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 |
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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 | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013873 |
E.1.2 | Term | Dupuytren's contracture operation |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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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 |
The objectives of this study are to evaluate the safety and efficacy of Xiapex® in subjects with Dupuytren’s contracture, the recovery and associated use of Health Care in patients in Europe when physicians and subjects are given the opportunity to treat the affected hand (or hands) by selecting which cord and joint to treat with each injection |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subject eligibility should be reviewed and documented by an appropriately qualified member of the investigator’s study team before subjects are included in the study. Subjects must meet all of the following criteria to enter the study:
1. At least 18 years of age and ≤ 70 years.
2. Presenting with a Dupuytren’s contracture of at least 20 degrees caused by a palpable cord in at least one finger other than the thumb.
3. Positive “table top test” defined as the inability to simultaneously place the affected finger(s) and palm flat against a table top.
4. In good health, based upon the results of a medical history, physical examination, and safety laboratory profile.
5. Female subjects of child bearing potential must use a highly effective contraception method or be surgically sterilized or be a post menopausal female (ie, no menses for at least 1 year). A pregnancy test will be performed prior to enrollment in the study.
6. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
7. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. |
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E.4 | Principal exclusion criteria |
Subjects presenting with any of the following will not be included in the study:
1. Nursing or pregnant, or plan to become pregnant during the treatment phase.
2. On an investigational drug within 30 days prior to the first dose of study drug.
3. Received a treatment on the selected joint, within 90 days of enrollment in the study, for Dupuytren’s contracture including needle aponeurotomy or any surgical procedure.
4. Patients with a known systemic hypersensitivity to collagenase or any of the other product excipients.
5. On anticoagulant medication or has received anticoagulant medication (except aspirin less than 150 mg daily) within 7 days before the first injection.
6. Has any clinically significant medical history or condition(s), including conditions that affect the hands, that would, in the opinion of the investigator, substantially increase the risk associated with the subject’s participation in the protocol or compromise the scientific objectives of the study.
7. Has a chronic muscular, neurological, or neuromuscular disorder that affects the hands.
8. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
9. Has jewelry on the hand to be treated that cannot be removed. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety:
• AEs, vital signs, clinical laboratory parameters and ADAs.
Efficacy:
• TPED and PED (Total Passive Extension Deficit: a sum of the Passive Extension Deficits (PED) in the MP, PIP, and DIP joints).
• Change in TPED and PED for each joint treated.
• ROM.
• Patient and physician global assessment of treatment satisfaction and disease severity.
Recovery, Hand functionality and HCRU:
• Type and amount of concomitant pain medications.
• Total recovery time, time to use the hand, daily activities, work vs. hobbies.
• Hand functionality – Unité Rhumatologique des Affections de la Main (URAM) scale will be completed only for subjects in France, the United Kingdom, Germany, Sweden and Hungary.
• Healthcare resource utilization questionnaire.
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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) |
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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 | Yes |
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 | No |
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 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 25 |
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 |
Denmark |
France |
Germany |
Hungary |
Italy |
Spain |
Sweden |
United Kingdom |
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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.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |