E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Juvenile Psoriatic Arthritis |
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E.1.1.1 | Medical condition in easily understood language |
Juvenile Psoriatic Arthritis |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10076674 |
E.1.2 | Term | Juvenile psoriatic arthritis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
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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 |
- Evaluate PK of ustekinumab in jPsA - Evaluate efficacy of ustekinumab in jPsA |
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E.2.2 | Secondary objectives of the trial |
- Evaluate PK of ustekinumab in jPsA - Evaluate efficacy of ustekinumab in jPsA - Evaluate safety of ustekinumab in jPsA - Evaluate immunogenicity of ustekinumab in jPsA |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. ≥5 to <18 years of age, inclusive. 2. Diagnosis of jPsA by Vancouver inclusion criteria, with exclusion of ERA. Diagnosis made ≥3 months prior to screening. Arthritis plus psoriasis, or arthritis plus at least 2 of the following: dactylitis, nail pits, family history in a first or second-degree relative, psoriasis-like rash. 3. Active disease in ≥3 joints at screening and at Week 0 (defined as swelling OR loss of motion with pain and/or tenderness). Swelling alone meets the criteria for an active arthritic joint. In the absence of swelling, loss of motion with pain or tenderness or both pain and tenderness meet the criteria for an active arthritic joint. 4. Medically stable on the basis of physical examination, medical history, and vital signs performed at screening. Any abnormalities must be consistent with the underlying illness in the study population and this determination must be recorded in the participant’s source documents and initialed by the investigator. 5. Medically stable on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel, hematology, or urinalysis are outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to not be clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator. |
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E.4 | Principal exclusion criteria |
1. Participants with enthesitis-related arthritis (ERA) 2. Taken any disallowed therapies as noted in Section 6.8, Concomitant Therapy of the study protocol before the planned first dose of study intervention. 3. If participants were nonresponders to previously received biologic treatment (excluding anti TNFαs), including, but not limited to, guselkumab, secukinumab (AIN457), tildrakizumab (MK3222), ixekizumab (LY2439821), brodalumab (AMG827), risankizumab (BI-655066), or other investigative biologic treatments for PsA or psoriasis. Prior nonresponse to an anti-TNFα inhibitor or to a Janus kinase (JAK) inhibitor is not an exclusion. 4. Received an investigational intervention (including investigational vaccines) or used an invasive investigational medical device within 4 months before the planned first dose of study intervention or is currently enrolled in an investigational study. Receipt of an investigational vaccine for COVID-19 is not an automatic exclusion criterion; discuss with medical monitor. 5. Have a history of latent or active granulomatous infection, including TB, histoplasmosis, or coccidioidomycosis, or have had a nontuberculous mycobacterial infection prior to screening. An exception is made for participants currently receiving treatment for latent TB with no evidence of active TB, or who have a history of latent TB and documentation of having completed appropriate treatment for latent TB within 3 years prior to the first administration of study agent (Section 5.1, Inclusion criterion 16.a of the study protocol). |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Observed steady-state trough concentrations and population PK model-predicted area under the curve at steady-state over a 12-week dosing interval (AUCss) at Week 28. 2. Percentage of participants with jPsA achieving the JIA ACR 30 criteria at Week 24. |
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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) |
PK 1. Observed steady-state trough concentrations and population PK model-predicted AUCss over a 12-week dosing interval at Week 52. Efficacy 2. Proportions of participants with JIA ACR 30 response at baseline and Weeks 4, 8, 12, 16, and 52. 3. Proportions of participants with JIA ACR 50 and 70 responses at baseline and Weeks 4, 8, 12, 16, 24, and 52. 4. Time to response measured using JIA ACR 30 from baseline through Week 24. 5. Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS) at Weeks 4, 8, 12, 16, 28, and 52. 6. Change from baseline in Psoriasis Area Severity Index (PASI) at Week 24. Safety 7. The occurrences and type of AEs, serious adverse events (SAEs), and reasonably related AEs including injection-site reactions and infections will be summarized. 8. The number of participants with abnormal laboratory parameters (hematology and chemistry) based on National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) toxicity grading will be summarized. Immunogenicity 9. The overall incidence of antibodies to ustekinumab (including peak titers) through Week 68. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1: Week 52 2: baseline and Weeks 4, 8, 12, 16, and 52 3: baseline and Weeks 4, 8, 12, 16, 24, and 52 4: from baseline through Week 24 5: Weeks 4, 8, 12, 16, 28, and 52 6: Week 24 7, 8, 9: from baseline through Week 68 |
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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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
Russian Federation |
Turkey |
United States |
France |
Germany |
Italy |
Spain |
United Kingdom |
Argentina |
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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.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |