- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07794735
A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins
Multicenter, Prospective, Open-Label Trial to Evaluate PK, Safety, and Tolerability of TAK-664 in IG Treatment-Naïve Participants With Primary Immunodeficiency Diseases
Primary immunodeficiency disease (or PIDD) is a group of conditions in which the immune system does not work properly. Some people with PIDD do not make enough antibodies. Antibodies are proteins that help to either protect the body from infections or fight infections. The main type of antibodies that helps protect the body from infection is IgG. People who do not make enough IgG often need medical treatment that gives the body those antibodies. This treatment is called IgG replacement therapy. It can be given through a vein (intravenous or IV) or under the skin (subcutaneous SC). TAK-664 is approved worldwide for SC IG replacement therapy.
The study wants to learn more about TAK-664 given to people with PIDD who have not yet been treated with IG (called 'treatment-naïve').
The main aim of the study is to check if giving TAK-664 daily for 5 days, and then once more 3 days later (Day 8), can raise IgG to the target level and keep IgG there.
Another aim of the study is to learn if TAK-664 given once a week can keep the IgG at the target levels during the study. The study also wants to find out how much the IgG levels raise and learn how many infections occur and how they are treated.
Studieoversikt
Status
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Fase
- Fase 4
Kontakter og plasseringer
Studiekontakt
- Navn: Takeda Contact
- Telefonnummer: +1-877-825-3327
- E-post: medinfoUS@takeda.com
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
- Eldre voksen
Tar imot friske frivillige
Beskrivelse
Inclusion Criteria:
- The participant or the participant's legally authorized representative is willing and able to understand and fully comply with trial procedures and requirements, in the opinion of the investigator.
- The participant or the participant's legally authorized representative has provided informed consent or assent, if applicable (that is, in writing, documented via a signed and dated informed consent form [ICF]), and any required privacy authorization before the initiation of any trial procedures.
- The participant is at least 6 years of age at the time of signing the ICF or assent, if applicable.
- The participant has a documented diagnosis of a form of primary humoral immunodeficiency involving a defect in antibody formation and requiring IG replacement, as defined according to the International Union of Immunological Societies (IUIS) Committee (Human Inborn Errors of Immunity: 2024 update on the phenotypic classification from the IUIS Expert Committee).
- The participant has never received immunoglobulin (IG) replacement treatment (that is, no prior IG replacement therapy).
- The participant must have an immunoglobulin G (IgG) level of less than or equal to (<=) 400 milligrams per deciliter (mg/dL) at screening.
- If a participant has the potential to become pregnant, they must have a negative pregnancy test at screening and agree to employ a highly effective contraceptive measure throughout the course of the trial and for at least 30 days after the last administration of TAK-664.
Exclusion Criteria:
- The participant has significant proteinuria (greater than or equal to [>=] 3 and/or known urinary protein loss greater than [>]1 gram per 24 [g/24] hours or nephrotic syndrome), has acute renal failure, is on dialysis, and/or has severe renal impairment on screening laboratory testing (blood urea nitrogen [BUN] or creatinine >2.5 × upper limit of the normal range [ULN]).
- The participant has immunoglobulin A (IgA) deficiency (IgA less than [<] 0.07 grams per liter [g/L]) associated with known anti-IgA antibodies and a history of hypersensitivity.
- The participant has a condition(s) that could alter protein catabolism and/or IgG use (for example, protein-losing enteropathies or nephrotic syndrome).
- The participant has a known history of a positive result or is positive at screening for one or more of the following: hepatitis B virus surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), or PCR for human immunodeficiency virus (HIV) Type 1 and Type 2. Note: Cured participants with a history of hepatitis C infection who have a negative PCR test at screening are eligible.
- The participant has a known history or current diagnosis of thromboembolic episodes, such as deep vein thrombosis, pulmonary embolism, myocardial infarction, ischemic stroke, transient ischemic attack, or peripheral artery disease, within 6 months before screening.
The participant has a history of malignancy with less than 2 years of complete remission before screening or active malignancy requiring chemotherapy and/or radiotherapy.
Note: Participants with adequately treated basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or stable prostate cancer not requiring treatment are eligible.
- The participant has congestive heart failure (New York Heart Association class III/IV), unstable angina, unstable cardiac arrhythmias, or uncontrolled hypertension (defined as diastolic blood pressure >100 millimeters of mercury (mm Hg) and/or systolic blood pressure >160 mm Hg during the screening period confirmed on 2 measures >30 minutes apart).
- The participant has an acquired or inherited thrombophilic disorder, such as protein C deficiency, protein S deficiency, antithrombin deficiency, or primary antiphospholipid antibody syndrome.
- The participant has malignancies of lymphoid cells, such as chronic lymphocytic leukemia and non-Hodgkin's lymphoma, which may lead to secondary hypogammaglobulinemia.
- The participant has a medical condition, laboratory finding, or physical examination finding that precludes participation or clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with the successful completion of the trial or place the participant at undue medical risk.
The participant has abnormal laboratory values at screening that meet any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
- Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2.5 × ULN for the testing laboratory.
- Persistent severe neutropenia (defined as an absolute neutrophil count (ANC) <=500 per cubic millimeters [/mm^3]).
- The participant has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site, at the discretion of the investigator.
- The participant has a known or suspected intolerance or hypersensitivity to compounds closely related to TAK-664 or any of the stated ingredients.
- The participant has an active infection and is receiving systemic antibiotic therapy for the treatment of infection at the time of screening.
The participant is required to take or has taken:
- Immunomodulatory/immunosuppressive agents that include but are not limited to specific complement inhibitors, rituximab, neonatal Fc receptor inhibitors (for example, efgartigimod), and chemotherapeutic drugs, within 12 months of screening or 5 times the half-life (t1/2) plus 6 months before screening, whichever is longer.
- Long-term systemic corticosteroids defined as a daily dose >1 mg/kg of prednisone-equivalent/day for >30 days within 3 months of screening. Note: Participants using short-pulse dose corticosteroid course and oral daily corticosteroids <=10 milligrams per day (mg/day) prednisone-equivalent are allowed.
- The participant has received a live-attenuated viral vaccination within 3 months of screening.
- The participant has known substance or prescription drug abuse within 12 months of screening.
- The participant is pregnant or breastfeeding at the time of screening or planning to become pregnant during participation in the trial.
- The participant has a current or relevant history of physical or psychiatric illness or any medical disorder that may require treatment or make the participant unlikely to fully complete the trial or any condition that presents undue risk from the trial intervention or procedures.
- The participant has participated or is scheduled to participate in another clinical trial involving a trial intervention or investigational device within 30 days before screening and during the trial.
- The participant is a trial site employee, an immediate family member (for example, spouse, parent, child, or sibling), or is in a dependent relationship with a trial site employee who is involved in the conduct of this trial or may consent under duress.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Eksperimentell: TAK-664, 150 mg/kg
Participants will receive a loading regimen of TAK-664 infusions consisting of 5 consecutive daily doses of 150 milligrams per kilogram (mg/kg) from Days 1 to 5 during the loading period, followed by maintenance period with the same dose of weekly infusion from Week 1 to Week 8.
|
Participants will receive TAK-664 infusion.
Andre navn:
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Percentage of Participants who Achieve a Total Serum IgG Trough Level of >=500 mg/dL
Tidsramme: At Day 15
|
Serum trough levels of total IgG will be determined by using validated assay methods.
|
At Day 15
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Percentage of Participants who Achieve a Total Serum IgG Trough level of >=500 mg/dL on Day 8
Tidsramme: At Day 8
|
Serum trough levels of total IgG will be determined by using validated assay methods.
|
At Day 8
|
|
Percentage of Participants who Achieve a Total Serum IgG Trough level of >=500 mg/dL at Weeks 4, 6, and 9
Tidsramme: At Weeks 4, 6, and 9
|
Serum trough levels of total IgG will be determined by using validated assay methods.
|
At Weeks 4, 6, and 9
|
|
Percentage of Participants who Achieve a Total Serum IgG Trough Level of >=700 mg/dL
Tidsramme: At Weeks 6, and 9
|
Serum trough levels of total IgG will be determined by using validated assay methods.
