Primary Immune DeficiencyPhase 4NCT05070455
An Open Label, Multicenter Study to Evaluate the Pharmacokinetics, Efficacy and Safety of ASCENIV™ (IGIV) in Pediatric Subjects With Primary Immunodeficiency Diseases (PIDD)
A Phase 4 study testing Asceniv™ in people with Primary Immune Deficiency, run by ADMA Biologics, Inc. at 3 sites in United States.
Trial summary
This is a Phase IV, multicenter, open-label study of Asceniv™ administered as an intravenous infusion of Asceniv™ (IGIV) 300-800 mg/kg every 21 or 28 days in approximately 12 pediatric subjects with Primary Immunodeficiency Diseases (PIDD). Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Primary Immune Deficiency
- What is being tested?
- Asceniv™
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Sep 2022to 30 Jun 2023
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subject and/or legal guardian must be able to understand the study procedures, have agreed to participate in the study and have voluntarily signed an IEC/IRB approved written informed consent. The consent form or a specific assent form, where required, will be signed and dated by minors.
- Have confirmed and documented clinical diagnosis of primary immunodeficiency disease including but not limited to: common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM syndrome, or antibody deficiencies.
- Be male or female, and ≥ 2 years and < 12 years at the time of informed consent by subject or legal guardian.
- Have been receiving IGIV at a dose that has not been changed by > 25% of the mean dose on a mg/kg basis for at least 3 months prior to study entry.
- Have two trough levels of IgG in the last year (screening level may be used), and maintained a trough serum IgG level > 500 mg/dL on the previous 2 assessments prior to receiving Asceniv™. (The trough level must be at least 300 mg/dL above pre-treatment serum IgG levels; with exception for cases of X-linked agammaglobulinemia where no pre-treatment value is available. Documentation will need to include dose, treatment interval and trade name of the IGIV products used for the three doses prior to the first Asceniv™ infusion in this study.
- 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. Females of non-childbearing potential are defined as prepubertal girls.
Who cannot join
- Have a known hypersensitivity to immunoglobulin or any excipient in Asceniv™.
- Have a history of any severe anaphylactic or anaphylactoid reaction to blood or any blood-derived product.
- Have a specific Immunoglobulin A (IgA) deficiency (IgA ≤ 5 mg/dL and normal IgG and IgM), history of allergic reaction to products containing IgA or has demonstrable antibodies to IgA.
- Have uncompensated, hemodynamically significant, congenital or other heart disease. Including but not limited to acute coronary syndromes and chronic stable angina.
- Have a medical condition that is known to cause secondary immune deficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, or HIV infection.
- Have a significant T-cell or granulocyte deficiency in number or function (chronic or recurrent absolute neutrophil count <1000 x 109/L).
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- Have significant renal impairment (defined as an estimated Glomerular Filtration Rate ≤ 50 mL/min/1.73m2); or have a history of acute renal failure.
- Have abnormal liver function, defined as ALT or AST ≥ 2.5 x ULN.
- Have any chronic lung disease (uncontrolled or chronic, severe asthma, etc.)
- Have an infusion port, catheter, or other foreign body present (excluding PE tubes). Long-standing, infection-free ports may be permitted at the discretion of the Medical Monitor.
- Be planned or scheduled to undergo surgery during the course of study participation.
- Have ongoing failure to thrive per PI assessment.
- Be receiving chronic anti-coagulation therapy.
- Have a history of DVT, thrombotic or thrombo-embolic event, or are at increased risk for thrombotic event due to presence of, but not limited to, atrial fibrillation, disease or injury requiring prolonged immobilization, or other risk factor(s) including significant proteinuria or protein losing enteropathy.
- Current daily use of the following medications:
- corticosteroids (> 0.15 mg/kg/day of prednisone equivalent) Note: Intermittent corticosteroid use during the study is allowable, if medically necessary and approved by the ADMA Medical Director: i.e. 1 mg/kg twice a day for ten days to a maximum of 40 mg per dose
- immunomodulatory drugs (e.g. TNF (inhibitors -Enbrel, Humira, etc.) Xolair and Dupixent administration permitted.
- immunosuppressive drugs (excluding topical pimecrolimus (Elidel) and tacrolimus (Protopic))
- Administration of a hyperimmune or specialty high titer Immunoglobulin product (e.g. Cytogam, VZIG, HBIG, etc.) within 30 days of screening, or expectation that a hyperimmune Immunoglobulin product will be given during the course of the study.
- Have uncontrollable arterial hypertension.
- Have anemia at screening (hemoglobin <10 g/dL).
- Have a history of hemolysis while undergoing treatment with IGIV therapy.
