Condition category
Cancer
Date applied
14/12/2013
Date assigned
20/01/2014
Last edited
26/06/2014
Prospective/Retrospective
Retrospectively registered
Overall trial status
Ongoing
Recruitment status
Recruiting

Plain English Summary

Background and study aims
In acute promyelocytic leukemia (APL), central nervous system (CNS) relapse occurs due to a lack of sufficient medication in the brain. The aim of our study is to enrol medium to high risk APL patients and patients with APL CNS relapse and to study whether mannitol-assisted prophylaxis (protective treatment) helps drugs penetrate the blood-brain barrier, thereby increasing the amount of the drugs in the CNS.

Who can participate?
Any APL patients (all age groups) at risk of CNS relapse or diagnosed with CNS relapse.

What does the study involve?
Our mannitol-assisted treatment strategy includes intravenous infusion (i.e., administered into a vein) of mannitol and arsenic trioxide (ATO). Patients at risk of CNS relapse will receive prophylaxis of mannitol and ATO; patients who have been diagnosed with CNS relapse will receive intrathecal chemotherapy (i.e., administered into the spine) plus mannitol and ATO. Long-term follow-up of the patients will be carried out.

What are the possible benefits and risks of participating?
Benefits are expected for the patients who will receive mannitol-assisted prophylaxis or treatment. Mannitol should help the ATO cross the blood-brain barrier, thereby increasing the amount of the drug in the CNS. The main risk of giving mannitol is to decrease the cerebral pressure (the pressure inside the skull). This could be prevented by letting the patients lie down for at least 10 hours during and after treatment.

Where is the study run from?
The study was set up at the First Affiliated Hospital of Harbin Medical University (China).

When is the study starting and how long is it expected to run for?
This study started in 1998 and is expected to run until 2018.

Who is funding the study?
China National Natural Science Foundation and China 863 Projects Foundation.

Who is the main contact?
Professor Jin Zhou, jinzhouh85@163.com
Professor Hong Wang, wh557@yahoo.com

Trial website

Contact information

Type

Scientific

Primary contact

Dr Jin Zhou

ORCID ID

Contact details

First Affiliated Hospital of Harbin Medical University
Department of Hematology
Youzheng Street
Nangang District
Harbin
150001
China
jinzhouh85@163.com

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

97-01-CHN

Study information

Scientific title

Retrospective study on mannitol-assisted prophylaxis and treatment for acute promyelocytic leukemia

Acronym

Study hypothesis

It was hypothesized that mannitol could help drugs enter the blood brain barrier (BBB). Thereby it could improve the clinical outcome of acute promyelocytic leukemia (APL) patients during prophylaxis and treatment.

Ethics approval

Harbin Medical University Ethics Committee, 16/10/1997, ref: HM970018

Study design

Retrospective study of a treatment's long-term outcome

Primary study design

Observational

Secondary study design

Cohort study

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

APL CNS relapse

Intervention

The study involved two groups of APL patients receiving different mannitol-assisted regimens:
1. Patients with CNS relapse received intrathecal chemotherapy plus mannitol-assisted arsenic trioxide (ATO)
2. Patients at risk of CNS relapse received mannitol-assisted ATO prophylaxis

Intervention type

Drug

Phase

Not Applicable

Drug names

Mannitol, arsenic trioxide

Primary outcome measures

1. Disease-free survival
2. Overall survival

Secondary outcome measures

1. Cerebrospinal fluid (CSF) ATO concentrations
2. CSF tests of APL burden
3. Drug side effects evaluation

Overall trial start date

01/01/1998

Overall trial end date

31/12/2018

Reason abandoned

Eligibility

Participant inclusion criteria

1. Any age APL patients, with either risks of central nervous system (CNS) relapse or already diagnosed with CNS relapse
2. Patients agreed to receive the prophylaxis or treatment

Participant type

Patient

Age group

Other

Gender

Both

Target number of participants

100

Participant exclusion criteria

1. Previous history of severe cardiovascular disease (coronary arterial disease, stroke, etc)
2. Severe chronic disease with poor prognosis (liver disease, kidney disease, etc)
3. Illegal drug use or chronic alcoholism
4. Physical limitations, mental or intellectual disabilities
5. Any condition that may affect the development of this trial

Recruitment start date

01/01/1998

Recruitment end date

31/12/2018

Locations

Countries of recruitment

China

Trial participating centre

First Affiliated Hospital of Harbin Medical University
Harbin
150001
China

Sponsor information

Organisation

First Affiliated Hospital of Harbin Medical University (China)

Sponsor details

c/o Dr Jin Zhou
Department of Hematology
Youzheng Street
Nangang District
Harbin
150001
China
jinzhouh85@163.com

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

China National Natural Science Foundation (China), No. 81070439

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Funder name

China 863 Projects Foundation (China), No. 2012AA020903

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

2014 results in: http://www.ncbi.nlm.nih.gov/pubmed/24963041

Publication citations

  1. Results

    Wang H, Cao F, Li J, Li L, Li Y, Shi C, Lan W, Li D, Zhao H, Zhang Y, Zhang Z, Liu X, Meng R, Yang B, Zhou J, Arsenic trioxide and mannitol for the treatment of acute promyelocytic leukemia relapse in central nervous system., Blood, 2014, doi: 10.1182/blood-2014-04-568121.

Additional files

Editorial Notes