A Randomized Controlled Trial of Mobile Technology-assisted Outpatient Maintenance Therapy in Children With Acute Lymphoblastic Leukaemia (ALL)
Phase: NA
Conditions: Pediatric Acute Leukemia
Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Clinical study protocol 
 
Title: 
A randomized controlled trial of mobile technology-assisted 
outpatient maintenance therapy in children with acute 
lymphoblastic leukaemia (ALL) 
 
Portocol number: 
mM-002 
Research unit： 
Institute of Hematology & Blood Diseases Hospital, 
Chinese Academy of Medical Sciences & Peking Union 
Medical College. 
 
Address： 
288 Nanjing Road, Heping district, Tianjin, China 
 
Version： 
3.0 
 
Version date： 
January 29, 2026 
 
 
 
 
Declaration of Secrecy 
 
This document is confidential information of Institute of Hematology & Blood Diseases 
Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College 
(Tianjin, China) and is only used for the purpose of this clinical study. It shall not be 
disclosed to anyone other than the participating researchers and members of the 
institutional review board. This information cannot be used for any purpose other than 
the evaluation or implementation of clinical studies without the prior written consent 
of Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical 
Sciences & Peking Union Medical College. 
 

1. Abstract 
Title 
A randomized controlled trial of mobile technology-assisted 
outpatient maintenance therapy in children with acute 
lymphoblastic leukaemia (ALL). 
Study objective 
To assess the efficacy of using mobile technology to improve 
the percentage of time wherein drug dosing is within the target 
range. 
Study design 
Single-center, randomized controlled trial. 
Study population 
Children with ALL receiving outpatient maintenance therapy. 
Inclusion criteria 
1) < 16 years old at diagnosis; 
2) having demonstrated ≥ 3 months of > 90% compliance with 
using a mobile app to log in records of daily medication and 
weekly blood test results; 
3) patient or legal guardian provides informed consent. 
Exclusion criteria 
None. 
Sample size 
≥ 200 subjects 
Dosage regimen 
A mobile application classifies each subject’s maintenance 
therapy dosing status as ‘On Track’, ‘Orange Alert’, or ‘Red 
Alert’ based on analysis of his/her electronic diary of 
medication and blood tests.  Subjects classified as ‘Orange 
Alert’ receive mobile-phone alert messages that instruct them 
to self-adjust dose of oral medication.  Subjects classified as 
‘Red Alert’ are directed to a mobile-phone portal for 
scheduling a telemedicine clinic visit and receive periodic 
model-phone messages instructing follow-up blood testing and 
dose adjustment. 
Study endpoints 
Primary endpoint:  
 
Percentage of time wherein white blood cell concentration 
is within the target range 2.0 – 3.0 × 109/L after 
randomization. 
 
Secondary endpoints:  
 
Percentage of time wherein white blood cell concentration 
is < 1.0 or > 5.0 × 109/L; 

Statistical analysis 
Wilcoxon test will be used to test the significance of change(s) 
in clinical endpoints.  

2. Research background 
 
Modern treatment for acute lymphoblastic leukaemia (ALL) in children 
lasts ≈ 2.5 years, consisting of inpatient chemotherapy lasting 1 year and 
ensuing outpatient maintenance therapy lasting 1.5 years.  During the 
outpatient maintenance therapy phase, subjects live at home, which are 
often ≥ hundreds of miles away from the ‘principal medical center’ (that is, 
the hospital where they receive inpatient chemotherapy), and they are to 
take oral 6-mercaptopurine daily and methotrexate weekly at home.  Also, 
subjects are to monitor their blood cell counts weekly at their local 
healthcare facilities.  Subjects and their guardians are instructed to self-
adjust dosage of maintenance therapy with the goal to maintain white blood 
cell concentration within the target range 2.0 – 3.0 × 109/L.  When white 
blood cell concentration is too low, drug dosage should be decreased.  On 
the other hand, when the white blood cell concentration is too high, dosage 
should be increased.  Only once every ≈3 months, subjects travel to the 
principal medical center for outpatient periodic check-up.  The principal 
medical center needs a method to better monitor subjects’ compliance of 
drug in-take and their adherence to the target range of white blood cell 
concentration.     
 
 
3. Study objective 
 
To assess whether the use of mobile technology can improve the 
percentage of time wherein outpatient drug dosing is ‘on target’.   
 
 
4. Study design 
 
Enrolled subjects enter electronic diaries of oral medication and outpatient 
blood test results in a mobile application.  The mobile application 
classifies each subject’s maintenance therapy dosing status as ‘On Track’, 
‘Orange Alert’, or ‘Red Alert’ based on his/her electronic diary. 
 
Subjects whose white blood cell concentration is 2.0 – 2.3 × 109/L in the 
most recent test or 3.5 – 5.0 × 109/L for < 3 most-recent weeks will be 
classified as ‘Orange Alert’.  Subjects whose white blood cell 
concentration is 3.5 – 5.0 × 109/L for ≥ 3 most-recent weeks or whose white 
blood cell concentration is < 2.0 or > 5.0 × 109/L in the most recent blood 

test are classified as ‘Red Alert’.  The other subjects are classified as ‘On 
Track’.   
 
Subjects classified as ‘Orange Alert’ receive mobile-phone alert messages 
that are tailored to their situations to help them self-adjust dose of oral 
medication.  Subjects classified as ‘Red Alert’ are directed to a mobile-
phone portal for scheduling an online doctor visit.   
 
 
5. Study population 
 
Children with ALL receiving outpatient maintenance therapy. 
 
 
6. Inclusion criteria 
 
1) < 16 years old at diagnosis; 
2) having demonstrated ≥ 3 months of > 90% compliance with using a 
mobile app to log in records of daily medication and weekly blood test 
results; 
3) patient or legal guardian provides informed consent. 
 
 
7. Exclusion criteria 
 
None. 
 
 
8. Drop-out and withdrawal criteria 
 
1) Drop-out criteria:  Relapse or death.   
 
2) Withdrawal criteria:  Any subject wishing to discontinue participating 
can withdraw from the study, and the date and reason for withdrawal 
shall be recorded.  
 
 
9. Follow-up 
 
≥ 3 months after randomization. 

 
 
10. Primary endpoint 
 
Percentage of time wherein white blood cell concentration is within the 
range 2.0 – 3.0 × 109/L. 
 
 
11. Secondary endpoints 
 
Percentage of time wherein white blood cell concentration is < 1.0 or > 5.0 
× 109/L;   
 
 
12. Sample size calculation 
 
To attain a 0.05 significance level and a 0.9 power at a presumed 5% 
dropout rate, we estimated ≥ 200 subjects will need to be enrolled.   
 
 
13. Statistical analysis 
 
Wilcoxon test will be used to test the significance of change(s) in clinical 
endpoints. 
 
 
14. Ethical review 
 
The Institute of Hematology, Chinese Academy of Medical Sciences 
(IHCAMS) has approved this study and its associated informed consent 
form.  The study will be conducted in accordance with the Declaration of 
Helsinki, regulations issued by the government of the People’s Republic of 
China, and additional precautions required by the Ethics Committee at the 
IHCAMS.  During the study, if there is any amendment to the study 
protocol, renewed approval shall be obtained from the IHCAMS Ethics 
Committee before continuation of the study. 
 
 
15. Preservation of research data 
 

All data of this study will be stored at the IHCAMS.  Data sharing among 
the researchers will abide by the regulations of the People’s Republic of 
China regarding subject privacy protection.