Targeted radiotherapy for AL-Amyloidosis – TRALA

Phase 1

Completed

• Conditions: Systemic AL-Amyloidosis; Nutritional, Metabolic, Endocrine; Amyloidosis

• Registration Number: ISRCTN13400668
• Lead Sponsor: niversity Hospital Southampton NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-11-15
• Study Completion: 2020-07-07
• Last Update Posted: 30 January 2023

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1. Aged =18 years
2. Diagnosis of systemic AL-amyloidosis, either as a new diagnosis or recurrent disease
3. Measurable clonal plasma cell dyscrasia
4. Amyloid related organ dysfunction or organ syndrome
5. Estimated life expectancy of at least 6 months (as defined at trial entry)
6. Sufficient stem cells for two transplant procedures
7. Bone Marrow (BM) cellularity >20%
8. Eligible for ASCT in AL amyloidosis defined as fulfilling all of the following criteria:
8.1. ECOG Performance Status of 0 or 1
8.2. Cardiac troponin-T <0.07 µg/L
8.3. NYHA heart failure class of <3
8.4. No more than 3 organs involved by amyloidosis by consensus guidelines
8.5. Creatinine clearance or isotope GFR =30ml/min
8.6. Bilirubin =1.5 times and alkaline phosphatase =3 x upper limit of normal
8.7. AST or ALT <2.5 x upper limit of normal range
8.8. Mean left ventricular wall thicknesses of <16mm by echocardiography
8.9. Absence of clinically important amyloid related autonomic neuropathy
8.10. Absence of clinically important amyloid related gastro intestinal haemorrhage
9. Capable of providing written, informed consent
10. Women of child bearing potential should use adequate forms of contraception
10.1. Intrauterine Device (IUD)
10.2. Hormonal based contraception (pill, contraceptive injection etc.)
10.3. Double Barrier contraception (condom and occlusive cap e.g. diaphragm or cervical cap with spermicide)
10.4. True abstinence (this is defined as refraining from heterosexual intercourse after receiving [111In] at the Dosimetry and Imaging visit through to final study visit

Exclusion Criteria

1. Overt symptomatic multiple myeloma
2. Amyloidosis of unknown or non AL type
3. Localised AL-amyloidosis (in which amyloid deposits are limited to a typical single organ, for example the bladder or larynx, in association with a clonal proliferative disorder within that organ)
4. Trivial or incidental AL amyloid deposits in the absence of a significant amyloid related organ syndrome (e.g., isolated carpal tunnel syndrome)
5. NYHA Class III or IV heart failure
6. Liver involvement by amyloid causing bilirubin >1.5 times upper limit of normal
7. Concurrent active malignancies, except surgically removed basal cell carcinoma of the skin or other in situ carcinomas
8. Pregnant, lactating or unwilling to use adequate contraception
9. Intolerance / sensitivity to any of the study drugs
10. Known positive Human anti-murine antibodies (HAMA)
11. Unable to provide written informed consent

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> 1. Specific organ toxicity as measured, following patient review, using the CTCAE version 4.0<br> 2. Adverse event rate is measured following patient review and interviews and is measured using the definitions of an adverse event under the clinical trials directive 2001/20/EC<br>