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Hydroxychloroquine in Previously Treated Patients With Metastatic Pancreatic Cancer

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ClinicalTrials.gov Identifier: NCT01273805
Recruitment Status : Completed
First Posted : January 11, 2011
Results First Posted : June 14, 2017
Last Update Posted : June 14, 2017
Sponsor:
Collaborators:
Brigham and Women's Hospital
Massachusetts General Hospital
Information provided by (Responsible Party):
Brian Wolpin, MD, MPH, Dana-Farber Cancer Institute

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pancreatic Cancer
Intervention Drug: Hydroxychloroquine
Enrollment 20
Recruitment Details 20 participants were enrolled between January 2011 and October 2012.
Pre-assignment Details  
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision. Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Period Title: Overall Study
Started 10 10
Completed 0 0
Not Completed 10 10
Reason Not Completed
Radiologic PD or Symptom Deterioration             10             10
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d. Total
Hide Arm/Group Description Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision. Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision. Total of all reporting groups
Overall Number of Baseline Participants 10 10 20
Hide Baseline Analysis Population Description
The analysis dataset is comprised of all enrolled patients.
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants 10 participants 20 participants
67.5
(56 to 83)
66
(58 to 76)
67
(56 to 83)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
Female
5
  50.0%
4
  40.0%
9
  45.0%
Male
5
  50.0%
6
  60.0%
11
  55.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 10 participants 20 participants
10 10 20
ECOG Performance Status (PS)   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
PS0 1 1 2
PS1 5 8 13
PS2 4 1 5
[1]
Measure Description: ECOG PS0: Fully active, able to carry on all pre-disease performance without restriction ECOG PS1: Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature ECOG PS2: Ambulatory and capable of all self-care but unable to carry out any work activities; Up and about more than 50% of waking hours
Site of Primary Tumor  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
Head or Head and Body 6 7 13
Body or Tail 4 3 7
Location of Metastasis  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 10 participants 10 participants 20 participants
Liver 8 7 15
Lung 4 3 7
Peritoneum 4 5 9
Other 2 2 4
1.Primary Outcome
Title 2-month Progression-Free Survival Rate
Hide Description 2-month progression-free survival rate was defined as the percentage of patients absent progression (PD) or death before 2 months. Patients were considered to have experienced PD if they demonstrated either clinical deterioration resulting in withdrawal or PD per RECIST 1.0 criteria: At least a 20% increase in the sum of longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. PD for the evaluation of non-target lesions is the appearance of one or more new lesions and/or unequivocal progression of non-target lesions.
Time Frame Disease was evaluated radiologically at baseline and at the first restaging at 2 months.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis dataset is comprised of all enrolled patients.
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description:
Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Overall Number of Participants Analyzed 10 10
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients
10
(.28 to 44.5)
10
(.28 to 44.5)
2.Secondary Outcome
Title Biochemical Response Rate
Hide Description Biochemical response rate was defined as the percentage of patients achieving on treatment a decrease in serum CA 19-9 by > 30% from baseline.
Time Frame Disease was evaluated radiologically at baseline and every 2 months on treatment. Median duration of treatment for this study cohort was 34 days.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Biochemical response could not be estimated due to insufficient longitudinal CA 19-9 measurements. This was directly related to the observed lack of activity of the study drug and corresponding short duration of therapy.
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description:
Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Tumor Response Rate
Hide Description Tumor response rate is the percentage of patients achieving complete or partial response on treatment based on RECIST 1.0 criteria. For target lesions, complete response (CR) is disappearance of all target lesions and partial response (PR) is at least a 30% decrease in the sum of longest diameter (LD) of target lesions, taking as reference baseline sum LD. CR for the evaluation of non-target lesions is the disappearance of non-target lesions and normalization of tumor marker level. Appearance of one or more new lesions is classified as progression of non-target lesions. CR or PR confirmation is required >/= 4 weeks.
Time Frame Disease was evaluated radiologically at baseline and every 2 months on treatment. Median duration of treatment for this study cohort was 34 days.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis dataset is comprised of all enrolled patients.
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description:
Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Overall Number of Participants Analyzed 10 10
Measure Type: Number
Unit of Measure: percentage of patients
0 0
4.Secondary Outcome
Title Overall Survival
Hide Description Overall survival estimated using Kaplan-Meier (KM) methods is defined as the time from study entry to death or date last known alive.
Time Frame All patients were followed until death. Median survival follow-up in this study cohort was 60 days (95% CI: 40-184).
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis dataset is comprised of all enrolled patients.
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description:
Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Overall Number of Participants Analyzed 10 10
Median (95% Confidence Interval)
Unit of Measure: days
51.5
(24 to 275)
83
(40 to 178)
5.Secondary Outcome
Title Progression-Free Survival
Hide Description Progression-free survival based on the Kaplan-Meier method is defined as the duration of time from study entry to time of objective progression on CT scan or the time of death for patients with clinical deterioration resulting in withdrawal from the trial. Per RECIST 1.0 criteria: progressive disease (PD) is at least a 20% increase in the sum of longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. PD for the evaluation of non-target lesions is the appearance of one or more new lesions and/or unequivocal progression of non-target lesions. Patients without an event were censored at date of last disease evaluation.
Time Frame Disease was evaluated radiologically at baseline and every 2 months on treatment. Median PFS follow-up in this study cohort was 46.5 days (95% CI 33-61).
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis dataset is comprised of all enrolled patients.
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description:
Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Overall Number of Participants Analyzed 10 10
Median (95% Confidence Interval)
Unit of Measure: days
51.5
(16 to 66)
44.5
(33 to 74)
6.Secondary Outcome
Title Grade 4-5 Treatment-Related Toxicity
Hide Description All grade 4-5 adverse events with treatment attribution of possibly, probably or definite based on CTCAEv3 as reported on case report forms.
Time Frame Adverse events were assessed each cycle throughout treatment. Participants were followed for the duration of treatment, an average of 34 days for this study population.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis dataset is comprised of all treated patients.
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description:
Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
Time Frame Adverse events (AE) were assessed each cycle throughout treatment. Participants were followed for the duration of treatment, an average of 34 days for this study population.
Adverse Event Reporting Description Maximum grade toxicity by type for a patient over time including only treatment-related events (possible, probable or definite attribution) was first calculated with patients therefore appearing once for a given type of toxicity. Serious and Other AEs were defined as events of grades 3-5 and grades 1-2, respectively, based on CTCAEv3.
 
