Pilot Study of Hydroxychloroquine for the Treatment of AIMSS

NCT07825454 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CTO-IUSCCC-0959
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the effectiveness of hydroxychloroquine sulfate (a type of drug commonly used to treat joint pain caused by inflammation, swelling) in reducing joint pain associated with Aromatase Inhibitor Associated Musculoskeletal Syndrome (AIMSS) for patients with breast cancer.

Conditions

Aromatase Inhibitor Associated Musculoskeletal Symptoms (AIMSS); Breast Cancer; Joint Pain

Keywords

Hydroxychloroquine; Joint Pain; Aromatase Inhibitor Associated Musculoskeletal Symptoms (AMISS)

Outcome Measures

Primary Outcomes (1)

• To evaluate the efficacy of hydroxychloroquine

  To evaluate the efficacy of hydroxychloroquine 400 mg daily in reducing joint pain associated with AIMSS as measured by change in Brief Pain Inventory (BPI) Average Pain score (scored from 0-10; a minimally important difference is a 2-point reduction or 30% from baseline).

  Day 1 and Week 12

Secondary Outcomes (8)

• Proportion of patients achieving a clinically meaningful improvement

  Day 1 and Week 12

• Changes in BPI Worst Pain and Pain Interference scores

  Day 1 and Week 12

• Changes in endocrine therapy related quality of life

  Day 1 and Week 12

• Changes in grip strength

  Day 1 and Week 12

• Patient-reported global impression of change

  Week 12

Study Arms (1)

Hydroxychloroquine sulfate

EXPERIMENTAL

Hydroxychloroquine sulfate, 400 mg orally once daily

Drug: Hydroxychloroquine Sulfate (HCQ)

Interventions

Hydroxychloroquine Sulfate (HCQ) DRUG

Drug: Hydroxychloroquine sulfate Dose: 400 mg orally once daily Duration: 12 weeks

Hydroxychloroquine sulfate

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years at the time of informed consent
• Ability to provide written informed consent and HIPAA authorization
• Diagnosis of DCIS or stage I, II, or III breast cancer
• Currently receiving adjuvant aromatase inhibitor therapy (anastrozole, letrozole or exemestane) for ≥ 4 weeks prior to enrollment Note: Concurrent use of ovarian suppression is allowed Note: Concurrent use of CDK4/6 inhibitors is not allowed due to cytopenia risk
• New or worsening self-reported musculoskeletal pain that began or significantly worsened after initiation of AI therapy
• BPI average pain score of ≥ 4 during screening
• ECOG PS 0-2
• Adequate organ function:
• Absolute neutrophil count ≥1,500/µL
• Hemoglobin ≥11.0 g/dL
• Platelet count ≥100,000/µL
• Serum creatinine ≤1.5× upper limit of normal (ULN) or eGFR ≥60 mL/min/1.73m2
• Total bilirubin ≤1.5× ULN (except in patients with documented Gilbert's disease, who must have a total bilirubin \< 3.0 mg/dL)
• AST and ALT ≤1.5× ULN
• Must agree to maintain stable doses of any analgesic medications or other AIMSS related therapies during the study period Note: rescue doses of acetaminophen or ibuprofen allowed on a non-daily basis, unless not new

You may not qualify if:

• Current or prior use of hydroxychloroquine or chloroquine within 6 months
• Known hypersensitivity to hydroxychloroquine, chloroquine, or 4-aminoquinoline compounds
• History of retinopathy or retinal vein occlusion issues Note: While there is an association between retinopathy and HCQ use, this occurs rarely and with long term use with higher cumulative doses that used here. Other clinical trials have not required retinal exam (example: CTO-TBCRC04627, approved by IU IRB)
• History of congestive heart failure (any NYHA class)
• QTc prolongation (\>450 msec) at screening or history of significant arrhythmias requiring treatment
• Moderate to severe renal impairment (eGFR \<60 mL/min/1.73m2)
• Use of a prohibited concomitant medication (refer to Section 6.3.2) that cannot be discontinued or changed to an alternate therapy
• Known glucose-6-phosphate dehydrogenase (G6PD) deficiency
• History of porphyria
• History of psoriasis
• History of antiepileptic medications
• Known history of inflammatory arthritis (example: rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis) or connective tissue disease (example: systemic lupus erythematosus, scleroderma, polymyositis)
• Recent initiation or dose change (within 4 weeks) of medications for pain management (NSAIDs, duloxetine, gabapentin, pregabalin, opioids) Note: Patients on stable doses for \>4 weeks are eligible
• Patients currently receiving or planned to receive high dose systemic treatment with corticosteroids defined as: cortisone \>50mg; hydrocortisone \>40mg, prednisone \>10mg, methylprednisolone \>8mg or dexamethasone \>1.5mg; or another immunosuppressive agent Note: Topical or inhaled corticosteroids are allowed
• Other active malignancy other than breast cancer requiring systemic therapy

Sponsors & Collaborators

• Indiana University: lead
• 100 Voices of Hope: collaborator

Study Sites (1)

Indiana University Melvin and Bren Simon Comprehensive Cancer Center

Indianapolis, Indiana, 46202, United States

Location

MeSH Terms

Conditions

Arthralgia; Breast Neoplasms

Interventions

Hydroxychloroquine

Condition Hierarchy (Ancestors)

Joint Diseases; Musculoskeletal Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Neoplasms by Site; Neoplasms; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Chloroquine; Aminoquinolines; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Tarah Ballinger, MD

  IUSCCC

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Niraj Shah, MS

CONTACT

317-278-3420; shahnir@iu.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: August 25, 2026
• First Posted: September 17, 2026
• Study Start: September 1, 2026
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: September 17, 2026

Record last verified: 2026-09

Locations

US (1)