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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/21/2021.

Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases

Clinicaltrials.gov identifier NCT03939533

Recruitment Status Recruiting

First Posted May 6, 2019

Last update posted July 29, 2020

Study Description

Brief summary:

CLINICAL PHASE 3 STUDY TO MONITOR THE SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (CUTAQUIG®) ADMINISTERED AT MODIFIED DOSING REGIMENS IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES

  • Condition or Disease:Primary Immune Deficiency Disorder
  • Intervention/Treatment: Drug: CUTAQUIG
  • Phase: Phase 3
Detailed Description

N/A

Study Design
  • Study Type: Interventional
  • Estimated Enrollment: 65 participants
  • Allocation: Randomized
  • Intervention Model: Parallel Assignment
  • Masking: None (Open Label) ()
  • Primary Purpose: Treatment
  • Official Title: Clinical Phase 3 Study to Monitor the Safety, Tolerability, and Efficacy of Subcutaneous Human Immunoglobulin (CUTAQUIG®) Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases
  • Actual Study Start Date: October 2019
  • Estimated Primary Completion Date: March 2021
  • Estimated Study Completion Date: March 2021
Arms and interventions
Arm Intervention/treatment
Experimental: Every Other Week Dosing Cohort - Cohort 3
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose
Drug: CUTAQUIG
Human normal immunoglobulin
Experimental: Increased Infusion Rate Cohort - Cohort 2
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
Drug: CUTAQUIG
Human normal immunoglobulin
Experimental: Increased Volume Cohort - Cohort 1
Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks
Drug: CUTAQUIG
Human normal immunoglobulin
Outcome Measures
  • Primary Outcome Measures: 1. IgG Trough Levels [ Time Frame: 25 weeks ]
    Change in individual total IgG trough levels from weekly infusions to every other week infusions
  • 2. AEs [ Time Frame: 25 weeks ]
    Occurrence of treatment-emergent AEs (TEAEs) throughout the entire Stabilization and Treatment Periods starting with the first infusion of investigational medicinal product (IMP)
  • 3. Temporally Associated TEAEs During Treatment Period [ Time Frame: 25 weeks ]
    Occurrence of TEAEs temporally associated with CUTAQUIG delivered during the Treatment Period
  • 4. Temporally Associated TEAEs During Stabilization Period [ Time Frame: 25 weeks ]
    Occurrence of TEAEs temporally associated with CUTAQUIG delivered during the Stabilization Period
  • 5. TEAEs by speed of infusion [ Time Frame: 25 weeks ]
    TEAEs by speed of infusion
  • 6. Number of local infusion-site reactions [ Time Frame: 25 weeks ]
    Patients will grade the overall perception of local reactions in their diaries after each infusion using a 4-point rating scale: 0=none, 1=mild, 2=moderate, 3=severe. Investigators will evaluate local reactions after infusion at every study visit, using a 4-point rating scale: 0=none, 1=mild, 2=moderate, 3=severe.
  • 7. CBC [ Time Frame: 25 weeks ]
    Complete blood count
  • 8. WBC [ Time Frame: 25 weeks ]
    White blood cells
  • 9. Differential WBC Count [ Time Frame: 25 weeks ]
    Measures amount of each type of white blood cell
  • 10. Hematocrit [ Time Frame: 25 weeks ]
    Changes in hematocrit
  • 11. Hemoglobin [ Time Frame: 25 weeks ]
    Changes in hemoglobin
  • 12. Blood sodium [ Time Frame: 25 weeks ]
    Changes in blood sodium
  • 13. Blood potassium [ Time Frame: 25 weeks ]
    Changes in blood potassium
  • 14. Blood glucose [ Time Frame: 25 weeks ]
    Changes in blood glucose
  • 15. ALT [ Time Frame: 25 weeks ]
    Changes in ALT
  • 16. AST [ Time Frame: 25 weeks ]
    Changes in AST
  • 17. LDH [ Time Frame: 25 weeks ]
    Changes in LDH
  • 18. Total bilirubin [ Time Frame: 25 weeks ]
    Changes in total bilirubin
  • 19. Blood urea nitrogen [ Time Frame: 25 weeks ]
    Changes in blood urea nitrogen
  • 20. Creatinine [ Time Frame: 25 weeks ]
    Changes in creatinine
  • 21. Urine pH [ Time Frame: 25 weeks ]
    Changes in urine pH
  • 22. Urine glucose [ Time Frame: 25 weeks ]
    Changes in urine glucose
  • 23. Urine ketones [ Time Frame: 25 weeks ]
    Changes in urine ketones
  • 24. Urine leukocytes [ Time Frame: 25 weeks ]
    Changes in urine leukocytes
  • 25. Urine hemoglobin [ Time Frame: 25 weeks ]
    Changes in urine hemoglobin
  • Secondary Outcome Measures: 1. Quality of Life questionnaire - CHQ-PF50 [ Time Frame: 25 weeks ]
    CHQ-PF50 (Child Health Questionnaire Parent Form 50) from parent or guardian of patients <14 years of age. Likert-type scale with higher scores indicating better or more positive health states. Scores transformed to 0 - 100 scale.
  • 2. Quality of Life questionnaire - SF-36 [ Time Frame: 25 weeks ]
    SF-36 (36-Item Short Form Survey) in patients ≥14 years of age SF-36: Likert-type scale with higher scores indicating better or more positive health states. Scores transformed to 0 - 100 scale.
  • 3. IgG trough levels [ Time Frame: 25 weeks ]
    Measurement of individual profiles of IgG trough levels over time (all cohorts)
  • 4. Infectious Disease Occurrence [ Time Frame: 25 weeks ]
    Monitoring for infectious disease occurrence
  • 5. Infectious Disease Resolution [ Time Frame: 25 weeks ]
    Monitoring for occurrences of infectious disease resolution
  • 6. Antibiotic use [ Time Frame: 25 weeks ]
    Monitoring for occurrences of antibiotic use
  • 7. Serious bacterial infections [ Time Frame: 25 weeks ]
    Occurrence of serious bacterial infections
  • 8. Blood Pressure [ Time Frame: 25 weeks ]
    Blood pressure including systolic and diastolic.
  • 9. Pulse [ Time Frame: 25 weeks ]
    Pulse
  • 10. Body temperature [ Time Frame: 25 weeks ]
    Body temperature
  • 11. Respiratory rate [ Time Frame: 25 weeks ]
    Respiratory rate
Eligibility Criteria
  • Ages Eligible for Study: 2 to 75 Years (Child, Adult, Older Adult)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
Criteria

Inclusion Criteria:

1. Age ≥2 years and ≤75 years.

2. Confirmed diagnosis of primary immunodeficiency (PI) disease as defined by the
European Society for Immunodeficiencies and Pan American Group for Immunodeficiency
and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or
agammaglobulinaemia. Note: The exact type of PI disease will be recorded.

3. Established on a consistent or stable mg/kg dose of any SCIG treatment for a minimum
of 3 months prior to Screening. Note: patients entering Cohort 3 must be on weekly
SCIG infusions for a minimum of 12 weeks.

4. Availability of the Immunoglobulin G (IgG) trough levels of 2 previous SCIG infusions
within 1 year of Screening, with 1 trough level obtained within 3 months prior to
enrollment, and maintenance of trough serum IgG levels

≥5.0 g/L in 2 previous infusions. Patients with no prior IgG trough level within 3
months prior to enrollment may use the Screening IgG trough level as their 2nd
reading.

5. Voluntarily given, fully informed signed informed consent. For patients under the
legal age of consent, voluntarily given, fully-informed, signed informed consent will
be provided by patient's parent or legal guardian, and assent will be provided by
patient (per age-appropriate Institutional Review Board [IRB] requirements).

6. Females of childbearing potential, who are not nursing and have no plans for pregnancy
during the course of the study, have been using at least 1 acceptable form of birth
control for a minimum of 30 days prior to the Screening visit and must agree to use at
least 1 acceptable method of contraception for 30 days after the last dose of
CUTAQUIG. Acceptable methods include: intrauterine device (IUD), hormonal
contraception, male or female condom, spermicide gel, diaphragm, sponge, cervical cap,
or abstinence.

7. For female patients of child-bearing potential, a negative result in a urine pregnancy
test conducted at the Screening visit.

8. Willingness to comply with all aspects of the protocol, including blood sampling, for
the duration of the study.

Exclusion Criteria:

1. Evidence of active infection within 4 weeks of Screening or during the Screening
Period.

2. Current or clinically-significant history of any cardiovascular, respiratory, hepatic,
renal, gastrointestinal, endocrine, neurological, immunological (excluding PI),
hematologic, and/or psychiatric disorder(s), or a history of any other illness that,
in the opinion of the Investigator, might confound the results of the study, or pose
additional risk to the patient by participation in the study.

3. Known history of adverse reactions to immunoglobulin A (IgA) in other products.

4. Body mass index (BMI) >40 kg/m2 for patients entering Cohort 2 or Cohort 3. There are
no BMI restrictions for Cohort 1.

5. Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived
products, or any component of the investigational product (such as Polysorbate 80).

6. Requirement of any routine premedication for IgG administration.

7. History of malignancies of lymphoid cells and immunodeficiency with lymphoma.

8. Severe liver function impairment (aspartate aminotransferase [AST] or alanine
aminotransferase [ALT] >3 times above upper limit of normal).

9. Known protein-losing enteropathies or clinically significant proteinuria.

10. Presence of renal function impairment (creatine >120 μM/L or creatinine >1.35 mg/dL),
or predisposition for acute renal failure (eg, any degree of preexisting renal
insufficiency or routine treatment with known nephritic drugs).

11. Treatment with oral or parenteral steroids for ≥30 days, or when given intermittently
or as bolus at daily doses ≥0.15 mg/kg when taken within 30 days of Screening. Note:
Short or intermittent courses of steroids (ie, a steroid burst) of >0.15 mg/kg/day is
allowed for treatment of a short-term condition such as an asthma exacerbation.

12. Treatment with immunosuppressive or immunomodulatory drugs (except Omalizumab).

13. Use of HYQVIA (Immune Globulin Infusion 10% [Human] with Recombinant Human
Hyaluronidase) within 3 months prior to first CUTAQUIG infusion.

14. Live viral vaccination (such as measles, rubella, mumps, and varicella) within 2
months prior to first CUTAQUIG infusion.

15. Exposure to blood or any blood product or derivative, other than subcutaneous IgG used
for regular PI disease treatment, within 3 months before the first CUTAQUIG infusion.

16. Treatment with any investigational medicinal product within 3 months prior to first
CUTAQUIG infusion. Note: Patients participating in Study SCGAM-03 will be allowed to
enter this study without the 3-month waiting period for an Investigational Product.
Patients receiving another SCIG product within 3 months prior to the first CUTAQUIG
infusion may be considered for enrollment after Sponsor approval.

17. Presence of any condition that is likely to interfere with the evaluation of CUTAQUIG
or satisfactory conduct of the trial.

18. Known or suspected to abuse alcohol, drugs, psychotropic agents, or other chemicals
within the past 12 months prior to first CUTAQUIG infusion.

19. Known active or chronic hepatitis B, hepatitis C, or HIV infection. Past hepatitis B
or hepatitis C infection that has been cured is allowed.

Contacts and Locations
Contacts

Contact: Mikaela Raymond 866-337-1868 ctgov@clinicalresearchmgt.com

Locations
Show 18 Study Locations
Sponsors and Collaborators

Octapharma

More Information
  • Responsible Party: Octapharma
  • ClinicalTrials.gov Identifier: NCT03939533 History of Changes
  • Other Study ID Numbers: SCGAM-06
  • First Posted: May 6, 2019 Key Record Dates
  • Last Update Posted: July 29, 2020
  • Last Verified: July 2020
  • Individual Participant
    Data (IPD) Sharing
    Statement:
  • Plan to Share IPD: Undecided
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Additional relevant MeSH terms: Immunologic Deficiency Syndromes