Chronic Idiopathic Thrombocytopenic PurpuraPhase 3NCT00504075
A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP)
A Phase 3 study testing Gammaplex, intravenous immunoglobulin in people with Chronic Idiopathic Thrombocytopenic Purpura, run by Bio Products Laboratory at 30 sites in 3 countries.
Trial summary
To determine if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. Also to assess the safety of GAMMAPLEX and determine if platelet counts are maintained at 50 x 109/L in subjects with chronic ITP for. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Chronic Idiopathic Thrombocytopenic Purpura
- What is being tested?
- Gammaplex, intravenous immunoglobulin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 30 sitesin 3 countries
- Study dates
- Sep 2007to Aug 2011
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females aged between 18 and 70 years.
- Confirmed diagnosis of chronic ITP of at least 6 months duration.
- Platelet count of less than or equal to 20 x 10 9/L at enrollment.
- Absence of other conditions that, in the opinion of the investigator, could cause thrombocytopenia.
- If subjects were treated with corticosteroids the treatment regimen/dose must have been stable (for a minimum of 2 weeks before screening). The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 6) If subjects were being treated with cyclophosphamide, azathioprine or attenuated androgens, the treatment regimen and dose must have been stable for a minimum of 2 months before Day 1. The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 7) Splenectomized subjects and both Rh(D)+ and Rh(D)- subjects may be included.
- 8) The subject has signed an informed consent form (subjects must be at least 18 years old), and/or the subject's legal guardian has signed the informed consent form if indicated 9) If a subject is a female of child-bearing potential, she must have a negative result on a urine-based HCG pregnancy test.
- 10) If a subject is a female who is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study.
Who cannot join
- A history of any severe or anaphylactic reaction to blood or any blood-derived product, or any severe reaction to IGIV or any other IgG preparation.
- Intolerance to any component of the investigational product.
- Received any live virus vaccine within the last 3 months prior to Day1.
- Received an IGIV preparation within 1 month prior to Day 1.
- Were currently receiving, or has received, any investigational agent within the 1 month prior to Day 1.
- Received any blood, blood product, or blood derivative within the 1 month prior to Day 1.
Show all 19Show fewer
- Received Rituximab within the 3 months before Day 1.
- Pregnant or nursing.
- Tested positive for any of the following at screening: HBsAg, NAT for HCV, NAT for HIV, Antibodies to HCV or HIV 1 or 2.
- Had levels greater than 2.5 times the upper limit of normal at screening, as defined by the central laboratory, of alanine aminotransferase or aspartate aminotransferase.
- Had severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or BUN greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); on dialysis; a history of acute renal failure.
- Known to have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
- History of deep vein thrombosis (DVT) or thrombotic complications of IGIV therapy.
- Any history or sign of hyperviscosity, transient ischemic attack (TIA), stroke, other thromboembolic event, or unstable angina.
- Suffered from any acute or chronic medical conditions (e.g., renal disease or predisposing conditions for renal disease, coronary artery disease, or protein losing enteropathy) that, in the opinion of the investigator, may interfere with the conduct of the study.
- An acquired medical condition, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (defined as an absolute neutrophil count (ANC) < 1 x 109/L).
- Non-controlled arterial hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >90 mmHg).
- Anemic (hemoglobin <10 g/dL) at screening.
- Unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative.
Trial locations and contacts
30 sites in 3 countries.
30 sites in 3 countries
India14 sites
Vedanta
Ahmedabad, Gujarat, 380009
Gurukrupa Hospital
Ahmedabad, Gujarat, 380061
Manipal Hospital
Bangalore, Karnataka, 560017
M S Patel Cancer Centre, Shree Krishna Hospital
Gokal Nagar, Karamsad, Gujarat, 388325
Apollo Hospitals
Hyderabad, Andhra Pradesh, 500003
Mahavir Hospital
Hyderabad, Andhra Pradesh, 500054
S.K. Soni Hospital Sect 5
Jaipur, Rajasthan, 300201
M. S Ramaiah Hospital
Karnataka, Bangalore, 560054
Vinaya Hospital & research Centre
Mangalore, Karnataka, 5750003
Kasturba Medical College, Manipal Acunova KMC Research Centre
Mangalore, Karnataka, 575001
Father Muller Medical College Hospital
Mangalore, Karnataka, 575002
Sir Ganga Ram Hospital
New Delhi, 110060
Deenanath Mangeshkar Hospital
Pune, Maharashtra, 411004
City Cancer Centre,
Suryarao Pet, Vijayawada, Andhra Pradesh, 520002
Argentina9 sites
I. A. D. T. (Instituto Argentino de Diagnóstico y Tratamiento)
Buenos Aires, C1122AAL
Hospital Britanico
Buenos Aires, C1280AEB
Centro de Educación Médica e Investigaciones Clinicas Dr. Norberto Quirno (CEMIC)
Buenos Aires, C1431FWO
Hospital Churruca
Capital Federal, Buenos Aires, C1437JCP
Hospital Privado de Cordoba
Córdoba, X5016KEH
J.R. Vidal Hospital
Corrientes
Hospital Italiano de La Plata
La Plata, Buenos Aires, B1900AXU
Instituto de Diagnóstico Hematológico Ambulatorio (IDHEA)
Rosario, Sante Fe, S2000JKR
CER San Juan
San Juan, San Juan Province, 5400
United States7 sites
Center for Cancer & Blood Disorders
Bethesda, Maryland, 20817
Rush University Medical Center
Chicago, Illinois, 60612
New York Hospital / Cornell University, Division of Pediatrics
New York, New York, 10021
Mid Florida Hematology & Oncology
Orange City, Florida, 32763
Oregon Health & Science University
Portland, Oregon, 97239
Cancer Care Centers of South Texas
San Antonio, Texas, 78229
Department of Pediatrics, SUNY at Stony Brook
Stony Brook, New York, 11794-8111
About this study
Official title: A Phase III, Multicenter, Open-Label Study To Evaluate the Efficacy and Safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura
To determine if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. Also to assess the safety of GAMMAPLEX and determine if platelet counts are maintained at 50 x 109/L in subjects with chronic ITP for.
Read the detailed description
The primary objective is to determine if BPL's GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. The secondary objectives are: 1) to determine the safety of GAMMAPLEX at the dosage used in this study. 2) to determine if GAMMAPLEX maintains platelet counts of ³ 50 x 109/L in subjects with chronic ITP for a period of time similar to that of a published data.
- Being tested
- Gammaplex, intravenous immunoglobulin
- Keywords
- Idiopathic Thrombocytopenic Purpura, Bleeding disorders, Immune System and Disorders
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Hemostatic Disorders, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gammaplex, intravenous immunoglobulinBiological
Also called Gammaplex
Dosage form: Gammaplex® is a sterile liquid of 5 % w/v normal immunoglobulin. Gammaplex® contains 5 g/100 mL of human normal immunoglobulin (i.e. 50 g/L, of which virtually 100% is IgG).
The first course of GAMMAPLEX will be administered as an intravenous infusion of 1 g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen may be administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
| Study group | Role | What they receive |
|---|---|---|
| Gammaplex (intravenous immunoglobulin) | Experimental | Not described Receives: Biological: Gammaplex, intravenous immunoglobulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L.
The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10^9/L by Day 9.
Measured: 9 days
Secondary outcomes (2)
The Safety of GAMMAPLEX at the Dosage Used in This Study.
The safety variables used to assess safety were the following:
- Adverse events
- The number and percent of infusions with at least 1 adverse event(AE) that occurs during an infusion or within 72 hours after the infusion stops
- Nature, severity, and frequency of AEs
- Suspected unexpected serious adverse reactions (SUSARs)
- Vital signs
- Clinical laboratory tests and Direct Coombs' Test
- Transmission of viruses
- Physical examination
Measured: AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90.
Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L.
Blood samples were collected to measure platelet counts and the duration of time for which the platelet count remained ≥50 x 10^9/L was measured.
Measured: Days 1, 2, 3, 5, 9, 14, 21, 32.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2007 (estimated)
- Study startSep 2007
- Primary completionAug 2011
- Study completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Idiopathic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bio Products Laboratory
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tim J Aldwinckle, MD, Principal investigator, Bio Products Laboratory
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00504075
- Sponsor's protocol ID
- GMX02
- Status last verified
- Feb 2013
- First submitted
- 18 Jul 2007
- Last updated
- 1 Mar 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Dash CH, Gillanders KR, Stratford Bobbitt ME, Gascoigne EW, Leach SJ. Safety and efficacy of Gammaplex(R) in idiopathic thrombocytopenic purpura (ClinicalTrials.gov--NCT00504075). PLoS One. 2014 Jun 3;9(6):e96600. doi: 10.1371/journal.pone.0096600. eCollection 2014.
