AzTrials
AzTrials

Primary ThrombocytopeniaPhase 3NCT01349790

Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

A Phase 3 study testing NewGam in people with Primary Thrombocytopenia, run by Octapharma at 1 site in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Primary Thrombocytopenia
What is being tested?
NewGam
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
1 siteGermany
Study dates
Oct 2011to Jul 2013
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age of ≥ 18 and ≤ 65 years.
  • Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) (diagnosed with a threshold platelet count < 100x10^9/L) of at least 12 months duration.
  • Platelet count of no more than 20x10^9/L with or without bleeding manifestations.
  • Freely given written informed consent from patient.
  • Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropin [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study.

Who cannot join

  • Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome [AIDS] or systemic lupus erythematosus [SLE]), or drug-related thrombocytopenia.
  • Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoietin receptor agonists, or other platelet enhancing drugs (including immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment, except for:
  • Long-term corticosteroid therapy when the dose has been stable during the preceding 3 weeks and no dosage change is planned until study Day 22.
  • Long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose has been stable during the preceding 3 months and no dosage change is planned until study Day 22.
  • Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.

Trial locations and contacts

1 site in 1 country.

Germany1 site

  • Abdulgabar Salama

    Berlin

Other trials in Germany

About this study

Official title: Prospective, Open-label, Non-controlled, Multicenter, Phase III Clinical Study to Evaluate the Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.

Read the detailed description

The primary objective of the study is to assess the efficacy of NewGam in correcting the platelet count. The secondary objective of the study is to evaluate the safety of NewGam. Safety will be assessed by monitoring vital signs, physical examination, evaluation of adverse events (AE) and laboratory parameters, and viral safety testing.

Being tested
NewGam
Keywords
ITP, Primary immune thrombocytopenia
Medical subject headings
Purpura, Thrombocytopenic, Idiopathic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NewGamDrug

    NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration.

Study groups in NCT01349790
Study groupRoleWhat they receive
NewGamExperimentalEach participant received 1 g/kg NewGam intravenously on 2 consecutive days.
Receives: Drug: NewGam

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Responders

    A responder is a study participant with an increase in platelets to ≥ 50x10^9/L within 7 days after the first infusion, ie, by study Day 8.

    Measured: Day 1 to Day 8

Secondary outcomes (15)

  1. Percentage of Alternative Responders

    An alternative responder is a study participant with an increase in platelets to ≥ 30x10^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

    Measured: Day 1 to Day 22

  2. Percentage of Complete Responders

    A complete responder is a study participant with an increase in platelets to ≥ 100x10^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

    Measured: Day 1 to Day 22

  3. Percentage of Alternative Responders Who Lost the Response

    An alternative responder who lost the response is a study participant who met the criterion for an alternative response but who then deteriorated, ie, their platelet count decreased to < 30x10^9/L, their platelet count decreased to less than double the baseline count, or bleeding occurred.

    Measured: Day 1 to Day 22

  4. Percentage of Complete Responders Who Lost the Response

    A complete responder who lost the response is a study participant who met the criterion for a complete response but who then deteriorated, ie, their platelet count decreased to < 100x10^9/L or bleeding occurred.

    Measured: Day 1 to Day 22

  5. Time to a Response

    A study participant had a response if their platelets increased to ≥ 50x10^9/L within 7 days after the first infusion, ie, by study Day 8.

    Measured: Day 1 to Day 8

  6. Time to an Alternative Response

    A study participant had a response if their platelets increased to ≥ 30x10^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

    Measured: Day 1 to Day 22

  7. Time to a Complete Response

    A study participant had a complete response if their platelets increased to ≥ 100x10^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

    Measured: Day 1 to Day 22

  8. Duration of a Response

    The duration of a response was defined as the time from when a response was achieved until the platelet count fell below 50x10^9/L.

    Measured: Day 1 to Day 22

  9. Duration of an Alternative Response

    The duration of an alternative response was defined as the time from when an alternative response was achieved until the platelet count fell below 50x10^9/L.

    Measured: Day 1 to Day 22

  10. Duration of a Complete Response

    The duration of a complete response was defined as the time from when a complete response was achieved until the platelet count fell below 50x10^9/L.

    Measured: Day 1 to Day 22

  11. Platelet Count by Visit

    The platelet count at each study visit are presented.

    Measured: Day 1 to Day 22

  12. Maximum Platelet Count

    The maximum platelet count achieved during the study is presented.

    Measured: Day 1 to Day 22

  13. Percentage of Responders Who Achieved a Normal Platelet Count

    The percentage of responders who achieved a normal platelet count is presented.

    Measured: Day 1 to Day 22

  14. Bleeding Intensity

    The percentage of participants with various intensities of overall bleeding, epistaxis (bleeding of the nose), oral bleeding, and skin bleeding graded as none, minor, mild, moderate, or severe at Baseline and Day 22 are reported.

    Measured: Day 1 to Day 22

  15. Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L

    The percentage of participants who achieved a platelet count > 30x10^9/L within 1 and 2 days after infusion is reported.

    Measured: Day 1 to Day 2

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 May 2011 (estimated)
  2. Study startOct 2011
  3. Primary completionJul 2013
  4. Study completionJul 2013

Resources

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
40 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01349790
Sponsor's protocol ID
NGAM-02
Other IDs
2009-014589-24, 2009
Status last verified
May 2017
First submitted
5 May 2011
Last updated
7 Jun 2017
Data monitoring committee
Yes

Publications

  • Arbach O, Taumberger AB, Wietek S, Cervinek L, Salama A. Efficacy and safety of a new intravenous immunoglobulin (Panzyga(R) ) in chronic immune thrombocytopenia. Transfus Med. 2019 Feb;29(1):48-54. doi: 10.1111/tme.12573. Epub 2019 Jan 27.

    PubMed 30687970

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing NewGam in people with Primary Thrombocytopenia, run by Octapharma at 1 site in Germany.

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 5 inclusion and 5 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: finished, as of the sponsor's last update on 7 Jun 2017. 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 Germany. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Jun 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Responders (time frame: Day 1 to Day 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01349790, which also lists 1 linked publication.

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 7 Jun 2017.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in