Safety and Tolerability of IMM01-STEM in Patients With Muscle Atrophy Related to Knee Osteoarthritis.

NCT05211986 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: STEM-MYO
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 2

Brief Summary

An open-label dose escalation study to assess the safety and tolerability of IMM01-STEM in participants with muscle atrophy related to knee osteoarthritis

Conditions

Muscle Atrophy

Outcome Measures

Primary Outcomes (1)

• Safety of IMMUNA(IMM01-STEM) treatment in study participants with muscle atrophy related to KOA

  Determined by the incidence and severity of dose-limiting toxicities (DLTs) and the incidence of treatment-emergent adverse events (TEAEs). Adverse events (AE) are classified based on Common Terminology Criteria for Adverse Events (CTCAE) as follows: Grade 0 = no adverse events, Grade 1= mild, Grade 2 = moderate, Grade 3 = severe, Grade 4 = life threatening and Grade 5 = fatal adverse events. A DLT is defined as any AE related to IMM01-STEM of a Grade 2 unresolved within 48 hours post-injection, or any Grade 3, 4, or 5 related to IMM01-STEM during any time of treatment or during the 48-hour, acute/subacute observation period.

  Day 0 to Day 28

Secondary Outcomes (2)

• Safety and tolerability of IMMUNA(IMM01-STEM) after 4 weeks of treatment in study participants with muscle atrophy related to KOA

  Day 28

• Functionality of the knee joint after 4 weeks of treatment with IMMUNA(IMM01-STEM) in study participants with muscle atrophy related to KOA

  Day 28

Study Arms (4)

Cohort A

ACTIVE COMPARATOR

Participants will receive twice weekly intramuscular (im) administration of IMM01-STEM for 4 weeks with a dose of 225μg.

Drug: IMM01-STEM

Cohort B

ACTIVE COMPARATOR

Participants will receive twice weekly intramuscular (im) administration of IMM01-STEM for 4 weeks with a dose of 450μg.

Drug: IMM01-STEM

Cohort C

ACTIVE COMPARATOR

Participants will receive twice weekly intramuscular (im) administration of IMM01-STEM for 4 weeks with a dose of 900μg.

Drug: IMM01-STEM

Cohort D

ACTIVE COMPARATOR

Participants will receive twice weekly intramuscular (im) administration of IMM01-STEM for 4 weeks with a dose of 2000μg.

Drug: IMM01-STEM

Interventions

IMM01-STEM DRUG

IMM01-STEM is a secretome product derived from partially differentiated pluripotent stem cells that contains regenerative molecules.

Cohort A; Cohort B; Cohort C; Cohort D

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Not a woman of childbearing potential (WOCBP). OR
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Immunis, Inc.: lead

Study Sites (2)

Orthopaedic Specialty Institute

Irvine, California, 92618, United States

RECRUITING

University of California, Irvine - Alpha Stem Cell Clinic

Orange, California, 92868, United States

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Muscular Atrophy

Condition Hierarchy (Ancestors)

Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Atrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Study Officials

• Tom Lane, PhD

  Chief Science Officer at Immunis, Inc.

  STUDY DIRECTOR

Central Study Contacts

Joelle Hafen, BS

CONTACT

9492662501; Joelle@immunisbiomedical.com

Erin Curry, PA

CONTACT

9492662501; Erin@immunisbiomedical.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 4 dose cohorts: Cohort A, IMM01-STEM 225μg; Cohort B, IMM01-STEM 450 μg; and Cohort C, IMM01-STEM 900 μg; Cohort D, IMM01-STEM 2000μg.

Study Record Dates

• First Submitted: November 29, 2021
• First Posted: January 27, 2022
• Study Start: September 13, 2022
• Primary Completion: March 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: October 30, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)