Date
19-08-2024
Processing Time Days
193
Number Of Sites
5
Number Of Participants
30

Sites

Site Name
Azienda Sanitaria Universitaria Friuli Centrale
Department Name
Rheumatology
Principal Investigator Name
Luca QUARTUCCIO
Principal Investigator Email
luca.quartuccio@asuiud.sanita.fvg.it
Contact Person Name
Luca QUARTUCCIO
Site Name
Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
Department Name
Rhumatology
Principal Investigator Name
Francesco CICCIA
Principal Investigator Email
francesco.ciccia@unicampania.it
Contact Person Name
Francesco CICCIA
Site Name
University Hospital Of Perugia
Department Name
Rhumatology
Principal Investigator Name
Elena BARTOLONI
Principal Investigator Email
elena.bartolonibocci@unipg.it
Contact Person Name
Elena BARTOLONI
Contact Person Email
elena.bartolonibocci@unipg.it
Site Name
Fondazione Policlinico Universitario Campus Bio-Medico
Department Name
Rhumatology
Principal Investigator Name
Roberto GIACOMELLI
Principal Investigator Email
r.giacomelli@unicampus.it
Contact Person Name
Roberto GIACOMELLI
Contact Person Email
r.giacomelli@unicampus.it
Site Name
Universita' Di Pisa
Department Name
Rhumatology
Principal Investigator Name
Chiara BALDINI
Principal Investigator Email
chiara.baldini74@gmail.com
Contact Person Name
Chiara BALDINI
Contact Person Email
chiara.baldini74@gmail.com

France

Earliest CTIS Part Ii Submission Date
08-02-2024
Latest Decision Or Authorization Date
23-07-2024
Processing Time Days
166
Number Of Sites
8
Number Of Participants
70

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Rhumatology
Principal Investigator Name
Véronique LE GUERN
Principal Investigator Email
veronique.le-guern@aphp.fr
Contact Person Name
Véronique LE GUERN
Contact Person Email
veronique.le-guern@aphp.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Internal Medicine
Principal Investigator Name
Eric HACHULLA
Principal Investigator Email
eric.hachulla@chru-lille.fr
Contact Person Name
Eric HACHULLA
Contact Person Email
eric.hachulla@chru-lille.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Rhumatology
Principal Investigator Name
Christophe RICHEZ
Principal Investigator Email
christophe.richez@chu-bordeaux.fr
Contact Person Name
Christophe RICHEZ
Site Name
Centre Hospitalier Le Mans
Department Name
Rheumatology / Immunology
Principal Investigator Name
Paul LEGENDRE
Principal Investigator Email
plegendre@ch-lemans.fr
Contact Person Name
Paul LEGENDRE
Contact Person Email
plegendre@ch-lemans.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Rhumatology
Principal Investigator Name
Jacques-Eric GOTTENBERG
Principal Investigator Email
jacques-eric.gottenberg@chru-strasbourg.fr
Contact Person Name
Jacques-Eric GOTTENBERG
Site Name
Assistance Publique Hopitaux De Paris (Le Kremlin-Bicetre)
Department Name
Rhumatology
Principal Investigator Name
Raphaèle SEROR
Principal Investigator Email
raphaele.seror@aphp.fr
Contact Person Name
Raphaèle SEROR
Contact Person Email
raphaele.seror@aphp.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Rhumatology
Principal Investigator Name
Jacques MOREL
Principal Investigator Email
j-morel@chu-montpellier.fr
Contact Person Name
Jacques MOREL
Contact Person Email
j-morel@chu-montpellier.fr
Site Name
Centre Hospitalier Regional Et Universitaire De Brest
Department Name
Rhumatology
Principal Investigator Name
Valérie DEVAUCHELLE-PENSEC
Principal Investigator Email
valerie.devauchelle-pensec@chu-brest.fr
Contact Person Name
Valérie DEVAUCHELLE-PENSEC

Spain

Earliest CTIS Part Ii Submission Date
08-02-2024
Latest Decision Or Authorization Date
23-07-2024
Processing Time Days
166
Number Of Sites
3
Number Of Participants
30

