A Phase I/IIa (early phase) Study of Targeted Radiotherapy alone for Stem Cell Transplant Conditioning in Systemic AL Amyloidosis

• Conditions: AL-Amyloidosis; MedDRA version: 18.0Level: LLTClassification code 10024460Term: Light chain disease myeloma associatedSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2015-002231-18-GB
• Lead Sponsor: niversity Southampton Hospital NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-09-23
• Last Update Posted: 18 April 2016

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

The main inclusion criteria is patients with systemic AL-amyloidosis with an indication for treatment who satisfy all standard transplant inclusion criteria (good organ function, no significant heart involvement, good performance status).

•Aged =18 years.
•Have a diagnosis of systemic AL-amyloidosis, either as a new diagnosis or recurrent disease.
•Measurable clonal plasma cell dyscrasia.
•Amyloid related organ dysfunction or organ syndrome.
•Estimated life expectancy of at least 6 months (as defined at trial entry).
•Sufficient stem cells for two transplant procedures .
•Bone Marrow (BM) cellularity >20%.
•Eligible for ASCT in AL amyloidosis defined as fulfilling all of the following criteria :
oECOG Performance Status of 0 or 1
oCardiac troponin-T <0.07 µg/L
oNYHA heart failure class of <3
oNo more than 3 organs involved by amyloidosis by consensus guidelines.
oCreatinine clearance or isotope GFR =30ml/min.
oBilirubin =1.5 times and alkaline phosphatase =3 x upper limit of normal.
oAST or ALT <2.5 x upper limit of normal range.
oMean left ventricular wall thicknesses of <16mm by echocardiography.
oAbsence of clinically important amyloid related autonomic neuropathy .
oAbsence of clinically important amyloid related gastro intestinal haemorrhage.
•Capable of providing written, informed consent.
•Women of child bearing potential should use adequate forms of contraception.
oIntrauterine Device (IUD)
oHormonal based contraception (pill, contraceptive injection etc.)
oDouble Barrier contraception (condom and occlusive cap e.g. diaphragm or cervical cap with spermicide)
oTrue abstinence

Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 18

Exclusion Criteria

Patients with poor performance, advanced organ involvement or significant cardiac involvement will be excluded from the study.

Patients with the following characteristics are ineligible for this study:
•Overt symptomatic multiple myeloma.
•Amyloidosis of unknown or non AL type.
•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).
•Trivial or incidental AL amyloid deposits in the absence of a significant amyloid related organ syndrome (e.g., isolated carpal tunnel syndrome).
•NYHA Class III or IV heart failure (appendix 1).
•Liver involvement by amyloid causing bilirubin >1.5 times upper limit of normal.
•Concurrent active malignancies, except surgically removed basal cell carcinoma of the skin or other in situ carcinomas.
•Pregnant, lactating or unwilling to use adequate contraception as listed above
•Intolerance / sensitivity to any of the study drugs.
•Known positive Human anti-murine antibodies (HAMA).
•Unable to provide written informed consent
•Involved in another IMP trial

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified