Immune ThrombocytopeniaPhase 3NCT02649504

Rituximab and Dexamethasone Followed by Mycophenolate Mofetil or Placebo in Patients With Immune Thrombocytopenia

A Phase 3 study testing Rituximab, Dexamethasone and Mycophenolate mofetil in people with Immune Thrombocytopenia, run by Weill Medical College of Cornell University.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Immune Thrombocytopenia
What is being tested?: Rituximab, Dexamethasone and Mycophenolate mofetil
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 0 participants
Where: No sites listed yet
Study dates: 1 Dec 2016to 1 Dec 2016
Phase: Phase 3
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male or female, greater than 18 years old
Signed written informed consent obtained prior to study entry
Immune Thrombocytopenia according to the American Society of Hematology guidelines, with other potential causes of thrombocytopenia excluded, platelets less than 30 x 109/L at study entry
Have adequate renal and hepatic function (creatinine and bilirubin less than 2 x Upper Limit of Normal (ULN), aspartate aminotransferase and alanine aminotransferase less than 3 x ULN)
No significant cytopenias (hemoglobin greater than10 g/dL without ongoing transfusional support, absolute neutrophil count greater than 1.0 x109/L at study entry).
If on corticosteroids, receiving less than or equal to 20 mg/day prednisone (or equivalent) with no change of dose for at least 2 weeks prior to study entry. No more than two 4-day courses of Dexamethasone prior to study entry. If on danazol, no change of dose for at least 2 weeks prior to study entry. (Thrombopoietin receptor agonist treatment will be allowed until the end of 8th week on study if started prior to study entry)

Show all 19Show fewer

No treatment with any known non-marketed drug substance or experimental therapy within 5 terminal half-lives or 4 weeks prior to enrollment, whichever is longer, or currently participating in any other interventional clinical study
No prior treatment with anti-B-lymphocyte antigen cluster of differentiation antigen 20 monoclonal antibody at a total dose greater than 375mg/m2
Screen for Hepatitis B and be negative for Hepatitis B surface antigen
Human Immunodeficiency Virus negative
No history of active hepatitis C infection or a past history hepatitis C (confirmed by negative anti- Hepatitis C Virus Antibody)
No past history of tuberculosis
No chronic or current infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment such as, but not limited to, chronic renal infection, or chronic chest infection with bronchiectasis
No current active hepatic or biliary disease (with exception of patients with Gilbert's syndrome, asymptomatic gallstones, or stable chronic liver disease meeting laboratory threshold limits per investigator discretion)
No significant concurrent, uncontrolled medical condition including but not limited to, renal, hepatic, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, which in the opinion of the investigator may represent a risk for the patient. Have no past or current malignancy for 3 years (excluding nonmelanoma skin cancers, e.g., basal cell carcinoma (BCC) and carcinoma in situ of the cervix)
Women of childbearing potential must have a negative pregnancy test at screening
No history of significant cerebrovascular disease in the past 6 months or ongoing event with active symptoms or sequelae.
No history of venous or arterial thromboembolism in the past year.
No clinically significant cardiac disease including unstable angina, acute myocardial infarction within six months prior to randomization, congestive heart failure (NYHA III-IV), and arrhythmia unless controlled by therapy, with the exception of extra systoles or minor conduction abnormalities

Who cannot join

Pregnant or lactating women
Women of childbearing potential and fertile men who are not practicing or who are unwilling to practice birth control while enrolled in the study or until at least 6 months after treatment. Women of childbearing potential must have a negative pregnancy test at screening. Women of childbearing potential, including women whose last menstrual period was less than one year prior to screening, unable or unwilling to use adequate contraception from study start to one year after the last dose of protocol therapy will be excluded from study. Adequate contraception is defined as intrauterine device, tubal sterilization, patient's partner had a vasectomy or total abstinence. Other contraceptive methods include hormonal contraceptive pills. Male subjects unable or unwilling to use adequate contraception methods from study start to one year after the last dose of protocol therapy will be excluded from the study as well.
Immunosuppressive agents or ITP treatments or other experimental treatments for ITP within 2 weeks. Intravenous Immunoglobulin, anti-Rh(D) prior to first day of rituximab treatment
Splenectomy or any other surgery within 4 weeks
Red blood cell transfusion or hospitalization for bleeding within 4 weeks
Concomitant anticoagulation therapy (warfarin, enoxaparin, dabigatran, rivaroxaban, apixaban, fondaparinux) or any platelet aggregation inhibitors including aspirin and clopidogrel (until platelets stable greater than 100x109/L)
New York Heart Classification III or IV heart disease. Other severe cardiovascular or cardiopulmonary disease, including chronic obstructive pulmonary disease
Other concurrent medical or psychiatric conditions that, in the Investigator's opinion, may be likely to confound study interpretation or prevent completion of study procedures and follow-up examinations

About this study

Official title: The Study of Immunotherapy With Rituximab and Pulse Dexamethasone Followed by With Mycophenolate Mofetil or Placebo in Adult Patients With Persistent and Chronic Immune Thrombocytopenia

The investigators will attempt to further increase the cure rate of ITP with medical therapy by providing maintenance therapy with Mycophenolate mofetil (MMF) to persistent/chronic ITP patients after treating them with induction therapy combining rituximab and dexamethasone. The investigators will randomly assign patients to MMF versus placebo in order to demonstrate safety (e.g., for infectious risk) and efficacy (platelet counts stably >50x109/L more than 1 year off therapy).

Read the detailed description

There are significant numbers of Immune Thrombocytopenia (ITP) patients 18 years of age or older who relapse and become refractory after short responses to initial therapy (usually corticosteroids) or who do not respond at all. Despite a variety of available therapies, there are patients with persistently low platelet counts and bleeding complications, and there are other patients who suffer from the side effects of current treatments. Thus, there still remains an unmet need for better treatments in these patients. Based on the investigators' experience, it is expected that most of ITP patients treated with intensive induction therapy (Dexamethasone combined with rituximab, R+3D) will increase their platelet counts, and the investigators hope that a further 6-month course of Mycophenolate Mofetil will help patients to maintain continuous response and even achieve a cure of ITP. The increase in platelet count will likely result in a decreased risk for bleeding and better health-related quality of life. In addition, responders potentially will be able to stop concomitant ITP medications and will not suffer from adverse events of various ITP therapies and will avoid undergoing splenectomy.

This multi-center, randomized study will help physicians determine the best treatment option for ITP patients and may help to establish a new standard of care.

The knowledge to be gained is that of:

whether R+3D and placebo confirms the previous data on R+3D
whether R+3D + MMF increases the cure rate of ITP
whether R+3D + MMF increases the risk of serious infections

The investigators do not believe that the triple therapy will result in a high rate of serious infections based on the past track record of R+3D and of MMF in combination with other stronger immunosuppressive medications such as cyclosporine and tacrolimus. Therefore the risk will be limited and worth the increase in cure rate that we expect.

Conditions: Immune Thrombocytopenia
Being tested: Rituximab, Dexamethasone, Mycophenolate mofetil, Placebo
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.

RituximabDrug
Also called anti- cluster of differentiation antigen 20 antibody
Rituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study
DexamethasoneDrug
Also called Decadron, Dexasone, Diodex, Hexadrol, Maxidex
Dexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study.
Mycophenolate mofetilDrug
Also called Cellcept
Patients allocated to the active drug arm will be given Mycophenolate mofetil starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.
PlaceboDrug
Also called inactive drug, fake pill
Patients allocated to the placebo arm will be given placebo starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.

Study groups in NCT02649504
Study group	Role	What they receive
R+3D, MMF	Active comparator	Rituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study. Dexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study. Mycophenolate mofetil (MMF) will be given starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks. Receives: Drug: Rituximab; Drug: Dexamethasone; Drug: Mycophenolate mofetil
R+3D, Placebo	Placebo comparator	Rituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study. Dexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study. Placebo will be given starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks. Receives: Drug: Rituximab; Drug: Dexamethasone; Drug: Placebo

What is being measured

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

Number of patients with a platelet count greater or equal to 50,000 for 5 of 6 platelet counts in the second half of second year (month 19 to month 24) with no rescue therapy
Measured: 2 years

