Drug Rediscovery for rare Immune Mediated Inflammatory Diseases (DRIMID)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

University Medical Center Utrecht

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

30 May 2024 → ongoing

Decision date (initial)

2023-12-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2022-502968-20-02

ClinicalTrials.gov

NCT06285539

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

To determine the effects of filgotinib, an approved JAK-inhibitor, on changes in quality of life, disease activity and safety in patients with refractory BD or IIM

Conditions and MedDRA coding

Behcet's disease

Study design 4 periods

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Age 18 years or older
2. Refractory disease, defined as symptomatic disease that persists despite a 12-week trial of corticoid therapy as well as lack of response to at least prednisone and one other immunosuppressive agent such as methotrexate (MTX), mycophenolate mofetil (MMF), azathioprine (AZA) or rituximab or intolerance to standard-of-care treatment, as defined by the treating physician.
3. No evidence of active or latent or inadequately treated infection with mycobacterium tuberculosis (TB) as defined by all of the following: both a negative QuantiFERON-TB Gold (QFT-G) In-Tube test and a Mantoux tuberculin skin test performed at or within 3 months prior to screening and no signs suggestive of active TB infection as determined (and documented) by a qualified radiologist or pulmonologist as per local standard of care on a chest radiograph and no history of either untreated or inadequately treated latent or active TB infection.
4. One of the following: (1) Diagnosis of Behçet’s disease without refractory life, organ or sight-threatening symtoms with active disease, defined as a BDCAF >2 or with active disease, based on clinical grounds (e.g. the need to start new or additional medication) or (2) Diagnosis of idiopathic inflammatory myopathy, according to diagnostic criteria: Dermatomyositis Classification Criteria according to the European Neuromuscular Centre guidelines 2018 or Anti-synthetase syndrome Classification Criteria according to the European Neuromuscular Centre guidelines 2003 or overlap/non-specific myositis, including polymyositis with active disease, defined as: dermatomyositis with a CDASI score of ≥5 or abnormal levels of at least 1 of the following enzymes: creatine kinase (≥ 4× upper limit of normal [ULN]), aldolase (≥4× ULN), lactate dehydrogenase (LDH ≥4× ULN), aspartate transaminase (AST ≥4× ULN), alanine aminotransferase (ALT ≥4× ULN) or MRI within the last 3 months indicative of active inflammation (e.g. edema signal pattern in affected proximal muscles) or active disease based on clinical grounds, e.g. the need to start new or additional medication

Exclusion criteria 26

1. Age <18 years
2. History of VTE
3. Concomitant malignancies or previous malignancies within the last five years (with exception of adequately treated basal or squamous cell carcinoma of the skin)
4. Kidney injury with estimated glomerular filtration rate <15mL/min/1.73m2
5. Liver failure Child Pugh C
6. Absolute neutrophil count <1*109
7. Absolute leukocyte count <0.5*109
8. Hemoglobin <5mmol/L
9. Inability to comply with study and/or follow-up procedures
10. Known recent substance abuse (drugs or alcohol).
11. Poor tolerability of venipuncture or lack of adequate venous access for required blood sampling during the study period.
12. Life expectancy less than 6 months
13. Previous non-adherence to immunosuppressants
14. Hypersensitivity to the active substance or to any of the excipients
15. Rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption
16. Myositis overlapping with other autoimmune diseases, immune mediated necrotizing myopathy (IMNM) or cancer-associated myositis
17. End-stage IIM wherein muscle weakness is most likely due to muscle damage, rather than myositis disease activity
18. Pregnancy or lactation
19. Previous use of other JAK-inhibitors
20. Use of any investigational drug within one month prior to screening or within five half-lives of the investigational agent, whichever is longer.
21. History of HIV
22. Presence of an active infection or hepatitis
23. Age ≥65 years
24. Increased risk of major cardiovascular problems
25. Current smoker or smoked for a long time in the past
26. Increased risk of cancer

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. EuroQol 5D-5L (EQ-5D-5L) form
2. Behçet’s Disease Current Activity Form (BDCAF)
3. Total Improvement Score (TIS) of the International Myositis Assessment Clinical Studies (IMACS) group

Secondary endpoints 10

1. AUC for number of oral ulcers
2. Cutaneous Dermatomyositis Disease Area and Severity index (CDASI)
3. Total number of flares in each disease, measured by a ≥1 point increase in Physician Global Assessment (PGA) on a scale from 0-3
4. Visual Analogue Scale (VAS) of disease activity, based on the clinical view of the local principal investigator
5. Glucocorticoid Toxicity Index (GTI)
6. Changes in glucocorticoid dose
7. VAS score of pain, patients perspective
8. Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F)
9. Patient Acceptable Symptom State (PASS)
10. Exposure-adjusted incidence rates for treatment-emergent adverse events

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

University Medical Center Utrecht

Sponsor organisation

University Medical Center Utrecht

Address

Heidelberglaan 100

City

Utrecht

Postcode

3584 CX

Country

Netherlands

Scientific contact point

Organisation

University Medical Center Utrecht

Contact name

Prof. dr. J.M. van Laar

Public contact point

Organisation

University Medical Center Utrecht

Contact name

Prof. dr. J.M. van Laar

Locations

1 EU/EEA country · 6 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 13 files

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

Application history

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