PubMed 24892422
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Multicenter, Open-Label Study To Evaluate the Efficacy and Safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT00504075
- Acronym / sponsor ID
- GMX02
- Keywords
- Idiopathic Thrombocytopenic Purpura, Bleeding disorders, Immune System and Disorders
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Bio Products LaboratoryUniversity, hospital or other
- Study start
- Sep 2007
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 19 Jul 2007 (estimated)
- Results
- Posted 6 Jun 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To determine if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. Also to assess the safety of GAMMAPLEX and determine if platelet counts are maintained at 50 x 109/L in subjects with chronic ITP for.
Detailed description
The primary objective is to determine if BPL's GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. The secondary objectives are: 1) to determine the safety of GAMMAPLEX at the dosage used in this study. 2) to determine if GAMMAPLEX maintains platelet counts of ³ 50 x 109/L in subjects with chronic ITP for a period of time similar to that of a published data.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gammaplex (intravenous immunoglobulin) | Experimental | Biological: Gammaplex, intravenous immunoglobulin |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Males and females aged between 18 and 70 years.
- Confirmed diagnosis of chronic ITP of at least 6 months duration.
- Platelet count of less than or equal to 20 x 10 9/L at enrollment.
- Absence of other conditions that, in the opinion of the investigator, could cause thrombocytopenia.
- If subjects were treated with corticosteroids the treatment regimen/dose must have been stable (for a minimum of 2 weeks before screening). The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 6) If subjects were being treated with cyclophosphamide, azathioprine or attenuated androgens, the treatment regimen and dose must have been stable for a minimum of 2 months before Day 1. The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 7) Splenectomized subjects and both Rh(D)+ and Rh(D)- subjects may be included.
Key exclusion criteria (19 listed)
- A history of any severe or anaphylactic reaction to blood or any blood-derived product, or any severe reaction to IGIV or any other IgG preparation.
- Intolerance to any component of the investigational product.
- Received any live virus vaccine within the last 3 months prior to Day1.
- Received an IGIV preparation within 1 month prior to Day 1.
- Were currently receiving, or has received, any investigational agent within the 1 month prior to Day 1.
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 |
|---|---|---|
| Gammaplex, intravenous immunoglobulinAlso: GammaplexDosage form: Gammaplex® is a sterile liquid of 5 % w/v normal immunoglobulin. Gammaplex® contains 5 g/100 mL of human normal immunoglobulin (i.e. 50 g/L, of which virtually 100% is IgG). The first course of GAMMAPLEX will be administered as an intravenous infusion of 1 g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen may be administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX. | Biological | Gammaplex (intravenous immunoglobulin) |
Primary outcome
The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L.
Time frame: 9 days
The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10^9/L by Day 9.
2 secondary outcomes
The Safety of GAMMAPLEX at the Dosage Used in This Study.
Time frame: AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90.
Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L.
Time frame: Days 1, 2, 3, 5, 9, 14, 21, 32.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 24892422Dash CH, Gillanders KR, Stratford Bobbitt ME, Gascoigne EW, Leach SJ. Safety and efficacy of Gammaplex(R) in idiopathic thrombocytopenic purpura (ClinicalTrials.gov--NCT00504075). PLoS One. 2014 Jun 3;9(6):e96600. doi: 10.1371/journal.pone.0096600. eCollection 2014.
- Sponsor websiteLink provided by the sponsor.
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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.
Tim J Aldwinckle, MD principal investigator
Bio Products Laboratory
30 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Gammaplex, intravenous immunoglobulin in people with Chronic Idiopathic Thrombocytopenic Purpura, run by Bio Products Laboratory at 30 sites in 3 countries.
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 7 inclusion and 19 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 1 Mar 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in India, Argentina, United States. 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 6 Jun 2012). 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?
The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L. (time frame: 9 days). 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 NCT00504075, 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 1 Mar 2013.