Chronic Inflammatory Demyelinating PolyradiculoneuropathyPhase 2NCT04658472
Proof-of-concept Study for SAR445088 in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
A Phase 2 study testing SAR445088 (IV) and SAR445088 (SC) in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy, run by Bioverativ, a Sanofi company at 32 sites in 10 countries.
Trial summary
Primary Objectives: Part A: Efficacy of SAR445088 across three subpopulations of CIDP patients: standard of care (SOC)-Treated, SOC-Refractory and SOC-Naive Part B:Long-term safety and tolerability of SAR445088 in CIDP Secondary Objectives: Part A: Safety and tolerability of SAR445088 in CIDP Immunogenicity of… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- What is being tested?
- SAR445088 (IV) and SAR445088 (SC)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 98 participants
- Where
- 32 sitesin 10 countries
- Study dates
- 28 Apr 2021to 1 Oct 2025
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults ≥18 years of age at the time of signing the informed consent.
- Documented definite or probable diagnosis of CIDP (typical CIDP, pure motor CIDP, or Lewis-Sumner Syndrome) according to the European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) Task Force first revision.
- Belonging to one of the following three groups: standard-of-care (SOC)-Treated, SOC-Refractory or SOC-Naive, as defined below.
- SOC-Treated (all criteria a-c must be met): a) Documented evidence of objective response to SOC, with clinically meaningful improvement. Clinically meaningful improvement is defined as one of the following: ≥1-point decrease in adjusted INCAT score, ≥4 points increase in RODS total score, ≥3 points increase in MRC Sum score, ≥8 kilopascal improvement in mean grip strength (one hand), or an equivalent improvement based on information documented in medical records and per the PI's judgement. b) Must be on stable SOC therapy, defined as no change greater than 10% in frequency or dose of immunoglobulin therapy or corticosteroids within 8 weeks prior to screening, remaining at stable SOC therapy until the time of first SAR445088 dosing. c) Evidence of clinically meaningful deterioration on interruption or dose reduction of SOC therapy within 24 months prior to screening, determined by clinical examination or medical records. Clinically meaningful deterioration is defined as one of the following: ≥1-point increase in adjusted INCAT score, decrease in RODS total score ≥4 points, decrease in MRC Sum score ≥3, mean grip strength worsening of ≥8 kilopascals (one hand), or an equivalent deterioration based on information from medical records and at the PI's judgement.
- SOC-Refractory (all criteria a-d must be met): a) Evidence of failure or inadequate response to SOC defined as no clinically meaningful improvement and persistent INCAT score ≥2 after treatment for a minimum of 12 weeks on SOC prior to screening. A clinically meaningful improvement is defined as one of the following: ≥1-point decrease in adjusted INCAT score, increase in RODS total score ≥4 points, increase in MRC Sum score ≥3, mean grip strength improvement of ≥8 kilopascals (one hand), or equivalent improvement based on information from medical records and at the PI's judgement.
- Or
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- Unable to receive or continue treatment with immunoglobulins or corticosteroids due to side effects.
- b) Patient has not received immunoglobulins (IVIg or SCIg) within 12 weeks prior to screening. c) Certain immunosuppressant drugs are allowed in this group if taken for ≥6 months and at a stable dose for ≥3 months prior to screening: azathioprine, methotrexate, mycophenolate mofetil and cyclosporine. Oral corticosteroids are allowed if on a stable dose of <20 mg/day of prednisone (or equivalent dose for other oral corticosteroids) for ≥3 months prior to screening. d) INCAT score: 2-9 (a score of 2 should be exclusively from leg disability component of INCAT).
- SOC-Naive (all criteria a-c must be met): a) Participants without previous treatment for CIDP or participants who received immunoglobulins (IVIg or SCIg) or corticosteroids but were stopped for reasons other than lack of response or side effects. b) Not treated with immunoglobulins (IVIg or SCIg) or corticosteroids for at least 6 months prior to screening. c) INCAT score: 2-9 (a score of 2 should be exclusively from leg disability component of INCAT.
- Documented vaccinations against encapsulated bacterial pathogens given within 5 years of enrollment or initiated a minimum of 14 days prior to first dose
- A female participant must use a double contraception method including a highly effective method of birth control from inclusion and up to 52 weeks plus 30 days after the last study dose and agree not to donate eggs, ova or oocytes during this period.
- A female participant must have a negative highly sensitive pregnancy test (urine or serum) as required by local regulations within 24 hours before the first dose of study intervention.
- Male participants, whose partners are of childbearing potential must accept to use, during sexual intercourse, a double contraceptive method according to the following: condom plus an additional highly effective contraception
- Male participants must have agreed not to donate sperm during the intervention and up to 52 weeks after the last dose.
- Capable of giving signed informed consent
Who cannot join
- Polyneuropathy of other causes, including but not limited to hereditary demyelinating neuropathies, neuropathies secondary to infection or systemic disease, diabetic neuropathy, drug- or toxin-induced neuropathies, multifocal motor neuropathy, polyneuropathy related to IgM monoclonal gammopathy, POEMS syndrome, lumbosacral radiculoplexus neuropathy, pure sensory CIDP and acquired demyelinating symmetric (DADS) neuropathy (also known as distal CIDP).
- Any other neurological or systemic disease that can cause symptoms and signs interfering with treatment or outcome assessments.
- Poorly controlled diabetes (HbA1c >7%).
- Serious infections requiring hospitalization within 30 days prior to screening and any active infection requiring treatment during screening.
- Clinical diagnosis of SLE.
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study. Specifically, history of any hypersensitivity reaction to SAR445088 or its components or of a severe allergic or anaphylactic reaction to any humanized or murine monoclonal antibody.
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- Participants with a history of suicidality in the six months prior to screening or currently at risk of committing suicide.
- Presence of conditions (medical history or laboratory assessments) that may predispose the participant to excessive bleeding or increased risk of infection.
- Evidence of CIDP relapse within 6 weeks after receiving a vaccination.
- Recent or planned major surgery that could confound the results of the trial or put the participant at undue risk.
- Treatment with plasma exchange within 12 weeks prior to screening.
- Prior treatment with rituximab or ocrelizumab in the 6 months prior to SAR445088 dosing or until return of B-cell counts to normal levels, whichever is longer.
- Immunosuppressive/chemotherapeutic medications such as azathioprine, methotrexate, cyclophosphamide, cyclosporine, mycophenolate mofetil, tacrolimus, interferon, TNF-alpha inhibitor: within 6 months prior to dosing (except for some cases as indicated in the SOC-Refractory group).
- Treatment (any time) with highly immunosuppressive/chemotherapeutic medications with sustained effects, eg, mitoxantrone, alemtuzumab, cladribine.
- Treatment (any time) with total lymphoid irradiation or bone marrow transplantation.
- Use of any specific complement system inhibitor (eg, eculizumab) within 12 weeks or 5 times the half-life of the product, whichever is longer, prior to screening.
- Pregnant (defined as positive β-HCG blood test) or lactating females.
- Positive result on any of the following tests: hepatitis B surface (HBsAg) antigen, anti-hepatitis B core antibodies (anti-HBc Ab)-unless anti-hepatitis B surface antibodies (anti-HBs Ab) are also positive , indicating natural immunity-, anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti-HIV2 antibodies).
- Evidence of IgG4 autoantibodies against paranodal proteins (NF155 and CNTN1).
- The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial locations and contacts
32 sites in 10 countries.
