Primary Immune Deficiency DisorderPhase 3NCT01888484

Study of Octanorm Subcutaneous IG in Patients With PID

A Phase 3 study testing octanorm 16.5% in people with Primary Immune Deficiency Disorder, run by Octapharma at 24 sites in 7 countries.

Completed

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

Official record

Search all trials

Trial summary

This is an open-label phase III study with a 12-week wash-in/wash-out period followed by a 12-month efficacy period. The main goals of the study are to assess the efficacy of octanorm in preventing serious bacterial infections (SBI) compared with historical control data and to evaluate the pharmacokinetic (PK)… Read more about this study

Who is it for?: Ages 2 to 75 years
Conditions the study is for: Primary Immune Deficiency Disorder
What is being tested?: octanorm 16.5%
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 75 participants
Where: 24 sitesin 7 countries
Study dates: Mar 2014to 9 Jun 2020
Phase: Phase 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 2 to 75 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Age of at least 2 years up to and including 75 years.
Confirmed diagnosis of PI as defined by the ESID and PAGID and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PI should be recorded.
Patients with at least 6 infusions on regular treatment with any IVIG, there of a minimum of the last 2 months on the same product prior to entering the study. Constant IVIG dose between 200 and 800 mg/kg body weight (±20% of the mean dose for the last 6 infusions).
Availability of the IgG trough levels of 2 previous IVIG infusions before enrollment, and maintenance of greater than or equal to 5.0 g/L in the trough levels of these 2 previous infusions.
Negative result on a pregnancy test (HCG-based assay in urine) for women of childbearing potential and use of a reliable method of contraception for the duration of the study.
For adult patients: freely given written informed consent. For minor patients: freely given written informed consent from parents/legal guardians and written informed assent from the child/adolescent in accordance with the applicable regulatory requirements.
Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

Who cannot join

Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
Known history of adverse reactions to IgA in other products.
Patients with body mass index ≥40 kg/m2.
Exposure to blood or any blood product or plasma derivatives, other than IVIG treatment for PID, within the past 3 months prior to the first infusion of octanorm.
Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product (such as Polysorbate 80).
Requirement of any routine premedication for IgG administration.

Show all 19Show fewer

History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
Severe liver function impairment (ALAT 3 times above upper limit of normal).
Known protein-losing enteropathies or proteinuria.
Presence of renal function impairment (creatinine greater than 120 uM/L), or creatinine greater than 1.35 mg/dL), or predisposition for acute renal failure (e.g., any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
Treatment with oral or parenteral steroids for greater than or equal to 30 days or when given intermittently or as bolus at daily doses greater than or equal to 0.15 mg/kg.
Treatment with immunosuppressive or immunomodulatory drugs.
Live viral vaccination (such as measles, rubella, mumps and varicella) within the last 2 months prior to first infusion of octanorm.
Treatment with any investigational medicinal product within 3 months prior to first infusion of octanorm.
Presence of any condition, that is likely to interfere with the evaluation of study medication or satisfactory conduct of the trial.
Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to first infusion of octanorm.
Known or suspected HIV, HCV, or HBV infection.
Pregnant or nursing women.
Planned pregnancy during the course of the study.

Trial locations and contacts

24 sites in 7 countries.

Country

City, postcode or hospital

24 sites in 7 countries

United States7 sites

Octapharma Research Site
Birmingham, Alabama, 35233
Octapharma Research Site
Centennial, Colorado, 80112
Octapharma Research Site
Frisco, Texas, 75034
Octapharma Research Site
Irvine, California, 92697
Octapharma Research Site
Omaha, Nebraska, 68046
Octapharma Research Site
San Diego, California, 92123
Octapharma Research Site
Toledo, Ohio, 43617

Other trials in United States

Czechia4 sites

Octapharma Research Site
Brno, 656 91
Octapharma Research Site
Olomouc, 775 20
Octapharma Research Site
Pilsen, 304 60
Octapharma Research Site
Prague, 150 06

Other trials in Czechia

Poland4 sites

Octapharma Research Site
Bialystok, 15-274
Octapharma Research Site
Krakow, 30-663
Octapharma Research Site
Krakow, 31-024
Octapharma Research Site
Lublin, 20-093

Other trials in Poland

Russia3 sites

Octapharma Research Site
Moscow, 117198
Octapharma Research Site
Saint Petersburg, 197101
Octapharma Research Site
Yekaterinburg, 620149

Other trials in Russia

Slovakia3 sites

Octapharma Research Site
Bratislava
Octapharma Research Site
Košice
Octapharma Research Site
Martin

Other trials in Slovakia

Canada2 sites

Octapharma Research Site
Edmonton, T6G 2V2
Octapharma Research Site
Montreal, H3H 1P3

Other trials in Canada

Hungary1 site

Octapharma Research Site
Budapest, 1097

Other trials in Hungary

About this study

Official title: Clinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency Diseases

Conditions: Primary Immune Deficiency Disorder
Being tested: octanorm 16.5%
Keywords: PID
Medical subject headings: Primary Immunodeficiency Diseases

What participants receive

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

octanorm 16.5%Biological
octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.

Study groups in NCT01888484
Study group	Role	What they receive
Octanorm 16.5%	Experimental	octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration. Receives: Biological: octanorm 16.5%

What is being measured

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

Rate of SBI Per Person-year
The primary efficacy outcome is the rate of SBI (Serious bacterial infections - defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.
Measured: Every 4 weeks until the final evaluation at week 65.
AUC(t) at Steady-State Conditions
The primary endpoint with respect to the PK investigations is the area under the curve AUC(t) (i.e., AUC from time 0 (start of the infusion) to the end of the nominal dosing period, standardised to 1 week) at PKSC2 at steady-state conditions.
Measured: Measured at Week 28 before the start of the SC infusion, 10 minutes before the end of the infusion, and at 2 h, 1, 2, 3, 4 and 7 days after the end of the infusion. Calculated and averaged.

Secondary outcomes (7)

The Annual Rate of All Infections of Any Kind or Seriousness.
The annual rate of all infections of any kind or seriousness.
Measured: Up to 65 weeks
Non-serious Infections
Non-serious infections (total and by category).
Measured: Up to 65 weeks
Cmax of Total IgG and IgG Subclasses
Cmax (Maximum Plasma Concentration) of total IgG and IgG Subclasses, where the mean value was calculated and reported
Measured: Measured at week 28
Tmax of Total IgG and IgG Subclasses
Tmax (Time to Maximum Plasma Concentration) of total IgG and IgG Subclasses
Measured: Measured at Week 28 for all patients, median value was calculated
AUC of Total IgG and IgG Subclasses
AUC (Area Under the Concentration-Time Curve) of total IgG and IgG Subclasses calculated for all patients and mean value was calculated and reported
Measured: Measured at Week 28
Trough Levels of Serum IgG
Trough levels of serum IgG, IgG1, IgG2, IgG3, IgG4 at PK 7 days after 28th infusion of octanorm measured for all patients and median value was calculated and reported
Measured: Measured once at Week 28, seven days after 28th infusion of octanorm
IVIG to Octanorm DCF (Based on the Area Under the Concentration-time Curve [AUCτ])
IVIG to Octanorm Dose Conversion Factor (based on the area under the concentration-time curve [AUCτ]) -- determined by least-squares regression Model which was without restriction of the intercept.
Measured: AUC Measured at Week 28

Timeline

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

Mar 20149 Jun 2020

First posted27 Jun 2013 (estimated)
Study startMar 2014
Primary completion9 Jun 2020
Study completion9 Jun 2020

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 (1)Papers the sponsor has linked to this study.
Other trials for Primary Immune Deficiency DisorderStudies of the same condition, open and under way.
More trials run by OctapharmaThe 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 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 75 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01888484
Sponsor's protocol ID: SCGAM-01
Status last verified: Jul 2021
First submitted: 21 Jun 2013
Last updated: 17 Aug 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Kobayashi RH, Gupta S, Melamed I, Mandujano JF, Kobayashi AL, Ritchie B, Geng B, Atkinson TP, Rehman S, Turpel-Kantor E, Litzman J. Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig(R)]) in the Treatment of Patients With Primary Immunodeficiencies. Front Immunol. 2019 Feb 4;10:40. doi: 10.3389/fimmu.2019.00040. eCollection 2019.
PubMed 30778345

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

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Study at a glance

From the registry record, last updated by the sponsor on 17 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Clinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency Diseases
ClinicalTrials.gov ID: NCT01888484
Acronym / sponsor ID: SCGAM-01
Conditions: Primary Immune Deficiency Disorder
Keywords: PID
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 75 participants (actual)
Lead sponsor: OctapharmaIndustry (pharma, biotech, devices)
Study start: Mar 2014
Primary completion: 9 Jun 2020 (actual)
Study completion: 9 Jun 2020 (actual)
First posted: 27 Jun 2013 (estimated)
Results: Posted 17 Aug 2021

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: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Octanorm 16.5%	Experimental	Biological: octanorm 16.5%octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.

