Safety, Tolerability and Efficacy of GcMAF in Patients With Chronic Inflammatory Diseases

S.LAB (SOLOWAYS)

Status and phase: Begins enrollment in 4 months; Phase 2; Phase 1

Conditions: Inflammation

Treatments: Biological: GcMAF; Other: Placebo Injection (Saline)

Study type: Interventional

Funder types: Other

Identifiers

NCT06877364

SW024

Details and patient eligibility

About

This study, titled "A Multicenter Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GcMAF in Patients With Chronic Inflammatory Diseases" will investigate Gc Macrophage Activating Factor, a protein derived by enzymatic deglycosylation of vitamin D-binding protein. GcMAF activates macrophages and dendritic cells, modulates M1/M2 profiles, and shows anti-inflammatory effects. Phase I (open-label, dose-escalation) will assess safety, tolerability, and determine a Recommended Dose (RD). Phase II (randomized, double-blind, placebo-controlled) will evaluate efficacy and further confirm safety in a larger patient population with conditions such as moderate rheumatoid arthritis or chronic cystitis, refractory to standard therapies.

Full description

This trial is designed to evaluate GcMAF, an immunomodulatory protein generated from vitamin D-binding protein (DBP) via specific enzymatic deglycosylation, in adults (≥18 years) with a confirmed chronic inflammatory disease (e.g., moderate rheumatoid arthritis, chronic cystitis) who have experienced inadequate response or intolerance to standard anti-inflammatory treatments (NSAIDs, corticosteroids, etc.). GcMAF's primary mechanism of action involves activating macrophages and dendritic cells, regulating cytokine production, and potentially offering anti-inflammatory as well as antitumor benefits, as indicated in preclinical studies on animal models with conditions such as adjuvant arthritis and hemorrhagic cystitis.

The study will be conducted in two phases. Phase I is an open-label, dose-escalation investigation comprising 3-4 cohorts receiving increasing doses of GcMAF (administered subcutaneously or intramuscularly). The primary objectives for Phase I are to determine the safety profile, tolerability, and maximum tolerated dose (MTD) or Recommended Dose (RD). This will involve continuous safety monitoring, assessment of adverse events (AEs) using CTCAE criteria, and evaluation of lab parameters (hematology, biochemistry, inflammatory markers). Secondary endpoints in Phase I include preliminary efficacy measures (changes in CRP, ESR, cytokines, and disease-activity scores) and (Cmax, Tmax, AUC, T½), along with possible measurement of antibodies to GcMAF.

Upon establishing the RD in Phase I, Phase II will be a randomized, double-blind, placebo-controlled trial enrolling approximately 60-120 patients. They will be allocated (1:1 or 1:1:1 if multiple GcMAF doses are tested) to receive either GcMAF (RD) or placebo for 12-16 weeks, followed by a 4-8-week observation period. The key inclusion criteria stipulate adult patients with documented chronic inflammatory disease and the ability to adhere to the protocol. Exclusion criteria include pregnancy or lactation, severe systemic illnesses (e.g., liver/kidney failure), active serious infections (HIV, TB, or active hepatitis B/C), recent participation in other experimental studies (<3 months), and hypersensitivity to protein products.

Phase II primary endpoints focus on clinical efficacy, measured by validated scores (e.g., DAS28 for rheumatoid arthritis, symptom indices for cystitis) and the proportion of patients achieving clinically significant improvement (e.g., DAS28 improvement ≥1.2 points). Secondary endpoints include ongoing safety assessments, laboratory markers of inflammation (CRP, ESR, cytokine profile), pharmacodynamic data (such as M1/M2 macrophage ratio), and patient-reported outcomes (e.g., SF-36, HAQ-DI). Statistical analyses will include descriptive methods in Phase I for safety and tolerability, and for Phase II comparative tests (t-test, ANOVA, or nonparametric equivalents) for continuous data and chi-square or Fisher's exact test for categorical response rates. Ultimately this trial seeks to confirm whether GcMAF offers a clinically meaningful reduction of inflammatory disease activity while maintaining an acceptable safety profile.

Enrollment: 120 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Inclusion criteria
• Age ≥ 18 years, informed consent.
• Confirmed diagnosis of a chronic inflammatory disease (e.g., moderate rheumatoid arthritis or chronic cystitis, or another validated nosology) according to recognized criteria.
• Insufficient effect or intolerance of standard anti-inflammatory therapy (NSAIDs, GCS, etc.).
• Ability to comply with the study protocol.

Exclusion criteria

• Pregnancy, lactation (lack of safety data in this group).
• Severe systemic diseases (liver/renal failure, severe cardiovascular diseases, uncontrolled arterial hypertension).
• Active serious infections (HIV, tuberculosis, hepatitis B/C in the active phase).
• Previous participation in other experimental studies < 3 months ago.
• Severe allergy to protein components of the drug.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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120 participants in 2 patient groups, including a placebo group

Experimental: GcMAF

Experimental group

Description:

This arm includes patients receiving GcMAF at the recommended dose (determined in the open-label, dose-escalation Phase I portion of the study). GcMAF will be administered (e.g., subcutaneously) once or twice-weekly for approximately 12 - 16 weeks in the randomized Phase II portion. Efficacy will be measured using validated clinical scales (e.g., DAS28 for rheumatoid arthritis) and laboratory markers (CRP, ESR, etc.). Safety and tolerability will be monitored throughout the treatment period and in a follow-up phase (4 - 8 weeks).

Treatment:

Biological: GcMAF

Placebo Comparator: Saline Injection

Placebo Comparator group

Description:

This arm includes patients receiving a placebo injection (e.g., saline) under the same schedule (once or twice-weekly for 12-16 weeks) in the randomized Phase II portion of the study. Participants and investigators will be blinded to the treatment assignment. Clinical outcomes and safety profiles will be compared with those of the GcMAF arm to evaluate efficacy and confirm the overall safety of GcMAF.

Treatment:

Other: Placebo Injection (Saline)