Arm/Group Title Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Hide Arm/Group Description Patients received 400 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision. Patients received 600 mg hydroxychloroquine orally twice per day. Cycle duration was 4 weeks. Patients remained on treatment indefinitely without the occurrence of disease progression, unacceptable adverse events, patient withdrawal, or discontinuation per MD decision.
All-Cause Mortality
Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)   0/10 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Affected / at Risk (%) Affected / at Risk (%)
Total   1/10 (10.00%)   1/10 (10.00%) 
Investigations     
Elevated alanine aminotransferase  1  1/10 (10.00%)  0/10 (0.00%) 
lymphopenia  2  0/10 (0.00%)  1/10 (10.00%) 
1
Term from vocabulary, CTCAE (3.0)
2
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Hydroxychloroquine 400 mg b.i.d. Hydroxychloroquine 600 mg b.i.d.
Affected / at Risk (%) Affected / at Risk (%)
Total   10/10 (100.00%)   10/10 (100.00%) 
Blood and lymphatic system disorders     
Anemia  1  0/10 (0.00%)  1/10 (10.00%) 
Eye disorders     
Blurred vision  1  0/10 (0.00%)  1/10 (10.00%) 
Gastrointestinal disorders     
Abdominal pain  1  1/10 (10.00%)  1/10 (10.00%) 
Constipation  1  1/10 (10.00%)  0/10 (0.00%) 
Diarrhea  1  1/10 (10.00%)  5/10 (50.00%) 
Flatulence  1  0/10 (0.00%)  1/10 (10.00%) 
Nausea  1  0/10 (0.00%)  5/10 (50.00%) 
Vomiting  1  0/10 (0.00%)  4/10 (40.00%) 
General disorders     
Facial edema  1  1/10 (10.00%)  0/10 (0.00%) 
Fatigue  1  3/10 (30.00%)  1/10 (10.00%) 
Investigations     
Elevated alanine aminotransferase  1  1/10 (10.00%)  0/10 (0.00%) 
Elevated alkaline phosphatase  1  3/10 (30.00%)  0/10 (0.00%) 
Elevated aspartate aminotransferase  1  3/10 (30.00%)  0/10 (0.00%) 
Hyperbilirubinemia  1  1/10 (10.00%)  0/10 (0.00%) 
Leukopenia  1  1/10 (10.00%)  0/10 (0.00%) 
Metabolism and nutrition disorders     
Anorexia  1  3/10 (30.00%)  2/10 (20.00%) 
Hyperglycemia  1  1/10 (10.00%)  0/10 (0.00%) 
Hypoalbuminemia  1  1/10 (10.00%)  0/10 (0.00%) 
Skin and subcutaneous tissue disorders     
Pruritus  1  2/10 (20.00%)  0/10 (0.00%) 
Rash, maculo-papular  1  1/10 (10.00%)  0/10 (0.00%) 
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Brian M. Wolpin
Organization: Dana-Farber Cancer Institute
Phone: 617.632.6942
Responsible Party: Brian Wolpin, MD, MPH, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT01273805     History of Changes
Other Study ID Numbers: 10-310
First Submitted: January 7, 2011
First Posted: January 11, 2011
Results First Submitted: April 11, 2017
Results First Posted: June 14, 2017
Last Update Posted: June 14, 2017