- Be morbidly obese as indicated by a Body Mass Index (BMI) ≥40.
- Have an active viral or bacterial infection or symptoms/signs consistent with such an infection, within the two weeks prior to the Screening Visit. Subjects may be receiving antibiotics as long as signs/symptoms of infection have been absent for two weeks prior to the initial infusion of investigational product (IP).
- Have received any blood product (other than Immunoglobulin G) within 3 months prior to screening.
- Have received any RSV specific products, including palivizumab (Synagis®) within 3 months prior to screening.
- Have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
- Have an acute or chronic medical condition that, in the opinion of the investigator, may interfere with the conduct of the study.
- Have any condition judged by the study physician to preclude participation in the study, including any psychological disorder, which might hinder compliance.
- Have any laboratory assessment result that, in the opinion of the investigator, warrants exclusion from participation in the study.
- Are currently pregnant or nursing.
- Have acute hepatitis A, acute or chronic Hepatitis B or C, or HIV infection.
- Have received investigational product within 3 weeks of the anticipated first infusion of Asceniv™.
Trial locations and contacts
3 sites in 1 country.
Amy Doman contact
United States3 sites, 3 recruiting
- Not yet recruiting
Immunoe Research Centers
Centennial, Colorado, 80112
Coordinator
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Coordinator
- Not yet recruiting
University of Utah
Salt Lake City, Utah, 84112
Coordinator
About this study
This is a Phase IV, multicenter, open-label study of Asceniv™ administered as an intravenous infusion of Asceniv™ (IGIV) 300-800 mg/kg every 21 or 28 days in approximately 12 pediatric subjects with Primary Immunodeficiency Diseases (PIDD). The study will be conducted at 5-7 centers in the United States, with subjects receiving six (28 day cycle) or seven (21 day cycle) doses of Asceniv™ during the study.
- Conditions
- Primary Immune Deficiency
- Being tested
- Asceniv™
- 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.
Asceniv™Biological
Each subject will receive an intravenous infusion of Asceniv™ on Study Day 1 (required to be within 28 days of screening) and every 21 or 28 days thereafter according to their current interval of IGIV treatment. Subjects will receive Asceniv™ at the same dose or higher dose if medically appropriate (300-800 mg/kg), every 21 or 28 days for five months (seven or six doses respectively).
| Study group | Role | What they receive |
|---|---|---|
| Asceniv | Experimental | Asceniv™ will be given as an intravenous infusion at the same dose, or higher dose where medically appropriate, as the subject's previous IV Immunoglobulin G treatment (300-800 mg/kg) every 21 or 28 days. Receives: Biological: Asceniv™ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cmax
Pharmacokinetic measure at 6th or 7th infusion
Measured: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Tmax
Pharmacokinetic measure at 6th or 7th infusion
Measured: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
AUC(0-ʈ)
Pharmacokinetic measure at 6th or 7th infusion
Measured: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
AUC(0-∞)
Pharmacokinetic measure at 6th or 7th infusion
Measured: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Terminal phase elimination half-life (ʈ½)
Pharmacokinetic measure at 6th or 7th infusion
Measured: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Terminal phase elimination rate (λZ)
Pharmacokinetic measure at 6th or 7th infusion
Measured: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Secondary outcomes (7)
Total IgG Trough
Levels taken before each infusion
Measured: At each visit through study completion, up to approximately 7 months
IgG Subclasses
Levels of subclasses 1-4 before infusion
Measured: Prior to first and last infusions
Antibodies
Levels of specific antibodies (anti-pneumococcal capsular polysaccharide, anti-haemophilus influenzae b, and anti-RSV neutralizing antibody)
Measured: Prior to first and last infusions
Infections
Number of infections of any kind, serious and non-serious
Measured: Up to approximately 7 months
Serious Bacterial Infections
Incidence of Serious Bacterial Infections
Measured: Up to approximately 7 months
Other Infections
Incidence of infections other than Serious Bacterial Infections
Measured: Up to approximately 7 months
Hospitalizations
Number of hospitalizations due to infections
Measured: Up to approximately 7 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2021
- Study start1 Sep 2022
- Primary completion31 Mar 2023 (estimated)
- Study completion30 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune DeficiencyStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ADMA Biologics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05070455
- Sponsor's protocol ID
- ADMA-004
- Status last verified
- Sep 2022
- First submitted
- 23 Sep 2021
- Last updated
- 16 Sep 2022
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Multicenter Study to Evaluate the Pharmacokinetics, Efficacy and Safety of ASCENIV™ (IGIV) in Pediatric Subjects With Primary Immunodeficiency Diseases (PIDD)
- ClinicalTrials.gov ID
- NCT05070455
- Acronym / sponsor ID
- ADMA-004
- Conditions
- Primary Immune Deficiency
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- ADMA Biologics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 31 Mar 2023 (estimated)
- Study completion
- 30 Jun 2023 (estimated)
- First posted
- 7 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase IV, multicenter, open-label study of Asceniv™ administered as an intravenous infusion of Asceniv™ (IGIV) 300-800 mg/kg every 21 or 28 days in approximately 12 pediatric subjects with Primary Immunodeficiency Diseases (PIDD). The study will be conducted at 5-7 centers in the United States, with subjects receiving six (28 day cycle) or seven (21 day cycle) doses of Asceniv™ during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Asceniv | Experimental | Biological: Asceniv™Asceniv™ will be given as an intravenous infusion at the same dose, or higher dose where medically appropriate, as the subject's previous IV Immunoglobulin G treatment (300-800 mg/kg) every 21 or 28 days. |
Who may qualify
- Ages
- 2 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Subject and/or legal guardian must be able to understand the study procedures, have agreed to participate in the study and have voluntarily signed an IEC/IRB approved written informed consent. The consent form or a specific assent form, where required, will be signed and dated by minors.
