Efficacy of intradiscal injection of autologous BM-MSC in subjects with chronic LBP due to multilevel lumbar IDD

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutics [E02]

Trial duration

13 Feb 2025 → 31 Oct 2025

Decision date (initial)

2025-02-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-519112-14-00

EudraCT number

2019-002749-40

ClinicalTrials.gov

NCT05066334

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

Evaluate the effectiveness of intradiscal injection of autologous BM-MSCs in reducing chronic LBP due to multilevel (max. 3 levels) lumbar IDD after 12 months of treatment in terms of pain relief (VAS), functionality (ODI) and quality of life (SF36)

Secondary objectives 4

1. Evaluate regenerative changes of the treated IVD
2. Assess modification of employment and work status between baseline and months 12.
3. Assess safety and tolerability
4. Evaluate consumption of medications to relieve pain such as type and dose of analgesics

Conditions and MedDRA coding

low back pain

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Age between 18 and 65 years
2. Signed informed consent
3. Symptomatic chronic LBP due to moderate IDD [modified Pfirrmann score 3-4 (Pfirrmann et al., 2001), Griffith score 3-7(Griffith et al., 2007)] at max.3 levels of the lumbar spine unresponsive to conservative treatment, physical and medical for at least 6 months. Physical treatment includes physiotherapy. Medical treatments includes NSAID, paracetamol, opioids and myorelaxant.
4. Annulus fibrosus intact, demonstrated by MRI
5. Pain baseline > 40 mm on VAS (0- 100)
6. NSAID washout of at least 2 days before screening
7. Painkillers washout of at least 24 hours before screening
8. For females of childbearing potential (see definitions in paragraph 6.5), a negative pregnancy test must be documented at Screening
9. Men and women should use effective contraception during treatment and for at least 24 months after BM-MSC discontinuation. The complete list of contraceptive methods is described in the patient information sheet and in the paragraph 6.5. As a precautionary measure, breast-feeding should be discontinued during treatment with BM-MSC and should not be restarted after discontinuation of BM-MSC.

Exclusion criteria 24

1. Congenital or acquired diseases leading to spine deformations that may upset cell application (scoliosis, isthmus lesion, sacralization and hemisacralization, degenetative spondilolisthesis
2. Spinal segmental instability assessed by dynamic X-Ray
3. Symptomatic facet joints syndrome on MRI (facet joints iperintensity and ipertrophy evaluated at coronal T2 weighted MRI).
4. Prior to the screening visit, has received: - Oral corticosteroid therapy within the previous 1 month, OR
5. Intramuscular, intravenous or epidural corticosteroid therapy within the previous 1 month.Presence of a 4th level with symptomatic IDD (modified Pfirrmann score 3- 4, Griffith score 3-7) in the lumbar spine
6. Presence of a 4th level with symptomatic IDD (modified Pfirrmann score 3-4, Griffith score 3-7) in the lumbar spine.
7. Spinal canal stenosis (Schizas score > B).
8. History of spinal infection
9. Lumbar disc herniation and sciatica
10. Endplate abnormality such as Schmorl’s Nodes
11. Previous discal puncture or previous spine surgery.
12. IDD with Modic III changes on MRI images
13. Patients not eligible to the intravertebral disc surgery
14. Patients who have the risk to undergo a surgery in the next 6 months
15. Patients with local infusion device/devices for corticosteroids
16. Obesity with body mass index (BMI in Kg/size in m2) greater than 35 (obesity grade II)
17. Participation in another clinical trial or treatment with another investigational product within 30 days prior to inclusion in the study
18. Abnormal blood tests: hepatic (alanine aminotransferase [ALT] and/or aspartate aminotransferase [AST] >1.5 × upper limit of normal [ULN]), renal, pancreatic or biliary disease, blood coagulation disorders, anemia or platelet count of <100 × 109/L.
19. Pregnant or lactating women, or premenopausal women not using an acceptable form of birth control, are ineligible for inclusion. Contraception will be maintained during treatment and until the end of relevant systemic exposure. Additional pregnancy testing will be performed at the end of relevant systemic exposure. The patients will be required to use contraception from initial treatment administration until 24 months after the last dose of study drug.
20. In each case of delayed menstrual period (over one month between menstruations) confirmation of absence of pregnancy is strongly recommended. The complete list of contraceptive methods is described in the patient information sheet and in the paragraph 6.5. Positive serology for following infection: Syphilis, HIV, Hepatitis B, or C.
21. Contraindication to MRI assessed by the investigator
22. Intolerance or allergy to local anaesthesia
23. Any history of Cancer or immunodeficiency disease
24. Previous transplantation.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. To evaluate the efficacy of intradiscal injection of autologous BM-MSC using the visual analog scale (VAS) and functional status assessed by the Oswestry disability index (ODI) after 12 months of treatment, defining responders in case of at least 30% improvement in VAS or ODI at month 12 compared to baseline.

Secondary endpoints 4

1. To evaluate changes of the treated IVD induced by regenerative therapy by quantitative Magnetic Resonance Imaging (MRI) signal measurements in T2 between baseline and 1, 3, 6 and 12 months
2. To evaluate modification of disability (ODI) and pain relief (VAS) considered as continuos measures, between baseline and 1, 3 and 6 months. • To evaluate modification of quality of life (SF-36 scores) and work ability index (WAI) considered as continuos measures, between baseline and 1, 3, 6 and 12 months.
3. Safety and tolerability will be evaluated by recording adverse events (AEs) and serious AEs (SAEs) throughout the study till 12 months. Number of participants with adverse events, the correlation of the AE to the IMP and their severity will be used as a measure of safety and tolerability
4. To evaluate consumption of medications to relieve pain such as type and dose of analgesics will be evaluated. Paracetamol (acetaminophen) and levels 2 analgesics will be assessed throughout the study at each visit

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon

Sponsor organisation

Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon

Address

Via Alvaro Del Portillo N 200

City

Rome

Postcode

00128

Country

Italy

Scientific contact point

Organisation

Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon

Contact name

Gianluca Vadalà

Public contact point

Organisation

Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon

Contact name

Gianluca Vadalà

Locations

1 EU/EEA country · 1 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 7 files

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

Application history

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