Paroxysmal Nocturnal HemoglobinuriaPhase 1NCT06326814
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults
A Phase 1 study testing Humanized anti-Factor Bb monoclonal antibody in people with Paroxysmal Nocturnal Hemoglobinuria, run by Sanofi at 2 sites in United States.
Trial summary
Primary objective The tolerability and safety of SAR443809 Secondary The PK parameters of SAR443809 The PD activity of SAR443809 The immunogenicity of SAR443809 Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Humanized anti-Factor Bb monoclonal antibody
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 54 participants
- Where
- 2 sitesUnited States
- Study dates
- 11 Oct 2021to 5 May 2023
- Phase
- Phase 1
- 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 18 to 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Inclusion Criteria: Having given written informed consent prior to undertaking any study-related procedure Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:
Any participant who, in the judgment of the Investigator, is likely to be noncompliant during the study, or unable to cooperate because of a language problem or poor mental development.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Parexel International Site Number : 8400003
Baltimore, Maryland, 21255
Parexel International Site Number : 8400002
Glendale, California, 91206
About this study
Official title: A First-in-human, Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Ascending Single Doses of SAR443809 in Healthy Adult Subjects
Primary objective
- The tolerability and safety of SAR443809 Secondary
- The PK parameters of SAR443809
- The PD activity of SAR443809
- The immunogenicity of SAR443809
Read the detailed description
Screening: up to 56 days (Day -56 to Day -2). Treatment: 1 day (treatment on Day 1, study observation period from Day -1 to Day 3). Follow-up and end of study: 105 days after IMP administration (follow up visits from Day 5 to Day 78; End of study visit on Day 106). Total study duration for each participant: approximately 23 weeks.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Humanized anti-Factor Bb monoclonal antibody, Placebo
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Humanized anti-Factor Bb monoclonal antibodyDrug
Also called SAR443809
Pharmaceutical form:Solution for injection-Route of administration:IV and SC routes of administration
PlaceboDrug
Pharmaceutical form:Solution for injection-Route of administration:IV and SC routes of administration
| Study group | Role | What they receive |
|---|---|---|
| SAR443809 and placebo dose 1 Arm | Experimental | 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 1 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
| SAR443809 and placebo dose 2 Arm | Experimental | 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 2 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
| SAR443809 and placebo dose 3 Arm | Experimental | 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 3 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
| SAR443809 and placebo dose 4 Arm | Experimental | 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 4 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
| SAR443809 and placebo dose 5 Arm | Experimental | 6 participants receiving SAR443809 and 2 receiving placebo, subcutaneous administration dose 5 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
| SAR443809 and placebo dose 6 Arm | Experimental | Optional: 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 6 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
| SAR443809 and placebo dose 7 Arm | Experimental | Optional: 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 7 Receives: Drug: Humanized anti-Factor Bb monoclonal antibody; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)
Measured: Baseline up to 23 weeks
Incidence of potentially Clinical laboratory abnormalities
Measured: Baseline up to 23 weeks
Secondary outcomes (14)
PK parameters of SAR443809 for IV and SC administrations: Maximum plasma concentration observed (Cmax
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: First time to reach Cmax (tmax
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to tlast (AUClast)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Area under the plasma concentration versus time curve extrapolated to infinity (AUC0-∞)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Terminal half-life associated with the terminal slope (λz) (t1/2z)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Time corresponding to the last concentration above the limit of quantification (Clast tlast)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: total body clearance of a drug from the plasma calculated by dividing dose by AUC (CL)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for SC administrations: apparent total body clearance of the SC formulation (CL/F)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: volume of distribution at steady-state (Vss)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for SC administrations: apparent volume of distribution at steady-state (Vss/F)
Measured: Baseline up to 23 weeks
PK parameters of SAR443809 for SC administrations: absolute bioavailability (F)
Measured: Baseline up to 23 weeks
Complement alternative pathway activity (Wieslab AP and alternative pathway hemolytic activity [AH50])
Ex vivo activity of the alternative pathway of complement in serum using the WIESLAB® Complement System Alternative Pathway kit and a hemolytic assay (AH50)
Measured: Baseline up to 23 weeks
Complement classical pathway activity (Wieslab CP)
Ex vivo activity of the alternative pathway of complement in serum using the WIESLAB® Complement System Classical Pathway kit
Measured: Baseline up to 23 weeks
Incidence of treatment -emergent Anti-SAR443809 antibodies
Measured: Baseline up to 23 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2024
- Study start11 Oct 2021
- Primary completion5 May 2023
- Study completion5 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by SanofiThe 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06326814
- Sponsor's protocol ID
- TDU16837
- Other IDs
- U1111-1298-7281
- Status last verified
- Mar 2024
- First submitted
- 5 Mar 2024
- Last updated
- 22 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First-in-human, Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Ascending Single Doses of SAR443809 in Healthy Adult Subjects
- ClinicalTrials.gov ID
- NCT06326814
- Acronym / sponsor ID
- TDU16837
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 54 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 11 Oct 2021 (actual)
- Primary completion
- 5 May 2023 (actual)
- Study completion
- 5 May 2023 (actual)
- First posted
- 22 Mar 2024 (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.
Primary objective
- The tolerability and safety of SAR443809 Secondary
- The PK parameters of SAR443809
- The PD activity of SAR443809
- The immunogenicity of SAR443809
Detailed description
Screening: up to 56 days (Day -56 to Day -2). Treatment: 1 day (treatment on Day 1, study observation period from Day -1 to Day 3). Follow-up and end of study: 105 days after IMP administration (follow up visits from Day 5 to Day 78; End of study visit on Day 106). Total study duration for each participant: approximately 23 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| SAR443809 and placebo dose 1 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: Placebo6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 1 |
| SAR443809 and placebo dose 2 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: Placebo6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 2 |
| SAR443809 and placebo dose 3 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: Placebo6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 3 |
| SAR443809 and placebo dose 4 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: Placebo6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 4 |
| SAR443809 and placebo dose 5 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: Placebo6 participants receiving SAR443809 and 2 receiving placebo, subcutaneous administration dose 5 |
| SAR443809 and placebo dose 6 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: PlaceboOptional: 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 6 |
| SAR443809 and placebo dose 7 Arm | Experimental | Drug: Humanized anti-Factor Bb monoclonal antibody, Drug: PlaceboOptional: 6 participants receiving SAR443809 and 2 receiving placebo, intravenous administration dose 7 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
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 |
|---|---|---|
| Humanized anti-Factor Bb monoclonal antibodyAlso: SAR443809Pharmaceutical form:Solution for injection-Route of administration:IV and SC routes of administration | Drug | SAR443809 and placebo dose 1 Arm, SAR443809 and placebo dose 2 Arm, SAR443809 and placebo dose 3 Arm, SAR443809 and placebo dose 4 Arm, SAR443809 and placebo dose 5 Arm, SAR443809 and placebo dose 6 Arm, SAR443809 and placebo dose 7 Arm |
| PlaceboPharmaceutical form:Solution for injection-Route of administration:IV and SC routes of administration | Drug | SAR443809 and placebo dose 1 Arm, SAR443809 and placebo dose 2 Arm, SAR443809 and placebo dose 3 Arm, SAR443809 and placebo dose 4 Arm, SAR443809 and placebo dose 5 Arm, SAR443809 and placebo dose 6 Arm, SAR443809 and placebo dose 7 Arm |
Primary outcome
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)
Time frame: Baseline up to 23 weeks
Incidence of potentially Clinical laboratory abnormalities
Time frame: Baseline up to 23 weeks
14 secondary outcomes
PK parameters of SAR443809 for IV and SC administrations: Maximum plasma concentration observed (Cmax
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: First time to reach Cmax (tmax
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to tlast (AUClast)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Area under the plasma concentration versus time curve extrapolated to infinity (AUC0-∞)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Terminal half-life associated with the terminal slope (λz) (t1/2z)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: Time corresponding to the last concentration above the limit of quantification (Clast tlast)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: total body clearance of a drug from the plasma calculated by dividing dose by AUC (CL)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for SC administrations: apparent total body clearance of the SC formulation (CL/F)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for IV and SC administrations: volume of distribution at steady-state (Vss)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for SC administrations: apparent volume of distribution at steady-state (Vss/F)
Time frame: Baseline up to 23 weeks
PK parameters of SAR443809 for SC administrations: absolute bioavailability (F)
Time frame: Baseline up to 23 weeks
Complement alternative pathway activity (Wieslab AP and alternative pathway hemolytic activity [AH50])
Time frame: Baseline up to 23 weeks
Complement classical pathway activity (Wieslab CP)
Time frame: Baseline up to 23 weeks
Incidence of treatment -emergent Anti-SAR443809 antibodies
Time frame: Baseline up to 23 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2024.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Humanized anti-Factor Bb monoclonal antibody in people with Paroxysmal Nocturnal Hemoglobinuria, run by Sanofi at 2 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?
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 22 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs) (time frame: Baseline up to 23 weeks). The study lists 2 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 22 Mar 2024; AzTrials last refreshed its copy on 6 Nov 2026.