Condition category
Haematological Disorders
Date applied
20/02/2013
Date assigned
27/02/2013
Last edited
27/02/2013
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Background and study aims
In this study, it will be assessed, whether NewGam [immunoglobulin intravenous (human)] corrects (i.e. increases) the platelet count.

Who can participate?
Patients with confirmed diagnosis of chronic Primary Immune Thrombocytopenia (ITP) of at least 12 months duration, age >=18 years and <=65 years, and a platelet count of <20x10 exp9/L with or without bleeding manifestations.

What does the study involve?
Beside examination at study start all patients will receive two intravenous infusions of NewGam at two consecutive days. Over a period of 63 days participants will be asked to give a small amount of blood for assessment of platelet count and safety parameters.

What are the possible benefits and risks of participating?
Patient will receive NewGam for a quick increase in platelet count. During this study the patients’ health condition will be assessed in shorter intervals than normal.
The most common side effects when people take intravenous (IV) immunoglobulin are headache, chills, back pain, chest pain, myalgia, fever, cutaneous reactions, fatigue, various minor allergic and hypersensitivity type of reactions, hot flushes and nausea.

Where is the study run from?
The study has been set up by Octapharma AG, a pharmaceutical company.

When is study starting and how long is it expected to run for?
Study started in June 2011 and expected to end in June 2013.

Who is funding the study?
Octapharma AG (Switzerland)

Who is the main contact?
Birgit Taumberger
Birgit.taumberger@octapharma.com

Trial website

Contact information

Type

Scientific

Primary contact

Prof Abdulgabar Salama

ORCID ID

Contact details

Universitätsklinikum Charité
Med. Fakultät der Humboldt-Universität zu Berlin
Institut für Transfusionsmedizin
Campus Virchow-Klinikum
Augustenburger Platz 1
Berlin
13353
Germany

Additional identifiers

EudraCT number

ClinicalTrials.gov number

NCT01349790

Protocol/serial number

NGAM-02

Study information

Scientific 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

Acronym

Study hypothesis

To assess the efficacy of NewGam in correcting the platelet count.

Ethics approval

1. Ethics Committee of the Institute for Clinical and Experimental Medicine and Thomayer Hospital, Prague, Czech Republic, 11.05.2011, ref: 710/11 (M11-25)
2. Ethics Committee for Multicentre Trials, 8 Damyan Gruev Str., 1303 Sofia, Bulgaria, 3.10.2011, KE-107/22.06.2011
3. Comité de Protection des personnes Sud Méditérrannée I, Institut Paoli Calmettes, 232 Boulevard Ste Marguerite, 13009 Marseille; France; 30. 01. 2012, Ref: 11 47
4. Ethik-Kommission des Landes Berlin Landesamt für Gesundheit und Soziales Berlin, Fehrbelliner Platz 1, 10707 Berlin; 07.09. 2011, 11/0218 ZS EK
5. Independent Bioethical Committee for Scientific Research at Medical University in Gdañsk, 3a M. Sk³odowskiej - Curie str.; 80-210 Gdañsk, 07.07.2011
6. National Ethics Committee, 48 Av. Sanatescu St., District 1, Bucharest, Romania, 20.07.2011
7. Ministry of Healthcare and Social Development, Ethics Committee, 8, Petrovskiy avenue,127051, Moscow, Russia, 24.08.2011
8. Central Ethics Commitee, 03680 Kiev, st. Narodnogo Opolchenia, 5, Ukraine, 5.12-1035/KE, 07.09.2011
9. lnstitutional Ethics committee for Sahyadri Hospitals, 30C, Erandawane, Karve Road, Pune 411004, India, 18.08.2011

Study design

Prospective open-label non-controlled multicenter phase III clinical study

Primary study design

Interventional

Secondary study design

Non randomised controlled trial

Trial setting

Hospitals

Trial type

Screening

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

Primary Immune Thrombocytopenia (ITP)

Intervention

All patients will receive 1 g/kg/day NewGam by intravenous infusion for 2 consecutive days, for a total of 2 g/kg.

Intervention type

Drug

Phase

Phase III

Drug names

Immunoglobulin intravenous (human) 10% (NewGam)

Primary outcome measures

The primary efficacy measure is defined as an increase in platelets to atleast 50x109/L within 7 days after the first infusion, i.e. by study Day 8 (at least once prior to Day 9).

Secondary outcome measures

Additional response rates will be calculated on basis of an alternative definition for response and for additionally defined criteria for complete response and loss of response.

Overall trial start date

30/06/2011

Overall trial end date

30/06/2013

Reason abandoned

Eligibility

Participant inclusion criteria

1. Age of >=18 years years and <=65 years
2. Confirmed diagnosis of chronic primary ITP of at least 12 months duration (diagnosed with threshold platelet count less than 100x109/L)
3. Platelet count of <=20x10exp9/L with or without bleeding manifestations

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

95

Participant exclusion criteria

1. Thrombocytopenia secondary to other diseases (such as Acquired Immunodeficiency Syndrome [AIDS] or systemic lupus erythematosus [SLE]) or drug-related thrombocytopenia.
2. Administration of intravenous immunoglobulin (IGIV), anti-D or thrombopoetin receptor agonists or other platelet enhancing drugs (incl. immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment
3. Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.
4. Severe liver or kidney disease (alanine aminotransferase [ALAT] 3x > upper limit of normal, creatinine >120 µmol/L).
5. Patients with risk factors for thromboembolic events in whom the risks outweigh the potential benefit of NewGam treatment.

Recruitment start date

30/06/2011

Recruitment end date

30/06/2013

Locations

Countries of recruitment

Bulgaria, Czech Republic, France, Germany, India, Poland, Romania, Russian Federation, Ukraine

Trial participating centre

Universitätsklinikum Charité
Berlin
13353
Germany

Sponsor information

Organisation

Octapharma AG (Switzerland)

Sponsor details

Seidenstrasse 2
Lachen
CH-8853
Switzerland

Sponsor type

Industry

Website

http://www.octapharma.com

Funders

Funder type

Industry

Funder name

Octapharma AG (Switzerland)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes