evaluation of respons to hydroxycobalamine in patients with methylmalonic acidemia
Design
all patients with isolated methylmalonic acidemia or methylmalonic acidemia with hemocystinuria confirmed on ms/ms and urine organic acid or genetic that are about 15 patients. this study no need to randomization and it is phase 3 clinical trail.
Settings and conduct
patients with isolated methylmalonic acidemia or methylmalonic acidemia with hemocyctinuria confirmed on ms/ms and urine organic acid or genetic in mashhad city at 1400 that are about 15 patients.after obtaining informed consent, they will enter the study and asked them if they received hydroxycobalamine,discontinue this drug about 1 week before test.then we received 5cc urine for mma, then in stable patients we administered hydroxycobalamine acetate amp from sterop company 1mg im daily for 3 consecutive days.in unstable patients we adimistered hydroxycobalamine 2mg iv for 7 days.the day after injections we will repeat urine mma. this study no need to blinding.
Participants/Inclusion and exclusion criteria
entry condition: patients with isolated methylmalonic acidemia or methylmalonic acidemia with hemocyctinuria confirmed on ms/ms and urine organic acid or genetic in mashhad city at 1400 that are about 15 patients.
no entry condition: patients that are suspicious to this disease and haven,t certain diagnose or poor controlled patients that haven,t enough cooperation.
Intervention groups
patients with methylmalonic acidemia
Main outcome variables
urine methylmalonic acid
General information
Reason for update
Acronym
MMA
IRCT registration information
IRCT registration number:IRCT20211207053311N1
Registration date:2022-03-07, 1400/12/16
Registration timing:registered_while_recruiting
Last update:2022-03-07, 1400/12/16
Update count:0
Registration date
2022-03-07, 1400/12/16
Registrant information
Name
Afsane Nazari
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 51 3501 2620
Email address
nazari.afsane@gmail.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2022-02-04, 1400/11/15
Expected recruitment end date
2022-06-05, 1401/03/15
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of response rate to hydroxycobalamin treatment in patients with methylmalonic acidemia
Public title
Evaluation of response rate to hydroxycobalamin treatment in patients with methylmalonic acidemia
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
patients with isolated methylmalonic acidemia or methylmalonic acidemia with hemocyctinuria confirmed in MS/MS and urine organic acid or genetic
Exclusion criteria:
suspicious patients that have not certain diagnosis and poor control patients that have not cooperation
Age
To 18 years old
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
15
More than 1 sample in each individual
Number of samples in each individual:
2
urine methylmalonic acid
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 mashhad university of medical sciences
Street address
Daneshgah Ave, mashhad university of medical sciences
City
mashhad
Province
Razavi Khorasan
Postal code
9138813944
Approval date
2022-01-25, 1400/11/05
Ethics committee reference number
IR.MUMS.MEDICAL.REC.1400.828
Health conditions studied
1
Description of health condition studied
methylmalonic acidemia
ICD-10 code
E71.120
ICD-10 code description
E71.120
Primary outcomes
1
Description
decrease more than 50 percent in urine methylmalonic acid considered positive and the patient is responsible to hydroxycobalamine.
Timepoint
urine methylmalonic acid before starting the intervention and then 3 days after received hydroxycobalamine in stable patients and after 7 days in unstable patients.
Method of measurement
evaluation of urine methylmalonic acid
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group:
Category
Treatment - Other
Recruitment centers
1
Recruitment center
Name of recruitment center
Akbar hospital
Full name of responsible person
Afsane Nazari
Street address
Kaveh Blvd, Akbar hospital
City
mashhad
Province
Razavi Khorasan
Postal code
9177897157
Phone
+98 51 3871 3803
Fax
+98 51 3870 9201
Email
ak.pr@mums.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
majid ghayour mobarhan
Street address
Daneshgah ave, mashhad university of medical sciences
City
mashhad
Province
Razavi Khorasan
Postal code
9138813944
Phone
+98 51 3841 1538
Fax
+98 51 3843 0249
Email
vcresraech@mums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Mashhad University of Medical Sciences
Proportion provided by this source
100
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
Mashhad University of Medical Sciences
Full name of responsible person
afsane nazari
Position
fellowship
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
No.18, pajuhesh 1 Ave, vakil abad 79 Blvd, mashhad, Iran
City
mashhad
Province
Razavi Khorasan
Postal code
9189835784
Phone
+98 51 3501 2620
Email
nazari.afsane@gmail.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
afsane nazari
Position
fellowship
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
No.18, pajuhesh 1 Ave, vakil abad 79 Blvd, mashhas, Iran
City
mashhad
Province
Razavi Khorasan
Postal code
9189835784
Phone
+98 51 3501 2620
Email
nazari.afsane@gmail.com
Person responsible for updating data
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
afsane nazari
Position
fellowship
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
No.18, pajuhesh 1 ave,vakil abad 79 Blvd, mashhad,Iran
City
mashhad
Province
Razavi Khorasan
Postal code
9189835784
Phone
+98 51 3501 2620
Email
nazari.afsane@gmail.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Undecided - It is not yet known if there will be a plan to make this available
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Undecided - It is not yet known if there will be a plan to make this available
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
Data Dictionary
Undecided - It is not yet known if there will be a plan to make this available