Amyotrophic Lateral Sclerosis (Healthy Volunteers)Phase 1NCT05795907
Single & Multiple Ascending Dose Study of SAR443820 in Healthy Adult Participants
A Phase 1 study testing SAR443820 in people with Amyotrophic Lateral Sclerosis (Healthy Volunteers), run by Sanofi at 1 site in United States.
Trial summary
This is a Phase 1, single-center study conducted in 2 parts: Part 1a, single ascending dose (SAD-TDU16519): Double-blind, randomized, placebo-controlled sequential ascending single oral doses including up to 6 cohorts. Each cohort will include 8 participants (6 receiving SAR443820 and 2 placebo). Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (Healthy Volunteers)
- What is being tested?
- SAR443820
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 84 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Nov 2020to 20 Jul 2021
- 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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Male and/or female participant, between 18 and 55 years of age, inclusive. Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m2, inclusive.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical examination).
- Having given written informed consent prior to undertaking any study-related procedure.
- Not under any administrative or legal supervision or under institutionalization due to regulatory or juridical order.
Who cannot join
- Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteo-muscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness.
- Personal medical history of seizure.
- Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month).
- Any medication (including St John's Wort) within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy; any vaccination within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion.
- Positive result for hepatitis B, C or HIV
- Positive result on urine drug screen
Show all 11Show fewer
- Positive alcohol test.
- Any consumption of citrus fruits or their juices within 5 days before inclusion.
- Current psychiatric disorder, suicidal ideation in the previous 6 months (as assessed by the C-SSRS), or a lifetime suicide attempt.
- Additional exclusion criteria applied, and specially for Part 1b, criteria related to the study procedure of lumbar puncture.
- 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
1 site in 1 country.
United States1 site
Prism Research-Site Number:8400001
Saint Paul, Minnesota, 55144
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability and Pharmacokinetics of Ascending Single and 14-day Repeated Oral Doses of SAR443820 in Healthy Adult Participants.
This is a Phase 1, single-center study conducted in 2 parts:
Part 1a, single ascending dose (SAD-TDU16519): Double-blind, randomized, placebo-controlled sequential ascending single oral doses including up to 6 cohorts. Each cohort will include 8 participants (6 receiving SAR443820 and 2 placebo).
Part 1b (TDU16519): - Open label, single SAR443820 dose in one or two separated cohort(s) for SAR443820 measurements in CSF and in plasma.
Part 2, multiple ascending dose (MAD -TDR16520): Double-blind, randomized, placebo-controlled, sequential ascending repeated oral doses for 14 days, including up to 4 cohorts. Each cohort will include 10 participants (8 receiving SAR443820 and 2 placebo).
Read the detailed description
The duration of the study for a participant will include:
Screening Period: up to 28 days
Part 1a:
Treatment in fasted condition: 1 day (Day 1). Study observation Period from Day -2/Day -1 to Day 3. Follow-up with the end of study: from Day 5 to Day 7. Total duration from screening per participant: up to 5 weeks.
Part 1b:
Treatment in fed condition: 1 day (Day 1). Study observation Period from Day -1/Day1 to Day 2. Follow-up with the end of study: from Day 5 to Day 7. Total duration from screening per participant: up to 5 weeks.
Part 2:
Treatment: 14 days (Day 1 to Day 14). Study observation Period from Day -2/Day -1 to Day 17. Follow-up with the end of study: from Day 19 to Day 21. Total duration from screening per participant: up to 7 weeks.
- Being tested
- SAR443820, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAR443820Drug
Capsule / Oral
PlaceboDrug
Matching Capsule / Oral
| Study group | Role | What they receive |
|---|---|---|
| SAR443820 | Experimental | Single dose oral ascending dose (parts 1a and 1b) and multiple ascending oral dose (part 2) of SAR443820 Receives: Drug: SAR443820 |
| Placebo | Placebo comparator | Single dose oral ascending dose (part 1a) and multiple ascending oral dose (part 2) of matching placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parts 1a and 1b: Number of participants with adverse events
Measured: Day1 up to Day 7 (end of study visit)
Part 2: Number of participants with adverse events
Measured: Day1 up to Day 21 (end of study visit)
Secondary outcomes (10)
Parts 1a and 1b: Assessment of pharmacokinetic parameter of SAR443820: Cmax in plasma
Parts 1a and 1b: Maximum plasma concentration
Measured: Day1
Parts 1a and 1b: Assessment of pharmacokinetic parameter of SAR443820: tmax in plasma
Parts 1a and 1b: time to reach Cmax
Measured: Day 1
Parts 1a and 1b : Assessment of pharmacokinetic parameter of SAR443820: AUC in plasma
Parts 1a and 1b: Area under the plasma concentration versus time
Measured: Day1
Parts 1a and 1b : Assessment of pharmacokinetic parameter of SAR443820: t1/2z in plasma
Terminal half-life in plasma
Measured: Day1
Part1b: SAR443820 concentrations in cerebrospinal fluid (CSF) samples
Part 1b: CSF to plasma concentration ratio
Measured: Day1
Part 2: Assessment of pharmacokinetic parameter of SAR443820: Cmax in plasma
Part 2: Maximum plasma concentration
Measured: Day1 and Day14
Part 2: Assessment of pharmacokinetic parameter of SAR443820: tmax in plasma
Part 2: Time to reach Cmax
Measured: Day1 and Day14
Part 2: Assessment of pharmacokinetic parameter of SAR443820: AUC tau in plasma
Part 2: Area under the plasma concentration versus time during a dosing interval
Measured: Day1 and Day14
Part 2 Assessment of pharmacokinetic parameter of SAR443820: t1/2z in plasma
Terminal half-life in plasma
Measured: Day14
Part 2: Day14/Day1 of 4β-hydroxycholesterol ratio in plasma
D14/D1 of 4β-hydroxycholesterol ratio
Measured: Day1 and Day14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2023
- Study start30 Nov 2020
- Primary completion20 Jul 2021
- Study completion20 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral Sclerosis (Healthy Volunteers)Studies 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
- Model description
- Part 1b with open label study design
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Trial Transparency email recommended (Toll free for US & Canada), Study director, SanofiClinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05795907
- Sponsor's protocol ID
- TDU16519-TDR16520
- Other IDs
- U1111-1254-0034
- Status last verified
- Mar 2023
- First submitted
- 8 Mar 2023
- Last updated
- 3 Apr 2023
- 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
Publications
Hincelin-Mery A, Nicolas X, Cantalloube C, Pomponio R, Lewanczyk P, Benamor M, Ofengeim D, Krupka E, Hsiao-Nakamoto J, Eastenson A, Atassi N. Safety, pharmacokinetics, and target engagement of a brain penetrant RIPK1 inhibitor, SAR443820 (DNL788), in healthy adult participants. Clin Transl Sci. 2024 Jan;17(1):e13690. doi: 10.1111/cts.13690. Epub 2023 Dec 11.
PubMed 38010108
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability and Pharmacokinetics of Ascending Single and 14-day Repeated Oral Doses of SAR443820 in Healthy Adult Participants.
- ClinicalTrials.gov ID
- NCT05795907
- Acronym / sponsor ID
- TDU16519-TDR16520
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 30 Nov 2020 (actual)
- Primary completion
- 20 Jul 2021 (actual)
- Study completion
- 20 Jul 2021 (actual)
- First posted
- 3 Apr 2023 (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 1, single-center study conducted in 2 parts:
Part 1a, single ascending dose (SAD-TDU16519): Double-blind, randomized, placebo-controlled sequential ascending single oral doses including up to 6 cohorts. Each cohort will include 8 participants (6 receiving SAR443820 and 2 placebo).
Part 1b (TDU16519): - Open label, single SAR443820 dose in one or two separated cohort(s) for SAR443820 measurements in CSF and in plasma.
Part 2, multiple ascending dose (MAD -TDR16520): Double-blind, randomized, placebo-controlled, sequential ascending repeated oral doses for 14 days, including up to 4 cohorts. Each cohort will include 10 participants (8 receiving SAR443820 and 2 placebo).
Detailed description
The duration of the study for a participant will include:
Screening Period: up to 28 days
Part 1a:
Treatment in fasted condition: 1 day (Day 1). Study observation Period from Day -2/Day -1 to Day 3. Follow-up with the end of study: from Day 5 to Day 7. Total duration from screening per participant: up to 5 weeks.
Part 1b:
Treatment in fed condition: 1 day (Day 1). Study observation Period from Day -1/Day1 to Day 2. Follow-up with the end of study: from Day 5 to Day 7. Total duration from screening per participant: up to 5 weeks.
Part 2:
Treatment: 14 days (Day 1 to Day 14). Study observation Period from Day -2/Day -1 to Day 17. Follow-up with the end of study: from Day 19 to Day 21. Total duration from screening per participant: up to 7 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Part 1b with open label study design
| Arm | Type | Receives |
|---|---|---|
| SAR443820 | Experimental | Drug: SAR443820Single dose oral ascending dose (parts 1a and 1b) and multiple ascending oral dose (part 2) of SAR443820 |
| Placebo | Placebo comparator | Drug: PlaceboSingle dose oral ascending dose (part 1a) and multiple ascending oral dose (part 2) of matching placebo |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Male and/or female participant, between 18 and 55 years of age, inclusive. Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m2, inclusive.
- Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical examination).
- Having given written informed consent prior to undertaking any study-related procedure.
- Not under any administrative or legal supervision or under institutionalization due to regulatory or juridical order.
Key exclusion criteria (11 listed)
- Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteo-muscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness.
- Personal medical history of seizure.
- Frequent headaches and/or migraine, recurrent nausea and/or vomiting (for vomiting only: more than twice a month).
- Any medication (including St John's Wort) within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy; any vaccination within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion.
- Positive result for hepatitis B, C or HIV
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 |
|---|---|---|
| SAR443820Capsule / Oral | Drug | SAR443820 |
| PlaceboMatching Capsule / Oral | Drug | Placebo |
Primary outcome
Parts 1a and 1b: Number of participants with adverse events
Time frame: Day1 up to Day 7 (end of study visit)
Part 2: Number of participants with adverse events
Time frame: Day1 up to Day 21 (end of study visit)
10 secondary outcomes
Parts 1a and 1b: Assessment of pharmacokinetic parameter of SAR443820: Cmax in plasma
Time frame: Day1
Parts 1a and 1b: Assessment of pharmacokinetic parameter of SAR443820: tmax in plasma
Time frame: Day 1
Parts 1a and 1b : Assessment of pharmacokinetic parameter of SAR443820: AUC in plasma
Time frame: Day1
Parts 1a and 1b : Assessment of pharmacokinetic parameter of SAR443820: t1/2z in plasma
Time frame: Day1
Part1b: SAR443820 concentrations in cerebrospinal fluid (CSF) samples
Time frame: Day1
Part 2: Assessment of pharmacokinetic parameter of SAR443820: Cmax in plasma
Time frame: Day1 and Day14
Part 2: Assessment of pharmacokinetic parameter of SAR443820: tmax in plasma
Time frame: Day1 and Day14
Part 2: Assessment of pharmacokinetic parameter of SAR443820: AUC tau in plasma
Time frame: Day1 and Day14
Part 2 Assessment of pharmacokinetic parameter of SAR443820: t1/2z in plasma
Time frame: Day14
Part 2: Day14/Day1 of 4β-hydroxycholesterol ratio in plasma
Time frame: Day1 and Day14
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 3 Apr 2023.
- Derived from this study: PubMed 38010108Hincelin-Mery A, Nicolas X, Cantalloube C, Pomponio R, Lewanczyk P, Benamor M, Ofengeim D, Krupka E, Hsiao-Nakamoto J, Eastenson A, Atassi N. Safety, pharmacokinetics, and target engagement of a brain penetrant RIPK1 inhibitor, SAR443820 (DNL788), in healthy adult participants. Clin Transl Sci. 2024 Jan;17(1):e13690. doi: 10.1111/cts.13690. Epub 2023 Dec 11.
- 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.
Trial Transparency email recommended (Toll free for US & Canada) study director
Sanofi
Clinical Sciences & Operations study director
Sanofi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SAR443820 in people with Amyotrophic Lateral Sclerosis (Healthy Volunteers), run by Sanofi at 1 site 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 4 inclusion and 11 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 3 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. 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?
Parts 1a and 1b: Number of participants with adverse events (time frame: Day1 up to Day 7 (end of study visit)). 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?
On the ClinicalTrials.gov record for NCT05795907, which also lists 1 linked publication.
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 3 Apr 2023.