E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Dupuytren's Contracture |
Contrattura di Dupuytren's |
|
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 | 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 |
|
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 |
• Evaluate the impact of Xiapex on the Dupuytren’s contracture, the Range of Motion (ROM) and the patient and physician reported treatment satisfaction and disease severity and their relationship to ROM. • Assess the recovery to normal activities. • Recovery time (How long overall, Time to use hand, time to return to work or daily activities, amount of work or daily activity time missed or reduced and affects on productivity and daily activities) - assessed via patient diary. • Use of concomitant analgesic medications. • Evaluate total healthcare resource utilization (HCRU) (eg, medical and allied healthcare visits and resources, use of splinting). • Assess the pattern of treatment when multiple joints and or/fingers are treated. |
- Valutare l’impatto di Xiapex sulla contrattura di Dupuytren, sul ROM (Range of Motion, escursione articolare), sulla soddisfazione del paziente e del medico riguardo al trattamento, sulla gravita' della malattia e sulla loro relazione nei confronti del ROM.
- Valutare il ritorno alle attivita' normali. Il tempo di recupero (tempo complessivo, tempo fino all’uso della mano, tempo per ritornare al lavoro o alle attivita' quotidiane, tempo di lavoro o dedicato alle attivita' quotidiane perso o ridotto ed effetti sulla produttivita' e sulle attivita' quotidiane) viene valutato per mezzo del diario del paziente.
-Valutare l’uso di analgesici concomitanti.
- Valutare lo schema di trattamento nel caso siano trattate diverse articolazioni e/o dita.
- Valutare l’utilizzo complessivo di servizi sanitari (per es. visite mediche e paramediche e servizi sanitari correlati, uso di stecche)
-ecc. |
|
E.2.2 | Secondary objectives of the trial |
Not Applicable |
non applicabile |
|
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 an highly effective method of birth control 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. |
1. Eta' di almeno 18 anni e ≤70 anni.
2. Manifestare una contrattura di Dupuytren con un valore di almeno 20° causata da una corda palpabile in almeno un dito (ad eccezione del pollice).
3. “Test del tavolo” positivo, definito come incapacita' di appoggiare contemporaneamente il dito o le dita affette e il palmo della mano sul piano di un tavolo.
4. Essere in buona salute in base ai risultati di anamnesi medica, esame obiettivo generale e profilo di sicurezza di laboratorio.
5. Le donne fertili devono usare un metodo contraccettivo riconosciuto o devono essere sterilizzate chirurgicamente oppure essere in postmenopausa (ossia non avere le mestruazioni da almeno 1 anno). Prima di essere arruolate nello studio sara' eseguito un test di gravidanza.
6. Presenza di un foglio di consenso informato firmato e datato personalmente che indichi che il soggetto (o un rappresentante legalmente riconosciuto) e' stato informato di tutti gli aspetti attinenti allo studio.
7. Essere soggetti che desiderano e sono in grado di osservare le visite programmate, lo schema terapeutico, i test di laboratorio e le altre procedure dello studio. |
|
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. |
1. In allattamento o in gravidanza o intenzione di iniziare una gravidanza durante la fase di trattamento.
2. In trattamento con un farmaco sperimentale nei 30 giorni che precedono la somministrazione della prima dose del farmaco in studio.
3. Sottoposti a trattamenti dell’articolazione prescelta contro la contrattura di Dupuytren entro 90 giorni dall’arruolamento nello studio. Questi trattamenti comprendono l’aponeurotomia con ago o una procedura chirurgica qualsiasi.
4. Con nota ipersensibilita' sistemica alla collagenasi o a uno qualsiasi degli altri eccipienti del prodotto.
5. In terapia anticoagulante o che hanno ricevuto un farmaco anticoagulante (eccetto l’aspirina in dose inferiore a 150 mg al giorno) nei 7 giorni precedenti alla prima iniezione.
6. Con anamnesi medica o disturbi clinicamente significativi, comprese affezioni delle mani che secondo l’opinione dello sperimentatore aumenterebbero sostanzialmente il rischio associato alla partecipazione del soggetto al protocollo o comprometterebbero gli obiettivi scientifici dello studio.
7. Presenza di disturbi muscolari, neurologici o neuromuscolari cronici a carico delle mani.
8. Presenza di altre condizioni organiche o psichiche acute o croniche gravi che possono incrementare il rischio associato alla partecipazione allo studio o alla somministrazione del prodotto sperimentale, oppure che potrebbero interferire con l’interpretazione dei risultati dello studio e che, a giudizio dello sperimentatore, renderebbero il soggetto non idoneo alla partecipazione a questo studio.
9. Con gioielli che non possono essere rimossi dalla mano da trattare. |
|
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 and HCRU: • Type and amount of concomitant pain medications. • Total recovery time, time to use the hand, daily activities, work vs. hobbies. • Healthcare resource utilization questionnaire. |
Sicurezza:
#AEs;
#segni vitali
#paramemtri clinici di laboratorio;
#ADAs;
Efficacia:
#TPDE e PED(deficit di stensione totale passiva: somma del deficit passivo di estensione -PED- nelle giunzioni metacarpofalangee-MP-, interfalangee prossimali-PIP- e interfalangee distali-DIP;
# midifiche in TPED e PED in ciascuna giunzione trattata;
#ROM;
#soddisfazione del paziente e del medico riguardo al trattamento, sulla gravita' della malattia |
|
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 | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 5 |
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 | 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
|
|
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 | 19 |
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 | 19 |
E.8.9.2 | In all countries concerned by the trial days | 0 |