Immune Thrombocytopenic PurpuraPhase 2NCT00718692
Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP)
A Phase 2 study testing Sym001 in people with Immune Thrombocytopenic Purpura, run by Symphogen A/S at 59 sites in 12 countries.
Trial summary
This trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenic Purpura
- What is being tested?
- Sym001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 59 sitesin 12 countries
- Study dates
- Jul 2008to Oct 2011
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit.
- History of isolated ITP
- RhD-positive serology.
- Previous treatment and response to first line therapy for ITP
Who cannot join
- Known clinical picture suggestive of other causes of thrombocytopenia, especially systematic lupus erythematosus,antiphospholipid syndrome, Evans syndrome, immunodeficiency states, lymphoproliferative disorders, liver disease,ingestion of drugs such as quinidine/quinine, heparin and sulfonamides and hereditary thrombocytopenia confirmed by relevant laboratory findings.
- Suspected infection with HIV, Hepatitis C, H. pylori.
- Clinical splenomegaly
- History of abnormal bone marrow examination.
- Ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale.
- Underlying haemolytic condition
- History of splenectomy.
- Subject is pregnant, breast feeding or intends to become pregnant.
Trial locations and contacts
59 sites in 12 countries.
59 sites in 12 countries
Germany7 sites
Research Site 4906, 4907
Berlin
Research Site 4902
Cologne
Research Site 4903
Duisburg
Research Site 4905
Essen
Reseach Site 4908
Halle
Research Site 4901
Hanover
Research Site 4904
Regensburg
India7 sites
St. John's Medical College Hospital, 9106
Bangalore, 560034
MS Ramaiah Memorial Hospital, 9108
Bangalore, 560054
Narayana Hrudayalaya Hospitals, 9105
Bangalore, 560099
Columbia Asia Referral Hospital-Yeshwanthpur, 9101
Bangalore, 576104
Aysha Hospital Pvt. Ltd., 9109
Chennai, 600010
Apollo Hospital, 9104
Hyderabad, 500033
Kasturba Medical College Hospital, 9103
Udupi, 576104
Poland7 sites
Research site 4801
Gdansk
Research Site 4806
Krakow
Research Site 4805
Lodz
Instytut Hematologii i Transfuzjologii, 4807
Warsaw
Research Site 4802, 4808
Warsaw
Research Site 4803
Warszava
Research Site 4804
Wroclaw
Israel6 sites
Bnai-Zion Medical Center, 9721
Haifa, 31048
Rambam Medical Center, 9723
Haifa, 35254
The Edith Wolfson Medical Center, 9725
Holon, 58100
Western Galilee - Nahariya Hospital, 9722
Nahariya, 22100
Rabin Medical Center, 9724
Petah Tikva, 49100
Tel-Aviv Sourasky Medical Center, 9726
Tel Aviv, 64239
Spain5 sites
Reseach Site 3403
Barcelona
Hospital Gregorio Maranon, 3405
Madrid
Hospital La Princesa, 3404
Madrid
Research Site 3401
Madrid
Research Site 3402
Valencia
Ukraine5 sites
Cherkassy regional oncology center, 3906
Cherkassy, 180009
Gusak Academy of AMS Ukraine, 3905
Donetsk, 83045
Khmelnytskyi Regional Hospital, 3903
Khmelnytskyi, 29000
City Hospital #9 Kyiv Clinical Hospital, 3904
Kiev, 04112
Vinnytsya Regional Clinical Hospital, 3902
Vinnytsia, 21018
United Kingdom5 sites
St. James's University Hospital, 4403
Leeds, LS9 7TF
Hammersmith Hosptial, 4402
London, W12 OHS
University College, 4401
London, WC1E 6HX
Royal Victoria Infirmery, 4404
Newcastle upon Tyne, NE1 4LP
Singleton Hospital, 4405
Swansea, SA2 8QL
Romania4 sites
Brasov Country Hospital, 4002
Brasov
Institutul Clinic Fundeni, 4003
Bucharest
"Prof. Dr. Ion Chiricuta" Oncolgy Institute, 4001
Cluj-Napoca
Spitalul Clinic de Urgente Sfantu Spiridon, 4004
Iași
Russia4 sites
Regional Clinical Hospital of Nizhny Novgorod, 7003
Nizhny Novgorod, 603126
Saint Petersburg State Institution Of Healthcare, 7001
Saint Petersburg, 198205
Regional Clinical Hospital na MI Kalinin, 7005
Samara, 443095
State Healthcare Institution Tula Regional Clin. Hosp., 7002
Tula, 300053
United States3 sites
2085 Henry Tecklenburg Drive, 1001
Charleston, South Carolina, 29414
Univ Nebraska Med Ctr. UNMC, 1005
Omaha, Nebraska, 68198-7680
Hospital of the University of Pennsylvania, Site 1002
Philadelphia, Pennsylvania, 19104
Belgium3 sites
Research Site 3203
Bruges
Research Site 3202
Leuven
Research Site 3201
Yvoir
Serbia3 sites
Clinical Center Nis 3802
Niš, 18000
Clinical center Vojvodina 3801
Novi Sad, 2100
Clinical Center Zemun 3803
Zemun, 11080
About this study
Official title: A Multi-centre, Single Intravenous Dose, Exploratory Dose-finding, Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP) in RhD Positive, Non-splenectomized Adult Subjects.
This trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001.
- Conditions
- Immune Thrombocytopenic Purpura
- Being tested
- Sym001
- Keywords
- Idiopathic, ITP
- 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.
