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AZD0530 Difumarate

Phase 2

Fibrodysplasia Ossificans Progressiva | Small Molecules | Rare Disease |AstraZeneca PLC|Trials Updated: Sep 2, 2026

AZD0530 Difumarate development status

Highest phase Phase 2
Registered trials 2 across 2 sponsors since Sep 2018

AZD0530 Difumarate target and mechanism

ModalitySmall Molecules

Also known as Saracatinib

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment20

FDA Designations

No designations recorded

AZD0530 Difumarate clinical trials

AZD0530 Difumarate · 1 trial · 1 indication

Phase 2 1
NCT04307953Saracatinib Trial TO Prevent FOPFibrodysplasia Ossificans Progressiva
ACTIVE NOT_RECRUITING20 Analytics
PHASE2ACTIVE NOT_RECRUITING
Saracatinib Trial TO Prevent FOP
Fibrodysplasia Ossificans Progressiva
NCT0430795320
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Study Endpoints

Primary Endpoints

Number of new active HO lesions on [18F] NaF PET/ CT
Baseline, month 6

The change between the two arms in development of new active heterotopic bone lesions measured by \[18F\] NaF PET/low-dose whole body CT during the initial 6-month RCT

Secondary Endpoints

Safety and tolerability assessments are the incidence and severity of adverse events (AE) during the RCT at the end of week 26.
Baseline, month 6 (+overall duration study)
New active HO lesions of the placebo arm during 26 weeks RCT compared to its roll over to 26-week open label treatment
Baseline, month 6, month 12
The change in heterotopic bone volume measured by low-dose whole body CT over twelve-months treatment during open-label extension of AZD0530 compared to the historical data of Clementia (NCT02322255)
Baseline, month 6, month 12, month 18
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AZD0530EXPERIMENTAL -
Placebo/AZD0530EXPERIMENTAL -

Interventions

NameTypeDescription
AZD0530 DifumarateDRUGAZD0530 for the duration of the trial
Matching placeboDRUGMatching placebo during 6 month RCT, AZD0530 thereafter
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Male or female aged 18-65 with a clinical diagnosis of FOP at screening, including congenital malformation of the great toes and a history of spontaneous or injury-induced heterotopic ossification (HO), and have a confirmed classic FOP phenotype by the documentation of an ACV...

Countries:GermanyNetherlandsUnited Kingdom
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