E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cutaneous T Cell Lymphoma |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 22.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028483 |
E.1.2 | Term | Mycosis fungoides |
E.1.2 | System Organ Class | 100000004864 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the efficacy and safety of two concentrations (0.03% and 0.06%) of resiquimod gel (CD11301 gel) in the treatment of CTCL (stage IA, IB, or IIA) versus placebo. |
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E.2.2 | Secondary objectives of the trial |
To compare and characterize the pharmacokinetic profile of two concentrations of resiquimod gel (0.03% and 0.06%) applied topically on up to 10% Body Surface Area (BSA) in subjects with early stage CTCL.
To assess a systemic effect of resiquimod gel on lesions distant from the treatment area(s). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Adults ≥ 18 years of age at the screening visit.
2. Have a clinical diagnosis of cutaneous T cell lymphoma (CTCL) stage IA, IB, or IIA including documentation of a skin biopsy within the last twelve (12) months with histological findings consistent with CTCL (Olsen et al, 2007). If the histological documentation is not available, a (non-target lesion) skin biopsy may be performed at the Screening visit for confirmation.
3. For stage IIA, only subjects with a classification of N0 (No clinically abnormal peripheral lymph nodes) or N1 (clinically abnormal lymph node(s) histopathology Dutch grade 1 or NCI LN0-2) can be enrolled.
4. Subjects must be B0 (absence of significant blood involvement: 5% of peripheral blood lymphocytes or <250/mcL are atypical (Sezary) cells, fewer than 20% Sezary cells or fewer than 250 Sezary cells/μL.
One of the following can be substituted for Sezary cells: up to 1,600/μL CD4 cells and lower than 250/μL CD4+/CD26- or CD4+/CD7- cells
5. Subjects may only be included when therapies, according to the S2k – Leitlinie – Kutane Lymphome, Stand 08/2017, are either contraindicated and/or ineffective and/or not well tolerated. |
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E.4 | Principal exclusion criteria |
1. Participation in previous studies with resiquimod.
2. Known or suspected allergies or sensitivities to any component of the resiquimod gel.
3. History of clinically meaningful allergic reactions to imiquimod.
4. History of autoimmune disease including (but not limited to) rheumatoid arthritis, autoimmune hepatitis, autoimmune thyroiditis, Sjögren syndrome, psoriasis, or systemic lupus erythematosus. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall response rate (complete and partial response) of target lesions: based upon Modified Composite Assessment of Index Lesion Severity (CAILS) score. |
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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) |
1) Overall response rate (CR and PR) based upon mSWAT composite score.
2) Time to overall response (CR or PR) response based on mCAILS score.
3) Duration of overall (CR or PR) response based on mCAILS score
4) Time to progressive disease using mSWAT
5) Skindex29 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
at Week 12 except 2, 3, 4, 5 |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |