Autologous BMA vs Saline and LAM + LP-PRP vs Saline Evaluations in Knee OA

NCT ID: NCT05517434

Last Updated: 2025-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

148 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-01-01

Study Completion Date

2026-12-31

Brief Summary

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ABLE OA is a Health Canada authorized (phase II/III) trial \[Parent Control #: 263591\]. A multi-center, prospective, double-blinded, randomized, placebo-controlled adaptive trial to evaluate the efficacy of two minimally manipulated autologous cellular preparations i) bone marrow aspirate (BMA) injection; and, ii) combined lipoaspirate micronized (LAM) and leukocyte poor (LP) platelet-rich plasma (PRP) injections for the treatment of knee osteoarthritis (OA).

BMA, LAM from lipoaspirate (LA), and LP-PRP from whole blood will be prepared using the Cervos Marrow Cellution™ Bone Marrow Aspiration System, Cervos LIPO-PRO™ Adipose Transfer System, and Cervos KEYPRP Platelet Separator System, respectively.

Patient-reported outcome (PRO) measures will be collected using web- or paper-based questionnaires administered at baseline (pre-injection) as well as at 3, 6 and 12 months (post-injection). Blood, synovial fluid, and urine samples will be collected at baseline pre-injection and 6 months post-injection only.

Detailed Description

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Trial interventions will occur in two independent studies under a single protocol where each experimental treatment will be compared to a placebo control.

Our primary hypothesis is that BMA or LAM + LP-PRP injection is 35% more effective than placebo saline injection control in terms of response rates in Numeric Pain Rating Scale (NPRS) scores as measured by their difference.

PRIMARY OBJECTIVE:

To determine the efficacy of an intra-articular injection of BMA or LAM + LP-PRP in patients with knee OA by comparing each of the two treatments to a placebo saline injection control arm. Efficacy will be measured by a pain intensity improvement of a minimum of 2 points in NPRS scores at 6 months after injection relative to baseline. The study endpoint is 6 months post-injection.

KEY SECONDARY OBJECTIVE:

To determine efficacy measured by improvements in the Knee Injury and Osteoarthritis Outcome Score (KOOS) function activities of daily living (ADL) subscale scores at 6 months after injection relative to baseline by comparing each of the treatment (BMA or LAM + LP-PRP) arm to a placebo saline control arm.

OTHER SECONDARY OBJECTIVES:

* To evaluate if an injection of BMA or LAM + LP-PRP into the knee joint shows greater improvements in other pain outcomes (including KOOS pain) compared to placebo injection at 3, 6 and 12 months post-injection relative to baseline.
* To evaluate the safety of BMA or LAM + LP-PRP injection into the knee joint compared to placebo injection. Adverse events will be collected at baseline, 3, 6 and 12 months post-injection, and deleterious effects on the joint will be assessed by X-ray at 6 months post-injection only.
* To evaluate the health profile and overall self-rated health status of patients in treatment and placebo arms at 3, 6 and 12 months post-injection relative to baseline.
* To evaluate overall patient satisfaction in treatment and placebo arms at 6 months post-injection.
* To evaluate health care consumption and health-related productivity losses of paid and unpaid work using the iMTA Medical Consumption Questionnaire (iMCQ) and iMTA Productivity Cost Questionnaire (iPCQ), respectively at 6 and 12 months post-injection relative to baseline.
* To determine the quality-adjusted-life years (QALYs) using the 5-level EuroQol five-dimensional (EQ-5D-5L) health state utility scores based on the Canadian preference weights to inform cost-effective estimates.

EXPLORATORY OBJECTIVES:

To determine the correlation between changes in NPRS/KOOS pain scores and KOOS ADL function at 6 months relative to baseline and the heterogeneity in:

1. the cellular composition and soluble factors in BMA, LAM and LP-PRP autologous cellular preparations \[in BMA or LAM + LP-PRP groups only\]
2. the levels of local and systemic immune cell and inflammatory profiles of patients (based on synovial fluid, blood, and urine readouts) \[in BMA or LAM + LP-PRP and placebo groups\]

A total of approximately 84 eligible participants in each study will be randomized in a 1:1 ratio, which allows for 42 participants per group (treatment vs. placebo). This sample size considers a potential drop-out rate of 10% for each study. Three recruitment centres (Toronto Western Hospital (TWH), University Health Network (UHN); Women's College Hospital (WCH); Cleveland Clinic Canada (CCC)) and one treatment centre (TWH, UHN) will be involved in these two studies. Stratification will occur by centre, baseline NPRS of 4-6 (moderate pain) or 7-10 (severe pain), and KL grade of 2 (minimal OA) or 3 (moderate OA). Additionally, the need to re-estimate the required sample size will be evaluated using the information available at interim. At the interim analysis, contingent on observed response rates and corresponding statistical signal, the required sample size may increase, ranging from 100 to 288 patients in total for each of the two studies.

