E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Allergy to peanuts or peanut-containing foods |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
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 |
The primary objective is to assess the safety and tolerability of AR101 [Characterized Peanut Allergen (CPNA)] when used in an oral immunotherapy (OIT) paradigm over an extended period (≥ 18 months) in peanut-allergic children and young adults who initiated OIT between the ages of 4 to 26 years, inclusive. |
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E.2.2 | Secondary objectives of the trial |
• To confirm and extend prior observations from ARC001 on the efficacy of Characterized Peanut Allergen in OIT, as assessed through reduction in clinical reactivity to limited amounts of peanut allergen. • To evaluate the immunological effects of peanut OIT • To determine the time course of tolerated up-dosing • To evaluate safety based on physician global assessment of disease activity The exploratory objective is to assess a higher degree of desensitization, based on an Open-label Food Challenge (OFC) up to a cumulative dose of 4043 mg of total peanut protein. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Completion of study ARC001 • Written informed consent from subject and/or parent/guardian • Written assent from all subjects as appropriate • Use of birth control for females of child-bearing potential • No change in the status of any longitudinally applicable ARC001 inclusion criteria |
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E.4 | Principal exclusion criteria |
• Early termination from ARC001 • For former active AR101 (CPNA) treatment subjects from ARC001 (Group 2), failure to tolerate with no or mild symptoms 300 mg of peanut protein in their ARC001 exit DBPCFC • Pregnancy or lactation • For former placebo subjects from ARC001 (Group 1), a lapse in dosing of more than 10 days from completion of ARC001 • Change in the status of any longitudinally applicable ARC001 exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary end-point is the incidence of treatment-related adverse events and dosing symptoms occurring with peanut OIT over a protracted treatment period comprising at least 18 months. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
After approximately 18 months of treatment |
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E.5.2 | Secondary end point(s) |
• The proportion of subjects who tolerate at least 300 mg (443 mg cumulative) of peanut protein with no more than mild symptoms during DBPCFC • The proportion of subjects who tolerate at least 600 mg (1043 mg cumulative) of peanut protein with no more than mild symptoms during DBPCFC • The proportion of subjects who tolerate 1000 mg (2043 mg cumulative) of peanut protein with no more than mild symptoms during DBPCFC • Change from baseline (carried over from ARC001) in maximum dose of peanut protein tolerated with no, or only mild, symptoms during DBPCFC • Maximum dose of peanut protein tolerated with no, or only mild, symptoms during DBPCFC • Changes in peanut-specific immunoglobulin E (IgE) and immunoglobulin G4 (IgG4), changes in skin prick test (SPT) mean wheal diameters will also be assessed. • Physician global assessment: Disease activity as measured on a 100 mm visual analogue scale (VAS) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.6 | Pharmacokinetic | No |
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 | 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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | Yes |
E.8.6.3 | Specify the countries outside of the EEA 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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Last assessment for the last subject in the study |
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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 | 0 |
E.8.9.2 | In all countries concerned by the trial months | 18 |
E.8.9.2 | In all countries concerned by the trial days | 0 |