Localized SclerodermaPhase 2DupiMorphNCT04200755
Clinical Trial to Evaluate Efficacy and Safety of Dupilumab in Localized Scleroderma
A Phase 2 study testing Dupilumab 300Mg Solution for Injection in people with Localized Scleroderma, run by University of Cologne at 4 sites in Germany.
Trial summary
The DupiMorph study evaluates the efficacy of Dupilumab in localized scleroderma patients. Dupilumab is approved in the US and EU for the treatment of moderate/severe atopic dermatitis and since 2018 in the US for severe asthma therapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Localized Scleroderma
- What is being tested?
- Dupilumab 300Mg Solution for Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 4 sitesGermany
- Study dates
- 19 May 2020to 30 Nov 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject is a male or female ≥18 years of age on the day the study informed consent is signed
- Out-patient status
- Caucasian
- Morphea (plaque type) or Generalized localized scleroderma (affecting at least three anatomic sites)
- At least one lesions with lilac ring (active phase of the disease);
- Activity of LS within the last 12 month (as defined by progression of size or new developing plaque)
Show all 9Show fewer
- For women of childbearing potential: negative pregnancy test at Visit 1
- For women of childbearing potential: Use of effective method of contraception from 4 weeks prior to enrolment, throughout study treatment until 12 weeks after the last IMP dose.
- Written informed consent signed
Who cannot join
- Systemic immunosuppressive therapy or UV therapy less than 3 months before enrollment.
- Participation in another trial of IMPs or devices parallel to, or less than 6 months before or previous participation in this trial
- Pregnancy or breastfeeding mother
- Diagnosis of other significant chronic inflammatory or autoimmune disorders. Patients with the following autoimmune disorders are excluded from the study: Multiple sclerosis, primary biliary cirrhosis, type I diabetes mellitus. Patients with the following autoimmune disorders are regarded as eligible: Lichen sclerosus, vitiligo, alopecia arthritis, thyroid diseases (e.g. Hashimoto disease). Patients with any autoimmune disorder not listed above should only be included after consultation with the principal coordinating investigator.
- Topical immunosuppressive therapy less than 1 month before enrollment
- Concurrent phototherapy
Show all 15Show fewer
- Known infection with helminths (helminthosis)
- Any condition or laboratory abnormality that, in the judgment of the investigator, would put the subject at unacceptable risk for participation in the study or may interfere with the assessments included in the study. E.g. uncontrolled psychiatric illness or history of clinical relevant drug abuse.
- Known hypersensitivity to any components of the IMP
- Treatment with a live (attenuated) vaccine within 3 months prior to enrollment
- History of malignancy (except patients with completely treated in situ carcinoma of the cervix, completely treated and resolved non-metastatic squamous or basal cell carcinoma of the skin)
- Known diagnosis of active tuberculosis or non-tuberculous mycobacterial infection or latent untreated tuberculosis unless it is well documented by a specialist that the patient has been adequately treated
- Known diagnosis of HIV, HBV or HCV infection
- Regular use (more than 2 visits per week) of a tanning booth/parlor
- Known diagnosis of asthma
Trial locations and contacts
4 sites in 1 country.
Germany4 sites
Charité - Universitätsmedizin Berlin Klinik für Dermatologie, Venerologie und Allergologie
Berlin, 10117
Uniklinik Köln, Klinik für Dermatologie und Venerologie
Cologne, 50924
Helios St. Elisabeth Klinik Oberhausen, Klinik für Dermatologie, Venerologie und Allergologie
Oberhausen, 46045
Universitäts-Hautklinik Tübingen
Tübingen, 72076
About this study
Official title: A Randomized, Placebo-controlled Phase IIa Clinical Trial to Evaluate the Efficacy and Safety of Subcutaneous Dupilumab in Localized Scleroderma
The DupiMorph study evaluates the efficacy of Dupilumab in localized scleroderma patients. Dupilumab is approved in the US and EU for the treatment of moderate/severe atopic dermatitis and since 2018 in the US for severe asthma therapy.
Read the detailed description
Localized scleroderma (LS) comprises a heterogenous group of sclerotic skin disorders. The incidence of LS is reported to be approximately 27 cases/ 1x106 and is hence approximately 2-3-fold higher compared to systemic scleroderma. Although in most cases not lethal the disease can significantly impact quality of life. Depending on the location of fibrosis, the disorder can cause bone deformities, alopecia, skin atrophy or lesions with severe hypo-/hyperpigmentation.