Conditions

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Osteoarthritis, Knee

Keywords

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placebo intra-articular injection autologous therapy bone marrow aspirate lipoaspirate platelet-rich plasma leukocyte-poor pain

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

STUDY 1 (N=74): All patients will undergo a bone marrow aspiration. A BMA injection \[Arm A, n=37\] will be compared to a saline injection \[Arm C, n=37\].

STUDY 2 (N=74): All patients will undergo a blood collection and lipoaspiration. A LAM injection followed by LP-PRP injection \[Arm B, n=37\] will be compared to two consecutive saline injections \[Arm D, n=37\].
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
A research nurse/staff will prepare the minimally manipulated cellular preparations and injection syringe(s), as well as deliver the corresponding syringe(s) based on group allocation to the clinician to administer the injection into the patient's knee joint. The syringe(s) containing the active treatment or saline solution will be obscured by a non-transparent, adhesive label hiding its content in order to maintain the blind for both the patient and clinician.

Study Groups

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For STUDY 1 (ARM A): Bone Marrow Aspirate (BMA)

This group will undergo a bone marrow aspiration and receive an ultrasound guided intra-articular injection of BMA (a single dose of cellular suspension of 9 mL or less)

Group Type EXPERIMENTAL

Bone Marrow Aspirate (BMA): Minimally manipulated autologous cellular preparation

Intervention Type BIOLOGICAL

Participants will undergo a bone marrow aspiration. About 10 mL of BMA will be collected from the posterior iliac spine i.e., ipsilateral and/or contralateral iliac crest using the Cervos Marrow Cellution™ kit. The BMA does not require processing using a centrifuge after collection. 9 mL (or less) of BMA is injected into the osteoarthritic knee joint after collection (Arm A, Study 1).

For STUDY 1 (ARM C): Saline Injection

This group will undergo a bone marrow aspiration and receive an ultrasound guided intra-articular injection of saline solution (9 mL)

Group Type PLACEBO_COMPARATOR

Saline (Placebo Comparator for BMA)

Intervention Type OTHER

Participants will undergo a bone marrow aspiration to collect about 10 mL of BMA from the posterior iliac spine i.e., ipsilateral and/or contralateral iliac crest. However, 0.9% sodium chloride (NaCl) Baxter or equivalent (9 mL) is injected into the osteoarthritic knee joint (Arm C, Study 1).

For STUDY 2 (ARM B): Lipoaspirate Micronized + Leukocyte-Poor Platelet-Rich Plasma (LAM + LP-PRP)

This group will undergo a blood collection plus lipoaspiration and receive an ultrasound guided intra-articular injection of LAM (a single dose of cellular suspension of 9 mL or less) followed by LP-PRP (a single dose of cellular suspension of 2 mL or less)

Group Type EXPERIMENTAL

Lipoaspirate Micronized + Leukocyte-Poor Platelet-Rich Plasma (LAM + LP-PRP): Minimally manipulated autologous cellular preparations

Intervention Type BIOLOGICAL

Participants will undergo a lipoaspiration. 40 mL of lipoaspirate (LA) will be collected from subcutaneous adipose tissue. LA will be processed using the Cervos LIPO-PRO™ kit and a centrifuge. Participants will also undergo a blood draw. About 30 mL of whole blood will be collected from the antecubital fossa. Whole blood will be processed using the Cervos KEYPRP kit and a centrifuge. After processing, 9 mL (or less) of LAM is injected first followed immediately by 2 mL (or less) of LP-PRP into the osteoarthritic knee joint (Arm B, Study 2).

For STUDY 2 (ARM D): Saline Injection

This group will undergo a blood collection plus lipoaspiration and receive ultrasound guided intra-articular injections of saline solution (9 mL followed by 2 mL)

Group Type PLACEBO_COMPARATOR

Saline (Placebo Comparator for LAM + LP-PRP)

Intervention Type OTHER

Participants will undergo a lipoaspiration to collect 40 mL of LA and a blood draw to collect about 30 mL of whole blood. However, 0.9% of sodium chloride (NaCl) Baxter or equivalent is injected twice (9 mL + 2 mL) into the osteoarthritic knee joint (Arm D, Study 2).

Interventions

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Bone Marrow Aspirate (BMA): Minimally manipulated autologous cellular preparation

Participants will undergo a bone marrow aspiration. About 10 mL of BMA will be collected from the posterior iliac spine i.e., ipsilateral and/or contralateral iliac crest using the Cervos Marrow Cellution™ kit. The BMA does not require processing using a centrifuge after collection. 9 mL (or less) of BMA is injected into the osteoarthritic knee joint after collection (Arm A, Study 1).

