Adipose-derived mesenchymal stem cells as local treatment of ulcerative colitis

2024-518643-38-00 Phase I and Phase II (Integrated) - Other Ended

Start 30 Jun 2023 · End 22 Oct 2025 · Status Ended · 1 EU/EEA countries · 1 sites

Overview

Sponsor-declared trial summary

Phase Phase I and Phase II (Integrated) - Other
Status Ended
Participants planned 16
Countries 1
Sites 1

ulcerative colitis

To investigate treatment with injection of allogeneic adipose-derived mesenchymal stem cells locally in the mucosa of patients with ulcerative colitis (UC) reduces the degree of mucosal inflammation and intestinal symptoms.

Key facts

Sponsor
Region Midtjylland
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Digestive System Diseases [C06]
Trial duration
30 Jun 2023 → 22 Oct 2025
Decision date (initial)
2024-11-11
Transition trial
Yes
Low-intervention
No
Rare-disease indication
No
Vulnerable population
No
Funding sources
Innovation Fond Denmark

External identifiers

EU CT number
2024-518643-38-00
EudraCT number
2022-002325-99

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To investigate treatment with injection of allogeneic adipose-derived mesenchymal stem cells locally in the mucosa of patients with ulcerative colitis (UC) reduces the degree of mucosal inflammation and intestinal symptoms.

Conditions and MedDRA coding

ulcerative colitis

VersionLevelCodeTermSystem organ class
20.1 LLT 10045365 Ulcerative colitis 10017947

Study design 1 period

#TitleAllocationBlindingRoles blindedArms
1 Adipose-derived mesenchymal stem cells as local treatment of ulcerative colitis
All patients serve as their own control, as they are both treated with injections of mesenchymal stem cells, as well as placebo injections with salt water. The method is as follows: For the first two patients, a dose of 2.5 x 10^6 cells is used, and if this dose is tolerated, 5 x 10^6 cells are used for the next two patients. Provided use of these doses is uncomplicated, increase the dose to 10 x 10^6 cells in the next six patients, and if this dose is also tolerated, a dose of 20 x 10^6 cells is used for the last six patients. Evaluation of dose tolerance is carried out 14 days after administration. In the absence of significant side effects in all patients at a given dose level, the inclusion of patients at next dose step. Dose selection is based on the doses used in several fistula studies (7-13), as well as on preliminary results mentioned above. The respective doses are distributed over 5 injection sites, each 0.5 mL, either at the bottom or at the top of the rectal mucosa. As a placebo control, above or below is given injections of 0.5 ml NaCl without containing stem cells. If stem cell injections or placebo control placed at the top or bottom is determined by drawing lots.
 2 Double [{"id":90131,"code":4,"name":"Analyst"},{"id":90130,"code":1,"name":"Subject"},{"id":90134,"code":3,"name":"Monitor"},{"id":90133,"code":2,"name":"Investigator"},{"id":90132,"code":5,"name":"Carer"}]

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

  1. Age 18-70 years
  2. Known ulcerative colitis with spread limited to the left side of the colon
  3. Mild-moderate activity in UC assessed by complete Mayoscore > 2 and < 10
  4. Speaks and understands Danish
  5. Fully informed consent

Exclusion criteria 8

  1. Severe disease activity assessed on the Mayoscore (score > 10)
  2. Acute severe colitis according to the Truelove-Witt criteria
  3. Disease spread proximal to the left flexure (extensive colitis)
  4. Active malignant disease
  5. Pregnancy (checked with pregnancy test (urine HCG), if fertile woman)
  6. Smoker (defined as smoking cessation < 12 weeks)
  7. People allergic to penicillin (the final product contains traces of penicillin)
  8. Fertile women who do not use very safe contraception (defined as birth control pills, IUDs or sexual abstinence) or are sterilized

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Reduced inflammatory activity assessed with endoscopic Mayoscore 14 days after treatment with injection of allogeneic adipose-derived mesenchymal stem cells.