The disease is pathomechanistically poorly understood and no effective therapy is currently approved. The most promising treatments up to date include methotrexate ± pulsed corticosteroids or phototherapy with PUVA or UVA1. Yet, the number of treated patients in these studies is low. The response rates are low and inconsistent with approximately 50% of patients treated with UVA1 experience a recurrence within three years. There are no studies on efficacy of topical corticosteroids in LS. Small pilot studies and few case reports describe regression of lesions after topical calcineurin inhibitors. In addition, current therapies can only be applied for a short time during the acute phase due to the side effect profile after long-term use (e.g. skin atrophy in response to topical steroids, skin cancer in response to long term UV therapy, multiple side effects by long- term use of methotrexate and/or corticosteroids). Hence, this study evaluates, in comparison with placebo, the efficacy of Dupilumab administered subcutaneously every 14 days in patients with Morphea (plaque type) or generalized localized scleroderma (affecting at least three anatomic sites).
- Conditions
- Localized Scleroderma
- Being tested
- Dupilumab 300Mg Solution for Injection, Placebo
- Keywords
- Morphea
- Medical subject headings
- Scleroderma, Localized
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dupilumab 300Mg Solution for InjectionDrug
Also called Dupixent
First dose: 600 mg (2 syringes); subsequent doses: 300 mg (1 syringe)
PlaceboOther
First dose: 2 syringes, no active substance; subsequent doses: 1 syringe, no active substance
| Study group | Role | What they receive |
|---|---|---|
| Dupilumab | Experimental | 30 patients; Dupilumab s.c. injection; 2 ready-to-use syringes (600 mg) initial (V1), 1 ready-to-use syringe (300 mg) every 14 days (V2- V13) Dupilumab s.c. injection in healthy skin, 24 weeks Receives: Drug: Dupilumab 300Mg Solution for Injection |
| Placebo | Placebo comparator | 15 patients; placebo s.c. injection; 2 ready-to-use syringes initial (V1), 1 ready-to-use syringe every 14 days (V2-V13) placebo s.c. injection in healthy skin, 24 weeks Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
LoSCAT target lesion
Treatment response is assessed using the LoSCAT (Localized Scleroderma Cutaneous Assessment Tool). Target lesion will be assessed at Baseline and End of Treatment. Score reduction by 50% after 24 weeks (End of Treatment Visit V14) compared to Baseline Visit (V1) is defined as treatment response.
Measured: Baseline to End of Treatment Visit, 24 weeks
Secondary outcomes (19)
mLoSSI all lesions
Change in mLoSSI (Localized Scleroderma Skin Activity Index) of all existing lesions during treatment, at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
LoSDI all lesions
Change in LoSDI (Localized Scleroderma Skin Damage Index) of all existing lesions during treatment, at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
Number of lesions
Count of all existing non-target and new lesions on the entire integument during treatment, at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
DLQI
Change in DermatoLogy Quality of life Index (DLQI). The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Measured: Baseline to Follow-Up Visit, 48 weeks
RNAseq
Gene signature of localized scleroderma after Dupilumab treatment: RNA- seq of tissue biopsies obtained from the lesion (lilac ring, optional: center) before (baseline visit V1) and after treatment (EoT V14) and healthy skin before treatment
Measured: Baseline to End of Treatment Visit, 24 weeks
RT-qPCR
Quantification of change in gene expression of genes identified by RNAseq in tissue of localized scleroderma patients after Dupilumab treatment: RT-qPCR of tissue biopsies obtained from the lesion (lilac ring, optional: center) before (baseline visit V1) and after treatment (End of Treatment Visit V14) and healthy skin before treatment
Measured: Baseline to End of Treatment Visit, 24 weeks
Adverse events (AEs)
Adverse events will be documented throughout the study
Measured: Baseline to Follow-Up Visit, 48 weeks
Physical examination
Physical examination (general appearance including skin, head and throat (head, eyes, ears, nose, and throat), lymph nodes, respiratory, cardiovascular, musculoskeletal, and neurological systems) will be documented throughout the study.
Measured: Baseline to Follow-Up Visit, 48 weeks
Body weight
Body weight will be documented throughout the study.
Measured: Baseline to Follow-Up Visit, 48 weeks
Blood pressure
Blood pressure will be documented throughout the study.
Measured: Baseline to Follow-Up Visit, 48 weeks
Pulse rate
Pulse rate will be documented throughout the study.
Measured: Baseline to Follow-Up Visit, 48 weeks
Body temperature
Body temperature will be documented throughout the study.
Measured: Baseline to Follow-Up Visit, 48 weeks
Haematocrit (HcT)
Haematocrit (HcT) will be documented at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
Haemoglobin
Haemoglobin (Hgb) will be documented at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
Blood cell count
Platelet count and differential White blood cell count will be documented at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
Blood Enzymes
Clinical Chemistry parameters (Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), Gamma-glutamyl transferase (GGT), Lactic dehydrogenase (LDH)) will be documented at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
Clinical Chemistry
Clinical Chemistry parameters (Creatinine) will be documented at End of Treatment Visit and during follow-up
Measured: Baseline to Follow-Up Visit, 48 weeks
Anti-nuclear antibodies (ANAs) levels
Change in anti-nuclear antibodies (ANAs) levels
Measured: Baseline to Follow-Up Visit, 48 weeks
Serum cytokine levels
Change in serum levels of IL-4, IL-5, IL-13, periostin, dipeptidyl peptidase-4
Measured: Baseline to Follow-Up Visit, 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2019
- Study start19 May 2020
- Primary completion30 Nov 2023
- Study completion30 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Localized SclerodermaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, multi-center, double-blind, placebo controlled, parallel group, multiple dose, phase IIa trial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Cologne
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sabine Eming, University of Cologne
- Investigators
- Sabine Eming, Prof. Dr., Principal investigator, University of Cologne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04200755
- Sponsor's protocol ID
- Uni-Koeln-3815
- Other IDs
- 2019-002036-90, 2019
- Acronym
- DupiMorph
- Status last verified
- Oct 2024
- First submitted
- 10 Dec 2019
- Last updated
- 26 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Eming SA, Kreuter A, Hahn M, Ghoreschi K, Moinzadeh P, Hunzelmann N, Becker K, Reiche N, Adams A, Shah-Hosseini K, Hellmich M, Willenborg S, Sanin DE, Rocken M, Krieg T. Interleukin 4-receptor-alpha blockade in localised scleroderma: a phase 2 proof-of-concept study. Ann Rheum Dis. 2026 Oct 29:S0003-4967(26)00605-9. doi: 10.1016/j.ard.2026.09.022. Online ahead of print.
PubMed 42957460
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-controlled Phase IIa Clinical Trial to Evaluate the Efficacy and Safety of Subcutaneous Dupilumab in Localized Scleroderma
- ClinicalTrials.gov ID
- NCT04200755
- Acronym / sponsor ID
- DupiMorph / Uni-Koeln-3815
- Conditions
- Localized Scleroderma
- Keywords
- Morphea
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- University of CologneUniversity, hospital or other
- Study start
- 19 May 2020 (actual)
- Primary completion
- 30 Nov 2023 (actual)
- Study completion
- 30 Nov 2023 (actual)
- First posted
- 16 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The DupiMorph study evaluates the efficacy of Dupilumab in localized scleroderma patients. Dupilumab is approved in the US and EU for the treatment of moderate/severe atopic dermatitis and since 2018 in the US for severe asthma therapy.
Detailed description
Localized scleroderma (LS) comprises a heterogenous group of sclerotic skin disorders. The incidence of LS is reported to be approximately 27 cases/ 1x106 and is hence approximately 2-3-fold higher compared to systemic scleroderma. Although in most cases not lethal the disease can significantly impact quality of life. Depending on the location of fibrosis, the disorder can cause bone deformities, alopecia, skin atrophy or lesions with severe hypo-/hyperpigmentation.
The disease is pathomechanistically poorly understood and no effective therapy is currently approved. The most promising treatments up to date include methotrexate ± pulsed corticosteroids or phototherapy with PUVA or UVA1. Yet, the number of treated patients in these studies is low. The response rates are low and inconsistent with approximately 50% of patients treated with UVA1 experience a recurrence within three years. There are no studies on efficacy of topical corticosteroids in LS. Small pilot studies and few case reports describe regression of lesions after topical calcineurin inhibitors. In addition, current therapies can only be applied for a short time during the acute phase due to the side effect profile after long-term use (e.g. skin atrophy in response to topical steroids, skin cancer in response to long term UV therapy, multiple side effects by long- term use of methotrexate and/or corticosteroids). Hence, this study evaluates, in comparison with placebo, the efficacy of Dupilumab administered subcutaneously every 14 days in patients with Morphea (plaque type) or generalized localized scleroderma (affecting at least three anatomic sites).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Randomized, multi-center, double-blind, placebo controlled, parallel group, multiple dose, phase IIa trial
| Arm | Type | Receives |
|---|---|---|
| Dupilumab | Experimental | Drug: Dupilumab 300Mg Solution for Injection30 patients; Dupilumab s.c. injection; 2 ready-to-use syringes (600 mg) initial (V1), 1 ready-to-use syringe (300 mg) every 14 days (V2- V13) Dupilumab s.c. injection in healthy skin, 24 weeks |
| Placebo | Placebo comparator | Other: Placebo15 patients; placebo s.c. injection; 2 ready-to-use syringes initial (V1), 1 ready-to-use syringe every 14 days (V2-V13) placebo s.c. injection in healthy skin, 24 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subject is a male or female ≥18 years of age on the day the study informed consent is signed
- Out-patient status
- Caucasian
- Morphea (plaque type) or Generalized localized scleroderma (affecting at least three anatomic sites)
- At least one lesions with lilac ring (active phase of the disease);
Key exclusion criteria (15 listed)
- Systemic immunosuppressive therapy or UV therapy less than 3 months before enrollment.
