Phase I/IIa study to test the safety and efficacy of MSC from umbilical cord tissue (UC-MSC) for the treatment of cartilage damage in the knee joint - BP CC 001

Overview

Sponsor-declared trial summary

Key facts

Sponsor

BIONCaRT GmbH

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05], Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutics [E02]

Trial duration

23 Oct 2025 → ongoing

Decision date (initial)

2025-04-24

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Sächsische Aufbaubank - Förderbank

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

Evaluation of the safety of the investigational product BP CC 001 (10 x 106 UC-MSC in 1 ml cell culture suspension) applied to a collagen support structure (Chondro-Gide®)

Secondary objectives 1

1. Evaluation of the efficacy of the investigational medicinal product

Conditions and MedDRA coding

Cartilage defect of the knee joint

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Patients of any gender with an age of ≥ 18 years to ≤ 60 years
2. Symptomatic grade III/IV cartilage defect according to ICRS or osteochondral defect with a maximum depth of 3 mm
3. A cartilage defect requiring treatment with a defect size of 2 - 6 cm² on the femoral condyles or patella or trochlea
4. Affected and contralateral knee (if known): Kellgren-Lawrence (K/L) osteoarthritis severity < grade III
5. KOOS pain sub-score of ≤ 70 (out of max. 100) in the affected knee joint and > 80 (out of max. 100) in the contralateral knee joint measured without taking analgesics within the last 24 hours
6. BMI < 35 kg/m²
7. Written informed consent

Exclusion criteria 20

1. Known varus or valgus malalignment of the affected leg of ≥ 5°
2. Antero-posterior or medio-lateral instability
3. Meniscus loss of more than 20% in the affected compartment
4. Patella instability
5. Simultaneous surgery on other joint structures (cruciate ligament replacement, meniscus suture, partial meniscus resection > 20%, osteotomy)
6. Simultaneous higher-grade (ICRS grade III/IV) cartilage damage on the corresponding joint surfaces to the treated cartilage damage
7. Intra-articular application of hyaluronic acid or glucocorticoids or thrombocyte concentrates within the last 6 months
8. Joint replacement in the contra-lateral knee or hip within the last 12 months
9. Rheumatoid arthritis or other inflammatory diseases such as autoimmune diseases, seronegative spondyloarthritis, gout, pseudogout
10. Previous fracture in the affected knee joint
11. Osteoporosis
12. Relevant concomitant diseases that increase the risk of surgery, e.g. heart failure, coronary heart disease, coagulation disorders, diabetes mellitus, liver cirrhosis, renal insufficiency, etc.
13. History of hypersensitivity to porcine collagens, human albumin, glucose or components of fibrin adhesives used in orthopaedics or surgery
14. Simultaneous participation in another interventional clinical trial (incl. within the last 4 weeks before inclusion)
15. Addictions or other illnesses that do not allow the person con-cerned to assess the nature and extent of the clinical trial and its possible consequences
16. Any state that excludes adherence with the trial protocol (e.g. lack of compliance)
17. Persons who are placed in an institution by court or official order
18. Persons dependent on the sponsor
19. Pregnant or breastfeeding women
20. Women of childbearing potential, except women who meet one of the following criteria: a) post-menopausal (12 months natural amenorrhoea) b) postoperative (6 weeks after bilateral ovarectomy with or with-out hysterectomy, bilateral salpingectomy) c) negative pregnancy test (urine) and regular and correct use of a contraceptive method with an Pearl Index < 1% per year - Combined (oestrogen and progestogen-containing) hormonal contraception - Hormonal contraception containing progestogens (oral, injected, implanted) - IUD (hormonal IUD, copper IUD) d) sexual abstinance e) vasectomy of the partner

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of patients with a serious adverse event within 24 months after surgery

Secondary endpoints 7

1. Regeneration of damaged cartilage measured by MOCART score (relative change in the value 12 months after surgery compared to the initial value before surgery)
2. Relative change in knee joint pain 12 and 24 months after the surgery compared to the baseline value before the surgery measured using the KOOS Pain Subscore
3. Relative change in knee-related quality of life 12 and 24 months after surgery compared to baseline before surgery measured by KOOS-QoL subscore
4. Relative change in symptoms and stiffness 12 and 24 months after surgery compared to pre-surgery baseline as measured by KOOS symptom subscore
5. Relative change in activities of daily life 12 and 24 months after surgery compared to baseline before surgery measured by KOOS-ADL subscore
6. Relative change in physical resilience during sporting activities 12 and 24 months after the surgery compared to the baseline value before the surgery measured by KOOS-Excercise /Rec-Subscore
7. AE, AR, SAE, SAR, SUSAR: Description of all adverse events (AE, AR, SAE, SAR, SUSAR) within 24 months after surgery in terms of severity (assessed according to National Cancer Institute - Common Terminology Criteria for Adverse Events Version 5.0 (NCI-CTCAE V 5.0)), causality, outcome

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

BIONCaRT GmbH

Sponsor organisation

BIONCaRT GmbH

Address

Fuerstenweg 8, Seidewitz Seidewitz

City

Grimma

Postcode

04668

Country

Germany

Scientific contact point

Organisation

BIONCaRT GmbH

Contact name

Dr. André Gerth

Public contact point

Organisation

BIONCaRT GmbH

Contact name

Dr. André Gerth

Locations

1 EU/EEA country · 7 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Documents 9 files

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

Application history

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