Sym001Drug
Each cohort will receive one single dose of Sym001 according the assigned dose level.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Patients treated with Sym001 Receives: Drug: Sym001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs)
Measured: 6 weeks post dosing
Secondary outcomes (1)
Measurements of platelet counts
Measured: From day 1 through week 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2008 (estimated)
- Study startJul 2008
- Primary completionAug 2011
- Study completionOct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Symphogen A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Swedish Orphan Biovitrum
- Responsible party
- Sponsor
- Investigators
- Mario Von Depka Prondzinski, PD Dr., Principal investigator, Werlhoff Institut Hannover, GermanyAnn Janssens, Dr., Principal investigator, ZU Gasthuisberg, Leuven, BelgiumJavier Loscertales Pueyo, Dra, Principal investigator, Hospital La Princesa, Madrid, SpainWieslaw Wiktor Jedrzrjczak, Prof., Principal investigator, Katedra i klinika Hematologii, Warszawa, PolandAndrei Cucucianu, Dr., Principal investigator, Prof. Dr. Ion Chiricuta Oncology Institute, Cluj-Napoca, RomaniaMarie Scully, Prof. Dr. MD, Principal investigator, University College London Hospitals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00718692
- Sponsor's protocol ID
- Sym001-03
- Other IDs
- 2007-006081-15
- Status last verified
- Jan 2019
- First submitted
- 18 Jul 2008
- Last updated
- 30 Jan 2019
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Robak T, Windyga J, Trelinski J, von Depka Prondzinski M, Giagounidis A, Doyen C, Janssens A, Alvarez-Roman MT, Jarque I, Loscertales J, Rus GP, Hellmann A, Jedrzejczak WW, Kuliczkowski K, Golubovic LM, Celeketic D, Cucuianu A, Gheorghita E, Lazaroiu M, Shpilberg O, Attias D, Karyagina E, Svetlana K, Vilchevska K, Cooper N, Talks K, Prabhu M, Sripada P, Bharadwaj TP, Naested H, Skartved NJ, Frandsen TP, Flensburg MF, Andersen PS, Petersen J. Rozrolimupab, a mixture of 25 recombinant human monoclonal RhD antibodies, in the treatment of primary immune thrombocytopenia. Blood. 2012 Nov 1;120(18):3670-6. doi: 10.1182/blood-2012-06-438804. Epub 2012 Aug 20.
PubMed 22915649
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-centre, Single Intravenous Dose, Exploratory Dose-finding, Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP) in RhD Positive, Non-splenectomized Adult Subjects.
- ClinicalTrials.gov ID
- NCT00718692
- Acronym / sponsor ID
- Sym001-03
- Conditions
- Immune Thrombocytopenic Purpura
- Keywords
- Idiopathic, ITP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Symphogen A/SIndustry (pharma, biotech, devices)
- Collaborators
- Swedish Orphan Biovitrum
- Study start
- Jul 2008 (actual)
- Primary completion
- Aug 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 21 Jul 2008 (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.
This trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Sym001Patients treated with Sym001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit.
- History of isolated ITP
- RhD-positive serology.
- Previous treatment and response to first line therapy for ITP
Key exclusion criteria (8 listed)
- Known clinical picture suggestive of other causes of thrombocytopenia, especially systematic lupus erythematosus,antiphospholipid syndrome, Evans syndrome, immunodeficiency states, lymphoproliferative disorders, liver disease,ingestion of drugs such as quinidine/quinine, heparin and sulfonamides and hereditary thrombocytopenia confirmed by relevant laboratory findings.
- Suspected infection with HIV, Hepatitis C, H. pylori.
- Clinical splenomegaly
- History of abnormal bone marrow examination.
- Ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale.
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 |
|---|---|---|
| Sym001Each cohort will receive one single dose of Sym001 according the assigned dose level. | Drug | 1 |
Primary outcome
Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs)
Time frame: 6 weeks post dosing
1 secondary outcome
Measurements of platelet counts
Time frame: From day 1 through week 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2019.
- Results publication: PubMed 22915649Robak T, Windyga J, Trelinski J, von Depka Prondzinski M, Giagounidis A, Doyen C, Janssens A, Alvarez-Roman MT, Jarque I, Loscertales J, Rus GP, Hellmann A, Jedrzejczak WW, Kuliczkowski K, Golubovic LM, Celeketic D, Cucuianu A, Gheorghita E, Lazaroiu M, Shpilberg O, Attias D, Karyagina E, Svetlana K, Vilchevska K, Cooper N, Talks K, Prabhu M, Sripada P, Bharadwaj TP, Naested H, Skartved NJ, Frandsen TP, Flensburg MF, Andersen PS, Petersen J. Rozrolimupab, a mixture of 25 recombinant human monoclonal RhD antibodies, in the treatment of primary immune thrombocytopenia. Blood. 2012 Nov 1;120(18):3670-6. doi: 10.1182/blood-2012-06-438804. Epub 2012 Aug 20.
- Individual participant dataSharing planned: No
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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.
Mario Von Depka Prondzinski, PD Dr. principal investigator
Werlhoff Institut Hannover, Germany
Ann Janssens, Dr. principal investigator
ZU Gasthuisberg, Leuven, Belgium
Javier Loscertales Pueyo, Dra principal investigator
Hospital La Princesa, Madrid, Spain
59 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sym001 in people with Immune Thrombocytopenic Purpura, run by Symphogen A/S at 59 sites in 12 countries.
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 4 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: finished, as of the sponsor's last update on 30 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 59 sites in Germany, India, Poland, Israel, Spain and 7 more countries. 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 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs) (time frame: 6 weeks post dosing). 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 NCT00718692, which also lists 1 linked publication.
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 30 Jan 2019.