Amyloid Transthyretin CardiomyopathyPhase 1NCT04360434
First-in-Human Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy
A Phase 1 study testing NI006 in people with Amyloid Transthyretin Cardiomyopathy, run by Neurimmune AG at 6 sites in 4 countries.
Trial summary
A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloid Transthyretin Cardiomyopathy
- What is being tested?
- NI006
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 6 sitesin 4 countries
- Study dates
- 10 Feb 2020to 26 Jul 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent obtained from the subject prior to any trial-related procedure indicating that he/she understands the purpose of, and procedures required for the trial and is willing to participate in it
- Male or female subjects aged ≥18 years (and < 85 years only for cohort 7) at the time of obtaining informed consent and with confirmed availability for the scheduled trial visits
- Confirmed ATTR-Cardiomyopathy diagnosis established by:
- Polarizing light microscopy of green birefringent material in Congo red-stained tissue specimens and confirmed diagnosis of ATTR amyloidosis by IHC or mass spectrometry OR
- positive bone scintigraphy using either DPD, HMDP or PYP, with cardiac signal intensity indicative of ATTR-Cardiomyopathy (early phase imaging: cardiac mediastinum ratio > 1.21; late phase imaging: Perugini Grade 2 or 3) and absence of gammopathy (negative serum and urine immunofixation electrophoresis plus normal free light chain serum ratio). If a gammopathy is detected, diagnosis must be established based on tissue biopsy as indicated above
- Known genotype as follows:
Show all 20Show fewer
- Known pathogenic TTR mutation for subjects with hereditary ATTR- Cardiomyopathy
- Known negative genetic testing for a TTR mutation for subjects with sporadic, WT- ATTR-Cardiomyopathy
- Chronic Heart Failure with all of the following characteristics:
- LVEF ≥40%
- LVWT ≥14 mm, measured by echocardiography
- NT-proBNP level ≥600 pg/mL
- Able to walk ≥150 meter in the 6-MWT
- NYHA class III (applicable only for cohort 7)
- No hospitalizations for cardiac disease for at least 30 calendar days prior to screening
- General health status acceptable for a participation in a clinical trial with a Karnofsky Performance Status ≥60%
- Stable pharmacological treatment of any other chronic condition for at least 30 calendar days prior to screening, with the exclusion of immunomodulatory and immunosuppressive treatments
- ANC ≥1000 cells/mm³, platelet count ≥100,000 cells/mm³, and hemoglobin ≥10 g/dL
- Women of childbearing potential must have a negative serum pregnancy test at screening and must agree to use highly effective physician-approved contraception from screening to 5 months after ending trial participation
- Males must be surgically sterile or must agree to use highly effective physician-approved contraception throughout of the trial participation, and for 5 months after ending trial participation
Who cannot join
- Amyloid light-chain amyloidosis or any other non ATTR amyloidosis
- Heart failure corresponding to NYHA class IV
- Uncontrolled hypertension with systolic pressure ≥180 mmHg or diastolic pressure ≥110 mmHg confirmed by 3 measurements in supine position recorded with 5 minutes break in between the measurements
- Hypotension with systolic pressure ≤ 90 mmHg or diastolic pressure ≤ 60 mmHg confirmed by 3 measurements in supine position recorded with 5 minutes break in between the measurements
- NT-proBNP ≥6'000 pg/mL (NT-proBNP ≥8'500 pg/mL applicable only for cohort 7)
- Heart failure not predominantly caused by ATTR-Cardiomyopathy
Show all 33Show fewer
- Any severe uncorrected valve disease
- Chronic liver disease with liver function test abnormalities:
- ALT and AST > 2.5 × ULN
- Total bilirubin > 2 × ULN
- Respiratory insufficiency requiring oxygen therapy
- Renal insufficiency with eGFR < 30 mL/min/1.73 m2 using the CKD-EPI equation
- Active malignancy with exception of the following:
- Adequately treated basal cell carcinoma
- Squamous cell carcinoma of the skin
- In situ cervical cancer
- Low risk prostate cancer with Gleason score < 7 and prostate specific antigen < 10 mg/mL
- Any other cancer from which the subject has been disease-free for ≥ 2 years
- Uncontrolled infection as per Investigator's judgement
- Known HIV infection, seropositivity for HIV, hepatitis B and C as well as active hepatitis A
- Autoimmune disease requiring immunosuppressive/modulating treatment in the last 2 years
- History of organ transplantation or ventricular assist device
- Polyneuropathy disability score > IIIA
- Suspected or known intolerance/allergy to proteins or any components of the investigational medicinal product
- Concomitant immunosuppressant therapy e.g., corticosteroids, prednisone, dexamethasone except as indicated in low dose (i.e., up to 10 mg prednisone or equivalent daily is allowed) for other medical conditions such as inhaled steroid for asthma
- Use of the following drugs acting on TTR or ATTR: tolcapone, diflunisal, patisiran, inotersen, and long-term doxycycline, in the 30 calendar days prior to signing informed consent form. Tafamidis is permitted if it is given as standard of care in a stable dose for at least 30 calendar days prior to signing the informed consent form
- Participation in another investigational clinical trial or intake of investigational drug within 30 calendar days before signing the informed consent form
- Suspected or known drug or alcohol abuse
- Serious psychiatric or any other medical condition (including laboratory abnormalities), which, in the opinion of the Investigator, makes the subject unsuitable for inclusion and puts the subject at an unacceptable risk
- Subject is nursing or is considering becoming pregnant during the trial or in the 5 months after ending trial participation
- Unwillingness or inability to adhere to the trial requirements
- If subject is in any way dependent on Neurimmune AG or the principal Investigator or if the subject is accommodated in an establishment on judicial or administrative order
- Employee or immediate family (spouse, parent, child or sibling, whether biological or legally adopted) of an employee of Neurimmune AG, the contract research organization or the trial site
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
France3 sites
Hôpital Henri Mondor
Créteil, 94000
CHU de Rennes - Hôpital Pontchaillou
Rennes, 35033
CHU Toulouse - Hôpital Rangueil
Toulouse, 31059
Spain1 site
Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda, 28222
About this study
Official title: A Phase 1, First-in-Human, Double-Blind, Placebo-Controlled, Multicenter, Single and Multiple Ascending Dose Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy Followed by an Open-Label Extension
A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM).
Read the detailed description
This phase 1, randomized, placebo-controlled, double-blind trial in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM) consists of single-ascending dose (SAD) and multiple-ascending dose (MAD) phases, followed by an open-label extension (OLE) phase.
In the SAD phase subjects are randomized in a 4:2 ratio to receive a single infusion of NI006 or placebo.
Subjects completing the SAD phase will be enrolled in the MAD phase upon evaluation of all available safety data and receive a maximum of 3 additional infusions of NI006 or placebo every 28 days.
Subjects completing the MAD phase will have the possibility to continue in an OLE phase with treatment up-titrations and switch from placebo to NI006 and receive up to 8 infusions of NI006 every 28 days.
Subjects of cohort 1 to 5 who received at least one dose of NI006 during the OLE phase will have the possibility for a second OLE phase (OLE2) after completing the OLE phase and receive up to 10 additional infusions of NI006 every 28 days.
In total, about 42 subjects are planned to be enrolled in 7 cohorts of 6 subjects each, at 6 ascending dose levels.
- Conditions
- Amyloid Transthyretin Cardiomyopathy
- Being tested
- NI006, Placebo
- Keywords
- ATTR, Transthyretin, Amyloidosis, Cardiomyopathy, ATTR-CM
- Medical subject headings
- Amyloidosis, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NI006Drug
NI006 will be administered intravenously
PlaceboDrug
Formulation buffer of NI006, matching volume of NI006 doses will be administered intravenously
| Study group | Role | What they receive |
|---|---|---|
| NI006 | Experimental | Dose escalation in up to 6 dose cohorts. Subjects will be administered a single dose of NI006 in the SAD, multiple doses of NI006 in the MAD and OLE phases. Receives: Drug: NI006 |
| Placebo | Placebo comparator | Subjects will be administered a single dose of placebo in the SAD phase and multiple doses of placebo in the MAD phase.
In the OLE phase, all subjects will be administered multiple doses of NI006. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters (hematology, clinical chemistry, immunology, urinalysis), vital signs, electrocardiogram and echocardiogram
Measured: 4 months
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters (hematology, clinical chemistry, immunology, urinalysis), vital signs, electrocardiogram and echocardiogram
Measured: 12 months
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters (hematology, clinical chemistry, immunology, urinalysis), vital signs, electrocardiogram and echocardiogram
Measured: additional up to 10 months
Secondary outcomes (14)
NI006 pharmacokinetic profile and parameters - Cmax
Maximum observed serum concentration (Cmax) of NI006
Measured: 4 months
NI006 pharmacokinetic profile and parameters - Tmax
Time to maximum observed serum concentration (Tmax) of NI006
Measured: 4 months
NI006 pharmacokinetic profile and parameters - AUCinf
Area under the serum concentration-time curve from zero to infinity (AUCinf) of NI006
Measured: 1 month
NI006 pharmacokinetic profile and parameters - CL
Serum clearance (CL) of NI006
Measured: 4 months
NI006 pharmacokinetic profile and parameters - Vz
NI006 apparent volume of distribution during terminal phase (Vz)
Measured: 4 months
NI006 pharmacokinetic profile and parameters - Vss
NI006 apparent volume of distribution at steady state (Vss)
Measured: 4 months
NI006 pharmacokinetic profile and parameters - t½
Terminal elimination half-life (t½) of NI006 in serum
Measured: 4 months
NI006 pharmacokinetic profile and parameters - AUCtau
Area under the serum concentration-time curve from time zero to the end of the dosing interval after the first dose (AUCtau) of NI006
Measured: 4 months
NI006 pharmacokinetic profile and parameters - RaccCmax
Accumulation ratio for maximum concentration (RaccCmax) of NI006 in serum
Measured: 4 months
NI006 pharmacokinetic profile and parameters - RaccAUC
Accumulation ratio calculated from AUC (RaccAUC) of NI006 in serum
Measured: 4 months
NI006 pharmacokinetic profile and parameters - Ctrough
Minimum observed concentration (Ctrough) of NI006 in serum
Measured: 12 months
NI006 OLE2 pharmacokinetic profile and parameters - Ctrough
Minimum observed concentration (Ctrough) of NI006 in serum
Measured: up to 10 months
NI006 pharmacokinetic profile and parameters - dose-normalized Ctrough
Dose-normalized minimum observed concentration (Ctrough) of NI006 in serum
Measured: 12 months
NI006 OLE2 pharmacokinetic profile and parameters - dose-normalized Ctrough
Dose-normalized minimum observed concentration (Ctrough) of NI006 in serum
Measured: up to 10 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2020
- Study start10 Feb 2020
- Primary completion26 Jul 2023
- Study completion26 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Amyloid Transthyretin CardiomyopathyStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurimmune AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04360434
- Sponsor's protocol ID
- NI006-101
- Other IDs
- 2019-001932-80, 2019
- Status last verified
- Nov 2023
- First submitted
- 11 Mar 2020
- Last updated
- 3 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Kahr PC, Aus dem Siepen F, Lairez O, Donal E, Damy T, Kristen AV, Quarta CC, Mercuri MF, Keppner-Witter S, Herrmann-Keiner E, Tichy M, Michalon A, Grimm J, Nitsch RM, Hock C, Garcia-Pavia P, van der Meer P. Cliramitug for depletion of cardiac amyloid transthyretin: long-term follow-up of the NI006-101 trial. Nat Med. 2026 Jul;32(7):2639-2646. doi: 10.1038/s41591-026-04487-3. Epub 2026 Jun 26.
PubMed 42362866Garcia-Pavia P, Aus dem Siepen F, Donal E, Lairez O, van der Meer P, Kristen AV, Mercuri MF, Michalon A, Frost RJA, Grimm J, Nitsch RM, Hock C, Kahr PC, Damy T. Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid. N Engl J Med. 2023 Jul 20;389(3):239-250. doi: 10.1056/NEJMoa2303765. Epub 2023 May 20.
PubMed 37212440
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, First-in-Human, Double-Blind, Placebo-Controlled, Multicenter, Single and Multiple Ascending Dose Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy Followed by an Open-Label Extension
- ClinicalTrials.gov ID
- NCT04360434
- Acronym / sponsor ID
- NI006-101
- Conditions
- Amyloid Transthyretin Cardiomyopathy
- Keywords
- ATTR, Transthyretin, Amyloidosis, Cardiomyopathy, ATTR-CM
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Neurimmune AGIndustry (pharma, biotech, devices)
- Study start
- 10 Feb 2020 (actual)
- Primary completion
- 26 Jul 2023 (actual)
- Study completion
- 26 Jul 2023 (actual)
- First posted
- 24 Apr 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.
A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM).
Detailed description
This phase 1, randomized, placebo-controlled, double-blind trial in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM) consists of single-ascending dose (SAD) and multiple-ascending dose (MAD) phases, followed by an open-label extension (OLE) phase.
In the SAD phase subjects are randomized in a 4:2 ratio to receive a single infusion of NI006 or placebo.
Subjects completing the SAD phase will be enrolled in the MAD phase upon evaluation of all available safety data and receive a maximum of 3 additional infusions of NI006 or placebo every 28 days.
Subjects completing the MAD phase will have the possibility to continue in an OLE phase with treatment up-titrations and switch from placebo to NI006 and receive up to 8 infusions of NI006 every 28 days.
Subjects of cohort 1 to 5 who received at least one dose of NI006 during the OLE phase will have the possibility for a second OLE phase (OLE2) after completing the OLE phase and receive up to 10 additional infusions of NI006 every 28 days.
In total, about 42 subjects are planned to be enrolled in 7 cohorts of 6 subjects each, at 6 ascending dose levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NI006 | Experimental | Drug: NI006Dose escalation in up to 6 dose cohorts. Subjects will be administered a single dose of NI006 in the SAD, multiple doses of NI006 in the MAD and OLE phases. |
| Placebo | Placebo comparator | Drug: PlaceboSubjects will be administered a single dose of placebo in the SAD phase and multiple doses of placebo in the MAD phase. In the OLE phase, all subjects will be administered multiple doses of NI006. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Written informed consent obtained from the subject prior to any trial-related procedure indicating that he/she understands the purpose of, and procedures required for the trial and is willing to participate in it
- Male or female subjects aged ≥18 years (and < 85 years only for cohort 7) at the time of obtaining informed consent and with confirmed availability for the scheduled trial visits
- Confirmed ATTR-Cardiomyopathy diagnosis established by:
- Known genotype as follows:
- Chronic Heart Failure with all of the following characteristics:
Key exclusion criteria (33 listed)
- Amyloid light-chain amyloidosis or any other non ATTR amyloidosis
- Heart failure corresponding to NYHA class IV
- Uncontrolled hypertension with systolic pressure ≥180 mmHg or diastolic pressure ≥110 mmHg confirmed by 3 measurements in supine position recorded with 5 minutes break in between the measurements
- Hypotension with systolic pressure ≤ 90 mmHg or diastolic pressure ≤ 60 mmHg confirmed by 3 measurements in supine position recorded with 5 minutes break in between the measurements
- NT-proBNP ≥6'000 pg/mL (NT-proBNP ≥8'500 pg/mL applicable only for cohort 7)
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 |
|---|---|---|
| NI006NI006 will be administered intravenously | Drug | NI006 |
| PlaceboFormulation buffer of NI006, matching volume of NI006 doses will be administered intravenously | Drug | Placebo |
Primary outcome
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram
Time frame: 4 months
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters (hematology, clinical chemistry, immunology, urinalysis), vital signs, electrocardiogram and echocardiogram
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram
Time frame: 12 months
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters (hematology, clinical chemistry, immunology, urinalysis), vital signs, electrocardiogram and echocardiogram
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram
Time frame: additional up to 10 months
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters (hematology, clinical chemistry, immunology, urinalysis), vital signs, electrocardiogram and echocardiogram
14 secondary outcomes
NI006 pharmacokinetic profile and parameters - Cmax
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - Tmax
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - AUCinf
Time frame: 1 month
NI006 pharmacokinetic profile and parameters - CL
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - Vz
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - Vss
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - t½
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - AUCtau
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - RaccCmax
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - RaccAUC
Time frame: 4 months
NI006 pharmacokinetic profile and parameters - Ctrough
Time frame: 12 months
NI006 OLE2 pharmacokinetic profile and parameters - Ctrough
Time frame: up to 10 months
NI006 pharmacokinetic profile and parameters - dose-normalized Ctrough
Time frame: 12 months
NI006 OLE2 pharmacokinetic profile and parameters - dose-normalized Ctrough
Time frame: up to 10 months
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 Nov 2023.
- Derived from this study: PubMed 42362866Kahr PC, Aus dem Siepen F, Lairez O, Donal E, Damy T, Kristen AV, Quarta CC, Mercuri MF, Keppner-Witter S, Herrmann-Keiner E, Tichy M, Michalon A, Grimm J, Nitsch RM, Hock C, Garcia-Pavia P, van der Meer P. Cliramitug for depletion of cardiac amyloid transthyretin: long-term follow-up of the NI006-101 trial. Nat Med. 2026 Jul;32(7):2639-2646. doi: 10.1038/s41591-026-04487-3. Epub 2026 Jun 26.
- Derived from this study: PubMed 37212440Garcia-Pavia P, Aus dem Siepen F, Donal E, Lairez O, van der Meer P, Kristen AV, Mercuri MF, Michalon A, Frost RJA, Grimm J, Nitsch RM, Hock C, Kahr PC, Damy T. Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid. N Engl J Med. 2023 Jul 20;389(3):239-250. doi: 10.1056/NEJMoa2303765. Epub 2023 May 20.
- Individual participant dataSharing planned: Undecided
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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.
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NI006 in people with Amyloid Transthyretin Cardiomyopathy, run by Neurimmune AG at 6 sites in 4 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 20 inclusion and 33 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 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France, Germany, Netherlands, Spain. 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?
Number and proportion of treatment emergent adverse events and serious adverse events and clinically significant changes in laboratory parameters, vital signs, electrocardiogram and echocardiogram (time frame: 4 months). 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 NCT04360434, which also lists 2 linked publications.
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 Nov 2023.