An open-label study to evaluate biomarkers and safety in systemic sclerosis patients treated with ABR-215757 (paquinimod)

• Register: EUCTR
• Last refreshed on: 27 October 2014
• Main ID: EUCTR2011-001667-44-SE
• Date of registration: 18/05/2011
• Prospective Registration: Yes
• Primary sponsor: Active Biotech AB
• Public title: An open-label study to evaluate biomarkers and safety in systemic sclerosis patients treated with ABR-215757 (paquinimod)
• Scientific title: An open-label study to evaluate biomarkers and safety in systemic sclerosis patients treated with ABR-215757 (paquinimod)
• Date of first enrolment: 28/06/2011
• Target sample size: 20
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001667-44
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Germany, Sweden, Switzerland

Contacts

• Name: Clinical Trials at Active Biotech
• Affiliation: Active Biotech AB

• Name: Clinical Trials at Active Biotech
• Affiliation: Active Biotech AB

Key inclusion & exclusion criteria

Inclusion criteria:
1. Age = 18 years at the time of signing the informed consent form
2. Clinical Diagnosis of SSc according to ACR criteria
3. Progressive SSc fulfilling at least one of the following:
- STPR (Skin Thickness Progression Rate) = 40, calculated as the mRSS at screening divided by time (in years) since the start of skin involvement. as reported by the patient (Denton 2007).
- Worsening of mRSS within the last 6 months as judged by the physician together with the patient, with involvement of at least two new anatomical sites as defined in the mRSS score (e.g. upper arm and thorax) or progression by at least two points in at least two anatomical sites as defined by the mRSS
4. Presence of SSc skin lesions on one or both forearms
5. Modified Rodnan Skin score (mRSS) =16 at baseline
6. ANA-positive

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Ongoing Severe SSc manifestations, such as pulmonary arterial hypertension (PAH) with dyspnea NYHA III or more, scleroderma renal crisis.
2. Vital capacity < 60% as measured within 6 months prior to the first dose of study medication
3. GFR < 30% of normal measured within 6 months prior to the first dose of study medication.
4. Treatment with Rituximab within 12 months or other biologic agent within 6 months, Mycophenolate mofetil (MMF) or Cyclophosphamide within 6 months, Methotrexate, Azathioprine or other immunosuppressants within 3 months prior to the first dose of study medication.
5. History of myocardial infarction or current uncontrolled angina, severe uncontrolled ventricular arrhythmias, symptomatic congestive heart failure, unstable angina pectoris, or electrocardiographic evidence of acute ischemia.
6. Marked baseline prolongation of QT/QTc interval (eg, repeated demonstration of a QTc interval >450 milliseconds
7. History of additional risk factors for torsade de pointes (eg, heart failure, hypokalemia, family history of long QT syndrome)
8. Treatment with concomitant medications that prolong the QT interval.
9. History of, or current ischemic CNS disease.
10. Current malignancy. A 5-year cancer-free period is required with the exception of skin basal or squamous cell carcinoma or cervical cancer in situ that has been excised.
11. Current severe infection
12. Known positive serology for HIV or active or latent hepatitis infection.
13. Treatment with endothelin receptor antagonist within 6 weeks prior to the first dose of study medication
14. Drug abuse
15. Major surgery within 3 weeks prior to study entry.
16. Known or suspected hypersensitivity to ABR-215757 or excipients.
17. Female patient of child-bearing potential who is not using a medically accepted safe method of contraception. All female patients of child-bearing potential must have a negative urine pregnancy test at the Screening and Baseline Visits. As interaction studies between ABR-215757 and hormonal contraceptives have not yet been performed, women using hormonal contraceptives such as the contraceptive pill, must also use a complementary contraceptive device, i.e. barrier method, during the treatment period and for at least 1 month thereafter.
18. Female patient of child-bearing potential who is pregnant or lactating.
19. Simultaneous participation or participation within 4 months or 5 half lives (whichever is longer) prior to study entry in any other study involving investigational drugs or other experimental therapy.
20. Other significant, unstable medical disease not related to SSc that in the investigator’s opinion would confound the study result or put the patient at risk
21. Patients likely to receive oral or intravenous steroids or immunosuppressant for other non-SSc condition during the study duration, as this will confound the study result.
22. Vaccination within 4 weeks prior to the first dose of study medication.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Systemic Sclerosis
MedDRA version: 14.0 Level: PT Classification code 10042953 Term: Systemic sclerosis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 14.0 Level: LLT Classification code 10036814 Term: Progressive systemic sclerosis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Intervention(s)

Product Name: paquinimod
Product Code: ABR-215757
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: paquinimod
CAS Number: 248282-01-1
Current Sponsor code: ABR-215757
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1.5-

Primary Outcome(s)

Main Objective: To study changes in disease related biomarkers in patients with progressive SSc during treatment with ABR-215757

Primary end point(s): • Changes in SSc disease activity related biomarkers

Secondary Objective: To assess the safety and tolerability of ABR-215757 in progressive SSc patients
To assess disease activity and quality of life (QoL) in patients with progressive SSc during treatment with ABR-215757
To assess the plasma levels of ABR-215757 during the study

Timepoint(s) of evaluation of this end point: baseline, 2, 4 and 8 weeks of treatment

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: baseline, 2, 4 and 8 weeks of treatment (In the open label continuation, safety and disease activity will be evaluated every 12 weeks)

Secondary end point(s): • Adverse events and changes in laboratory safety parameters.
• Disease activity
• Quality of life measured by the SF-36 health survey and by the scleroderma health assessment questionnaire (SHAQ)
• Plasma levels of ABR-215757

Secondary ID(s)

11575705

Source(s) of Monetary Support

Active Biotech AB