Study of Octanorm Subcutaneous IG in Patients With PID

NCT01888484 | Completed Phase 3 Results DMC

• 1 other identifier: SCGAM-01
• Study Type: interventional
• Target: 75
• Locations: 7 countries
• Sites: 24

Brief 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) characteristics of octanorm.

Conditions

Primary Immune Deficiency Disorder

Keywords

PID

Outcome Measures

Primary Outcomes (2)

• 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.

  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 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.

  Up to 65 weeks

• Non-serious Infections

  Up to 65 weeks

• Cmax of Total IgG and IgG Subclasses

  Measured at week 28

• Tmax of Total IgG and IgG Subclasses

  Measured at Week 28 for all patients, median value was calculated

• AUC of Total IgG and IgG Subclasses

  Measured at Week 28

Study Arms (1)

Octanorm 16.5%

EXPERIMENTAL

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

Biological: octanorm 16.5%

Interventions

octanorm 16.5% BIOLOGICAL

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

Octanorm 16.5%

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (24)

Octapharma Research Site

Birmingham, Alabama, 35233, United States

Location

Octapharma Research Site

Irvine, California, 92697, United States

Location

Octapharma Research Site

San Diego, California, 92123, United States

Location

Octapharma Research Site

Centennial, Colorado, 80112, United States

Location

Octapharma Research Site

Omaha, Nebraska, 68046, United States

Location

Octapharma Research Site

Toledo, Ohio, 43617, United States

Location

Octapharma Research Site

Frisco, Texas, 75034, United States

Location

Octapharma Research Site

Edmonton, T6G 2V2, Canada

Location

Octapharma Research Site

Montreal, H3H 1P3, Canada

Location

Octapharma Research Site

Brno, 656 91, Czechia

Location

Octapharma Research Site

Olomouc, 775 20, Czechia

Location

Octapharma Research Site

Pilsen, 304 60, Czechia

Location

Octapharma Research Site

Prague, 150 06, Czechia

Location

Octapharma Research Site

Budapest, 1097, Hungary

Location

Octapharma Research Site

Bialystok, 15-274, Poland

Location

Octapharma Research Site

Krakow, 30-663, Poland

Location

Octapharma Research Site

Krakow, 31-024, Poland

Location

Octapharma Research Site

Lublin, 20-093, Poland

Location

Octapharma Research Site

Moscow, 117198, Russia

Location

Octapharma Research Site

Saint Petersburg, 197101, Russia

Location

Octapharma Research Site

Yekaterinburg, 620149, Russia

Location

Octapharma Research Site

Bratislava, Slovakia

Location

Octapharma Research Site

Košice, Slovakia

Location

Octapharma Research Site

Martin, Slovakia

Location

Related Publications (1)

• 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.

  PMID: 30778345 DERIVED

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Results Point of Contact

• Title: Mikaela Raymond
• Organization: Clinical Research Management Group

ctgov@cllinicalresearchmgt.com

866-337-1868

Study Officials

• Wolfgang Frenzel

  International Medical Director

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2013
• First Posted: June 27, 2013
• Study Start: March 1, 2014
• Primary Completion: June 9, 2020
• Study Completion: June 9, 2020
• Last Updated: August 17, 2021
• Results First Posted: August 17, 2021

Record last verified: 2021-07

Locations

CA (2); PL (4); US (7); HU (1); RU (3); SL (3); CZ (4)