E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Raynaud’s phenomenon secondary to systemic sclerosis |
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E.1.1.1 | Medical condition in easily understood language |
Raynaud’s phenomenon due to systemic sclerosis |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10037912 |
E.1.2 | Term | Raynaud's phenomenon |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To explore the effect of a single subcutaneous (SC) dose of CAM2043 on skin temperature (an indirect measure of digital vascular perfusion), as evaluated by thermography following controlled cold challenge in patients with Raynaud’s phenomenon secondary to systemic sclerosis |
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E.2.2 | Secondary objectives of the trial |
To explore the effect of a single SC dose of CAM2043 on skin temperature
To assess the pharmacokinetics (PK) of treprostinil following a single SC dose of CAM2043 to patients with Raynaud’s phenomenon secondary to systemic sclerosis
To assess the effect of a single SC dose of CAM2043 on patient reported outcomes in patients with Raynaud’s phenomenon secondary to systemic sclerosis
To explore the PK/pharmacodynamic (PD) relationships between treprostinil concentration and AUC and maximum skin temperature for thermography
To assess the safety and tolerability of a single SC dose of CAM2043 in patients with Raynaud’s phenomenon secondary to systemic sclerosis
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients with a diagnosis of systemic sclerosis, as defined by 2013 American College of Rheumatology (ACR) criteria / European League against Rheumatism (EULAR) criteria.
Patients with Raynaud’s phenomenon secondary to systemic sclerosis with a minimum of 5 Raynaud’s phenomenon attacks per week.
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E.4 | Principal exclusion criteria |
Presence of active digital tip ulceration that has developed within 4 weeks before the Screening Visit. Active digital tip ulceration is defined by 1) visible loss of epithelial integrity or 2) an ulcer covered with eschar which is associated with ongoing moderate pain. Chronic ulcers with overlying eschar but with absent to mild pain are considered eligible.
A variability of more than ±20% between 2 assessments of thermography which are separated by at least 3 hours at the Screening Visit.
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E.5 End points |
E.5.1 | Primary end point(s) |
- Change from Baseline to 6 h post-dose in the area under the curve (AUC) for reperfusion/rewarming (AUCtherm) as derived from thermography measurements |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Plasma concentrations of treprostinil
- Change from baseline in Raynaud’s Condition Score
- Change from baseline in Patient Global Assessment
- Adverse events (AEs)
- Clinical chemistry and hematology (including coagulation)
- Urinalysis
- Vital signs
- Electrocardiogram (ECG) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Days -14 to Day 0, Day 1, Day 2, Day 4, Day 8, Day 15
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 5 |
E.8.9.2 | In all countries concerned by the trial days | 0 |