Clinical Trials

Numerous clinical trials sponsored by organizations including Italfarmaco, ITF Therapeutics LLC, and Radboud University Medical Center have evaluated Givinostat (ITF-2357) across Phase 1 through Phase 3. Spanning completed, recruiting, active not recruiting, and terminated recruitment statuses, these studies assess indications such as Duchenne muscular dystrophy (DMD), Becker muscular dystrophy, polycythemia vera, chronic myeloproliferative neoplasms, and juvenile idiopathic arthritis, alongside pharmacokinetic and drug-interaction profiles in healthy volunteers. Major evaluations focus on Phase 3 efficacy trials in ambulant and non-ambulant DMD cohorts and Phase 1/2 studies in JAK2V617F-positive polycythemia vera.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06769633 RECRUITING
Duchenne Muscular Dystrophy
Italfarmaco
 2025-01-02 PHASE2
NCT03373968 RECRUITING
Duchenne Muscular Dystrophy
Italfarmaco
 2017-10-24 PHASE2; PHASE3
NCT05933057 RECRUITING
Duchenne Muscular Dystrophy
Italfarmaco
 2024-02-19 PHASE3
NCT01761968 ACTIVE_NOT_RECRUITING
Chronic Myeloproliferative Neoplasms
Italfarmaco
 2013-03 PHASE2
NCT06093672 RECRUITING
Polycythemia Vera
Italfarmaco
 2024-03-26 PHASE3
NCT06736223 COMPLETED
Hepatic Impairment
Italfarmaco
 2025-05-29 PHASE1
NCT05933057 Recruiting
Duchenne Muscular Dystrophy
Italfarmaco
 2024-02-19 Phase 3
NCT05492318 COMPLETED
Drug-Drug Interaction; Heathy Volunteer
Italfarmaco
 2022-03-21 PHASE1
NCT05845567 COMPLETED
Drug Drug Interaction
Italfarmaco
 2022-03-21 PHASE1
NCT05860114 COMPLETED
Drug Drug Interaction
Italfarmaco
 2022-03-21 PHASE1
NCT05860114 Completed
Drug Drug Interaction
Italfarmaco
 2022-03-21 Phase 1
NCT05845567 Completed
Drug Drug Interaction
Italfarmaco
 2022-03-21 Phase 1
NCT02851797 COMPLETED
Duchenne Muscular Dystrophy
Italfarmaco
 2017-06-06 PHASE3
NCT04821063 COMPLETED
Duchenne and Becker Muscular Dystrophy; Polycytemia Vera
Italfarmaco
 2021-04-13 PHASE1
NCT03238235 COMPLETED
Becker Muscular Dystrophy
Italfarmaco
 2018-01-09 PHASE2
NCT01901432 COMPLETED
Polycythemia Vera
Italfarmaco
 2013-10 PHASE1; PHASE2
NCT01761292 COMPLETED
Duchenne Muscular Dystrophy (DMD)
Italfarmaco
 2013-04 PHASE1; PHASE2
NCT01557452 TERMINATED
Juvenile Idiopathic Arthritis
Italfarmaco
 2011-12-28
NCT01901432 Completed
Polycythemia Vera
Italfarmaco
 2013-10 Phase 1|Phase 2
NCT01261624 TERMINATED
Polyarticular Course Juvenile Idiopathic Arthritis
Italfarmaco
 2010-10 PHASE2
NCT00928707 COMPLETED
Polycythemia Vera
Italfarmaco
 2009-06 PHASE2
NCT00792467 COMPLETED
Hodgkin's Lymphoma
Italfarmaco
 2008-02 PHASE1; PHASE2
NCT00792506 TERMINATED
Multiple Myeloma
Italfarmaco
 2008-10 PHASE2
NCT00792831 TERMINATED
Chronic Lymphocytic Leukemia
Italfarmaco
 2008-02 PHASE2
NCT00496431 TERMINATED
Hodgkin's Lymphoma
Italfarmaco
 2007-05 PHASE1; PHASE2
NCT00606307 COMPLETED
Myeloproliferative Diseases
Italfarmaco
 2007-12 PHASE2
NCT00792740 TERMINATED
Crohn's Disease
Italfarmaco
 2007-10-22 PHASE1; PHASE2
NCT00570661 COMPLETED
Active Systemic; Onset Juvenile Idiopathic Arthritis
Italfarmaco
 2006-09-12 PHASE2
NCT00442182 UNKNOWN
Autoinflammatory Syndromes; HIDS; TRAPS; Schnitzler's Syndrome
Radboud University Medical Center
 2006-09 PHASE2

(data from https://clinicaltrials.gov, updated on 2025-07-10)

Check the ITF-2357 (Givinostat) Hydrochloride Monohydrate product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

ITF-2357 (Givinostat) functions as a potent inhibitor of class I and class II histone deacetylases (HDACs), blocking enzymatic deacetylation of histone and non-histone proteins to modulate gene transcription and suppress pro-inflammatory cytokine expression. This molecular inhibition decreases tissue inflammation and muscle fibrosis while inducing apoptosis in hyperproliferative cells, which underlies its therapeutic relevance in clinical trial conditions such as Duchenne muscular dystrophy and polycythemia vera.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.