Who may qualify

Ages: 2 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (7 listed)

Age of at least 2 years up to and including 75 years.
Confirmed diagnosis of PI as defined by the ESID and PAGID and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PI should be recorded.
Patients with at least 6 infusions on regular treatment with any IVIG, there of a minimum of the last 2 months on the same product prior to entering the study. Constant IVIG dose between 200 and 800 mg/kg body weight (±20% of the mean dose for the last 6 infusions).
Availability of the IgG trough levels of 2 previous IVIG infusions before enrollment, and maintenance of greater than or equal to 5.0 g/L in the trough levels of these 2 previous infusions.
Negative result on a pregnancy test (HCG-based assay in urine) for women of childbearing potential and use of a reliable method of contraception for the duration of the study.

Key exclusion criteria (19 listed)

Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
Known history of adverse reactions to IgA in other products.
Patients with body mass index ≥40 kg/m2.
Exposure to blood or any blood product or plasma derivatives, other than IVIG treatment for PID, within the past 3 months prior to the first infusion of octanorm.
Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product (such as Polysorbate 80).

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
octanorm 16.5%octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.	Biological	Octanorm 16.5%

Primary outcome

Rate of SBI Per Person-year
Time frame: Every 4 weeks until the final evaluation at week 65.
The primary efficacy outcome is the rate of SBI (Serious bacterial infections - defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.
AUC(t) at Steady-State Conditions
Time frame: Measured at Week 28 before the start of the SC infusion, 10 minutes before the end of the infusion, and at 2 h, 1, 2, 3, 4 and 7 days after the end of the infusion. Calculated and averaged.
The primary endpoint with respect to the PK investigations is the area under the curve AUC(t) (i.e., AUC from time 0 (start of the infusion) to the end of the nominal dosing period, standardised to 1 week) at PKSC2 at steady-state conditions.

7 secondary outcomes

The Annual Rate of All Infections of Any Kind or Seriousness.
Time frame: Up to 65 weeks
Non-serious Infections
Time frame: Up to 65 weeks
Cmax of Total IgG and IgG Subclasses
Time frame: Measured at week 28
Tmax of Total IgG and IgG Subclasses
Time frame: Measured at Week 28 for all patients, median value was calculated
AUC of Total IgG and IgG Subclasses
Time frame: Measured at Week 28
Trough Levels of Serum IgG
Time frame: Measured once at Week 28, seven days after 28th infusion of octanorm
IVIG to Octanorm DCF (Based on the Area Under the Concentration-time Curve [AUCτ])
Time frame: AUC Measured at Week 28

Results and safety

Results posted to the registry on 17 Aug 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Children ≥2 Years <6 Years	12	12
Children Aged ≥6 to <12 Years	14	14
Adolescents ≥12 Years <17 Years	12	8
Adults ≥17 Years ≤75 Years	37	34

Primary outcome: Rate of SBI Per Person-year

Time frame: Every 4 weeks until the final evaluation at week 65.. Number. Unit: SBIs per person year

Measure	All Patients n = 75
Value	0

Population analysed: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group

Primary outcome: AUC(t) at Steady-State Conditions

Time frame: Measured at Week 28 before the start of the SC infusion, 10 minutes before the end of the infusion, and at 2 h, 1, 2, 3, 4 and 7 days after the end of the infusion. Calculated and averaged.. Mean (Standard Deviation). Unit: h*g/L

Measure	Total Patients n = 24
Value	2232.61 (585.842)

Population analysed: In several cases, AUCτ could not be calculated due to very flat time-concentration profiles.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: For the duration of the 65 week treatment period starting with the first infusion of octanorm.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
All Patients	9 of 75 (12%)	61 of 75 (81.3%)	0 of 75

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 30778345Kobayashi RH, Gupta S, Melamed I, Mandujano JF, Kobayashi AL, Ritchie B, Geng B, Atkinson TP, Rehman S, Turpel-Kantor E, Litzman J. Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig(R)]) in the Treatment of Patients With Primary Immunodeficiencies. Front Immunol. 2019 Feb 4;10:40. doi: 10.3389/fimmu.2019.00040. eCollection 2019.

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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.

Wolfgang Frenzel study director

International Medical Director

24 sites in 7 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing octanorm 16.5% in people with Primary Immune Deficiency Disorder, run by Octapharma at 24 sites in 7 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 7 inclusion and 19 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 17 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 sites in United States, Czechia, Poland, Russia, Slovakia and 2 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 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 17 Aug 2021). 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?

Rate of SBI Per Person-year (time frame: Every 4 weeks until the final evaluation at week 65.). The study lists 2 primary outcomes in all. 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 NCT01888484, 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 17 Aug 2021.