Intervention Type BIOLOGICAL

Saline (Placebo Comparator for BMA)

Participants will undergo a bone marrow aspiration to collect about 10 mL of BMA from the posterior iliac spine i.e., ipsilateral and/or contralateral iliac crest. However, 0.9% sodium chloride (NaCl) Baxter or equivalent (9 mL) is injected into the osteoarthritic knee joint (Arm C, Study 1).

Intervention Type OTHER

Lipoaspirate Micronized + Leukocyte-Poor Platelet-Rich Plasma (LAM + LP-PRP): Minimally manipulated autologous cellular preparations

Participants will undergo a lipoaspiration. 40 mL of lipoaspirate (LA) will be collected from subcutaneous adipose tissue. LA will be processed using the Cervos LIPO-PRO™ kit and a centrifuge. Participants will also undergo a blood draw. About 30 mL of whole blood will be collected from the antecubital fossa. Whole blood will be processed using the Cervos KEYPRP kit and a centrifuge. After processing, 9 mL (or less) of LAM is injected first followed immediately by 2 mL (or less) of LP-PRP into the osteoarthritic knee joint (Arm B, Study 2).

Intervention Type BIOLOGICAL

Saline (Placebo Comparator for LAM + LP-PRP)

Participants will undergo a lipoaspiration to collect 40 mL of LA and a blood draw to collect about 30 mL of whole blood. However, 0.9% of sodium chloride (NaCl) Baxter or equivalent is injected twice (9 mL + 2 mL) into the osteoarthritic knee joint (Arm D, Study 2).

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Male or female at least 30 years of age at the time of screening
* Willingness and ability to comply with study procedures and visit schedules and able to follow oral and written instructions
* Signed consent for study participation
* Baseline NPRS ≥ 4 points
* Presence of chronic, symptomatic knee pain in at least one knee; if both knees are affected, the knee with greater severity will be selected for treatment
* KL grade 2 or 3 knee OA based on standing knee X-ray assessment
* Body mass index ≤ 30 kg/m2

Exclusion Criteria

* Approved anti-inflammatory therapy injections (corticosteroid, Synvisc, PRP, nSTRIDE-Autologous Protein Solution) within the previous 6 months in the knee
* Major axial deviation (varus \>10°, valgus \>10°)
* Any concomitant knee lesion causing pain or effusion (i.e., ligamentous or meniscal injury, osteochondral lesion)
* Presence of clinically observed active infection in the index knee
* Diagnosed with rheumatoid arthritis, Reiter's syndrome, psoriatic arthritis, gout, ankylosing spondylitis, or arthritis secondary to other inflammatory diseases; chondrocalcinosis, Paget's disease, or villonodular synovitis
* Diagnosed with leukemia or other hematologic cancers, known presence of metastatic malignant cells, or ongoing or planned chemotherapeutic treatment
* Presence of venous or lymphatic stasis in the index leg
* A history of local anesthetic allergy
* Medical conditions such as hemophilia or other blood clotting disorders
* Arthroscopic knee surgery within the previous 6 months
* Daily opioid use for the past 3 months, use of non-steroidal anti-inflammatory drugs within 1 week of the procedure, unable to hold anti-platelet medications
* Use of systemic corticosteroids for treatment of a chronic medical condition within the past 3 months
* Immunosuppression or acute infective processes

Study Treatment Exclusion:

* For Study 1: Inability to tolerate the bone marrow aspiration procedure resulting in insufficient collection of BMA (\<10 mL) after two successive aspiration attempts
* For Study 2: Inability to tolerate the lipoaspiration procedure resulting in insufficient collection of LA (\<40 mL) after two successive aspiration attempts
Minimum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Women's College Hospital

OTHER

Sponsor Role collaborator

University Health Network, Toronto

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sowmya Viswanathan, PhD

Role: PRINCIPAL_INVESTIGATOR

University Health Network, Toronto

Christian Veillette, MD, MSc, FRCSC

Role: STUDY_DIRECTOR

University Health Network, Toronto

Christopher Kim, HBSc, MSc, MD, FRCSC, PhD(c)

Role: PRINCIPAL_INVESTIGATOR

University Health Network, Toronto

Locations

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Women's College Hospital

Toronto, Ontario, Canada

Site Status NOT_YET_RECRUITING

Toronto Western Hospital, University Health Network

Toronto, Ontario, Canada

Site Status RECRUITING

Countries

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Canada

Central Contacts

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Shoba Singh

Role: CONTACT

Phone: 416-634-7240

Email: [email protected]

Facility Contacts

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Ananya Pathak

Role: primary

Shoba Singh

Role: primary

Other Identifiers

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22-5959

Identifier Type: -

Identifier Source: org_study_id

22-5792 (ABLE OA, study 2)

Identifier Type: OTHER

Identifier Source: secondary_id