32 sites in 10 countries
United States5 sites
The University of Kansas Clinical Research Center Site Number : 8400003
Fairway, Kansas, 66205
University of Southern California Site Number : 8400004
Los Angeles, California, 90033
University of Minnesota Site Number : 8400006
Minneapolis, Minnesota, 55414
Columbia University Site Number : 8400005
New York, New York, 10032
University of California Irvine Site Number : 8400002
Orange, California, 92868
France5 sites
Investigational Site Number : 2500003
Bordeaux
Investigational Site Number : 2500004
Garches, 92380
Investigational Site Number : 2500005
Le Kremlin-Bicêtre, 94275
Investigational Site Number : 2500001
Marseille, 13385
Investigational Site Number : 2500002
Nice, 06002
Germany4 sites
Investigational Site Number : 2760001
Düsseldorf, 40225
Investigational Site Number : 2760003
Essen, 45147
Investigational Site Number : 2760002
Göttingen, 37075
Investigational Site Number : 2760004
Tübingen, 72076
Italy4 sites
Investigational Site Number : 3800003
Genova, 16132
Investigational Site Number : 3800001
Milan, 20132
Investigational Site Number : 3800002
Rome, Lazio, 00168
Investigational Site Number : 3800005
Rozzano, Milano, 20089
China3 sites
Investigational Site Number : 1560002
Fuzhou, 350001
Investigational Site Number : 1560001
Shanghai, 200040
Investigational Site Number : 1560004
Wuhan, 430030
Serbia3 sites
Investigational Site Number : 6880001
Belgrade, 11000
Investigational Site Number : 6880002
Belgrade, 11000
Investigational Site Number : 6880003
Niš, 18000
Spain3 sites
Investigational Site Number : 7240002
Barcelona, Barcelona [Barcelona], 08035
Investigational Site Number : 7240001
Barcelona, Barcelona [Barcelona], 08041
Investigational Site Number : 7240003
Valencia, 46026
Canada2 sites
Investigational Site Number : 1240001
Gatineau, Quebec, J8Y 1W2
Investigational Site Number : 1240002
Québec, G1J 1Z4
Netherlands2 sites
Investigational Site Number : 5280001
Amsterdam, 1105AZ
Investigational Site Number : 5280002
Utrecht, 3584 CX
Poland1 site
Investigational Site Number : 6160001
Lublin, Lublin Voivodeship, 20-954
About this study
Official title: A Phase 2, Multicenter, Open-label, Non-randomized, Proof-of-concept Study Evaluating the Efficacy, Safety, and Tolerability of SAR445088 in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Primary Objectives:
- Part A: Efficacy of SAR445088 across three subpopulations of CIDP patients: standard of care (SOC)-Treated, SOC-Refractory and SOC-Naive
- Part B:Long-term safety and tolerability of SAR445088 in CIDP
Secondary Objectives:
- Part A:
- Safety and tolerability of SAR445088 in CIDP
- Immunogenicity of SAR445088
- Efficacy of SAR445088 with overlapping SOC (SOC-Treated group)
- Part B:
- Durability of efficacy during long-term treatment with SAR445088 in CIDP
- Long-term immunogenicity of SAR445088 in CIDP
Read the detailed description
The duration of the study for a participant will include:
Part A Screening period: up to 6 weeks. Treatment period: once successfully screened, enrolled participants will receive study intervention for 24 weeks.
Safety follow-up visit: participants who do not enroll (rollover) into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24, ie, approximately at Week 46.
Part B Treatment period (extension): for all groups, this period will consist of 52 weeks of treatment with SAR445088 (Weeks 24 to 76; Part A and B total treatment period of 76 weeks).
Safety follow-up visit: participants who do not enroll (rollover) into Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (Week 98).
Part C Treatment period: participants who complete Part B will be reassessed for continuing eligibility, defined as having successfully completed Part B (received IMP until the Week 76 visit and have responded to SAR445088 based on Investigator's medical judgement), with no new safety concerns. Eligible participants will be given the option of rolling into Part C, where they will continue receiving SAR445088 until end of study.
End of study has been defined as the last safety follow-up visit for the last patient which occurs 22 weeks after last dose.
In addition, there is a follow-up call 56 weeks ±14 days after last dose to confirm negative result of urine pregnancy test for women of childbearing potential who are participating in the study, or to query male participants regarding pregnancy of partners who are women of childbearing potential.
- Being tested
- SAR445088 (IV), SAR445088 (SC)
- Medical subject headings
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAR445088 (IV)Drug
Pharmaceutical form: Solution for Injection Route of Administration: Intravenous (IV)
SAR445088 (SC)Drug
Pharmaceutical form: Solution for Injection Route of Administration: Subcutaneous (SC)
| Study group | Role | What they receive |
|---|---|---|
| SOC-Refractory Initial Dose | Experimental | Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately (~)at Week 46).
Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given the option of rolling into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (approximately week 98) if they will not continue in Part C.
Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study.
Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose. Receives: Drug: SAR445088 (IV); Drug: SAR445088 (SC) |
| SOC-Refractory Low Dose | Experimental | Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately at Week 46).
Part B: Participants who successfully complete Part A, will be reassessed for continued eligibility and will be given the option to roll into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (approximately week 98) if they do not continue in Part C.
Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study.
Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose. Receives: Drug: SAR445088 (IV); Drug: SAR445088 (SC) |
| SOC-Treated Initial Dose | Experimental | Part A: Eligible participants will receive SAR445088 for 24 weeks. Weeks 1-12 (overlap period): Participants will be given SAR445088 with superimposing effects of SOC therapy; Weeks 13-24: SAR445088 given.
Participants who do not enroll into Part B will attend final safety follow-up visit at 22 weeks after Week 24 (~Week 46).
Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given option of rolling into Part B, and continue receiving SAR445088 for 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (~week 98) if they do not continue in Part C.
Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until end of study.
Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit at 22 weeks after the last SAR445088 dose. Receives: Drug: SAR445088 (IV); Drug: SAR445088 (SC) |
| SOC-Naive | Experimental | Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately at Week 46).
Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given the option of rolling into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after last SAR445088 dose (approximately week 98) if they do not continue in Part C.
Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after last SAR445088 dose. Receives: Drug: SAR445088 (IV); Drug: SAR445088 (SC) |
| SOC-Treated Low Dose | Experimental | Part A: Eligible participants will receive SAR445088 for 24 weeks. Weeks 1-12 (overlap period): Participants will be given SAR445088 with superimposing effects SOC therapy; Weeks 13-24: SAR445088. Participants who do not enroll into Part B will attend a final safety follow-up visit that will take place 22 weeks after Week 24 (~Week 46).
Part B: Participants who successfully complete Part A, will be reassessed for continued eligibility, will be given the option of rolling into Part B, and continue receiving SAR445088 for 52 weeks. At the end of the Part B treatment period, participants will attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (~week 98) if they do not continue in Part C.
Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until end of study. Participants who discontinue at any time in Part C will attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose. Receives: Drug: SAR445088 (IV); Drug: SAR445088 (SC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A, SOC-Treated: Percentage of participants relapsing after withdrawal of SOC and during the SAR445088 treatment period
Relapse will be defined as ≥1-point increase in adjusted Inflammatory neuropathy cause and treatment (INCAT) disability score. The Investigator/rater will evaluate the level of impairment in participants? arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Measured: Day 1 up to 24 weeks
Part A, SOC-Refractory (initial and low dose group) and SOC-Naive: Percentage of participants responding during the SAR445088 treatment period
Response will be defined as ≥1-point decrease in adjusted INCAT disability score. The Investigator/rater will evaluate the level of impairment in participants? arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Measured: Day 1 up to 24 weeks
Part B: Number of participants reported with adverse events
Number of participants reported with adverse events during 76 weeks of treatment and 22 weeks of follow-up.
Measured: Day 1 up to Week 98
Secondary outcomes (6)
Part A: Number of participants reported with adverse events
Number of participants reported with adverse events during 24 weeks of treatment period and if not enrolled to Part B, 22 weeks of follow-up period.
Measured: Day 1 up to 46 Weeks
Part A: Number of participants with incidence and titer of anti-SAR445088 antibodies (ADA)
Incidence and titer of anti-SAR445088 antibodies (ADA) will be assessed during the 24 weeks of treatment period and if not enrolled to Part B, 22 weeks of follow-up period.
Measured: Day 1 up to 46 Weeks
Part A: Percentage of participants in the SOC-Treated group improving during the overlap treatment period
Improvement will be defined as ≥1 point decrease in adjusted INCAT disability score. The Investigator/rater will evaluate the level of impairment in participants? arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Measured: Day 1 up to 12 Weeks
Part B, SOC-Treated (initial dose group): Percentage of participants relapse-free during the treatment extension period
Relapse-free will be defined as no increase in adjusted INCAT disability score >2 points. The Investigator/rater will evaluate the level of impairment in participants? arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Measured: Week 24 up to Week 76
Part B, SOC-Refractory (initial and low dose group) and SOC-Naive: Percentage of participants with sustained response during the treatment extension period
Maintenance of response will be defined as no increase in adjusted INCAT disability score greater than or equal to 2 points. The Investigator/rater will evaluate the level of impairment in participants arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Measured: Week 24 up to Week 76
Part B: Long-term immunogenicity
Incidence and titer of anti-SAR445088 antibodies during the entire SAR445088 treatment period and follow-up period.
Measured: Day 1 Up to Week 98
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2020
- Study start28 Apr 2021
- Primary completion1 Oct 2025
- Study completion1 Oct 2025
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 Chronic Inflammatory Demyelinating PolyradiculoneuropathyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 98 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ, a Sanofi company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04658472
- Sponsor's protocol ID
- PDY16744
- Other IDs
- U1111-1246-7023, 2024-512345-16, 2020-004006-54
- Status last verified
- Oct 2025
- First submitted
- 1 Dec 2020
- Last updated
- 28 Oct 2025
- 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
Querol L, Lewis RA, Hartung HP, Van Doorn PA, Wallstroem E, Luo X, Alonso-Alonso M, Atassi N, Hughes RAC. An innovative phase 2 proof-of-concept trial design to evaluate SAR445088, a monoclonal antibody targeting complement C1s in chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2023 Jun;28(2):276-285. doi: 10.1111/jns.12551. Epub 2023 May 31.
PubMed 37119056
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Open-label, Non-randomized, Proof-of-concept Study Evaluating the Efficacy, Safety, and Tolerability of SAR445088 in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- ClinicalTrials.gov ID
- NCT04658472
- Acronym / sponsor ID
- PDY16744
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 98 participants (actual)
- Lead sponsor
- Bioverativ, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 28 Apr 2021 (actual)
- Primary completion
- 1 Oct 2025 (actual)
- Study completion
- 1 Oct 2025 (actual)
- First posted
- 8 Dec 2020 (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 Objectives:
- Part A: Efficacy of SAR445088 across three subpopulations of CIDP patients: standard of care (SOC)-Treated, SOC-Refractory and SOC-Naive
- Part B:Long-term safety and tolerability of SAR445088 in CIDP
Secondary Objectives:
- Part A:
- Safety and tolerability of SAR445088 in CIDP
- Immunogenicity of SAR445088
- Efficacy of SAR445088 with overlapping SOC (SOC-Treated group)
- Part B:
- Durability of efficacy during long-term treatment with SAR445088 in CIDP
- Long-term immunogenicity of SAR445088 in CIDP
Detailed description
The duration of the study for a participant will include:
Part A Screening period: up to 6 weeks. Treatment period: once successfully screened, enrolled participants will receive study intervention for 24 weeks.
Safety follow-up visit: participants who do not enroll (rollover) into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24, ie, approximately at Week 46.
Part B Treatment period (extension): for all groups, this period will consist of 52 weeks of treatment with SAR445088 (Weeks 24 to 76; Part A and B total treatment period of 76 weeks).
Safety follow-up visit: participants who do not enroll (rollover) into Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (Week 98).
Part C Treatment period: participants who complete Part B will be reassessed for continuing eligibility, defined as having successfully completed Part B (received IMP until the Week 76 visit and have responded to SAR445088 based on Investigator's medical judgement), with no new safety concerns. Eligible participants will be given the option of rolling into Part C, where they will continue receiving SAR445088 until end of study.
End of study has been defined as the last safety follow-up visit for the last patient which occurs 22 weeks after last dose.
In addition, there is a follow-up call 56 weeks ±14 days after last dose to confirm negative result of urine pregnancy test for women of childbearing potential who are participating in the study, or to query male participants regarding pregnancy of partners who are women of childbearing potential.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| SOC-Refractory Initial Dose | Experimental | Drug: SAR445088 (IV), Drug: SAR445088 (SC)Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately (~)at Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given the option of rolling into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (approximately week 98) if they will not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose. |
| SOC-Refractory Low Dose | Experimental | Drug: SAR445088 (IV), Drug: SAR445088 (SC)Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately at Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continued eligibility and will be given the option to roll into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (approximately week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose. |
| SOC-Treated Initial Dose | Experimental | Drug: SAR445088 (IV), Drug: SAR445088 (SC)Part A: Eligible participants will receive SAR445088 for 24 weeks. Weeks 1-12 (overlap period): Participants will be given SAR445088 with superimposing effects of SOC therapy; Weeks 13-24: SAR445088 given. Participants who do not enroll into Part B will attend final safety follow-up visit at 22 weeks after Week 24 (~Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given option of rolling into Part B, and continue receiving SAR445088 for 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (~week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit at 22 weeks after the last SAR445088 dose. |
| SOC-Naive | Experimental | Drug: SAR445088 (IV), Drug: SAR445088 (SC)Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately at Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given the option of rolling into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after last SAR445088 dose (approximately week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after last SAR445088 dose. |
| SOC-Treated Low Dose | Experimental | Drug: SAR445088 (IV), Drug: SAR445088 (SC)Part A: Eligible participants will receive SAR445088 for 24 weeks. Weeks 1-12 (overlap period): Participants will be given SAR445088 with superimposing effects SOC therapy; Weeks 13-24: SAR445088. Participants who do not enroll into Part B will attend a final safety follow-up visit that will take place 22 weeks after Week 24 (~Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continued eligibility, will be given the option of rolling into Part B, and continue receiving SAR445088 for 52 weeks. At the end of the Part B treatment period, participants will attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (~week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until end of study. Participants who discontinue at any time in Part C will attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Adults ≥18 years of age at the time of signing the informed consent.
- Documented definite or probable diagnosis of CIDP (typical CIDP, pure motor CIDP, or Lewis-Sumner Syndrome) according to the European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) Task Force first revision.
- Belonging to one of the following three groups: standard-of-care (SOC)-Treated, SOC-Refractory or SOC-Naive, as defined below.
- SOC-Treated (all criteria a-c must be met): a) Documented evidence of objective response to SOC, with clinically meaningful improvement. Clinically meaningful improvement is defined as one of the following: ≥1-point decrease in adjusted INCAT score, ≥4 points increase in RODS total score, ≥3 points increase in MRC Sum score, ≥8 kilopascal improvement in mean grip strength (one hand), or an equivalent improvement based on information documented in medical records and per the PI's judgement. b) Must be on stable SOC therapy, defined as no change greater than 10% in frequency or dose of immunoglobulin therapy or corticosteroids within 8 weeks prior to screening, remaining at stable SOC therapy until the time of first SAR445088 dosing. c) Evidence of clinically meaningful deterioration on interruption or dose reduction of SOC therapy within 24 months prior to screening, determined by clinical examination or medical records. Clinically meaningful deterioration is defined as one of the following: ≥1-point increase in adjusted INCAT score, decrease in RODS total score ≥4 points, decrease in MRC Sum score ≥3, mean grip strength worsening of ≥8 kilopascals (one hand), or an equivalent deterioration based on information from medical records and at the PI's judgement.
- SOC-Refractory (all criteria a-d must be met): a) Evidence of failure or inadequate response to SOC defined as no clinically meaningful improvement and persistent INCAT score ≥2 after treatment for a minimum of 12 weeks on SOC prior to screening. A clinically meaningful improvement is defined as one of the following: ≥1-point decrease in adjusted INCAT score, increase in RODS total score ≥4 points, increase in MRC Sum score ≥3, mean grip strength improvement of ≥8 kilopascals (one hand), or equivalent improvement based on information from medical records and at the PI's judgement.
Key exclusion criteria (20 listed)
- Polyneuropathy of other causes, including but not limited to hereditary demyelinating neuropathies, neuropathies secondary to infection or systemic disease, diabetic neuropathy, drug- or toxin-induced neuropathies, multifocal motor neuropathy, polyneuropathy related to IgM monoclonal gammopathy, POEMS syndrome, lumbosacral radiculoplexus neuropathy, pure sensory CIDP and acquired demyelinating symmetric (DADS) neuropathy (also known as distal CIDP).
- Any other neurological or systemic disease that can cause symptoms and signs interfering with treatment or outcome assessments.
- Poorly controlled diabetes (HbA1c >7%).
- Serious infections requiring hospitalization within 30 days prior to screening and any active infection requiring treatment during screening.
- Clinical diagnosis of SLE.
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 |
|---|---|---|
| SAR445088 (IV)Pharmaceutical form: Solution for Injection Route of Administration: Intravenous (IV) | Drug | SOC-Naive, SOC-Refractory Initial Dose, SOC-Refractory Low Dose, SOC-Treated Initial Dose, SOC-Treated Low Dose |
| SAR445088 (SC)Pharmaceutical form: Solution for Injection Route of Administration: Subcutaneous (SC) | Drug | SOC-Naive, SOC-Refractory Initial Dose, SOC-Refractory Low Dose, SOC-Treated Initial Dose, SOC-Treated Low Dose |
Primary outcome
Part A, SOC-Treated: Percentage of participants relapsing after withdrawal of SOC and during the SAR445088 treatment period
Time frame: Day 1 up to 24 weeks
Relapse will be defined as ≥1-point increase in adjusted Inflammatory neuropathy cause and treatment (INCAT) disability score. The Investigator/rater will evaluate the level of impairment in participants? arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Part A, SOC-Refractory (initial and low dose group) and SOC-Naive: Percentage of participants responding during the SAR445088 treatment period
Time frame: Day 1 up to 24 weeks
Response will be defined as ≥1-point decrease in adjusted INCAT disability score. The Investigator/rater will evaluate the level of impairment in participants? arms and legs each on a scale of 0 (least impaired) to 5 (most impaired), in the context of a neurological examination, and will record the added scores (range 0 to 10) per the INCAT reporting instructions. A low score reflects no or minimal disability eg, no arm dysfunction or walking normally, whereas higher scores indicate more disability eg, wheelchair bound or no purposeful arm movement.
Part B: Number of participants reported with adverse events
Time frame: Day 1 up to Week 98
Number of participants reported with adverse events during 76 weeks of treatment and 22 weeks of follow-up.
6 secondary outcomes
Part A: Number of participants reported with adverse events
Time frame: Day 1 up to 46 Weeks
Part A: Number of participants with incidence and titer of anti-SAR445088 antibodies (ADA)
Time frame: Day 1 up to 46 Weeks
Part A: Percentage of participants in the SOC-Treated group improving during the overlap treatment period
Time frame: Day 1 up to 12 Weeks
Part B, SOC-Treated (initial dose group): Percentage of participants relapse-free during the treatment extension period
Time frame: Week 24 up to Week 76
Part B, SOC-Refractory (initial and low dose group) and SOC-Naive: Percentage of participants with sustained response during the treatment extension period
Time frame: Week 24 up to Week 76
Part B: Long-term immunogenicity
Time frame: Day 1 Up to Week 98
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 28 Oct 2025.
- Derived from this study: PubMed 37119056Querol L, Lewis RA, Hartung HP, Van Doorn PA, Wallstroem E, Luo X, Alonso-Alonso M, Atassi N, Hughes RAC. An innovative phase 2 proof-of-concept trial design to evaluate SAR445088, a monoclonal antibody targeting complement C1s in chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2023 Jun;28(2):276-285. doi: 10.1111/jns.12551. Epub 2023 May 31.
- PDY16744 Plain Language Results SummariesLink provided by the sponsor.
- 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.
Clinical Sciences & Operations study director
Sanofi
32 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SAR445088 (IV) and SAR445088 (SC) in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy, run by Bioverativ, a Sanofi company at 32 sites in 10 countries.
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 15 inclusion and 20 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 28 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, France, Germany, Italy, China and 5 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Part A, SOC-Treated: Percentage of participants relapsing after withdrawal of SOC and during the SAR445088 treatment period (time frame: Day 1 up to 24 weeks). The study lists 3 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 NCT04658472, 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 28 Oct 2025.