Sites

Site Name
Hospital Clinic De Barcelona
Department Name
Rhumatology
Principal Investigator Name
Manel RAMOS CASALS
Principal Investigator Email
mramos@clinic.cat
Contact Person Name
Manel RAMOS CASALS
Contact Person Email
mramos@clinic.cat
Site Name
Parc Tauli Hospital Universitari
Department Name
Rhumatology
Principal Investigator Name
Maria Begoña
Principal Investigator Email
bmari@tauli.cat
Contact Person Name
Maria Begoña
Contact Person Email
bmari@tauli.cat
Site Name
Vall D'hebron Institut De Recerca
Department Name
Rhumatology
Principal Investigator Name
Roser SOLANS
Principal Investigator Email
rsolanslaq@gmail.com
Contact Person Name
Roser SOLANS
Contact Person Email
rsolanslaq@gmail.com

Norway

Earliest CTIS Part Ii Submission Date
20-02-2024
Latest Decision Or Authorization Date
25-07-2024
Processing Time Days
156
Number Of Sites
2
Number Of Participants
25

Sites

Site Name
Helse Bergen HF
Department Name
Rhumatology
Principal Investigator Name
Sten DANIEL
Principal Investigator Email
sten.daniel.hammenfors@helse-bergen.no
Contact Person Name
Sten DANIEL
Site Name
Helse Stavanger HF
Department Name
Medicine
Principal Investigator Name
Katrine braekke norheim
Principal Investigator Email
katrine.brekke.norheim@sus.no
Contact Person Name
Katrine braekke norheim
Contact Person Email
katrine.brekke.norheim@sus.no

Greece

Earliest CTIS Part Ii Submission Date
29-03-2024
Latest Decision Or Authorization Date
20-11-2024
Processing Time Days
236
Number Of Sites
2
Number Of Participants
25

Sites

Site Name
National And Kapodistrian University Of Athens
Department Name
Department of Pathophysiology School of Medicine
Principal Investigator Name
Andreas GOULES
Principal Investigator Email
agoules@med.uoa.gr
Contact Person Name
Andreas GOULES
Contact Person Email
agoules@med.uoa.gr
Site Name
University General Hospital Of Ioannina
Department Name
Department of Internal Medicine
Principal Investigator Name
Paraskevi VOULGARI
Principal Investigator Email
pvoulgar@uoi.gr
Contact Person Name
Paraskevi VOULGARI
Contact Person Email
pvoulgar@uoi.gr

Sponsor

Primary sponsor

Full Name
Assistance Publique Hopitaux De Paris
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
France

Contract research organisations

Name
Pharmacist M. Ltd.
Responsibilities
Sponsor/third-party duties (codes: 1,5,7,8,9); contact: i.koukli@pharmassist-cro.com

Third parties

  • {"country":"Greece","full_name":"Pharmacist M. Ltd.","duties_or_roles":"sponsorDuties codes: 1,5,7,8,9","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
PLAQUENIL 200 mg, comprimé pelliculé
Active Substance
Hydroxychloroquine sulfate
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Authorised (Marketing authorisation number: 34009 364 414 6 0)
Maximum Dose
400 mg per day
Investigational Product Name
Arava 20 mg film-coated tablets
Active Substance
Leflunomide
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Authorised (Marketing authorisation number: EU/1/99/118/007)
Maximum Dose
20 mg per day
Investigational Product Name
Mycophenolate mofetil Teva 500 mg film-coated tablets
Active Substance
Mycophenolate mofetil
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Authorised (Marketing authorisation number: EU/1/07/439/003)
Maximum Dose
2000 mg per day
Investigational Product Name
Placebo of hydroxychloroquine
Modality
Other
Investigational Product Name
Placebo of leflunomide
Modality
Other
Investigational Product Name
Placebo of mycophenolate mofetil
Modality
Other
Combination Treatment
Yes

Related trials

Other published trials that may interest you.