- Participation in another trial of IMPs or devices parallel to, or less than 6 months before or previous participation in this trial
- Pregnancy or breastfeeding mother
- Diagnosis of other significant chronic inflammatory or autoimmune disorders. Patients with the following autoimmune disorders are excluded from the study: Multiple sclerosis, primary biliary cirrhosis, type I diabetes mellitus. Patients with the following autoimmune disorders are regarded as eligible: Lichen sclerosus, vitiligo, alopecia arthritis, thyroid diseases (e.g. Hashimoto disease). Patients with any autoimmune disorder not listed above should only be included after consultation with the principal coordinating investigator.
- Topical immunosuppressive therapy less than 1 month before enrollment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dupilumab 300Mg Solution for InjectionAlso: DupixentFirst dose: 600 mg (2 syringes); subsequent doses: 300 mg (1 syringe) | Drug | Dupilumab |
| PlaceboFirst dose: 2 syringes, no active substance; subsequent doses: 1 syringe, no active substance | Other | Placebo |
Primary outcome
LoSCAT target lesion
Time frame: Baseline to End of Treatment Visit, 24 weeks
Treatment response is assessed using the LoSCAT (Localized Scleroderma Cutaneous Assessment Tool). Target lesion will be assessed at Baseline and End of Treatment. Score reduction by 50% after 24 weeks (End of Treatment Visit V14) compared to Baseline Visit (V1) is defined as treatment response.
19 secondary outcomes
mLoSSI all lesions
Time frame: Baseline to Follow-Up Visit, 48 weeks
LoSDI all lesions
Time frame: Baseline to Follow-Up Visit, 48 weeks
Number of lesions
Time frame: Baseline to Follow-Up Visit, 48 weeks
DLQI
Time frame: Baseline to Follow-Up Visit, 48 weeks
RNAseq
Time frame: Baseline to End of Treatment Visit, 24 weeks
RT-qPCR
Time frame: Baseline to End of Treatment Visit, 24 weeks
Adverse events (AEs)
Time frame: Baseline to Follow-Up Visit, 48 weeks
Physical examination
Time frame: Baseline to Follow-Up Visit, 48 weeks
Body weight
Time frame: Baseline to Follow-Up Visit, 48 weeks
Blood pressure
Time frame: Baseline to Follow-Up Visit, 48 weeks
Pulse rate
Time frame: Baseline to Follow-Up Visit, 48 weeks
Body temperature
Time frame: Baseline to Follow-Up Visit, 48 weeks
Haematocrit (HcT)
Time frame: Baseline to Follow-Up Visit, 48 weeks
Haemoglobin
Time frame: Baseline to Follow-Up Visit, 48 weeks
Blood cell count
Time frame: Baseline to Follow-Up Visit, 48 weeks
Blood Enzymes
Time frame: Baseline to Follow-Up Visit, 48 weeks
Clinical Chemistry
Time frame: Baseline to Follow-Up Visit, 48 weeks
Anti-nuclear antibodies (ANAs) levels
Time frame: Baseline to Follow-Up Visit, 48 weeks
Serum cytokine levels
Time frame: Baseline to Follow-Up Visit, 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2024.
- Derived from this study: PubMed 42957460Eming SA, Kreuter A, Hahn M, Ghoreschi K, Moinzadeh P, Hunzelmann N, Becker K, Reiche N, Adams A, Shah-Hosseini K, Hellmich M, Willenborg S, Sanin DE, Rocken M, Krieg T. Interleukin 4-receptor-alpha blockade in localised scleroderma: a phase 2 proof-of-concept study. Ann Rheum Dis. 2026 Oct 29:S0003-4967(26)00605-9. doi: 10.1016/j.ard.2026.09.022. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sabine Eming, Prof. Dr. principal investigator
University of Cologne
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dupilumab 300Mg Solution for Injection in people with Localized Scleroderma, run by University of Cologne at 4 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 15 exclusion criteria. 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 26 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Germany. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
LoSCAT target lesion (time frame: Baseline to End of Treatment Visit, 24 weeks). 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 NCT04200755, which also lists 1 linked publication.
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 26 Oct 2024.