IMM01-STEM to treat muscle loss from knee osteoarthritis

An Open-label Dose Escalation Study to Assess the Safety and Tolerability of IMM01-STEM in Participants With Muscle Atrophy Related to Knee Osteoarthritis

This will test whether twice-weekly IMM01-STEM injections into the thigh are safe and tolerable for people with muscle loss related to knee osteoarthritis.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 28 (estimated)
• Ages: 50 Years to 75 Years
• Sex: All
• Sponsor: Immunis, Inc. (Industry-sponsored)
• Locations: 2 sites (Irvine, California and 1 other locations)
• Trial ID: NCT05211986 on ClinicalTrials.gov

What this trial studies

This is an open-label, dose-escalation Phase 1/2 study testing safety and tolerability of IMM01-STEM, a secretome product derived from partially differentiated pluripotent stem cells, in adults with quadriceps atrophy related to knee osteoarthritis. Up to 18 participants will be enrolled across up to four ascending dose cohorts (225, 450, 900, and 2000 μg) and will receive intramuscular injections twice weekly for 4 weeks (8 injections total). Participants are observed for 3 hours after each injection for early adverse reactions, with screening and baseline assessments performed before dosing. The primary focus is on safety and tolerability, with dose escalation guided by observed adverse events.

Who should consider this trial

Good fit: Adults with knee osteoarthritis (Kellgren-Lawrence grade 2–3) who have measurable quadriceps weakness, a BMI under 40, can ambulate more than 50 feet unassisted, and meet the study's screening criteria are ideal candidates.

Not a fit: People without measurable quadriceps weakness, with very advanced knee osteoarthritis (beyond KL grade 3), or who cannot receive intramuscular injections are unlikely to benefit from this study.

Why it matters

Potential benefit: If successful, IMM01-STEM could help preserve or restore quadriceps muscle around the knee, improving strength and function for people with knee osteoarthritis.

How similar studies have performed: This approach is novel: secretome and cell-derived regenerative therapies have shown promise in preclinical models but have limited clinical success so far.

Eligibility criteria

Inclusion Criteria:

* Has moderate KOA (defined as Kellgren-Lawrence \[KL\] grade 2 to 3) on affected limb
* Has quadriceps weakness (\<7.5N/kg)
* Can ambulate \>50 feet unassisted
* This criteria deleted with protocol amendment 6
* Has a body mass index (BMI) of \<40kg/m2
* A male must agree to use contraception during the treatment period and for at least 3 months after the last dose of study treatment and refrain from donating sperm during this period.
* A female is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:

  1. Not a woman of childbearing potential (WOCBP). OR
  2. A WOCBP who agrees to follow the protocol's contraceptive guidance during the treatment period and for at least 3 months after the last dose of study treatment.
* Female has a negative pregnancy test result at screening and prior to investigational medicinal product (IMP) administration
* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
* Willing and able to comply with all study requirements, according to the judgment of the Investigator
* Has discontinued systemic oral or intravenous steroid use for 6 months prior to Screening
* Has vital sign measurements within the following ranges at Baseline (predose at Visit 2): heart rate \>50 and \<100 bpm, systolic blood pressure \>100 and \<170 mmHg, diastolic blood pressure \>50 and \<90 mmHg, and blood oxygenation (by pulse-oximetry) \>95%
* Participant has undergone and failed at least 1 3-month or longer treatment regimen (ie, activity modification, weight loss, physical therapy, anti-inflammatory medications, or injection therapy) within a 2-year period prior to the Screening visit.
* Study participant is able to speak, read, and understand English, in order to understand the nature of this study.

Exclusion Criteria:

* Severe KOA (defined as KL grade \>3) on contralateral limb
* Has had prior total knee arthroplasty
* Has a known hypersensitivity to any components of the study medication or comparative drugs (and/or an investigational device) as stated in this protocol
* Has current or past history of malignancy (5y) excluding nonmelanoma skin cancer
* Has neurological, vascular, or cardiac condition that limit function, or, in the opinion of the investigator, could jeopardize or would compromise the study participant's ability to participate in this study
* Has uncontrolled comorbidities including diabetes (hemoglobin A1c level \>7.0%), Hypertension (resting heart rate \>100 bpm, systolic blood pressure \>170 mmHg, or diastolic blood pressure \>90 mmHg), cardiovascular disease, asthma, or COPD.
* Is known positive for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCVAb) or human immunodeficiency virus-1/2 antibody (HIV-1/2Ab). Patients with a negative RNA test for HCV are acceptable.
* Is taking a prohibited medication or has taken a prohibited medication (narcotic pain medication, local anti-inflammatory, other investigational drugs)
* Participant has had a change in medication to manage comorbid condition(s) (including diabestes, hypertension, asthma, and cardiovascular disease) within 1 month of the Screening visit.
* Participant received intra-articular cortisone or viscosupplementation product(eg, Synvisc®) injections within 3 months prior to the first dose of IMP.
* Has had administration of a live, attenuated vaccine within 28 days of starting study treatment or anticipation that such vaccine will be required during the study Prior/concurrent clinical study experience
* Has current or chronic history of liver disease or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones)
* This criteria was deleted with protocol amendment 6
* This criteria was deleted with protocol amendment 6
* This criteria was deleted with protocol amendment 6
* Has current or past history (10y) of smoking
* Has a history of chronic alcohol or drug abuse within the previous 3 months
* Participant is currently using a systemic oral or intravenous steroid regimen (eg, for asthma or other chronic respiratory condition) or brief course of systemic pulse steroid administration (eg, for flare up of nonarthritic condition or COVID-19).

Where this trial is running

Irvine, California and 1 other locations

• Orthopaedic Specialty Institute — Irvine, California, United States (Recruiting)
• University of California, Irvine - Alpha Stem Cell Clinic — Orange, California, United States (Active_not_recruiting)

Study contacts

• Study coordinator: Joelle Hafen, BS

Conditions Muscle Atrophy