Timeline

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

1 Dec 20161 Dec 2016

First posted7 Jan 2016 (estimated)
Study start1 Dec 2016 (estimated)
Primary completion1 Dec 2016 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
More trials run by Weill Medical College of Cornell 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 3
Primary purpose: Treatment
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: NCT02649504
Sponsor's protocol ID: 1501015858
Status last verified: Aug 2018
First submitted: 17 Dec 2015
Last updated: 14 Aug 2018
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Bussel JB, Lee CS, Seery C, Imahiyerobo AA, Thompson MV, Catellier D, Turenne IG, Patel VL, Basciano PA, Elstrom RL, Ghanima W. Rituximab and three dexamethasone cycles provide responses similar to splenectomy in women and those with immune thrombocytopenia of less than two years duration. Haematologica. 2014 Jul;99(7):1264-71. doi: 10.3324/haematol.2013.103291. Epub 2014 Apr 18.
PubMed 24747949
Stasi R, Pagano A, Stipa E, Amadori S. Rituximab chimeric anti-CD20 monoclonal antibody treatment for adults with chronic idiopathic thrombocytopenic purpura. Blood. 2001 Aug 15;98(4):952-7. doi: 10.1182/blood.v98.4.952.
PubMed 11493438
Stasi R, Stipa E, Forte V, Meo P, Amadori S. Variable patterns of response to rituximab treatment in adults with chronic idiopathic thrombocytopenic purpura. Blood. 2002 May 15;99(10):3872-3. doi: 10.1182/blood-2002-02-0392. No abstract available.
PubMed 12014370
Patel VL, Mahevas M, Lee SY, Stasi R, Cunningham-Rundles S, Godeau B, Kanter J, Neufeld E, Taube T, Ramenghi U, Shenoy S, Ward MJ, Mihatov N, Patel VL, Bierling P, Lesser M, Cooper N, Bussel JB. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012 Jun 21;119(25):5989-95. doi: 10.1182/blood-2011-11-393975. Epub 2012 May 7.
PubMed 22566601
Zaja F, Baccarani M, Mazza P, Bocchia M, Gugliotta L, Zaccaria A, Vianelli N, Defina M, Tieghi A, Amadori S, Campagna S, Ferrara F, Angelucci E, Usala E, Cantoni S, Visani G, Fornaro A, Rizzi R, De Stefano V, Casulli F, Battista ML, Isola M, Soldano F, Gamba E, Fanin R. Dexamethasone plus rituximab yields higher sustained response rates than dexamethasone monotherapy in adults with primary immune thrombocytopenia. Blood. 2010 Apr 8;115(14):2755-62. doi: 10.1182/blood-2009-07-229815. Epub 2010 Feb 3.
PubMed 20130241

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

All Immune Thrombocytopenia trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: The Study of Immunotherapy With Rituximab and Pulse Dexamethasone Followed by With Mycophenolate Mofetil or Placebo in Adult Patients With Persistent and Chronic Immune Thrombocytopenia
ClinicalTrials.gov ID: NCT02649504
Acronym / sponsor ID: 1501015858
Conditions: Immune Thrombocytopenia
Phase: Phase 3
Study type: Interventional
Recruitment status: Withdrawn
Enrolment: 0 participants (actual)
Lead sponsor: Weill Medical College of Cornell UniversityUniversity, hospital or other
Study start: 1 Dec 2016 (estimated)
Primary completion: 1 Dec 2016 (estimated)
First posted: 7 Jan 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.

Detailed description

This multi-center, randomized study will help physicians determine the best treatment option for ITP patients and may help to establish a new standard of care.

The knowledge to be gained is that of:

whether R+3D and placebo confirms the previous data on R+3D
whether R+3D + MMF increases the cure rate of ITP
whether R+3D + MMF increases the risk of serious infections

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
R+3D, MMF	Active comparator	Drug: Rituximab, Drug: Dexamethasone, Drug: Mycophenolate mofetilRituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study. Dexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study. Mycophenolate mofetil (MMF) will be given starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.
R+3D, Placebo	Placebo comparator	Drug: Rituximab, Drug: Dexamethasone, Drug: PlaceboRituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study. Dexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study. Placebo will be given starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (19 listed)

Male or female, greater than 18 years old
Signed written informed consent obtained prior to study entry
Immune Thrombocytopenia according to the American Society of Hematology guidelines, with other potential causes of thrombocytopenia excluded, platelets less than 30 x 109/L at study entry
Have adequate renal and hepatic function (creatinine and bilirubin less than 2 x Upper Limit of Normal (ULN), aspartate aminotransferase and alanine aminotransferase less than 3 x ULN)
No significant cytopenias (hemoglobin greater than10 g/dL without ongoing transfusional support, absolute neutrophil count greater than 1.0 x109/L at study entry).

