Long-term Follow-up Study With Darvadstrocel in the Treatment of Complex Perianal Fistula
- Conditions
- Complex Perianal FistulaCrohn's Disease
- Interventions
- Biological: Darvadstrocel
- Registration Number
- NCT04075825
- Lead Sponsor
- Takeda
- Brief Summary
The main aim is to follow-up on long term side effect and symptom improvement of Darvadstrocel in the treatment of complex perianal fistula in adults. Participants will not receive any drug in this study.
- Detailed Description
The drug being tested in this study is called darvadstrocel (Cx601). Darvadstrocel is being tested to treat people who have complex perianal fistula in CD. This study will look at the long-term safety and efficacy of darvadstrocel in the treatment of complex perianal fistula in CD.
The study will enroll approximately 150 patients. Participants who received darvadstrocel or placebo in study ADMIRE-CD II (Cx601-0303, NCT03279081) and who have completed the 52 weeks of the study will be enrolled in this long-term extension study.
This multi-center study will be conducted worldwide. The overall time to participate in this study is 104 weeks (in addition to the 52 weeks on ADMIRE-CD II study). Participants will make multiple visits to the clinic and will be contacted by telephone every 3 months for a follow-up assessment. After unblinding of the ADMIRE-CD II study, the LTE study will be conducted as an open-label study. Participants will remain in the treatment group assigned in the ADMIRE-CD II study.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 151
- Has participated in and completed the ADMIRE-CD II (NCT03279081) study (i.e., did not discontinue).
- Has been more than 3 months since the participant completed the ADMIRE-CD II study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Darvadstrocel Darvadstrocel Participants who received a single dose of darvadstrocel, 120 million cells, intralesionally or darvadstrocel matching placebo previously in the ADMIRE-CD II study will be observed for efficacy and safety. No drug administration in this study.
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events (AEs) Baseline (Week 52 of ADMIRE-CD II) up to Week 156 after investigational medicinal product (IMP) administration An AE is defined as any untoward medical occurrence in a clinical investigation participant administered an investigational medicinal product; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug.
Number of Participants With Specific Adverse Events of Special Interest (AESIs) Baseline (Week 52 of ADMIRE-CD II) up to Week 156 after IMP administration AESIs are AEs that are not solicited local or systemic AEs, they are predefined AEs that required close monitoring and prompt reporting to the sponsor. AESIs included protocol specified immunogenicity/alloimmune reactions, tumorgenicity and ectopic tissue formation.
Number of Participants With Serious Adverse Events (SAEs) Baseline (Week 52 of ADMIRE-CD II) up to Week 156 after IMP administration An SAE is defined as an untoward medical occurrence, significant hazard, contraindication, side effect or precaution that at any dose: results in death, is life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is medically significant.
- Secondary Outcome Measures
Name Time Method Change from Baseline of ADMIRE-CD II in Scores of Discharge Items of Perianal Disease Activity Index (PDAI) Score at Weeks 104 and 156 Weeks 104 and 156 The PDAI is a scoring system to evaluate the severity of perianal CD. From the 5-item instrument, discharge will be used. Each category is graded on a 5-point Likert scale ranging from no symptoms (score of 0) to severe symptoms (score of 4); a higher score indicates more severe disease.
Change from Baseline of ADMIRE-CD II in Scores of Pain Items of Perianal Disease Activity Index (PDAI) Score at Weeks 104 and 156 Weeks 104 and 156 The PDAI is a scoring system to evaluate the severity of perianal CD. From the 5-item instrument, pain will be used. Each category is graded on a 5-point Likert scale ranging from no symptoms (score of 0) to severe symptoms (score of 4); a higher score indicates more severe disease.
Percentage of Participants who Achieve Clinical Remission at Weeks 104 and 156 (After IMP Administration in ADMIRE-CD II Study) Baseline of ADMIRE-CD II and Weeks 104 and 156 Clinical remission is defined as closure of all treated external fistula openings that were draining at baseline of ADMIRE-CD II despite gentle finger compression.
Percentage of Participants With Relapse Up to Week 156 Relapse is defined as participants who were in combined remission at Week 52 of ADMIRE-CD II and who have either reopening of any of the treated fistula(s) external openings with active drainage as clinically assessed or, the development of a perianal fluid collection \>2 cm of the treated perianal fistulas confirmed by centrally read magnetic resonance imaging (MRI) assessment.
Percentage of Participants who Achieve Clinical Response at Weeks 104 and 156 (After IMP Administration in ADMIRE-CD II Study) Baseline of ADMIRE-CD II and Weeks 104 and 156 Clinical response is defined as closure of at least 50% of all treated external fistula openings that were draining at baseline of ADMIRE-CD II despite gentle finger compression.
Percentage of Participants who Achieve Combined Clinical Remission at Week 156 (After IMP Administration in ADMIRE-CD II Study) Baseline of ADMIRE-CD II and Week 156 Combined remission of complex perianal fistula(s) is defined as the clinical assessment of closure of all treated external openings that were draining at baseline of ADMIRE-CD II, 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.
