Phase 2
N=129
The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis
AL Amyloidosis
Bottom Line
View on ClinicalTrials.gov: NCT02632786 ↗Enrolled (actual)
129
Serious AEs
22.5%
Results posted
Apr 2019
Primary outcome: Primary: Number of Participants With Cardiac Response and Non-Response — 26; 30; 40; 33 Participants — p=0.3190
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- NEOD001 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Prothena Biosciences Ltd.
- Primary completion
- Mar 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Cardiac Response and Non-Response |
26; 30; 40; 33 | 0.3190 |
| SECONDARY SF-36v2 PCS Score |
0.19; 0.97 | 0.5563 |
| SECONDARY 6MWT Distance |
19.25; 8.00 | 0.8992 |
| SECONDARY Number of Participants With Renal Best Response and Non-Response |
7; 6; 6; 12 | 0.3529 |
| SECONDARY NIS-LL Total Score |
-1.2; -0.6 | 0.7570 |
| SECONDARY NT-proBNP Slope |
9.45; 81.41 | 0.0729 |
| SECONDARY Hepatic Best Response |
1; 0; 4; 4 | 0.4142 |
Summary
This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.
Eligibility Criteria
Inclusion Criteria
- Age ≥18 years
- Confirmed diagnosis of systemic AL amyloidosis
- ≥1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic response
- Cardiac involvement
- NT-proBNP ≥650
Exclusion Criteria
- Non-AL amyloidosis
- Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma
- NT-proBNP >5000
- Received Plasma cell directed chemotherapy within 6 months
- Received autologous stem cell transplant (ASCT) within 12 months
Data sourced from ClinicalTrials.gov (NCT02632786). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.