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At Weeks 6, and 9
|
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Change in Total Serum IgG Trough Level of >=100 mg/dL From Baseline to Days 8 and 15
Tidsramme: Baseline up to Days 8 and 15
|
Serum trough levels of total IgG will be determined by using validated assay methods.
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Baseline up to Days 8 and 15
|
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Annualized Rate of All Infections
Tidsramme: From first dose of study drug up to end of trial (EOT) (up to 10 weeks)
|
The annual rate of infections will be calculated as the mean number of infections per participant per year.
|
From first dose of study drug up to end of trial (EOT) (up to 10 weeks)
|
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Annualized Rate of Acute Serious Bacterial Infections (ASBIs)
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
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The ASBI rate will be calculated as the mean number of acute serious bacterial infections per participant per year.
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From first dose of study drug up to EOT (up to 10 weeks)
|
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Duration of Infections
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
|
The duration of an infection will be calculated as the number of days between the AE start date and the AE end date.
Infections will be extracted from adverse events (AE) reporting.
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From first dose of study drug up to EOT (up to 10 weeks)
|
|
Annualized Rate of Days on Oral or Parenteral Antibiotics for Prophylaxis or Treatment of Infections
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
|
Annualized rate of days on oral or parenteral antibiotics will be calculated as the total number of days participants received oral or parenteral antibiotics for prophylaxis or treatment of infections divided by the total participant-years of follow-up.
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From first dose of study drug up to EOT (up to 10 weeks)
|
|
Number of Hospitalizations Due to Infections
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
|
Number of hospitalizations due to infections will be standardized to per year.
|
From first dose of study drug up to EOT (up to 10 weeks)
|
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Total Number of Days of Hospital Stay Due to Infections
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
|
Total number of days of hospital stay due to infections will be standardized to per year.
|
From first dose of study drug up to EOT (up to 10 weeks)
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Related to TAK-664
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
|
An adverse event (AE) is any untoward medical occurrence in a clinical trial participant, temporally associated with the use of the trial intervention, whether or not the occurrence is considered related to the trial intervention.
TEAEs are defined as AEs that started at or after the initiation of the first administration of TAK-664.
Serious TEAEs are the AEs that result in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged in-patient hospitalization; congenital anomaly/birth defect or are otherwise considered medically important.
Any TEAE that is recorded by the investigator as related to TAK-664 is considered as TEAE related to TAK-664.
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From first dose of study drug up to EOT (up to 10 weeks)
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Number of Participants With TEAEs Temporally Associated With TAK-664
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
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Temporally associated TEAEs are defined as TEAEs which begin during or within 72 hours of the completion of TAK-664 infusion.
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From first dose of study drug up to EOT (up to 10 weeks)
|
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Number of Infusions of TAK-664 Without Infusion Rate Reduction, Interruption, or Infusion Withdrawals due to TAK-664-related TEAEs
Tidsramme: From first dose of study drug up to EOT (up to 10 weeks)
|
Number of infusions of TAK-664 without infusion rate reduction, interruption, or infusion withdrawals due to TAK-664-related TEAEs will be reported.
|
From first dose of study drug up to EOT (up to 10 weeks)
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Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Study Director, Takeda
Publikasjoner og nyttige lenker
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
- Genetiske sykdommer, medfødte
- Sykdommer i immunsystemet
- Immunologiske mangelsyndromer
- Medfødte, arvelige og neonatale sykdommer og abnormiteter
- Primære immunsviktsykdommer
- Aminosyrer, peptider og proteiner
- Proteiner
- Farmasøytiske preparater
- Immunoglobuliner
- Immunoproteiner
- Blodproteiner
- Serumglobuliner
- Globuliner
- gamma-globuliner
- Løsninger
- Hizentra
Andre studie-ID-numre
- TAK-664-4005
- 2026-000263-39 (EudraCT-nummer)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
Tilgangskriterier for IPD-deling
IPD-deling Støtteinformasjonstype
- STUDY_PROTOCOL
- SEVJE
- ICF
- CSR
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