Percentage of Participants With New Anal Abscess in Treated Fistula at Week 156 Week 156
Trial Locations
- Locations (58)
Hospital Clinico San Carlos
🇪🇸Madrid, Spain
Virginia Mason Medical Center - Gastroenterology
🇺🇸Seattle, Washington, United States
University of Miami Hospital
🇺🇸Miami, Florida, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
The Chaim Sheba Medical Center
🇮🇱Tel Hashomer, Israel
Indiana University - Colon and Rectal
🇺🇸Indianapolis, Indiana, United States
Morristown Medical Center - Gastroenterology
🇺🇸Morristown, New Jersey, United States
Lenox Hill Hospital
🇺🇸New York, New York, United States
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
FN Hradec Kralove
🇨🇿Hradec Kralove, Czechia
Northwell Health
🇺🇸Manhasset, New York, United States
University of Kansas Medical Center (KUMC) - University of Kansas Liver Center - Hepatology Clinic
🇺🇸Kansas City, Kansas, United States
Dartmouth Hitchcock Medical Center - Cancer Center
🇺🇸Lebanon, New Hampshire, United States
Massachussetts General Hospital - Gastroenterology
🇺🇸Boston, Massachusetts, United States
University of Virginia
🇺🇸Charlottesville, Virginia, United States
NH Hospital a.s.
🇨🇿Horovice, Czechia
Penn State Hershey Medical Center - Surgery
🇺🇸Hershey, Pennsylvania, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
GZA Sint-Vincentius
🇧🇪Antwerpen, Belgium
Hopital Saint Louis
🇫🇷Paris, France
Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus Gyermekgyogyaszati Klinika
ðŸ‡ðŸ‡ºDebrecen, Hungary
CHRU Hopital de Pontchaillou - Maladies De L'Appareil Digesti
🇫🇷Rennes, France
Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar
ðŸ‡ðŸ‡ºSzeged, Csongrad, Hungary
Rabin Medical Center, Beilinson Hospital -Gastroenterology
🇮🇱Petah Tikva, HaMerkaz, Israel
Hospital Universitario Son Espases
🇪🇸Palma de Mallorca, Baleares, Spain
C.H.U. de Pontevedra
🇪🇸Pontevedra, Spain
Wielospecjalistyczny Szpital Medicover
🇵🇱Warszawa, Poland
Complesso Integrato Columbus, Universita Cattolica del Sacro Cuore
🇮🇹Roma, Italy
Hospital Universitario de Fuenlabrada
🇪🇸Fuenlabrada, Madrid, Spain
Hospital Del Mar
🇪🇸Barcelona, Spain
Centrum Medyczne Melita Medical
🇵🇱Wroclaw, Dolnoslaskie, Poland
Fundacion Jimenez Diaz
🇪🇸Madrid, Spain
Parc Tauli Hospital Universitari
🇪🇸Sabadell, Barcelona, Spain
Hospital Clinic De Barcelona
🇪🇸Barcelona, Spain
H.U.V. del Rocio
🇪🇸Sevilla, Spain
Mayo Clinic College of Medicine - Division of Colon and Rectal Surgery - Division of Colon and Rectal Surgery
🇺🇸Rochester, Minnesota, United States
Cedar-Sinai Medical Center
🇺🇸West Hollywood, California, United States
Brown Surgical Associates,Inc.
🇺🇸Providence, Rhode Island, United States
Centre Hospitalier Lyon Sud
🇫🇷Pierre Benite Cedex, France
Semmelweis Egyetem Altalanos Orvostudomanyi Kar
ðŸ‡ðŸ‡ºBudapest, Hungary
AdventHealth Tampa
🇺🇸Tampa, Florida, United States
PU A. Gemelli, Universita Cattolica del Sacro Cuore
🇮🇹Roma, Italy
Yale University School of Medicine
🇺🇸New Haven, Connecticut, United States
USF Health South Tampa Center for Advanced Healthcare
🇺🇸Tampa, Florida, United States
Paris St. Joseph Hospital
🇫🇷Paris, Ile-de-France, France
Cleveland Clinic Florida
🇺🇸Fort Lauderdale, Florida, United States
UZ Leuven
🇧🇪Leuven, Vlaams Brabant, Belgium
Johns Hopkins School of Medicine
🇺🇸Baltimore, Maryland, United States
Universitair Ziekenhuis Gent
🇧🇪Gent, Oost-Vlaanderen, Belgium
CHRU de Brabois Hopitaux de Brabois
🇫🇷Vandoeuvre Les Nancy Cedex, Nancy, France
CHRU de Lille - Hopital Claude Huriez - Gastroenterologie
🇫🇷Lille, Nord, France
MH Egeszsegugyi Kozpont
ðŸ‡ðŸ‡ºBudapest, Pest, Hungary
Rambam Medical Centre
🇮🇱Haifa, Israel
CHU de Clermont-Ferrand - Estaing
🇫🇷Clermont-Ferrand cedex 1, France
Hadassah Medical Organization, Hadassah Medical Center, Ein-
🇮🇱Jerusalem, Yerushalayim, Israel
AOU Policlinico di Modena - Gastroenterologia
🇮🇹Modena, Italy
H.U. G.Trias i Pujol
🇪🇸Badalona, Barcelona, Spain