Primary Immune Deficiency DisorderPhase 3NCT03939533
Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases
A Phase 3 study testing CUTAQUIG in people with Primary Immune Deficiency Disorder, run by Octapharma at 18 sites in United States.
Trial 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 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?
- CUTAQUIG
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 18 sitesUnited States
- Study dates
- 17 Oct 2019to 3 Jan 2022
- 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 ≥2 years and ≤75 years.
- 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.
- 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.
- 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.
- 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).
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- 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.
- For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the Screening visit.
- Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
Who cannot join
- Evidence of active infection within 4 weeks of Screening or during the Screening Period.
- 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.
- Known history of adverse reactions to immunoglobulin A (IgA) in other products.
- Body mass index (BMI) >40 kg/m2 for patients entering Cohort 2 or Cohort 3. There are no BMI restrictions for Cohort 1.
- 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.
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- History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
- Severe liver function impairment (aspartate aminotransferase [AST] or alanine aminotransferase [ALT] >3 times above upper limit of normal).
- Known protein-losing enteropathies or clinically significant proteinuria.
- 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).
- 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.
- Treatment with immunosuppressive or immunomodulatory drugs (except Omalizumab).
- Use of HYQVIA (Immune Globulin Infusion 10% [Human] with Recombinant Human Hyaluronidase) within 3 months prior to first CUTAQUIG infusion.
- Live viral vaccination (such as measles, rubella, mumps, and varicella) within 2 months prior to first CUTAQUIG infusion.
- 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.
- 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.
- Presence of any condition that is likely to interfere with the evaluation of CUTAQUIG 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 CUTAQUIG infusion.
- Known active or chronic hepatitis B, hepatitis C, or HIV infection. Past hepatitis B or hepatitis C infection that has been cured is allowed.
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
Octapharma Research Site
Albany, Georgia, 31707
Octapharma Research Site
Asheville, North Carolina, 28801
Octapharma Research Site
Bellingham, Washington, 98225
Octapharma Research Site
Centennial, Colorado, 80112
Octapharma Research Site
Chevy Chase, Maryland, 20815
Octapharma Research Site
Cincinnati, Ohio, 45231
Octapharma Research Site
Dallas, Texas, 75225
Octapharma Research Site
Irvine, California, 92697
Octapharma Research Site
Louisville, Kentucky, 40215
Octapharma Research Site
Mayfield, Ohio, 44124
Octapharma Research Site
North Palm Beach, Florida, 33408
Octapharma Research Site
Papillion, Nebraska, 68046
Octapharma Research Site
Pittsburgh, Pennsylvania, 15241
Octapharma Research Site
Santa Barbara, California, 93105
Octapharma Research Site
Scottsdale, Arizona, 85251
Octapharma Research Site
St Louis, Missouri, 63141
Octapharma Research Site
St. Petersburg, Florida, 33701
Octapharma Research Site
Toledo, Ohio, 43617
About this study
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
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
- Conditions
- Primary Immune Deficiency Disorder
- Being tested
- CUTAQUIG
- 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.
CUTAQUIGDrug
Human normal immunoglobulin
| Study group | Role | What they receive |
|---|---|---|
| Increased Volume Cohort - Cohort 1 | Experimental | Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks Receives: Drug: CUTAQUIG |
| Increased Infusion Rate Cohort - Cohort 2 | Experimental | Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks Receives: Drug: CUTAQUIG |
| Every Other Week Dosing Cohort - Cohort 3 | Experimental | Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose Receives: Drug: CUTAQUIG |
What is being measured
The primary outcome is the main question the study is designed to answer.
IgG Trough Levels From Baseline to End of Study (28 Weeks)
Mean change from baseline in individual total IgG trough levels in cohort 3 from weekly infusions to end of study (28 weeks) every other week infusions, and for cohort 1 and cohort 2 (weekly infusions) change from baseline through study completion (28 weeks)
Measured: Through study completion, up to 28 weeks
Secondary outcomes (4)
Serious Bacterial Infection Rates
Number of subjects who reported SBIs during the study
Measured: Through study completion, up to 28 weeks
Time to Resolution of Infections
The amount of days it took for infectious disease occurrence and resolution for subjects
Measured: Through study completion, 28 weeks
Antibiotic Usage
Amount of subjects treated with antibiotics during the study
Measured: Through study completion, up to 28 weeks
Number of Antibiotic Treatment Episodes Annualized
Total number of treatment episodes annualized calculated as the sum of all unique episodes of antibiotics of all subjects from first dose day of cutaquig to last study visit/number of person years exposure
Measured: Through study completion, up to 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2019
- Study start17 Oct 2019
- Primary completion3 Jan 2022
- Study completion3 Jan 2022
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.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03939533
- Sponsor's protocol ID
- SCGAM-06
- Status last verified
- Nov 2023
- First submitted
- 3 May 2019
- Last updated
- 7 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03939533
- Acronym / sponsor ID
- SCGAM-06
- Conditions
- Primary Immune Deficiency Disorder
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 17 Oct 2019 (actual)
- Primary completion
- 3 Jan 2022 (actual)
- Study completion
- 3 Jan 2022 (actual)
- First posted
- 6 May 2019 (actual)
- Results
- Posted 7 Nov 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Increased Volume Cohort - Cohort 1 | Experimental | Drug: CUTAQUIGIncreased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks |
| Increased Infusion Rate Cohort - Cohort 2 | Experimental | Drug: CUTAQUIGIncreased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks |
| Every Other Week Dosing Cohort - Cohort 3 | Experimental | Drug: CUTAQUIGEvery other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Age ≥2 years and ≤75 years.
- 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.
- 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.
- 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.
Key exclusion criteria (19 listed)
- Evidence of active infection within 4 weeks of Screening or during the Screening Period.
- 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.
- Known history of adverse reactions to immunoglobulin A (IgA) in other products.
- Body mass index (BMI) >40 kg/m2 for patients entering Cohort 2 or Cohort 3. There are no BMI restrictions for Cohort 1.
- 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 |
|---|---|---|
| CUTAQUIGHuman normal immunoglobulin | Drug | Every Other Week Dosing Cohort - Cohort 3, Increased Infusion Rate Cohort - Cohort 2, Increased Volume Cohort - Cohort 1 |
Primary outcome
IgG Trough Levels From Baseline to End of Study (28 Weeks)
Time frame: Through study completion, up to 28 weeks
Mean change from baseline in individual total IgG trough levels in cohort 3 from weekly infusions to end of study (28 weeks) every other week infusions, and for cohort 1 and cohort 2 (weekly infusions) change from baseline through study completion (28 weeks)
4 secondary outcomes
Serious Bacterial Infection Rates
Time frame: Through study completion, up to 28 weeks
Time to Resolution of Infections
Time frame: Through study completion, 28 weeks
Antibiotic Usage
Time frame: Through study completion, up to 28 weeks
Number of Antibiotic Treatment Episodes Annualized
Time frame: Through study completion, up to 28 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing CUTAQUIG in people with Primary Immune Deficiency Disorder, run by Octapharma at 18 sites in United States.
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 9 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 7 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States. 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 7 Nov 2023). 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?
IgG Trough Levels From Baseline to End of Study (28 Weeks) (time frame: Through study completion, up to 28 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Nov 2023.