Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01814800
• Date of registration: 06/03/2013
• Prospective Registration: Yes
• Primary sponsor: ADMA Biologics, Inc.
• Public title: Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
• Scientific title: An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
• Date of first enrolment: February 2014
• Target sample size: 59
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01814800
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: James Mond, M.D., Ph.D.
• Affiliation: ADMA Biologics, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

To be eligible to participate in this study, the subjects must meet the following criteria:

1. Signed a written informed consent or a specific assent form for minors.

2. Have a diagnosis of primary immunodeficiency disease.

3. Be = 2 years and = 75 years.

4. Have body weight = 12 kg at screening.

5. Have been receiving IGIV at a dose that has not been changed by >50% of the mean dose
on a mg/kg basis for at least 3 months prior to study entry and have maintained a
trough serum Immunoglobulin G (IgG) level = 500 mg/dL on the previous 2 assessments
prior to receiving RI 002. The trough level must be at least 300 mg/dL above the
pre-treatment serum IgG level.

6. For female subjects, be of non-childbearing potential or have a negative pregnancy
test prior to study start and be deemed not at risk of becoming pregnant by adherence
to a reliable contraceptive method for the duration of the study.

Exclusion Criteria:

Subjects must be excluded if they meet any of the following criteria:

1. Have a known hypersensitivity to immunoglobulin or any excipient in RI-002.

2. Have a history of a severe anaphylactic or anaphylactoid reaction to blood or any
blood-derived product.

3. Have a specific Immunoglobulin A (IgA) deficiency, history of allergic reaction to
products containing IgA or has demonstrable antibodies to IgA.

4. Have uncompensated hemodynamically significant congenital or other heart disease.

5. Have a medical condition that is known to cause secondary immune deficiency.

6. Have a significant T-cell deficiency or deficiency of granulocyte number or function.

7. Have significant renal impairment or have a history of acute renal failure.

8. Have abnormal liver function.

9. Be receiving chronic anti-coagulation therapy.

10. Have a history of deep vein thrombosis (DVT), thrombotic or thrombo-embolic event, or
are at increased risk for thrombotic events.

11. Current daily use of the following medications:

- corticosteroids (> 7.5 mg (or equivalent dose on a mg/kg basis) of prednisone
equivalent per day for > 30 days)

- immunomodulatory drugs

- immunosuppressive drugs (excluding topical pimecrolimus (Elidel) and tacrolimus
(Protopic))

12. Administration of a hyperimmune or specialty high titer immunoglobulin product.

13. Have uncontrollable arterial hypertension.

14. Have a history of hemolysis or positive Coombs test while undergoing treatment with
IGIV therapy.

15. Be morbidly obese as indicated by a Body Mass Index (BMI) = 40

16. Have received any blood product (other than Immunoglobulin G) within 3 months prior to
screening.

17. Have received any Respiratory Syncytial Virus (RSV) specific products, including
palivizumab (Synagis®) within 3 months prior to screening.

18. Have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within
the past 12 months.

19. Have any condition or abnormal laboratory assessment judged by the investigator to
preclude participation in the study.

20. Are currently pregnant or nursing.

21. Have hepatitis A, B, or C.



Age minimum: 2 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Immune Deficiency Disorder

Intervention(s)

Biological: RI-002

Primary Outcome(s)

Number of Serious Bacterial Infections (SBIs) Per Subject Per Year (FDA Guidance for Industry (2008)) [Time Frame: One year]

Secondary Outcome(s)

Number of Hospitalizations Due to Infections - Per Subject-Year [Time Frame: Up to 1 year]

Number of Unscheduled Visits to Physician/ER Due to Infections - Per Subject-Year [Time Frame: Up to 1 year]

Time to Resolution of Infections - Duration Per Infection [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 5 [Time Frame: Up to 1 year]

Number of Unscheduled Visits to Physician/ER Due to Infections - Total Number of Visits [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 23F [Time Frame: Up to 1 year]

Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection) [Time Frame: Up to 1 year]

Number of Hospitalizations Due to Infections [Time Frame: Up to 1 year]

Days of Hospitalization Due to Infections - Per Subject-Year [Time Frame: Up to 1 year]

Incidence of All Infections (Serious and Non-serious) [Time Frame: Up to 1 Year]

Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Combined Days Lost [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 19A [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 6B [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Haemophilus Influenzae Type B [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - IgG [Time Frame: Up to 1 year]

Correlation Between Trough Level of RI-002 and Serious and Non-serious Infections [Time Frame: Up to 1 year]

Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection) - Per Subject-Year [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 9V [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 3 [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Tetanus [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Respiratory Syncytial Virus (RSV) [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 1 [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 14 [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 19F [Time Frame: Up to 1 year]

Days of Hospitalization Due to Infections [Time Frame: Up to 1 year]

Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Per Subject-Year [Time Frame: Up to 1 year]

Time to Resolution of Infections - Infection Days Per Subject [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 18C [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 4 [Time Frame: Up to 1 year]

Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 7F [Time Frame: Up to 1 year]

Secondary ID(s)

ADMA-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/10/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01814800