Familial platelet disorder with associated myeloid malignancy

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At a Glance

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Regulatory Watchboard

4 events
Jun 2024Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder

M.D. Anderson Cancer Center — PHASE2

TrialRECRUITING
May 2024Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder

M.D. Anderson Cancer Center — PHASE1

TrialRECRUITING
Feb 2021

Pepaxto: FDA approved

Treatment, in combination with dexamethasone, of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody (Indication withdrawn)

FDAcompleted
Mar 2019Longitudinal Studies of Patient With FPDMM

National Human Genome Research Institute (NHGRI)

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

2 programs

FINANCIAL LANDSCAPE SUMMARY

2

Total programs

2

Open now

1

Copay cards

Copay Assistance1

Pepaxto

Oncopeptides AB

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Patient Assistance Programs1

Neulasta

Amgen, Inc.

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Approved Treatments

1 FDA-approved

Bosulif

(bosutinib)Orphan drugstandard

PF PRISM C.V.

12.1 Mechanism of Action Bosutinib is a TKI. Bosutinib inhibits the BCR-ABL kinase that promotes CML; it is also an inhibitor of Src-family kinases in...

FDA label ↗

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Clinical Trial Landscape

3 active trials
1Phase 2
1Phase 1
1Unknown
3Total recruiting
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Specialist Network

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