Clinical study to evaluate the efficacy and safety of immunoglobulin intravenous (human) 10% (NewGam) in Primary Immune Thrombocytopenia
| ISRCTN | ISRCTN37539930 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN37539930 |
| ClinicalTrials.gov number | NCT01349790 |
| Secondary identifying numbers | NGAM-02 |
- Submission date
- 20/02/2013
- Registration date
- 27/02/2013
- Last edited
- 10/04/2019
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Plain English summary of protocol
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
Contact information
Scientific
Universitätsklinikum Charité
Med. Fakultät der Humboldt-Universität zu Berlin
Institut für Transfusionsmedizin
Campus Virchow-Klinikum
Augustenburger Platz 1
Berlin
13353
Germany
Study information
| Study design | Prospective open-label non-controlled multicenter phase III clinical study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Screening |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| 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 |
| Study objectives | To assess the efficacy of NewGam in correcting the platelet count. |
| Ethics approval(s) | 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 |
| Health condition(s) or problem(s) studied | 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 |
| Pharmaceutical study type(s) | |
| Phase | Phase III |
| Drug / device / biological / vaccine name(s) | Immunoglobulin intravenous (human) 10% (NewGam) |
| Primary outcome measure | 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 study start date | 30/06/2011 |
| Completion date | 30/06/2013 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 95 |
| Key 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 |
| Key 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. |
| Date of first enrolment | 30/06/2011 |
| Date of final enrolment | 30/06/2013 |
Locations
Countries of recruitment
- Bulgaria
- Czech Republic
- France
- Germany
- India
- Poland
- Romania
- Russian Federation
- Ukraine
Study participating centre
13353
Germany
Sponsor information
Industry
Seidenstrasse 2
Lachen
CH-8853
Switzerland
| Website | http://www.octapharma.com |
|---|---|
"ROR" |
https://ror.org/002k5fe57 |
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/02/2019 | 10/04/2019 | Yes | No |
Editorial Notes
10/04/2019: Publication reference added.
