Long-term Follow-up Study With Darvadstrocel in the Treatment of Complex Perianal Fistula

• Register: EUCTR
• Last refreshed on: 22 April 2024
• Main ID: EUCTR2019-000333-39-CZ
• Date of registration: 03/09/2019
• Prospective Registration: Yes
• Primary sponsor: Takeda Development Center Americas, Inc
• Public title: Long-term Follow-up Study With Darvadstrocel in the Treatment of Complex Perianal Fistula
• Scientific title: A Follow-up of a Phase 3 Study to Evaluate the Long-term Safety and Efficacy of Darvadstrocel in the Treatment of Complex Perianal Fistula in Subjects With Crohn’s Disease Who Have Participated in ADMIRE II Study - Long-term Follow-up Study With Darvadstrocel in the Treatment of Complex Perianal Fistula
• Date of first enrolment: 15/10/2019
• Target sample size: 150
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-000333-39
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Belgium, Canada, Czech Republic, Czechia, France, Germany, Hungary, Israel, Italy, Poland, Spain, United Kingdom, United States

Contacts

• Name: Clinical Operations
• Affiliation: Takeda Development Center Americas, Inc

• Name: Clinical Operations
• Affiliation: Takeda Development Center Americas, Inc

Key inclusion & exclusion criteria

Inclusion criteria:
Subject eligibility is determined according to the following criteria before entry into the study:

1. In the opinion of the investigator, the subject is capable of understanding and complying withprotocol requirements.
2. The subject or, when applicable, the subject’s legally acceptable representative signs and dates a written ICF and any required privacy authorization before the initiation of any study procedures.
3. The subject has participated in and completed the ADMIRE-CD II study (ie, did not discontinue).

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 124
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 26

Exclusion criteria:
Subjects will not be eligible for inclusion in the study if:

1. It has been more than 3 months since the subject completed the ADMIRE-CD II study.
2. Subjects are currently receiving, have received any investigational drug in the last 3 months before the inclusion in the study, or are planning to receive any investigational drug during the duration of this LTE study, except for prior participation in the ADMIRE-CD II study.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Perianal fistulising Crohn´s disease
MedDRA version: 20.0 Level: PT Classification code 10002156 Term: Anal fistula System Organ Class: 10017947 - Gastrointestinal disorders

Intervention(s)

Trade Name: Alofisel
Product Name: Cx601
Product Code: Cx601
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: DARVADSTROCEL
Current Sponsor code: Allogenic eASCs
Other descriptive name: Expanded human allogenic mesenchymal adult stem cells extracted from adipose tissue (eASCs)
Concentration unit: Other
Concentration type: equal
Concentration number: 5000000-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intralesional use

Primary Outcome(s)

Primary end point(s): - AEs.
- SAEs.
- Specific AESIs:
– Immunogenicity/alloimmune reactions.
– Tumorgenicity.
– Ectopic tissue formation.

Secondary Objective: To evaluate the long-term efficacy of a single dose of darvadstrocel for the treatment of complex perianal fistula(s) in subjects with CD.

Main Objective: To evaluate the long-term safety of a single dose of darvadstrocel in subjects with CD and complex perianal fistula by evaluation of AEs, serious adverse events (SAEs), and adverse events of special interest (AESIs).

Timepoint(s) of evaluation of this end point: Examinations/assessments at week 0, 52, 104.
Telephone calls every 3 months.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Examinations/assessments at week 0, 52, 104.
Telephone calls every 3 months.

Secondary end point(s): Proportion of subjects who achieve clinical remission at Week 104 and Week 156 after IMP administration.
– Clinical remission is defined as closure of all treated external fistula openings that were draining at baseline despite gentle finger compression.
- Proportion of subjects who achieve clinical response at Week 104 and Week 156 after IMP administration.
– Clinical response is defined as closure of at least 50% of all treated external fistula openings that were draining at baseline despite gentle finger compression.
Proportion of subjects with a relapse where a relapse is defined as:
– Reopening of any of the treated fistula(s) external openings with active drainage as clinically assessed in subjects who were in clinical remission at Week 52, or
– The development of a perianal fluid collection >2 cm of the treated perianal fistulas confirmed by centrally read magnetic resonance imaging (MRI) assessment.
- Proportion of subjects who achieve combined remission at Week 156 after IMP
administration.
–- Combined remission of complex perianal fistula(s), defined as the clinical assessment of closure of all treated external openings that were draining at baseline (ie, screening visit), despite gentle finger compression, and
–- Absence of collection(s) >2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by centrally read blinded MRI assessment.
- Proportion of subjects with new anal abscess in treated fistula.
- Change from baseline to Week 104 and Week 156 after IMP administration in in scores of discharge and pain items of Perianal Disease Activity Index (PDAI) score.

Secondary ID(s)

Darvadstrocel-3003

Source(s) of Monetary Support

Takeda Development Center Americas, Inc

Ethics review

Status: Approved
Approval date: 02/10/2019
Contact: