A prospective, multicentre, open label, exploratory study to investigate the ability of the Heidelberg assay panel and the B-Cell/antibody response panel to predict the clinical effect of Octagam® 5% in subjects with relapsing/remitting multiple sclerosis

ISRCTN	ISRCTN57377482
DOI	https://doi.org/10.1186/ISRCTN57377482
Secondary identifying numbers	GAM-25

Submission date: 18/03/2009
Registration date: 18/03/2009
Last edited: 10/04/2013

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Nervous System Diseases

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Stefan Wietek
Scientific

Octapharma PPG
Vienna CR&D Office
Oberlaaer Str. 235
Vienna
1100
Austria

Phone	+43 (0)1 61032 1778
Email	stefan.wietek@octapharma.com

Study information

Study design	Prospective exploratory open label multicentre phase II trial
Primary study design	Interventional
Secondary study design	Non randomised controlled trial
Study setting(s)	Hospital
Study type	Treatment
Participant information sheet	Not available in web format, please use the contact details below to request a patient information sheet
Scientific title
Study objectives	To find a panel of laboratory parameters which might be able to predict the clinical outcome of treatment of patients with relapsing-remitting multiple sclerosis (MS) with Octagam®.
Ethics approval(s)	1. EC Medical University of Innsbruck (Austria) - includes approval for Linz and Klagenfurt; approval received on the 9th January 2009 and final approval for additional study materials received on 22nd January 2009 2. EC Heidelberg (Germany) - includes approval for study sites at Heidelberg, Asbach, Berlin, Hamburg, Regensburg, Rostock; approval received on the 31st March 2009
Health condition(s) or problem(s) studied	Multiple sclerosis
Intervention	Octagam® 5% (12 infusions 0.4 g/kg every 4 weeks).
Intervention type	Other
Primary outcome measure	A panel of lab parameters (components of cells of the immune system, serum proteins, gene expression, single nucleotide polymorphism [SNP] analysis) will be analysed at baseline, 12 - 24 hours after end of the first infusion, and 24 and 48 weeks after the first Octagam® administration and the clinical course of the disease will be monitored by relapse activity, EDSS and Multiple Sclerosis Functional Composite (MSFC) at predefined time points. Statistical analysis will test whether any of the lab parameters of the HAP panel or the B-cell antibody response panel might be able to predict the clinical outcome observed following Octagam® 5% treatment. The proportion of subjects clinically responding to Octagam® 5% will be determined.
Secondary outcome measures	Lesion load in brain MRI imaging (T2 weighted, T1 weighted, Gd-enhancing lesion load) after treatment will be compared to lesion load before treatment.
Overall study start date	20/03/2009
Completion date	20/09/2010

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	Both
Target number of participants	30
Key inclusion criteria	1. Subjects aged greater than or equal to 18 years, either sex 2. Multiple sclerosis (MS) according to the revised McDonald criteria 3. Relapsing-remitting form of MS 4. First-line disease modifying treatments (interferon-beta [IFN-beta] or glatiramer acetate) are contraindicated or not tolerated 5. Kurtzke's Expanded Disability Status Scale (EDSS) between 0 and 3.5 (0 to less than 3.5) 6. Subjects who experienced at least one relapse during the last 12 months or at least two relapses in the last 24 months prior to study entry 7. Freely given, fully informed written consent obtained from subject
Key exclusion criteria	1. Subjects who have received treatment with immunoglobulins for any reason previously 2. Subjects who have received immuno-suppressive treatments (e.g. azathioprine, mitoxantrone, cyclophosphamide) for any reason previously except relapse treatment with corticosteroids 3. Subjects who have received disease modifying first-line treatments with IFN-beta during the last 8 weeks or with glatiramer acetate during the last 16 weeks 4. Subjects who have received any monoclonal antibody therapies (e.g. natalizumab) previously 5. Subjects who had a relapse within 3 months prior to study entry 6. Subjects with severe renal function impairment as defined by serum creatinine values greater than 24 mg/l 7. Subjects with known intolerance to homologous immunoglobulins, especially immunoglobulin A (IgA) deficiency, when the subject has antibodies against IgA 8. Subjects with a body weight of greater than 120 kg 9. Subjects with a history of anaphylaxis after previous transfusions of blood or blood products 10. Subjects for whom magnetic resonance imaging (MRI) is contraindicated or who are allergic to gadolinium 11. Pregnant or lactating women 12. Subjects who delivered a baby within 12 months before study entry (including miscarriage and stillbirth) 13. Subjects with a diagnosis of significant depression 14. Subjects with known chronic infectious diseases or malignant disease 15. Subjects with known antibody deficiencies or other autoimmune diseases other than MS 16. Subjects participating in another study during the course of this study or during the past 6 months or who have ever participated in a study investigating in new disease modifying or immunosuppressive drugs
Date of first enrolment	20/03/2009
Date of final enrolment	20/09/2010

Locations

Countries of recruitment

Austria
Germany

Study participating centre

Octapharma PPG

Vienna
1100
Austria

Sponsor information

Octapharma AG (Switzerland)
Industry

Seidenstr. 2
Lachen
CH-8853
Switzerland

Website	http://www.octapharma.com
"ROR"	https://ror.org/002k5fe57

Funders

Funder type

Industry

Octapharma AG (Switzerland)

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