Evaluation the advantages and disadvantages of PDlasta in Severe Congenital Neutropenia Patients with age older than 12 years
Design
After sign the inform consent form 10 Patients older than 12 years old that treated with PDlgrastim already and have all of the inclusion criteria enroll the study
Settings and conduct
Patients receive the PDlasta for free and treat with PDlasta according to protocol and visit weekly till 3 months and then monthly to evaluate clinical status, CBC and ANC (Absolute Neutrophil Count), PDlasta side effects and dosage adjustment
Participants/Inclusion and exclusion criteria
Inclusion criteria: All patients with severe congenital neutropenia older than 12 years old and treated with Filgrastim in past, arrest in myeloid maturation, without mutation in GCSF receptor gene ;Exclusion criteria: neutropenia due to other diseases, huge splenomegaly, coexistent disorders
Intervention groups
Patients receive the PDlasta and we evaluate the clinical sign and symptoms and also absolute neutrophil count
Main outcome variables
Increase in absolute neutrophil count and decrease in hospitalization due to infectious diseases.
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20150125020786N3
Registration date:2018-07-08, 1397/04/17
Registration timing:prospective
Last update:2018-07-08, 1397/04/17
Update count:0
Registration date
2018-07-08, 1397/04/17
Registrant information
Name
Marzieh Maddah
Name of organization / entity
Immunology, Asthma & Allergy Research Institute
Country
Iran (Islamic Republic of)
Phone
+98 21 6693 5855
Email address
pourpakz@tums.ac.ir
Recruitment status
Recruitment complete
Funding source
Pouyesh Darou
Expected recruitment start date
2018-07-23, 1397/05/01
Expected recruitment end date
2019-07-23, 1398/05/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Follow-up of Severe Congenital Neutropenia Patients Receiving PegGCSF (PD lasta); Study of Early and Late Drug Complications
Public title
Follow-up of Severe Congenital Neutropenia Patients Receiving PegGCSF (PD lasta); Study of Early and Late Drug Complications
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Adult patients and children with severe congenital neutropenia over 12 years
Severe Congenital Neutropenia Patients
Myeloid maturation arrest in bone marrow
Already successfully treated with Filgrastim
Negative mutation in GCSF receptor
Normal spleen and liver in abdominal sonography
Consent to Participate in study and sign inform consent
Cooperative patient for close follow-up
Exclusion criteria:
Secondary neutropenia due to chemotherapy or other diseases
Life-threatening complications such as anaphylaxis or splenic rupture due to Filgrastim
Uncooperative patient for close follow-up
Severe Spleenomegaly
patients with other chronic diseases such as heart or hepatic disease or ...
Age
From 12 years old
Gender
Both
Phase
1-2
Groups that have been masked
No information
Sample size
Target sample size:
10
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Immunology , Asthma and Allergy Research Institiute
Street address
4th Floor, Building No. 3, Tehran - Children's Hospital Medical Center, Keshavarz Blvd. Qarib Street
City
Tehran
Province
Tehran
Postal code
1419733151
Approval date
2014-01-04, 1392/10/14
Ethics committee reference number
182412
Health conditions studied
1
Description of health condition studied
Congenital Neutropenia
ICD-10 code
D70
ICD-10 code description
Neutropenia
Primary outcomes
1
Description
Absolute Neutrophil Count
Timepoint
first day of study , 7th day, 14th day, 21st day, 28th day, 35th day, 42nd day, 49th day, 56th day, 3th day, 70th day, 77th day, 84th day, 91st day then every month until reciving the drug
Method of measurement
Blood Sample
Secondary outcomes
1
Description
Infection
Timepoint
Monthly
Method of measurement
Clinical questionnare
2
Description
Hospitalization rate
Timepoint
Monthly
Method of measurement
Clinical questionnare
Intervention groups
1
Description
PegGCSF produced by Pouyesh Darou Prescribed weekly or every 2 weeks and the dose will be adjusted based on response to drug
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Immunology , Asthma and Allergy Research Institute
Full name of responsible person
Marzieh Maddah
Street address
4th Floor, Building No. 3, Children's Hospital Medical Center, Qarib Street, Keshavarz Blvd.
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6691 9589
Email
marzieh.maddah@gmail.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Pouyesh Darou biopharmaceutical Co.
Full name of responsible person
Dr Tabatabai
Street address
No. 13, 5th Ave., Fatemi St.
City
Tehran
Province
Tehran
Postal code
123456798
Phone
+98 21 8899 7248
Email
info@pooyeshdarou.com
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Pouyesh Darou biopharmaceutical Co.
Proportion provided by this source
50
Public or private sector
Private
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Industry
2
Sponsor
Name of organization / entity
Immunology, Asthma and Allergy research Institute
Full name of responsible person
Zahra Pourpak
Street address
4th Floor, Building No. 3, Children's Hospital Medical Center, Keshavarz Blvd., Qarib Street
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6691 9589
Email
pourpakz@tums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
No
Title of funding source
Immunology, Asthma and Allergy research Institute
Proportion provided by this source
50
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Immunology, Asthma and Allergy Research Institute
Full name of responsible person
Marzieh Maddah
Position
Researcher and MD , specialist
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
4th Floor, Building No. 3, Children's Hospital Medical Center, Keshavarz Blvd., Qarib Street
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6693 5855
Fax
Email
marzieh.maddah@gmail.com
Web page address
Person responsible for scientific inquiries
Contact
Name of organization / entity
Immunology, Asthma and Allergy Research Institute
Full name of responsible person
Mostafa Moin
Position
Professor, pediatrician, Immunology, Asthma and Allergy subspecilist
Latest degree
Subspecialist
Other areas of specialty/work
Immunology
Street address
4th Floor, Building No. 3, Children's Hospital Medical Center, Qarib Street
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6693 5855
Fax
Email
mmoin@tums.ac.ir
Web page address
Person responsible for updating data
Contact
Name of organization / entity
Immunology, Asthma and Allergy Research Institute
Full name of responsible person
Marzieh Maddah
Position
researcher
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
4th Floor, Building No. 3, Children's Hospital Medical Center, Qarib Street
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6693 5855
Fax
Email
marzieh.maddah@gmail.com
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)
No - There is not a plan to make this available
Justification/reason for indecision/not sharing IPD
Patients recruitment will be before January 1st 2019.
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Not applicable
Informed Consent Form
Undecided - It is not yet known if there will be a plan to make this available
Clinical Study Report
Undecided - It is not yet known if there will be a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available