Alloimmune ThrombocytopeniaPhase 2/3NCT00194987

A Trial of Antenatal Treatment of Alloimmune Thrombocytopenia

A Phase 2/3 study testing IVIG (Intravenous Immunoglobulin) and prednisone in people with Alloimmune Thrombocytopenia and Fetal Alloimmune Thrombocytopenia, run by Weill Medical College of Cornell University at 1 site in United States.

Completed

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

Official record

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Trial summary

The purposes of this study are to provide medical management by giving treatment to the mother that will bring up the fetal platelet count and to minimize the number of invasive procedures to the fetus (which may result in serious fetal injuries). Read more about this study

Who is it for?: Ages 18 to 50 years, women only
Conditions the study is for: Alloimmune Thrombocytopenia, Fetal Alloimmune Thrombocytopenia
What is being tested?: IVIG (Intravenous Immunoglobulin) and prednisone
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 102 participants
Where: 1 siteUnited States
Study dates: Apr 2001to 30 Jun 2015
Phase: Phase 2/3
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 50 years
Women only
No healthy volunteers
Adult

Who may be able to join

Pregnant women are eligible for inclusion into the Very High Risk Group if they:
are PLA-1 (platelet antigen A1) negative and have known platelet incompatibility with their fetus
have had a previous child who suffered an antenatal hemorrhage prior to 28 weeks gestation (as best as could be estimated)
are less than 19 weeks gestation
Pregnant women are eligible for inclusion into the High Risk Group if they:
are PLA-1 negative and have known platelet incompatibility with the fetus

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have had a previous child who suffered an antenatal hemorrhage after 28 weeks gestation (as best as could be estimated)
are between 12-30 weeks gestation
Pregnant women are eligible for inclusion into the Standard Risk Group if they:
are PLA-1 negative and have known platelet incompatibility with the fetus
have not had a previous child who suffered an antenatal hemorrhage
are between 20-30 weeks gestation

Who cannot join

Women are not eligible for inclusion into the Very High Risk Group if they:
have had a previous child who suffered an antenatal hemorrhage after 28 weeks gestation
are greater than 19 weeks gestation
Women are not eligible for inclusion into the High Risk Group if they:
have had a previous child who suffered an antenatal hemorrhage prior to 28 weeks gestation
are greater than 30 weeks gestation

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Women are not eligible for inclusion into the Standard Risk Group if they:
have had a previous child who suffered an antenatal hemorrhage
are greater than 30 weeks gestation

Trial locations and contacts

1 site in 1 country.

United States1 site

New York Presbyterian Hospital-Weill Cornell Medical Center
New York, New York, 10021

Other trials in United States

About this study

Official title: A Randomized Multicenter Trial of Antenatal Treatment of Alloimmune Thrombocytopenia

Conditions: Alloimmune Thrombocytopenia, Fetal Alloimmune Thrombocytopenia
Being tested: IVIG (Intravenous Immunoglobulin), prednisone
Keywords: Alloimmune thrombocytopenia, Fetal alloimmune thrombocytopenia

What participants receive

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

IVIG (Intravenous Immunoglobulin)Drug
one gram per kg of IVIG per infusion given either once or twice
prednisoneDrug
in arm with IVIG 1 gm/kg/wk will also receive prednisone 0.5 mg/kg/day

Study groups in NCT00194987
Study group	Role	What they receive
IVIG x 2	Experimental	IVIG 2 g/kg/wk divided into 2 infusions per week for women with a fetus affected by FNAIT Receives: Drug: IVIG (Intravenous Immunoglobulin)
IVIG x 1 + prednisone	Experimental	IVIG 1 g/kg/wk in 1 infusion per week + prednisone 0.5 mg po daily for women with a fetus affected by FNAIT Receives: Drug: IVIG (Intravenous Immunoglobulin); Drug: prednisone

What is being measured

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

Number of Newborns With a Birth Platelet Count > 50,000/uL
this uses the birth platelet count of the fetuses from the study when they are born
Measured: 32-40 weeks (the endpoint is the birth which is not at the same number of weeks for all of the babies. This is why the weeks are not listed specifically eg week 40

Secondary outcomes (2)

Intracranial Hemorrhage: Number Occurring in Fetuses and Newborns of Mothers in Study
number of ICH assessed by fetal and neonatal ultrasound with MRI back up most commonly in utero so range from 20-40 weeks fo gestation
Measured: time of ICH (range 20-40 wks)
Number of Fetal Platelet Counts > 50,000/uL
Number of Fetal Platelet Counts > 50,000/uL Among Those Who Underwent Fetal Blood Sampling and Had a Fetal Platelet Count Determined
Measured: 32 +/- 2 weeks

Timeline

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

Apr 200130 Jun 2015

First posted19 Sep 2005 (estimated)
Study startApr 2001
Primary completion30 Jun 2015
Study completion30 Jun 2015

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 (2)Papers the sponsor has linked to this study.
Other trials for Alloimmune 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 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: randomization between 2 groups
Masking: None (open label)
Enrolment: 102 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00194987
Sponsor's protocol ID: 0102004801
Status last verified: Oct 2018
First submitted: 13 Sep 2005
Last updated: 7 Nov 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Lakkaraja M, Jin JC, Manotas KC, Vinograd CA, Ferd P, Gabor J, Wissert M, Berkowitz RL, McFarland JG, Bussel JB. Blood group A mothers are more likely to develop anemia during antenatal intravenous immunoglobulin treatment of fetal and neonatal alloimmune thrombocytopenia. Transfusion. 2016 Oct;56(10):2449-2454. doi: 10.1111/trf.13779. Epub 2016 Sep 9.
PubMed 27611703
Berkowitz RL, Lesser ML, McFarland JG, Wissert M, Primiani A, Hung C, Bussel JB. Antepartum treatment without early cordocentesis for standard-risk alloimmune thrombocytopenia: a randomized controlled trial. Obstet Gynecol. 2007 Aug;110(2 Pt 1):249-55. doi: 10.1097/01.AOG.0000270302.80336.dd.
PubMed 17666597

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?

See locations

RecruitingPhase 3
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
RecruitingPhase 3
A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
Not yet recruitingObservational study
Neonatal Thrombocytopenia in the NICU
Thrombocytopenia, Neonatal Thrombocytopenia
RecruitingEarly Phase 1
An Clinical Study of CD19 CAR NK Cells for the Treatment of Refractory Primary Immune Thrombocytopenia
Thrombocytopenia Alloimmune

All Alloimmune Thrombocytopenia trials

Study at a glance

From the registry record, last updated by the sponsor on 7 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized Multicenter Trial of Antenatal Treatment of Alloimmune Thrombocytopenia
ClinicalTrials.gov ID: NCT00194987
Acronym / sponsor ID: 0102004801
Conditions: Alloimmune Thrombocytopenia, Fetal Alloimmune Thrombocytopenia
Keywords: Alloimmune thrombocytopenia, Fetal alloimmune thrombocytopenia
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Completed
Enrolment: 102 participants (actual)
Lead sponsor: Weill Medical College of Cornell UniversityUniversity, hospital or other
Collaborators: New York Presbyterian Hospital, Columbia University
Study start: Apr 2001
Primary completion: 30 Jun 2015 (actual)
Study completion: 30 Jun 2015 (actual)
First posted: 19 Sep 2005 (estimated)
Results: Posted 7 Nov 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2
Model description: randomization between 2 groups

Arm	Type	Receives
IVIG x 2	Experimental	Drug: IVIG (Intravenous Immunoglobulin)IVIG 2 g/kg/wk divided into 2 infusions per week for women with a fetus affected by FNAIT
IVIG x 1 + prednisone	Experimental	Drug: IVIG (Intravenous Immunoglobulin), Drug: prednisoneIVIG 1 g/kg/wk in 1 infusion per week + prednisone 0.5 mg po daily for women with a fetus affected by FNAIT

Who may qualify

Ages: 18 to 50 years
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (12 listed)

are PLA-1 (platelet antigen A1) negative and have known platelet incompatibility with their fetus
have had a previous child who suffered an antenatal hemorrhage prior to 28 weeks gestation (as best as could be estimated)
are less than 19 weeks gestation
are PLA-1 negative and have known platelet incompatibility with the fetus
have had a previous child who suffered an antenatal hemorrhage after 28 weeks gestation (as best as could be estimated)

Key exclusion criteria (9 listed)

have had a previous child who suffered an antenatal hemorrhage after 28 weeks gestation
are greater than 19 weeks gestation
have had a previous child who suffered an antenatal hemorrhage prior to 28 weeks gestation
are greater than 30 weeks gestation
have had a previous child who suffered an antenatal hemorrhage

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 (Intravenous Immunoglobulin)one gram per kg of IVIG per infusion given either once or twice	Drug	IVIG x 1 + prednisone, IVIG x 2
prednisonein arm with IVIG 1 gm/kg/wk will also receive prednisone 0.5 mg/kg/day	Drug	IVIG x 1 + prednisone

Primary outcome

Number of Newborns With a Birth Platelet Count > 50,000/uL
Time frame: 32-40 weeks (the endpoint is the birth which is not at the same number of weeks for all of the babies. This is why the weeks are not listed specifically eg week 40
this uses the birth platelet count of the fetuses from the study when they are born

2 secondary outcomes

Intracranial Hemorrhage: Number Occurring in Fetuses and Newborns of Mothers in Study
Time frame: time of ICH (range 20-40 wks)
Number of Fetal Platelet Counts > 50,000/uL
Time frame: 32 +/- 2 weeks

Results and safety

Results posted to the registry on 7 Nov 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Intravenous Gammglobulin (IVIG) 1g/kg Twice Per Week	51	51
IVIG 1g/k/Week and Prednisone 0.5 mg/kg/Day	51	48

Primary outcome: Number of Newborns With a Birth Platelet Count > 50,000/uL

Time frame: 32-40 weeks (the endpoint is the birth which is not at the same number of weeks for all of the babies. This is why the weeks are not listed specifically eg week 40. Number. Unit: number of newborns with >50,000 pets

Measure	IVIG 1g/kg Twice Per Week n = 51	IVIG 1g/k/Week and Prednisone 0.5 mg/kg/Day n = 51
Value	48	47

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: collected for study women from 20 weeks of gestation until 2 weeks after birth of newborn fetus and newborns were collected for the same period of time. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
IVIG X 2 Weekly	14 of 51 (27.5%)	12 of 51 (23.5%)	0 of 51
IVIG 1 g/kg/wk + Prednisone	12 of 51 (23.5%)	8 of 51 (15.7%)	0 of 51
IVIG X 2 Weekly--fetuses/Newborns	4 of 51 (7.8%)	0 of 51 (0%)	0 of 51
IVIG x 1 + Prednisone---fetuses/Newborns	3 of 51 (5.9%)	0 of 51 (0%)	0 of 51

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 27611703Lakkaraja M, Jin JC, Manotas KC, Vinograd CA, Ferd P, Gabor J, Wissert M, Berkowitz RL, McFarland JG, Bussel JB. Blood group A mothers are more likely to develop anemia during antenatal intravenous immunoglobulin treatment of fetal and neonatal alloimmune thrombocytopenia. Transfusion. 2016 Oct;56(10):2449-2454. doi: 10.1111/trf.13779. Epub 2016 Sep 9.
Derived from this study: PubMed 17666597Berkowitz RL, Lesser ML, McFarland JG, Wissert M, Primiani A, Hung C, Bussel JB. Antepartum treatment without early cordocentesis for standard-risk alloimmune thrombocytopenia: a randomized controlled trial. Obstet Gynecol. 2007 Aug;110(2 Pt 1):249-55. doi: 10.1097/01.AOG.0000270302.80336.dd.

Related trials for Alloimmune Thrombocytopenia

RecruitingPhase 3
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
RecruitingPhase 3
A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
Not yet recruitingObservational study
Neonatal Thrombocytopenia in the NICU
Thrombocytopenia, Neonatal Thrombocytopenia
RecruitingEarly Phase 1
An Clinical Study of CD19 CAR NK Cells for the Treatment of Refractory Primary Immune Thrombocytopenia
Thrombocytopenia Alloimmune

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

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 12 inclusion and 9 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 Nov 2018. 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 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 combined 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.

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 Nov 2018). 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?

Number of Newborns With a Birth Platelet Count > 50,000/uL (time frame: 32-40 weeks (the endpoint is the birth which is not at the same number of weeks for all of the babies. This is why the weeks are not listed specifically eg week 40). 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 NCT00194987, which also lists 2 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 7 Nov 2018.