- Have confirmed and documented clinical diagnosis of primary immunodeficiency disease including but not limited to: common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM syndrome, or antibody deficiencies.
- Be male or female, and ≥ 2 years and < 12 years at the time of informed consent by subject or legal guardian.
- Have been receiving IGIV at a dose that has not been changed by > 25% of the mean dose on a mg/kg basis for at least 3 months prior to study entry.
- Have two trough levels of IgG in the last year (screening level may be used), and maintained a trough serum IgG level > 500 mg/dL on the previous 2 assessments prior to receiving Asceniv™. (The trough level must be at least 300 mg/dL above pre-treatment serum IgG levels; with exception for cases of X-linked agammaglobulinemia where no pre-treatment value is available. Documentation will need to include dose, treatment interval and trade name of the IGIV products used for the three doses prior to the first Asceniv™ infusion in this study.
Key exclusion criteria (33 listed)
- Have a known hypersensitivity to immunoglobulin or any excipient in Asceniv™.
- Have a history of any severe anaphylactic or anaphylactoid reaction to blood or any blood-derived product.
- Have a specific Immunoglobulin A (IgA) deficiency (IgA ≤ 5 mg/dL and normal IgG and IgM), history of allergic reaction to products containing IgA or has demonstrable antibodies to IgA.
- Have uncompensated, hemodynamically significant, congenital or other heart disease. Including but not limited to acute coronary syndromes and chronic stable angina.
- Have a medical condition that is known to cause secondary immune deficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, or HIV infection.
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 |
|---|---|---|
| Asceniv™Each subject will receive an intravenous infusion of Asceniv™ on Study Day 1 (required to be within 28 days of screening) and every 21 or 28 days thereafter according to their current interval of IGIV treatment. Subjects will receive Asceniv™ at the same dose or higher dose if medically appropriate (300-800 mg/kg), every 21 or 28 days for five months (seven or six doses respectively). | Biological | Asceniv |
Primary outcome
Cmax
Time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Pharmacokinetic measure at 6th or 7th infusion
Tmax
Time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Pharmacokinetic measure at 6th or 7th infusion
AUC(0-ʈ)
Time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Pharmacokinetic measure at 6th or 7th infusion
AUC(0-∞)
Time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Pharmacokinetic measure at 6th or 7th infusion
Terminal phase elimination half-life (ʈ½)
Time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Pharmacokinetic measure at 6th or 7th infusion
Terminal phase elimination rate (λZ)
Time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule
Pharmacokinetic measure at 6th or 7th infusion
7 secondary outcomes
Total IgG Trough
Time frame: At each visit through study completion, up to approximately 7 months
IgG Subclasses
Time frame: Prior to first and last infusions
Antibodies
Time frame: Prior to first and last infusions
Infections
Time frame: Up to approximately 7 months
Serious Bacterial Infections
Time frame: Up to approximately 7 months
Other Infections
Time frame: Up to approximately 7 months
Hospitalizations
Time frame: Up to approximately 7 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2022.
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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.
Amy Doman contact
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Asceniv™ in people with Primary Immune Deficiency, run by ADMA Biologics, Inc. at 3 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 6 inclusion and 33 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: status not confirmed, as of the sponsor's last update on 16 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rebecca Avila (561-989-5853, reavila@admabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cmax (time frame: At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule). The study lists 6 primary outcomes in all. 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 16 Sep 2022.