Transthyretin Amyloid PolyneuropathyPhase 4NCT06940336
To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
A Phase 4 study testing Tafamidis Meglumine Soft Capsules in people with Transthyretin Amyloid Polyneuropathy, run by Qilu Pharmaceutical Co., Ltd. at 1 site in China.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Transthyretin Amyloid Polyneuropathy
- What is being tested?
- Tafamidis Meglumine Soft Capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 19 Jun 2025to Feb 2028
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female participants between the ages of 18 and 80 years.
- Participant has amyloid deposits confirmed by biopsy (Biopsy must have been performed within 5 years prior to enrollment).
- Participant must have a TTR mutation that is associated with ATTR-PN (TTR mutation test must have been performed within 5 years prior to enrollment).
- Participant has peripheral neuropathy at screening.
- Participant has a Karnofsky Performance Status Score ≥50.
- Stages of disease according to symptom severity-stage 1.
Who cannot join
- Participant has other causes of amyloidosis, such as light chain amyloidosis, AA amyloidosis.
- Participant has used tafamidis within 2 months prior to enrollment.
- Participant has used diflunisal, patisiran, inotersen, or other agents for familial amyloidosis within 30 days prior to enrollment or plan to use them during the study period.
- Participant has used non-protocol NSAIDs more than 4 times within 30 days prior to enrollment or plan to use them more than 4 times per month during the study period.
- Participant has used doxycycline,tauroursodeoxycholate within 14 days prior to enrollment or plan to use them during the study period.
- Participant has sensory motor neuropathy caused by other causes, such as chronic inflammatory demyelinating polyradiculopathy, chronic idiopathic axonal neuropathy, diabetic neuropathy, chronic alcoholic neuropathy, paraneoplastic neuropathy,Guillain-Barre syndrome, vitamin B12 deficiency.
- Participant has received liver or any other organ except cornea transplantation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Qilu Hospital of Shandong University
Jinan
chuanzhu yan, Doctor, 18560082288, zzl2002@medmail.com
About this study
Official title: A Single Arm, Multicenter, Open-label,Phase IV Clinical Study to Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients withTransthyretin Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy
- Conditions
- Transthyretin Amyloid Polyneuropathy
- Being tested
- Tafamidis Meglumine Soft Capsules
- Medical subject headings
- Amyloid Neuropathies, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tafamidis Meglumine Soft CapsulesDrug
20 mg orally once daily for 72 weeks
| Study group | Role | What they receive |
|---|---|---|
| Tafamidis Meglumine Soft Capsules | Experimental | Not described Receives: Drug: Tafamidis Meglumine Soft Capsules |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
Measured: Baseline to 72 Weeks
Secondary outcomes (2)
Change from Baseline NIS-LL at Week 24 and Week 48
Change from Baseline NIS-LL at Week 24 and Week 48
Measured: Baseline to 24 and 48 Weeks
Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72
Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72
Measured: Baseline to 4, 8, 12, 24, 36, 48 and 72 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2025
- Study start19 Jun 2025
- Primary completionJan 2028 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin Amyloid PolyneuropathyStudies of the same condition, open and under way.
- More trials run by Qilu Pharmaceutical Co., Ltd.The 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Qilu Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06940336
- Sponsor's protocol ID
- QLG1046-401
- Status last verified
- Apr 2025
- First submitted
- 8 Apr 2025
- Last updated
- 30 Jul 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm, Multicenter, Open-label,Phase IV Clinical Study to Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients withTransthyretin Amyloid Polyneuropathy
- ClinicalTrials.gov ID
- NCT06940336
- Acronym / sponsor ID
- QLG1046-401
- Conditions
- Transthyretin Amyloid Polyneuropathy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Qilu Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 19 Jun 2025 (actual)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 23 Apr 2025 (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.
The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tafamidis Meglumine Soft Capsules | Experimental | Drug: Tafamidis Meglumine Soft Capsules |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female participants between the ages of 18 and 80 years.
- Participant has amyloid deposits confirmed by biopsy (Biopsy must have been performed within 5 years prior to enrollment).
- Participant must have a TTR mutation that is associated with ATTR-PN (TTR mutation test must have been performed within 5 years prior to enrollment).
- Participant has peripheral neuropathy at screening.
- Participant has a Karnofsky Performance Status Score ≥50.
Key exclusion criteria (7 listed)
- Participant has other causes of amyloidosis, such as light chain amyloidosis, AA amyloidosis.
- Participant has used tafamidis within 2 months prior to enrollment.
- Participant has used diflunisal, patisiran, inotersen, or other agents for familial amyloidosis within 30 days prior to enrollment or plan to use them during the study period.
- Participant has used non-protocol NSAIDs more than 4 times within 30 days prior to enrollment or plan to use them more than 4 times per month during the study period.
- Participant has used doxycycline,tauroursodeoxycholate within 14 days prior to enrollment or plan to use them during the study period.
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 |
|---|---|---|
| Tafamidis Meglumine Soft Capsules20 mg orally once daily for 72 weeks | Drug | Tafamidis Meglumine Soft Capsules |
Primary outcome
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
Time frame: Baseline to 72 Weeks
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
2 secondary outcomes
Change from Baseline NIS-LL at Week 24 and Week 48
Time frame: Baseline to 24 and 48 Weeks
Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72
Time frame: Baseline to 4, 8, 12, 24, 36, 48 and 72 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2025.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tafamidis Meglumine Soft Capsules in people with Transthyretin Amyloid Polyneuropathy, run by Qilu Pharmaceutical Co., Ltd. at 1 site in China.
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 6 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 30 Jul 2025. 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 China. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact chuanzhu yan, Doctor (18560082288, zzl2002@medmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72 (time frame: Baseline to 72 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2025.