A Phase 1/2 interventional study of BAX 888 in Hemophilia A, sponsored by Baxalta now part of Shire. Completed at 26 sites in 6 countries. Open to male participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-09-11.
Sponsored by Baxalta now part of Shire (part of Takeda) · Phase 1/2, Interventional, and Prevention
The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults.
Participants will receive one infusion with BAX 888 at the hemophilia treatment center. During the study, participants will visit their study clinic multiple times.
This study consists of 3 cohorts. Participants will be assigned to 1 of 3 dose cohorts with a minimum of 24 hours between dosing of each participant. Initially, 2 participants will be dosed in a cohort, with up to a total of 5 participants if the cohort is expanded based on safety and activity levels data.
Dose escalation: After dosing first 2 participants in cohort 1 the decision will be made on the following: If week 4 FVIII activity levels of both participants are less than (\<) 2%, then dose escalation to cohort 2 will be triggered with no further dosing in cohort 1. If FVIII activity levels >=2% are observed in at least 1 participant among the 2 participants the decision to escalate dose or expand the cohort with dosing of additional participants will be based on all available data through Week 14.
Dose expansion: After dose escalation and administration of BAX 888 to the first 2 participants in 3 cohorts: If sustained Week 14 FVIII activity levels are >=30% are not achieved in both participants (first 2 participants in cohorts 1 and 2) then escalation to immediate next cohort will be triggered after Data Monitoring Committee (DMC) review of all available safety and FVIII activity levels data. For cohort 3 dosing of additional participants will be paused until further review of available data. If sustained Week 14 FVIII levels are >=30% in at least 1 of the 2 participants (first 2 participant in cohort 1, 2, 3) then expansion of cohorts 1, 2 (with up to 5 participants), 3 (with up to 3 additional participants) will be initiated with dosing or study could be completed with no further dosing.
23 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 4 is below the median of 30 across 499 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →Baxalta now part of Shire is the lead sponsor of 111 studies on the registry; none are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.
First submitted before Baxalta became part of Takeda.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Evidence of markers of potential underlying risk for mediated hepatic disease:
Markers of hepatic inflammation or cirrhosis as evidenced by 1 or more of the following:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0).
Drug: BAX 888
Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0).
Drug: BAX 888
Participants will receive a single peripheral IV infusion of BAX 888 in Cohort 1 and 2 Day 0.
Also known as: Adeno-associated virus serotype 8 (AAV8) vector expressing B-domain deleted Factor VIII (BDD-FVIII), BAX888
Number of Participants With BAX 888-Related Adverse Events (AEs)
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
Time frame: From first dose up to end of the study (approximately 6 years)
Change From Baseline in Circulating Plasma FVIII Activity Level
Change from baseline in circulating plasma FVIII activity level, based on one-stage clotting assay was assessed.
Time frame: Baseline, up to Month 60
Number of Participants With Clinically Significant Change From Baseline in Circulating Plasma FVIII Antigen Level
Change from baseline in circulating plasma FVIII antigen (protein) levels were to be assessed and number of participants with clinically significant change as determined by the principal investigator (PI) were reported.
Time frame: Baseline, up to Month 60
Annualized Bleed Rate (ABR)
ABR in comparison to before gene transfer will be assessed. A bleed is defined as subjective or objective evidence of bleeding which may or may not require treatment with FVIII. ABR was calculated as (number of bleeding episodes/observed treatment period in days)\*365.25.
Time frame: Up to approximately 6 years 4 months
Percentage of Participants With a Reduction in Consumption of Exogenous FVIII
The reduction in consumption of exogenous FVIII was assessed by comparing the amount of exogenous FVIII taken at earliest time point available (prior to BAX 888 infusion) with the amount taken at the last post-infusion timepoint available, during the study. Percentage of participants with reduction in consumption of exogenous FVIII are reported.
Time frame: Up to approximately 6 years 4 months
Number of Participants Who Developed Inhibitory Antibodies to FVIII
Participants were assessed to check if they developed inhibitory antibodies to FVIII.
Time frame: Up to approximately 6 years 4 months
Number of Participants Who Developed Total Binding Antibodies to FVIII
Participants were assessed to check if they developed total binding antibodies to FVIII (Immunoglobulin G \[IgG\], Immunoglobulin M \[IgM\]).
Time frame: Up to approximately 6 years 4 months
Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and FVIII Proteins
The humoral (antibody-mediated) and cell-mediated immune response to adeno-associated virus (AAV8) (the vector) and FVIII proteins, was assessed. Humoral Immune Response: It is indicated by presence of specific antibodies. The anti-AAV8 binding antibodies, IgG or IgM were measured by the enzyme-linked immunosorbent assay (ELISA) method. Neutralizing antibodies were measured by a cell-based luminescent assay. Cell-mediated Immune response: The AAV8 and FVIII specific cell mediated immunity was assessed using validated interferon-γ (IFN-γ) enzyme-linked immunosorbent spot (ELISpot) assays. This assay tests the human T-cell recall response to the AAV8 and FVIII proteins. These proteins were called antigens for these tests (AAV8 peptide pools 1, 2, 3 and two pooled test antigens (1 and 2) for FVIII). Number of participants who had humoral and/or cell mediated immune response to AAV8 and FVIII proteins, are reported by humoral and cell mediated immune response categories.
Time frame: Up to approximately 6 years 4 months
Surveillance of AAV8 Genome Shedding
Surveillance of AAV8 genome shedding in blood, saliva, semen, stool and urine until two consecutive negative results were assessed.
Time frame: Blood: Day 1, weekly at Clinic Visits between Weeks 1-15, and at Months 4 and 5; Saliva, Semen, and Stool: Day 1 and Week 1; Urine: Day 1 and Weeks 1,2,3
A total of 4 participants took part in the study globally from 27 February 2018 to 09 July 2024.
| Milestone | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Started | 2 | 2 |
| Completed | 2 | 1 |
| Not completed | 0 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
| Participants | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Number of Participants With BAX 888-Related Adverse Events (AEs) | 2 | 2 |
Change from baseline in circulating plasma FVIII activity level, based on one-stage clotting assay was assessed.
| International Units per deciliter(IU/dL) | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Change From Baseline in Circulating Plasma FVIII Activity Level | 11.40 ± 1.131 | 248.30 ± NA |
Change from baseline in circulating plasma FVIII antigen (protein) levels were to be assessed and number of participants with clinically significant change as determined by the principal investigator (PI) were reported.
| Participants | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Number of Participants With Clinically Significant Change From Baseline in Circulating Plasma FVIII Antigen Level | 2 | 0 |
ABR in comparison to before gene transfer will be assessed. A bleed is defined as subjective or objective evidence of bleeding which may or may not require treatment with FVIII. ABR was calculated as (number of bleeding episodes/observed treatment period in days)\*365.25.
| bleeds per year | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Annualized Bleed Rate (ABR) | 1.0 ± 1.41 | 0.5 ± 0.71 |
The reduction in consumption of exogenous FVIII was assessed by comparing the amount of exogenous FVIII taken at earliest time point available (prior to BAX 888 infusion) with the amount taken at the last post-infusion timepoint available, during the study. Percentage of participants with reduction in consumption of exogenous FVIII are reported.
| percentage of participants | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Percentage of Participants With a Reduction in Consumption of Exogenous FVIII | 0.0 | 0.0 |
Participants were assessed to check if they developed inhibitory antibodies to FVIII.
| Participants | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Number of Participants Who Developed Inhibitory Antibodies to FVIII | 0 | 0 |
Participants were assessed to check if they developed total binding antibodies to FVIII (Immunoglobulin G \[IgG\], Immunoglobulin M \[IgM\]).
| Participants | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Number of Participants Who Developed Total Binding Antibodies to FVIII | 0 | 0 |
The humoral (antibody-mediated) and cell-mediated immune response to adeno-associated virus (AAV8) (the vector) and FVIII proteins, was assessed. Humoral Immune Response: It is indicated by presence of specific antibodies. The anti-AAV8 binding antibodies, IgG or IgM were measured by the enzyme-linked immunosorbent assay (ELISA) method. Neutralizing antibodies were measured by a cell-based luminescent assay. Cell-mediated Immune response: The AAV8 and FVIII specific cell mediated immunity was assessed using validated interferon-γ (IFN-γ) enzyme-linked immunosorbent spot (ELISpot) assays. This assay tests the human T-cell recall response to the AAV8 and FVIII proteins. These proteins were called antigens for these tests (AAV8 peptide pools 1, 2, 3 and two pooled test antigens (1 and 2) for FVIII). Number of participants who had humoral and/or cell mediated immune response to AAV8 and FVIII proteins, are reported by humoral and cell mediated immune response categories.
| Participants | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Humoral: Binding Ab to AAV8 IgG Titer | 2 | 1 |
| Humoral: Neutralising Ab to AAV8 Titer | 2 | 1 |
| Cell-Mediated: AAV8 Peptide Pool 1 Mean | 0 | 1 |
| Cell-Mediated: AAV8 Peptide Pool 2 Mean | 1 | 1 |
| Cell-Mediated: AAV8 Peptide Pool 3 Mean | 1 | 1 |
| Cell-Mediated: FVIII Peptide Pool 1 Mean | 0 | 0 |
| Cell-Mediated: FVIII Peptide Pool 2 Mean | 1 | 1 |
Surveillance of AAV8 genome shedding in blood, saliva, semen, stool and urine until two consecutive negative results were assessed.
| Genome copies per 100 ng of sample | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| Blood: Day 1 | — | 3684434.0 ± NA |
| Blood: Week 1 | 3634.0 ± NA | 18608.0 ± 664.68 |
| Blood: Week 2 | 2076.0 ± 295.57 | 10670.0 ± 3900.40 |
| Blood: Week 3 | 2069.0 ± 741.05 | 9845.0 ± 6488.41 |
| Blood: Week 4 | 1098.0 ± NA | 9033.5 ± 4978.74 |
| Blood: Week 5 | 1483.0 ± 1097.43 | 7884.0 ± 288.50 |
| Blood: Week 6 | 904.0 ± 335.17 | 5372.5 ± 441.94 |
| Blood: Week 7 | 1004.0 ± 656.20 | 6808.0 ± 1121.47 |
| Blood: Week 8 | 828.5 ± 267.99 | 7384.0 ± 1011.16 |
| Blood: Week 9 | 714.5 ± 222.74 | 6384.5 ± 195.87 |
| Blood: Week 10 | 773.0 ± 9.90 | 6144.5 ± 939.74 |
| Blood: Week 11 | 755.0 ± 241.83 | 3935.0 ± 1547.15 |
| Blood: Week 12 | 728.0 ± 130.11 | 3893.5 ± 276.48 |
| Blood: Week 13 | 654.0 ± 22.63 | 3044.5 ± 1191.47 |
| Blood: Week 14 | 735.5 ± 82.73 | 2856.0 ± 1473.61 |
| Blood: Week 15 | 742.0 ± NA | 2812.0 ± 2083.14 |
| Blood: Month 4 | 422.0 ± 192.33 | 3066.0 ± NA |
| Blood: Month 5 | — | 210.0 ± NA |
| Saliva: Day 1 | 935.0 ± 268.70 | 2076.5 ± 473.05 |
| Saliva: Week 1 | — | 352.5 ± 12.02 |
| Semen: Day 1 | 55.0 ± NA | 230.5 ± 188.80 |
| Semen: Week 1 | — | 194.0 ± NA |
| Stool: Day 1 | — | 5985.0 ± NA |
| Stool: Week 1 | — | 7164.0 ± 7993.14 |
| Urine: Day 1 | NA ± NA | NA ± NA |
| Urine: Week 1 | NA ± NA | NA ± NA |
| Urine: Week 2 | NA ± NA | NA ± NA |
| Urine: Week 3 | NA ± NA | NA ± NA |
Collected over All-cause mortality: Up to approximately 6 years 4 months; SAEs and Other (Non-Serious) AEs: From first dose up to end of the study (approximately 6 years). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: BAX 888 2.0*10^12 cp/kg | 0/2 (0%) | 0/2 (0%) | 2/2 (100%) |
| Cohort 2: BAX 888 6.0*10^12 cp/kg | 0/2 (0%) | 1/2 (50%) | 2/2 (100%) |
| Event | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| HypophosphataemiaMetabolism and nutrition disorders | 0/2 | 1/2 |
| Event | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg |
|---|---|---|
| HypertransaminasaemiaHepatobiliary disorders | 2/2 | 2/2 |
| NasopharyngitisInfections and infestations | 2/2 | 0/2 |
| SARS-CoV-2 test positiveInvestigations | 2/2 | 0/2 |
| Abdominal pain upperGastrointestinal disorders | 0/2 | 1/2 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/2 | 1/2 |
| ArthritisMusculoskeletal and connective tissue disorders | 1/2 | 0/2 |
| AstheniaGeneral disorders | 1/2 | 0/2 |
| Back painMusculoskeletal and connective tissue disorders | 1/2 | 0/2 |
| COVID-19Infections and infestations | 1/2 | 1/2 |
| DermatitisSkin and subcutaneous tissue disorders | 0/2 | 1/2 |
The Safety Set consisted of all participants who received any amount of investigational product.
| Age, Continuous(years) | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg | Total |
|---|---|---|---|
| Mean | 29.5 ± 13.44 | 27.5 ± 3.54 | 28.5 ± 8.1 |
| Sex: Female, Male(Participants) | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 2 | 2 | 4 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 2 | 2 | 4 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1: BAX 888 2.0*10^12 cp/kg | Cohort 2: BAX 888 6.0*10^12 cp/kg | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 2 | 2 | 4 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — Takeda does not provide access to Individual Participant Data when a study is in a very limited (small) study population due to participant privacy concerns such as potential reidentification of study participants.
This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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