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A Phase I Study of Hydroxychloroquine With or Without Erlotinib in Advanced NSCLC

Phase 1
18 Years
Not Enrolling
Non-small Cell Lung Cancer

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Trial Information

A Phase I Study of Hydroxychloroquine With or Without Erlotinib in Advanced NSCLC

- The goal of this study is to find the highest dose of HCQ that can be given safely with
erlotinib. Therefore, not all participants will receive the same dose of HCQ. Small
groups of participants will be enrolled in steps in this trial. The first group will
be given a certain dose of HCQ. If they have few or manageable side effects, the next
small group of participants enrolled will receive a higher dose. This increase in
doses will continue until the research doctors find the highest dose of HCQ that can be
given without causing severe or unmanageable side effects.

- Both HCQ and erlotinib are pills that are taken orally. Treatment will be divided into
time periods called cycles. Each treatment cycle is 28 days. The exception to this 28
day cycle is when participants start taking the pills for the first time. Erlotinib is
started first for 7 days and then HCQ is added. When the HCQ begins, the first cycle
of 28 days begins.

- There are several tests and procedures that will be performed at specific time periods
during protocol treatment. These include: blood work, performance status assessment,
questions about medical history and medications, tumor assessment with CT or MRI and,
eye exams.

- Participants may continue to receive study treatment as long as they do not experience
unacceptable side effects or disease progression.

Inclusion Criteria:

- Pathologically confirmed diagnosis of non-small cell lung cancer

- Stage IIIB with pleural effusion or Stage IV disease by the American Joint Committee
on Cancer (AJCC) 6th edition staging criteria.

- At least 12 weeks of prior treatment with erlotinib, gefitinib, or another EGFR small
molecule TKI agent.

- Age equal to or greater than 18 years

- Measurable disease, defined according to RECIST criteria

- Performance status of 0, 1 or 2

- At least 2 weeks since prior radiation treatment

- At least 2 weeks since any prior chemotherapy or targeted therapy

- Adequate organ function as outlined in the protocol

- Approval for HCQ treatment by an eye doctor, based on a screening eye exam. Examples
of disqualifying baseline conditions include macular degeneration and other retinal

- Willingness to comply with protocol procedures including the blood-sampling schedule
for PK analyses and periodic eye examination

Exclusion Criteria:

- Current use of hydroxychloroquine for any reason

- Known hypersensitivity to chloroquine, hydroxychloroquine, or any other closely
related drug

- Known hypersensitivity to erlotinib, gefitinib, or any closely related drug

- Glucose-6-phosphate dehydrogenase (G6PD) deficiency, as HCQ may cause hemolytic
anemia in patients with G6PD deficiency

- Cataracts that would interfere with required funduscopic examinations, or severe
baseline visual impairment including macular degeneration, retinopathy or visual
field changes, or having only one functional eye. All patients must undergo a
screening eye exam prior to enrollment

- Pregnancy or breastfeeding. Female subjects of childbearing age and male subjects
must practice acceptable method of birth control

- Symptomatic CNS metastases or newly diagnosed CNS metastases that have not yet been
definitively treated with radiation and/or surgery

- Prior radiation therapy inclusive of all identified target lesions

- Any evidence of clinically active interstitial lung disease

- Malignancies within the past 3 years except for adequately treated carcinoma of the
cervix or basal or squamous cell carcinomas of the skin

- Although not an absolute exclusion criteria, caution should be exercised in patients
with a diagnosis of porphyria or non-light sensitive psoriasis, as HCQ can
significantly exacerbate both of those conditions

- Evidence of any other significant clinical disorder or laboratory finding that makes
it undesirable for the patient to participate in the study

- Use of any non-FDA approved or investigational agent within 2 weeks of enrolling onto
the trial, or failure to recover from the side effects of any of these agents

- Penicillamine use for Wilson's disease or any other indication, as concomitant use
with HCQ can increase toxicity to penicillamine

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Identify the dose limiting toxicities and maximum tolerated dose of HCQ plus erlotinib in patients with advanced NSCLC and to determine the overall safety profile.

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Lecia Sequist, MD, MPH

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachussets General Hospital


United States: Institutional Review Board

Study ID:




Start Date:

July 2007

Completion Date:

December 2012

Related Keywords:

  • Non-Small Cell Lung Cancer
  • erlotinib
  • tarceva
  • hydroxychloroquine
  • Carcinoma, Non-Small-Cell Lung
  • Lung Neoplasms



Massachusetts General Hospital Boston, Massachusetts  02114-2617