Efficacy and safety of repeated Rituximab infusions in patients with Active Systemic SClerosis and background medication, a one year randomized blinded placebo-controlled trial, the RASSc study.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Radboud universitair medisch centrum Stichting

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05], Diseases [C] - Immune System Diseases [C20]

Trial duration

2 Sep 2025 → ongoing

Decision date (initial)

2025-05-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

ZonMw

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy

The primary objectives of the RASSc study are to examine in patients with early or progressive SSc:
1) The effectiveness of (repeated) RTX compared to placebo in preventing the occurrence of organ involvement and halting progressive disease

Secondary objectives 1

1. The secondary objectives are to examine in patients treated with RTX compared to placebo the between group differences in: • incidence or progression of other internal organ involvement such as pulmonary arterial hypertension, renal crisis, gastric antral vascular ectasia, myositis, arthritis, digital ulcers, pitting scars or tendon friction rubs at 18 months. • derived composite outcome CRISS if applicable at 18 months. • capillary density and nailfold pattern at 18 months • change in functionality measured by SHAQ DI v2.0 at 18 months • quality of life measured by EQ-5D-5L, K-BILD, SF-36 and patient and physician global assessment over 18 months • in patient satisfaction as measured by PSQ-18 at 18 months • cumulative incidence of all-cause mortality and disease specific mortality at 18 months • incidence and types of patients with and total number of AE during 18 months • cost-effectiveness over 18 months

Conditions and MedDRA coding

Systemic sclerosis

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Patients with SSc, with early disease (<4 years) and/or progressive disease, on stable background therapy may be included.

Exclusion criteria 1

1. Patients treated with cyclophosphamide iv pulses, tocilizumab or other biologicals currently or in the past 3 months as well as patients treated with autologous hematopoietic stem cell transplantation in the last 2 years will be excluded. Patients with a malignancy or severe pulmonary hypertension (NYHA III and IV) and patients with a contra-indication for RTX such as active moderate or severe infections and severe heart failure will also be excluded.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary outcome will be between group differences of disease progression after 12 months follow-up. Disease progression is defined as a dichotomized composite outcome measure representing a clinical relevant worsening in skin and/or lung involvement, defined as: A) Increase in modified Rodnan Skin Score of 3 points and/or 15%, and/or B) Decrease ≥ 10% in FVC, or a decrease in FVC ≥ 5%-< 10% and a decline in DLCO ≥ 15%, and/or new ILD on HRCT-scan.

Secondary endpoints 1

1. Secondary outcomes will be assessed during follow-up, after 12 and 18 months. Secondary outcomes include between group differences in other organ involvement, the derived composite outcome CRISS if applicable, changes in nailfold capillaroscopic findings, change in functionality, quality of life, patient satisfaction, mortality, safety and cost-effectiveness with an economic evaluation.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Radboud universitair medisch centrum Stichting

Sponsor organisation

Radboud universitair medisch centrum Stichting

Address

Geert Grooteplein Zuid 10

City

Nijmegen

Postcode

6525 GA

Country

Netherlands

Scientific contact point

Organisation

Radboud universitair medisch centrum Stichting

Contact name

Madelon Vonk

Public contact point

Organisation

Radboud universitair medisch centrum Stichting

Contact name

Madelon Vonk

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications