Newly Diagnosed Pediatric ITPPhase 2NCT02669277
Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP
A Phase 2 study testing IVIG prepared by minipool technology and standard IVIG product in people with Newly Diagnosed Pediatric ITP, run by Ain Shams University at 1 site in Egypt.
Trial summary
To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks. The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10^9/L to ≥… Read more about this study
- Who is it for?
- Ages 12 months to 16 years
- Conditions the study is for
- Newly Diagnosed Pediatric ITP
- What is being tested?
- IVIG prepared by minipool technology and standard IVIG product
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteEgypt
- Study dates
- Aug 2015to Feb 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- All newly diagnosed ITP patients (< 2 weeks from onset of the disease), with no platelet enhancing therapy.
- Age eligible for study: 1-16 years old
- Gender eligible for study: both sexes
- Informed consent signed by patient or his legal guardian.
Who cannot join
- Patients started on steroid therapy.
- Platelet count<10,000/mm3.
- Having a life threatening bleeding.
- Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy.
- Treatment with immunosuppressive or other immunomodulatory drugs within 3 weeks prior to screening.
- Treatment with any other investigational drug within 7 days before study entry.
Trial locations and contacts
1 site in 1 country.
mohsen elalfy, MD contact
Egypt1 site, 1 recruiting
- Recruiting
hematology clinic ,pediatrics hospital, Ain Shams University hospital Cairo, Egypt
Cairo
Mohsen Saleh Elalfy, professor
Mohsen Saleh Elalfy, professor
About this study
Official title: Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP: A Prospective Randomized Study
To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks.
The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10^9/L to ≥ 100 x 10^9/L and absence of bleeding (complete response) or platelet count ≥ 50 x 10^9/L and at least 2-fold increase the base line count with absence of bleeding (partial response), within the specified time frame.
Read the detailed description
To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks.
The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10^9/L to ≥ 100 x 10^9/L and absence of bleeding (complete response) or platelet count ≥ 50 x 10^9/L and at least 2-fold increase the base line count with absence of bleeding (partial response), within the specified time frame.
Secondary:
- To assess safety of mini-pool IVIG in patients with newly diagnosed ITP by assessing frequency of adverse events related to mini-pool IgG administration
- To compare data on efficacy and safety of mini-pool IVIG with data obtained from literature on standard IVIG.
Study population:
Patients with newly diagnosed ITP attending Hematology clinic Ain Shams Children hospital
Sample size:
Seventy- two patients will be enrolled. They will be divided into 3 groups:
Group A; platelet non enhancing group Group B; group receiving standard IVIG Group C; group receiving IVIG New product 24 patients in each group in a ratio 1:1:1.
- Conditions
- Newly Diagnosed Pediatric ITP
- Being tested
- IVIG prepared by minipool technology, standard IVIG product
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVIG prepared by minipool technologyDrug
Also called IG plasma
Drug: To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks.
standard IVIG productDrug
to compare efficacy and safety with the minipool IVIG
| Study group | Role | What they receive |
|---|---|---|
| group A | No intervention | no platelet enhancing therapy |
| group B | Active comparator | Standard IVIG single dose 1.0 gm /kg/dose Receives: Drug: standard IVIG product |
| group C | Experimental | minipool IVIG product single dose 1.0 gm /kg/dose Receives: Drug: IVIG prepared by minipool technology |
What is being measured
The primary outcome is the main question the study is designed to answer.
bleeding score(SMOG)
change of bleeding score(SMOG) at day 3,week1,week 2 and week 4 after presentation
Measured: 4 weeks
complete blood count
change of complete blood count for number of platelets,absolute neutrophil count,absolute lymphocyte count and hemoglobin at day 3,week 1,week 2 and week 4 after presentation
Measured: 4 weeks
Secondary outcomes (1)
Number of participants with treatment-related adverse events
presence of side effects: anaphylaxis,fever,rash and any others
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2016 (estimated)
- Study startAug 2015
- Primary completionFeb 2016 (estimated)
- Study completionFeb 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Newly Diagnosed Pediatric ITPStudies of the same condition, open and under way.
- More trials run by Ain Shams UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mohsen Saleh Elalfy, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02669277
- Sponsor's protocol ID
- FMASU R30/2015
- Status last verified
- Jan 2016
- First submitted
- 20 Jan 2016
- Last updated
- 1 Feb 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP: A Prospective Randomized Study
- ClinicalTrials.gov ID
- NCT02669277
- Acronym / sponsor ID
- FMASU R30/2015
- Conditions
- Newly Diagnosed Pediatric ITP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- Aug 2015
- Primary completion
- Feb 2016 (estimated)
- Study completion
- Feb 2016 (estimated)
- First posted
- 1 Feb 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks.
The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10^9/L to ≥ 100 x 10^9/L and absence of bleeding (complete response) or platelet count ≥ 50 x 10^9/L and at least 2-fold increase the base line count with absence of bleeding (partial response), within the specified time frame.
Detailed description
To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks.
The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10^9/L to ≥ 100 x 10^9/L and absence of bleeding (complete response) or platelet count ≥ 50 x 10^9/L and at least 2-fold increase the base line count with absence of bleeding (partial response), within the specified time frame.
Secondary:
- To assess safety of mini-pool IVIG in patients with newly diagnosed ITP by assessing frequency of adverse events related to mini-pool IgG administration
- To compare data on efficacy and safety of mini-pool IVIG with data obtained from literature on standard IVIG.
Study population:
Patients with newly diagnosed ITP attending Hematology clinic Ain Shams Children hospital
Sample size:
Seventy- two patients will be enrolled. They will be divided into 3 groups:
Group A; platelet non enhancing group Group B; group receiving standard IVIG Group C; group receiving IVIG New product 24 patients in each group in a ratio 1:1:1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| group A | No intervention | –no platelet enhancing therapy |
| group B | Active comparator | Drug: standard IVIG productStandard IVIG single dose 1.0 gm /kg/dose |
| group C | Experimental | Drug: IVIG prepared by minipool technologyminipool IVIG product single dose 1.0 gm /kg/dose |
Who may qualify
- Ages
- 12 months to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- All newly diagnosed ITP patients (< 2 weeks from onset of the disease), with no platelet enhancing therapy.
- Age eligible for study: 1-16 years old
- Gender eligible for study: both sexes
- Informed consent signed by patient or his legal guardian.
Key exclusion criteria (6 listed)
- Patients started on steroid therapy.
- Platelet count<10,000/mm3.
- Having a life threatening bleeding.
- Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy.
- Treatment with immunosuppressive or other immunomodulatory drugs within 3 weeks prior to screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IVIG prepared by minipool technologyAlso: IG plasmaDrug: To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks. | Drug | group C |
| standard IVIG productto compare efficacy and safety with the minipool IVIG | Drug | group B |
Primary outcome
bleeding score(SMOG)
Time frame: 4 weeks
change of bleeding score(SMOG) at day 3,week1,week 2 and week 4 after presentation
complete blood count
Time frame: 4 weeks
change of complete blood count for number of platelets,absolute neutrophil count,absolute lymphocyte count and hemoglobin at day 3,week 1,week 2 and week 4 after presentation
1 secondary outcome
Number of participants with treatment-related adverse events
Time frame: 4 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2016.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
mohsen elalfy, MD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IVIG prepared by minipool technology and standard IVIG product in people with Newly Diagnosed Pediatric ITP, run by Ain Shams University at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact mohsen elalfy, MD (elalfym@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
bleeding score(SMOG) (time frame: 4 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Feb 2016.