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Purpura, Thrombocytopaenic, IdiopathicPhase 2NCT01440361

A Study to Investigate Belimumab for the Treatment of Chronic Immune Thrombocytopenia.

A Phase 2 study testing Belimumab in people with Purpura, Thrombocytopaenic, Idiopathic, run by GlaxoSmithKline.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

Chronic immune thrombocytopenia (ITP) is a longterm disease in which the blood does not clot normally. This is due to a low number of blood cell fragments called platelets. Platelets clot to seal small cuts or breaks on blood vessel walls and stop bleeding. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Purpura, Thrombocytopaenic, Idiopathic
What is being tested?
Belimumab
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
No sites listed yet
Study dates
Mar 2013to Apr 2015
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 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female,18-75 years old
  • Chronic ITP for a minimum of 6 months with a platelet count <75,000/uL at screening and a platelet count <75,000/uL 2 to 6 months before screening
  • Stable either on no treatment or on a stable dose of corticosteroids (10 milligrams(mg)/day prednisone or prednisone equivalent or less) and/or azathioprine (100mg/day or less) for a minimum of 30 days before screening
  • Single QTc <450 milliseconds (msec); or QTc <480 msec in subjects with Bundle Branch Block
  • A female subject is eligible to participate if she is not pregnant or nursing and at least one of the following conditions apply: a. Non-childbearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea. b.Child-bearing potential and agrees to use one of the contraception methods listed in the protocol

Who cannot join

  • Diagnosis of ITP is secondary to other conditions
  • Treated with any B cell targeted therapy at any time
  • Have received any of the following within 364 days prior to Day 0: Abatacept, A biologic investigational agent other than B cell targeted therapy
  • Have received any of the following within 180 days prior to Day 0: Intravenous (IV) cyclophosphamide, 3 or more courses of systemic corticosteroids for concomitant conditions
  • Have received any of the following within 90 days prior to Day 0: High dose corticosteroid for treatment of ITP, Splenectomy, plasmapheresis
  • Have received any of the following within 60 days, 5 half-lives or twice the duration of the biological effect of belimumab before Day 0: A non-biologic investigational agent, any other immunosuppressive/immunomodulatory agent with the exception of azathioprine and corticosteroids, Eltrombopag, romiplostim, any steroid injection
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  • Have received any of the following within 30 days before Screening: Intravenous immunoglobulin, Corticosteroids greater than 10mg/day (prednisone or prednisone equivalent) or azathioprine more than 100 mg/day, Changes to corticosteroid or azathioprine therapy
  • Have received a live vaccine within 30 days before Day 0
  • Subject could be at risk of haemorrhage that threatens a vital organ
  • History of a major organ transplant or hematopoietic stem cell/marrow transplant
  • History of malignant neoplasm within the last 5 years, except for adequately treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the uterine cervix
  • Required management of infections, as follows: Currently on any suppressive therapy for a chronic infection, Hospitalisation for treatment of infection within 60 days before Day 0, Use of parenteral antibiotics within 60 days before Day 0
  • Significant unstable or uncontrolled acute or chronic diseases not due to ITP or planned surgical procedure or a history of any other medical disease, laboratory abnormality, or condition that makes the subject unsuitable for the study
  • Positive screening Hepatitis C antibody result or Hepatitis B (HB) infection
  • Positive test for Human Immunodeficiency Virus (HIV) antibody at screening or historically
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) greater or equal to 2x upper limit of normal (ULN); alkaline phosphatase and bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin is <35%)
  • IgA deficiency (IgA <10mg/dL)
  • Abnormal lab results
  • Lymphocyte count <500/mm3
  • History of sensitivity to any of the study medications, or components thereof or a history of drug or other allergy including a previous anaphylactic reaction to parenteral administration contrast agents, human or murine proteins or monoclonal antibodies
  • Evidence of serious suicide risk
  • Current drug or alcohol abuse or dependence
  • Where participation in the study would result in donation of blood or blood products >500 mL within a 56 day period

About this study

Official title: A Clinical and Mechanistic Proof of Efficacy Study With Belimumab in Chronic Immune Thrombocytopenia (ITP) Patients.

Chronic immune thrombocytopenia (ITP) is a longterm disease in which the blood does not clot normally. This is due to a low number of blood cell fragments called platelets. Platelets clot to seal small cuts or breaks on blood vessel walls and stop bleeding. Normally the immune system makes proteins called antibodies to fight off harmful substances that enter the body. In ITP, the immune system produces antibodies that attack and destroy the body's platelets by mistake.

Patients can suffer from bleeding under the skin, nosebleeds, blood in urine or stools and in very severe cases bleeding in the brain. Patients have an increased frequency of death from bleeding complications compared to normal.

Read more

Chronic ITP is fairly rare , with an incidence of 32 new cases/million people each year.

Existing treatments work by lowering the activity of the immune system or directly increasing platelet count. These treatments do not work effectively in all patients and can have side effects. We hope that understanding how belimumab works in ITP will help in the development of future treatments for ITP and other autoimmune diseases.

We will test the safety, blood levels and effects of the study medication in people with chronic ITP. Patients will receive the study medication intravenously (through a needle inserted into a vein) and blood samples will be taken before and on several occasions afterwards.

Up to 40 patients with chronic ITP, aged 18 to 75 will participate. Approximately 11 patients will take dummy medicine instead of the study medicine neither they or their study doctor will know which one they are given. Participants will take up to 57 weeks to finish the study. They'll make 12 outpatient visits.

The study will take place in hospitals in the UK. Other sites in mainland Europe may also be initiated.

A pharmaceutical company, GlaxoSmithKline, is funding the study.

Being tested
Belimumab, Normal saline placebo
Medical subject headings
Purpura

What participants receive

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

  • BelimumabDrug

    Also called Benlysta/LymphoStat-B

    10 mg /kg given as an intravenous infusion every 4 weeks for 24 weeks (with an additional dose at Week 2)

  • Normal saline placeboDrug

    Also called Saline

    Placebo given as an intravenous infusion every 4 weeks for 24 weeks (with an additional dose at Week 2)

Study groups in NCT01440361
Study groupRoleWhat they receive
BelimumabExperimentalReconstituted solution for intravenous infusion
Receives: Drug: Belimumab
Normal salinePlacebo comparatorSolution for intravenous infusion
Receives: Drug: Normal saline placebo

What is being measured

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

  1. Platelet count

    Change in platelet count

    Measured: Baseline, week 28

  2. Anti-platelet autoantibodies

    Change in anti-platelet autoantibodies

    Measured: Baseline, week 28

Secondary outcomes (14)

  1. Platelet count (time)

    Time to first response of platelet count increase >20,000/microlitre (μl) from baseline

    Measured: Baseline, week 0, 2, 4, 8, 12, 16, 20, 24, 28

  2. Platelet count (incidence)

    Incidence of response of platelet count increase >20,000/μL from baseline

    Measured: Baseline, week 28

  3. Platelet count (incidence of complete response)

    Incidence of complete response as defined by platelet count to >100,000

    Measured: Baseline, week 28

  4. Platelet count (incidence of doubling)

    Incidence of subjects with ≥2 times baseline platelet count

    Measured: Baseline, week 28

  5. Vital signs

    Change in vital signs outside normal range

    Measured: Baseline, week 0, 2, 4, 8, 12, 16, 20, 24, 28, 40

  6. Clinical chemistry and haematology

    Change in clinical chemistry and haematology

    Measured: Baseline, week 2, 4, 8, 12, 16, 20, 24, 28, 40

  7. Immunogenicity

    Change in immunogenicity

    Measured: Baseline, week 12, 28, 40, 52

  8. Serum concentrations of belimumab

    Change in serum concentrations of belimumab

    Measured: Baseline, week 2, 8, 24, 28, 40, 52

  9. Serum and/or platelet bound anti-platelet antibodies

    Change in serum and/or platelet bound anti-platelet antibodies

    Measured: Baseline, week 4, 8, 12, 16, 20, 24, 28, 40, 52

  10. B cell and T cell sub-populations and B lymphocyte stimulator (BLyS) receptor

    Change in B cell and T cell sub-populations and BLyS receptor

    Measured: Baseline, week 4, 8, 16, 24, 40, 52

  11. Antigen-specific B cells and T cells

    Change in antigen-specific B cells and T cells

    Measured: Baseline, week 8, 16, 24, 40

  12. Serum cytokine/chemokine profile

    Change in serum cytokine/chemokine profile

    Measured: Baseline, week 8, 16, 24, 28, 40

  13. Transcriptome profile

    Change in transcriptome profile

    Measured: Baseline, week 8, 28

  14. Autoantibody profile

    Change in autoantibody profile

    Measured: Baseline, week 28

Timeline

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

  1. First posted26 Sep 2011 (estimated)
  2. Study startMar 2013
  3. Primary completionApr 2015 (estimated)
  4. Study completionApr 2015 (estimated)

Why it stopped: This study was Cancelled Before Active

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 2
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01440361
Sponsor's protocol ID
114870
Status last verified
Jul 2014
First submitted
22 Sep 2011
Last updated
28 Jul 2014
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Belimumab in people with Purpura, Thrombocytopaenic, Idiopathic, run by GlaxoSmithKline.

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 23 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: withdrawn, as of the sponsor's last update on 28 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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?

The study was withdrawn before enrolling participants, so there are no results.

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?

Platelet count (time frame: Baseline, week 28). 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?

On the ClinicalTrials.gov record for NCT01440361.

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 28 Jul 2014.

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