Health Trials

A Study to Test Whether Nerandomilast Helps People With Systemic Sclerosis

ConditionSystemic Sclerosis
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorBoehringer Ingelheim

About this trial

Nerandomilast is being developed to help people with systemic sclerosis by potentially improving symptoms and slowing disease progression. This study is open to adults who are at least 18 years old and have systemic sclerosis (SSc). People can join the study if they have limited or diffuse cutaneous SSc with disease onset within 7 years of the first non-Raynaud's symptom. The purpose of this study is to find out whether a medicine called nerandomilast helps people with systemic sclerosis. This study also aims to find out how well nerandomilast is tolerated in people with systemic sclerosis.

Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take the tablets twice a day.

Participants are in the study for 1 to about 4 years. During this time, they visit the study site regularly and get phone calls from the site staff. During study visits participants regularly have blood samples taken and doctors check changes in skin thickening, lung function, and internal organs, overall health and the safety and tolerability of study treatment in people with SSc. The results are compared between the groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial.

Patients must be at least 18 years of age and fulfil the 2013 American College of Rheumatology/European Alliance of Associations for Rheumatology (ACR/EULAR) criteria for SSc.

Patients must be diagnosed with limited cutaneous SSc (lcSSc) or diffuse cutaneous SSc (dcSSc), as defined by LeRoy et al. (1988).

Disease onset (defined by first non-RP [Raynaud's phenomenon] symptom) must be within 7 years of Visit 1.

Disqualifiers

Active, unstable, or uncontrolled vasculitis within 8 weeks prior to Visit 1 or during the screening period.

Any suicidal behaviour in the past 2 years.

Any suicidal ideation of type 4 or 5 on the C-SSRS in the past 3 months. Further exclusion criteria apply.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Placebo matching nerandomilast formulation 1

    Drug

    Film-coated tablets

  • Placebo matching nerandomilast formulation 2

    Drug

    Film-coated tablets

  • Nerandomilast formulation 1

    Drug

    Film-coated tablets

  • Nerandomilast formulation 2

    Drug

    Film-coated tablets

Treatment groups

448 Participants
are divided into 2 treatment groups
Group A: PlaceboPlacebo comparator 2 interventions
Group B: NerandomilastExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Time to the first occurrence of disease progression or all-cause death

Time frame
up to 4 years

Secondary outcomes

1

Change from baseline in mRSS at Week 52

The modified Rodnan Skin Score (mRSS) measures skin thickness and is the sum of scores from 17 surface anatomic areas rated on a 0-3 scale (0=normal skin; 1=mild thickness; 2=moderate thickness; 3=severe thickness with inability to pinch the skin into a fold). The total mRSS ranges from 0 (best possible outcome) to 51 (worst possible outcome).

Time frame
At baseline and at Week 52.
2

Change from baseline in HAQ-DI score at Week 52

Health Assessment Questionnaire Disability Index (HAQ-DI) is used frequently in rheumatological disorders including SSc, assessing function/activities of daily living with 20 items in 8 categories, namely dressing and grooming, hygiene, arising, reach, eating, grip, walking, and common daily activities. Each category has at least 2 sub-category questions. Within each category, patients report the amount of difficulty they have in performing the specific sub-category items. There are four response options ranging from "no difficulty" to "unable to do", scored 0 to 3. A global score will be calculated from the category scores with higher scores indicating more severe disability.

Time frame
At baseline and at Week 52.
3

Change from baseline in FVC [mL] at Week 52

Forced vital capacity (FVC)

Time frame
At baseline and at Week 52.
4

Disease improvement as defined by rCRISS-25 at Week 52

Revised composite response index in systemic sclerosis (rCRISS) is a composite outcome measure developed for SSc that includes PRO and clinician-reported outcome (ClinRO) measures: * mRSS * FVC % * HAQ-DI * Patient Global Assessment (PGA) of overall health status * Clinician Global Assessment (CGA) of overall health status For rCRISS-25 a patient has improvement on at least 2 of the 5 core set measures and without worsening on more than 1 core set measure. The improvement or worsening must be at least 25% relative change from the baseline for 4 core set measures (or ≥5% relative change from baseline for FVC % predicted).

Time frame
At baseline and at Week 52.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Boehringer Ingelheim

Lead sponsor

Source ClinicalTrials.gov
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