Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The primary objective for this study is to evaluate the efficacy of doxycycline + ursodeoxycholic acid (UDCA) on disease progression in Transthyretin Amyloidosis (ATTR) subjects with cardiomyopathy with or without neuropathy.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Doxycycline and UDCA | Experimental | Doxycycline (200 mg/day intermittently) and UDCA (750 mg/day continuously) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Doxycycline | Drug | 200 mg/day (100 mg twice daily, orally) for 4 weeks with a pause of 2 weeks in combination with UDCA |
|
| Measure | Description | Time Frame |
|---|---|---|
| The efficacy on serum N terminal proBNP (NT-proBNP) | The primary endpoint of the study is the response rate to doxycycline + UDCA treatment at month 12. A responder is an ATTR subject with: - a reduction of, or an increase in serum NT-proBNP concentration of less than 30% of pre-treatment level will be regarded as consistent with treatment efficacy | At 12 month treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Modified Body Mass Index (mBMI) reduction | mBMI-reduction of less than 10% | 12 month |
| Increase of septum thickness | Increase of septum thickness ≤ 2 mm |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Ole B Suhr, MD PhD Prof | Dept of Clinical Medicine and public Health, Umeå University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Dept of Clinical Medicin, Ptieå Hospital | Piteå | SE-941 28 | Sweden | |||
| Dept of clinical medicin, Skellefteå Hospital |
Not provided
| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D009202 | Cardiomyopathies |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D004318 | Doxycycline |
| D014580 | Ursodeoxycholic Acid |
| ID | Term |
|---|---|
| D013754 | Tetracyclines |
| D009279 | Naphthacenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Ursodeoxycholic acid | Drug | 750 mg/day (500 mg +250mg orally) continuously |
|
|
| 12 month |
| Neurologic Kumamoto Scale | To assess the change from baseline in the neurologic Kumamoto Scale | 6, 12 and 18 month |
| Number of patients with adverse events | To assess the tolerability and safety of the treatment, the number of patients with adverse reactions will be recorded. Monthly phone contacts will be performed for monitoring of the treatment safety. The safety profile of doxycycline + UDCA will be assessed through the recording, reporting and analysis of baseline medical conditions, physical examination findings including vital signs and laboratory tests. These will be compared to analysis results observed during the study. | During 12 month treatment and during 6 month follow-up |
| Blood work for potential drug-related adverse events | To assess the tolerability and safety of the treatment, blood work [e.g.complete blood count, creatinine and aspartate transaminase (AST), alkaline phosphatase(ALT)] for potential drug-related adverse events will be drawn at 1, 3, 6, 9, 12 and 18 month. The safety profile of doxycycline + UDCA will be assessed through the recording, reporting and analysis of baseline medical conditions, physical examination findings including vital signs and laboratory tests. These will be compared to analysis results observed during the study. | 18 months |
| Skellefteå |
| SE-931 86 |
| Sweden |
| Dept of Clinical Medicine, Umeå University Hospital | Umeå | SE-90185 | Sweden |
| D006844 |
| Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D011083 | Polycyclic Compounds |
| D003840 | Deoxycholic Acid |
| D002793 | Cholic Acids |
| D001647 | Bile Acids and Salts |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D002757 | Cholanes |