Key exclusion criteria (8 listed)

Pregnant or lactating women
Women of childbearing potential and fertile men who are not practicing or who are unwilling to practice birth control while enrolled in the study or until at least 6 months after treatment. Women of childbearing potential must have a negative pregnancy test at screening. Women of childbearing potential, including women whose last menstrual period was less than one year prior to screening, unable or unwilling to use adequate contraception from study start to one year after the last dose of protocol therapy will be excluded from study. Adequate contraception is defined as intrauterine device, tubal sterilization, patient's partner had a vasectomy or total abstinence. Other contraceptive methods include hormonal contraceptive pills. Male subjects unable or unwilling to use adequate contraception methods from study start to one year after the last dose of protocol therapy will be excluded from the study as well.
Immunosuppressive agents or ITP treatments or other experimental treatments for ITP within 2 weeks. Intravenous Immunoglobulin, anti-Rh(D) prior to first day of rituximab treatment
Splenectomy or any other surgery within 4 weeks
Red blood cell transfusion or hospitalization for bleeding within 4 weeks

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
RituximabAlso: anti- cluster of differentiation antigen 20 antibodyRituximab will be given at the standard dose of 375mg/m2 on days 1, 8, 15, 22 of the study	Drug	R+3D, MMF, R+3D, Placebo
DexamethasoneAlso: Decadron, Dexasone, Diodex, Hexadrol, MaxidexDexamethasone will be given at dose 40 mg/day on days 1-4, 15-18, and 29-32 (+/- 3 days) of the study.	Drug	R+3D, MMF, R+3D, Placebo
Mycophenolate mofetilAlso: CellceptPatients allocated to the active drug arm will be given Mycophenolate mofetil starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.	Drug	R+3D, MMF
PlaceboAlso: inactive drug, fake pillPatients allocated to the placebo arm will be given placebo starting on day 33, 500 mg twice daily for the first week and then escalation to 1000 mg twice daily for 24 weeks.	Drug	R+3D, Placebo

Primary outcome

Number of patients with a platelet count greater or equal to 50,000 for 5 of 6 platelet counts in the second half of second year (month 19 to month 24) with no rescue therapy
Time frame: 2 years

Results and safety

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

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2018.
Background: PubMed 24747949Bussel JB, Lee CS, Seery C, Imahiyerobo AA, Thompson MV, Catellier D, Turenne IG, Patel VL, Basciano PA, Elstrom RL, Ghanima W. Rituximab and three dexamethasone cycles provide responses similar to splenectomy in women and those with immune thrombocytopenia of less than two years duration. Haematologica. 2014 Jul;99(7):1264-71. doi: 10.3324/haematol.2013.103291. Epub 2014 Apr 18.
Background: PubMed 11493438Stasi R, Pagano A, Stipa E, Amadori S. Rituximab chimeric anti-CD20 monoclonal antibody treatment for adults with chronic idiopathic thrombocytopenic purpura. Blood. 2001 Aug 15;98(4):952-7. doi: 10.1182/blood.v98.4.952.
Background: PubMed 12014370Stasi R, Stipa E, Forte V, Meo P, Amadori S. Variable patterns of response to rituximab treatment in adults with chronic idiopathic thrombocytopenic purpura. Blood. 2002 May 15;99(10):3872-3. doi: 10.1182/blood-2002-02-0392. No abstract available.
Background: PubMed 22566601Patel VL, Mahevas M, Lee SY, Stasi R, Cunningham-Rundles S, Godeau B, Kanter J, Neufeld E, Taube T, Ramenghi U, Shenoy S, Ward MJ, Mihatov N, Patel VL, Bierling P, Lesser M, Cooper N, Bussel JB. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012 Jun 21;119(25):5989-95. doi: 10.1182/blood-2011-11-393975. Epub 2012 May 7.
Background: PubMed 20130241Zaja F, Baccarani M, Mazza P, Bocchia M, Gugliotta L, Zaccaria A, Vianelli N, Defina M, Tieghi A, Amadori S, Campagna S, Ferrara F, Angelucci E, Usala E, Cantoni S, Visani G, Fornaro A, Rizzi R, De Stefano V, Casulli F, Battista ML, Isola M, Soldano F, Gamba E, Fanin R. Dexamethasone plus rituximab yields higher sustained response rates than dexamethasone monotherapy in adults with primary immune thrombocytopenia. Blood. 2010 Apr 8;115(14):2755-62. doi: 10.1182/blood-2009-07-229815. Epub 2010 Feb 3.

Related trials for Immune Thrombocytopenia

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

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.

James B. Bussel, M.D principal investigator

Weill Medical College of Cornell University

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Rituximab, Dexamethasone and Mycophenolate mofetil in people with Immune Thrombocytopenia, run by Weill Medical College of Cornell University.

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 19 inclusion and 8 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 14 Aug 2018. 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 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?

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?

Number of patients with a platelet count greater or equal to 50,000 for 5 of 6 platelet counts in the second half of second year (month 19 to month 24) with no rescue therapy (time frame: 2 years). 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 NCT02649504, which also lists 5 linked publications.

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 14 Aug 2018.