Clinical Trial to Evaluate Efficacy and Safety of Dupilumab in Localized Scleroderma

NCT04200755 | Completed Phase 2 DMC

• 2 other identifiers: Uni-Koeln-38152019-002036-90
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Localized Scleroderma

Keywords

Morphea

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to End of Treatment Visit, 24 weeks

Secondary Outcomes (19)

• mLoSSI all lesions

  Baseline to Follow-Up Visit, 48 weeks

• LoSDI all lesions

  Baseline to Follow-Up Visit, 48 weeks

• Number of lesions

  Baseline to Follow-Up Visit, 48 weeks

• DLQI

  Baseline to Follow-Up Visit, 48 weeks

• RNAseq

  Baseline to End of Treatment Visit, 24 weeks

Study Arms (2)

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

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

Other: Placebo

Interventions

Dupilumab 300Mg Solution for Injection DRUG

First dose: 600 mg (2 syringes); subsequent doses: 300 mg (1 syringe)

Also known as: Dupixent

Dupilumab

Placebo OTHER

First dose: 2 syringes, no active substance; subsequent doses: 1 syringe, no active substance

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• University of Cologne: lead

Study Sites (4)

Charité - Universitätsmedizin Berlin Klinik für Dermatologie, Venerologie und Allergologie

Berlin, 10117, Germany

Location

Uniklinik Köln, Klinik für Dermatologie und Venerologie

Cologne, 50924, Germany

Location

Helios St. Elisabeth Klinik Oberhausen, Klinik für Dermatologie, Venerologie und Allergologie

Oberhausen, 46045, Germany

Location

Universitäts-Hautklinik Tübingen

Tübingen, 72076, Germany

Location

MeSH Terms

Conditions

Scleroderma, Localized

Interventions

dupilumab; Solutions; Injections

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Pharmaceutical Preparations; Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Sabine Eming, Prof. Dr.

  University of Cologne

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Permuted blocks of varying length with allocation ratio (verum:placebo = 2:1); double-blind, i.e. patients and investigators are masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Coordinating Investigator

Model Details: Randomized, multi-center, double-blind, placebo controlled, parallel group, multiple dose, phase IIa trial

Study Record Dates

• First Submitted: December 10, 2019
• First Posted: December 16, 2019
• Study Start: May 19, 2020
• Primary Completion: November 30, 2023
• Study Completion: November 30, 2023
• Last Updated: October 26, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

DE (4)