A phase II study of the safety and efficacy of doxycycline administered cyclically in patients suffering from Transthyretin Amiloidosis ATTR - ND

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-000012-24-IT

Enrollment

Registered

2006-03-15

Start date

2006-01-24

Completion date

Unknown

Last updated

2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin amyloidosis ATTR MedDRA version: 6.1 Level: HLGT Classification code 10034606

Interventions

Trade Name: BASSADO 10 CPR 100 MG Pharmaceutical Form: Tablet INN or Proposed INN: Doxycycline CAS Number: 24390-14-5 Concentration unit: mg milligram(s) Concentration type: equal Concentration number

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: age / 18 years, histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green birefringent material in Congo red-stained tissue specimens, NYHA class / III, contraception for women of potential childbearing, ... Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: prior liver transplantation or liver transplantation anticipated in 1 year, uncontrolled infection, any known active inflammatory process, significant acute gastrointestinal symptoms, active peptic ulceration, ...

Design outcomes

Primary

Measure	Time frame
Main Objective: Primary objective is to establish the clinical efficacy, in terms of response rate, of doxycycline in ATTR;Secondary Objective: The following variables will be assessed and evaluated in all patients safety profile time to progression of amyloidosis-associated clinical symptoms and/or organ dysfunction duration of response ;Primary end point(s): Response is the primary endpoint and will be evaluated according to response criteria listed below. Evaluation of treatment efficacy will be performed after 3 and 6 cycles. Improvement of one or more affected organ s as defined by Neuropathy clinical improvement according to the Kumamoto Neurologic Scale9 and/or to EMG studies. Patients will need at least 1 point improvement on their neuropathy disability score. Heart / 2 mm reduction in interventricular septal IVS thickness by echocardiogram or improvement of ejection fraction by / 20 echocardiogram must be performed at the same institution or 50 reduction of abnormal NT-proBNP concentration or return into reference interval. GI tract reduction of diarrhea to less than 50 of previous movements/day. Malabsorption will be evaluated by serial measurement of transthyretin concentration. It has been reported that increase of TTR 4 mg/dL/week reflect improved nutritional status. Only 1 parameter is required to satisfy the response criteria.	—

Measure

Time frame

Main Objective: Primary objective is to establish the clinical efficacy, in terms of response rate, of doxycycline in ATTR;Secondary Objective: The following variables will be assessed and evaluated in all patients safety profile time to progression of amyloidosis-associated clinical symptoms and/or organ dysfunction duration of response ;Primary end point(s): Response is the primary endpoint and will be evaluated according to response criteria listed below. Evaluation of treatment efficacy will be performed after 3 and 6 cycles. Improvement of one or more affected organ s as defined by Neuropathy clinical improvement according to the Kumamoto Neurologic Scale9 and/or to EMG studies. Patients will need at least 1 point improvement on their neuropathy disability score. Heart / 2 mm reduction in interventricular septal IVS thickness by echocardiogram or improvement of ejection fraction by / 20 echocardiogram must be performed at the same institution or 50 reduction of abnormal NT-proBNP concentration or return into reference interval. GI tract reduction of diarrhea to less than 50 of previous movements/day. Malabsorption will be evaluated by serial measurement of transthyretin concentration. It has been reported that increase of TTR 4 mg/dL/week reflect improved nutritional status. Only 1 parameter is required to satisfy the response criteria.

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Countries

Italy

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026