Secondary endpoints 6

  1. Reduced inflammatory activity assessed with endoscopic Mayoscore 4, 7, 14 and 30 days after treatment.
  2. Improvement in histological grading of inflammation in tissue samples day 4, 7, 14 and 30 after treatment.
  3. Improvement in symptoms 4, 7, 14 and 30 days after treatment assessed by complete Mayoscore
  4. Complications/adverse events during the treatment in relation to dose seeking
  5. Demonstrate the formation or absence of HLA antibodies, corresponding to the donor's HLA type, as a result of the treatment.
  6. Demonstrate differences between the ability of mesenchymal stem cells from different donors to immunomodulation using potency assays.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

MSC, adipose tissue

PRD11626814 · Product

Active substance
Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells Expanded
Substance synonyms
Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue
Other product name
Allogeneic Adipose-Derived Mesenchymal Stem Cells (AD-MSC) in suspension (20 million cells/ 2,5 mL saline)
Pharmaceutical form
SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE
Route of administration
INTRALESIONAL USE
Authorisation status
Not Authorised
MA holder
REGION MIDTJYLLAND
Paediatric formulation
No
Orphan designation
No

MSC, adipose tissue

PRD11626708 · Product

Active substance
Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells Expanded
Substance synonyms
Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue
Other product name
Allogeneic Adipose-Derived Mesenchymal Stem Cells (AD-MSC) in suspension (5 million cells/ 2,5 mL saline)
Pharmaceutical form
SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE
Route of administration
INTRALESIONAL USE
Authorisation status
Not Authorised
MA holder
REGION MIDTJYLLAND
Paediatric formulation
No
Orphan designation
No

MSC, adipose tissue

PRD11626103 · Product

Active substance
Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells Expanded
Substance synonyms
Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue
Other product name
Allogeneic Adipose-Derived Mesenchymal Stem Cells (AD-MSC) in suspension (2,5 million cells/ 2,5 mL saline)
Pharmaceutical form
SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE
Route of administration
INTRALESIONAL USE
Authorisation status
Not Authorised
MA holder
REGION MIDTJYLLAND
Paediatric formulation
No
Orphan designation
No

MSC, adipose tissue

PRD11626749 · Product

Active substance
Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells Expanded
Substance synonyms
Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue
Other product name
Allogeneic Adipose-Derived Mesenchymal Stem Cells (AD-MSC) in suspension (10 million cells/ 2,5 mL saline)
Pharmaceutical form
SUSPENSION FOR INJECTION IN PRE-FILLED SYRINGE
Route of administration
INTRALESIONAL USE
Authorisation status
Not Authorised
MA holder
REGION MIDTJYLLAND
Paediatric formulation
No
Orphan designation
No

Placebo 2

Natriumklorid ”B. Braun”

PRD563912 · Product

Active substance
Sodium Chloride
Pharmaceutical form
SOLUTION FOR INJECTION/INFUSION
Route of administration
INTRALESIONAL USE
Authorisation status
Authorised
ATC code
B05BB01 — ELECTROLYTES
Marketing authorisation
13341
MA holder
B.BRAUN MELSUNGEN AG
MA country
Denmark
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Natriumklorid Fresenius Kabi 9 mg/ml

PRD2128220 · Product

Active substance
Sodium Chloride
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRALESIONAL USE
Authorisation status
Authorised
ATC code
B05BB01 — ELECTROLYTES
Marketing authorisation
14043
MA holder
FRESENIUS KABI AB
MA country
Denmark
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Region Midtjylland

59 Total trials 36 Recruiting 13 Ended
Academic / Non-commercial
Sponsor organisation
Region Midtjylland
Address
Palle Juul-Jensens Boulevard 99
City
Aarhus N
Postcode
8200
Country
Denmark

Scientific contact point

Organisation
Region Midtjylland
Contact name
Klaus Krogh

Public contact point

Organisation
Region Midtjylland
Contact name
Klaus Krogh

Third parties 1

OrganisationCity, countryDuties
Aarhus Universitet
ORG-100028380
 Aarhus N, Denmark On site monitoring, Code 9

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Ended 16 1
Rest of world — 0 —

Investigational sites

Denmark

1 site · Ended
Region Midtjylland
Department of Hepatology and Gastroenterology, Palle Juul-Jensens Boulevard 99, 8200, Aarhus N

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Denmark 2023-06-30 2025-10-22 2023-11-27 2025-08-29

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protokol_2024-518643-38-00_redacted 3
Recruitment arrangements (for publication) K1_Recruitment arrangements 1
Subject information and informed consent form (for publication) L1_Informed_Consent_Form 1
Subject information and informed consent form (for publication) L1_Subject_Information_Sheet_redacted 3
Synopsis of the protocol (for publication) D1_Protokol_synopsis_DK_2024-518643-38-00 1

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-10-22 Denmark Acceptable
2024-11-08
 2024-11-11

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