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

• Register: ClinicalTrials.gov
• Last refreshed on: 13 November 2023
• Main ID: NCT03939533
• Date of registration: 03/05/2019
• Prospective Registration: Yes
• Primary sponsor: Octapharma
• Public title: Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases
• Scientific 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
• Date of first enrolment: October 17, 2019
• Target sample size: 64
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03939533
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

United States

Key inclusion & exclusion 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.



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

Health Condition(s) or Problem(s) studied

Primary Immune Deficiency Disorder

Intervention(s)

Drug: CUTAQUIG

Primary Outcome(s)

IgG Trough Levels From Baseline to End of Study (28 Weeks) [Time Frame: Through study completion, up to 28 weeks]

Secondary Outcome(s)

Antibiotic Usage [Time Frame: Through study completion, up to 28 weeks]

Serious Bacterial Infection Rates [Time Frame: Through study completion, up to 28 weeks]

Number of Antibiotic Treatment Episodes Annualized [Time Frame: Through study completion, up to 28 weeks]

Time to Resolution of Infections [Time Frame: Through study completion, 28 weeks]

Secondary ID(s)

SCGAM-06

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 07/11/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03939533