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Plerixafor

Phase 2

Lymphoma | Small molecule | Oncology |Sanofi|Last Updated: Sep 14, 2023

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment5

FDA Designations

No designations recorded

Clinical trial landscape

Plerixafor · 10 trials · 23 indications

Phase 2 3Phase 1 7
NCT01696461A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous PlerixaforRelated Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member
COMPLETED127 Analytics
NCT01753453An Exploratory Safety Study to Investigate the Extent of Tumor Cell Mobilization (TCM) After Use of G-CSF Alone or G-CSF Plus Plerixafor in Multiple Myeloma (MM) Patients Who May be Poor Mobilizers of Stem CellsMultiple Myeloma
COMPLETED23 Analytics
NCT00665314Evaluation of the Safety and Efficacy of the Addition of AMD3100 to a G-CSF Mobilization Regimen in Patients With Lymphoma (NHL and HD) and Multiple Myeloma (MM).Lymphoma
COMPLETED5 Analytics
PHASE2COMPLETED
A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor
Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family MemberUnlock trial analytics
PHASE2COMPLETED
An Exploratory Safety Study to Investigate the Extent of Tumor Cell Mobilization (TCM) After Use of G-CSF Alone or G-CSF Plus Plerixafor in Multiple Myeloma (MM) Patients Who May be Poor Mobilizers of Stem Cells
Multiple MyelomaUnlock trial analytics
PHASE2COMPLETED
Evaluation of the Safety and Efficacy of the Addition of AMD3100 to a G-CSF Mobilization Regimen in Patients With Lymphoma (NHL and HD) and Multiple Myeloma (MM).
LymphomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Donors Whose Cells Were Successfully Mobilized and Collected With a Sufficient CD34+ Cell Dose Using Plerixafor as the Mobilizing Agent, Using an Intention-to-treat Analysis.
donation

Donor mobilization following plerixafor was considered successful if ≥ 2.0x106 CD34+ cells/kg recipient weight was collected in no more than two leukapheresis collections.

The presence of myeloma tumor cells as measured by the percentage of myeloma tumor cells/CD34+ cells
Day 1 to Day 8 of the apheresis/treatment period

Peripheral blood parameters

The presence of myeloma tumor cells as measured by the percentage of myeloma tumor cells/plerixafor cumulative dose/kg body weights
Day 5 to Day 8 of the apheresis/treatment period

Peripheral blood parameters

The presence of myeloma tumor cells as measured by the percentage of myeloma tumor cells/G-CSF cumulative dose/kg body weight
Day 5 to Day 8 of the apheresis/treatment period

Peripheral blood parameters

The change in tumor cell mobilization(TCM) in the peripheral blood
Day 4 pre-G-CSF to Day 5 pre-G-CSF

Peripheral blood parameters

The number of myeloma tumor cells per patient at each apheresis
Day 1 to Day 8 of the apheresis/treatment period

Apheresis product parameters

The number of patients who mobilize at least 4.5x10^5 myeloma tumor cells/kg body weight as measured in each apheresis product
Day 5 to Day 8 of the apheresis/treatment period

Apheresis product parameters

To determine if patients reach a target of ≥ 2x10^6 CD34+ cells/kg within 2 days of apheresis in Non-Hodgkin's Lymphoma (NHL), Hodgkin's Disease (HD) or Multiple Myeloma (MM) patients who are proven poor mobilizer.
after last apheresis
Safety of Investigational Medicinal Product (IMP)
24 months

Determining the causality of adverse events (AEs) and serious adverse events (SAEs)

Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Up to 5 days
Time to Neutrophil Recovery
100 Days post Transplant

leukocytes \> 500/ul on 2 consecutive days

Time to Platelet Recovery
100 Days Post Transplant

platelet \> 20,000/ul on 2 consecutive days

Plerixafor-associated Adverse Events
100 Days Post Transplant
Pharmacokinetics as measured by maximum observed concentration (Cmax)
Pre-dose to 24 hours post-dose
Pharmacokinetics as measured by time to maximum concentration (Tmax)
Pre-dose to 24 hours post-dose
• Pharmacokinetics as measured by area under the concentration-time curve (AUC) from Time 0 to 24 hours post-dose
Pre-dose to 24 hours post-dose
Pharmacokinetics as measured by terminal half-life (t1/2)
Pre-dose to 24 hours post-dose
Pharmacokinetics as measured by apparent volume of distribution (Vz/F)
Pre-dose to 24 hours post-dose
Pharmacokinetics as measured by apparent total systemic clearance (CL/F)
Pre-dose to 24 hours post-dose
Determine the maximum tolerated dose of plerixafor when used in combination with cytarabine and daunorubicin and with and without GCSF
28 days from first dose and up to 42 days following last dose
The maximum tolerated dose of plerixafor when combined with rituximab as treatment for previously treated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)
29 Days
Dose-Normalized Maximum Concentration of Plerixafor (Cmax)
Pre-dose of plerixafor to 24 hours post-plerixafor

Evaluation of Cmax following a single dose of 240 µg/kg plerixafor administered on Day 1. Cmax was normalized by dose.

Dose-Normalized Area Under the Plerixafor Concentration Time Curve From Time 0 to 24 Hours Post-dose (AUC0-24h)
Pre-dose of plerixafor to 24 hours post-plerixafor

Evaluation of AUC0-24 hour following a single dose of 240 µg/kg plerixafor administered on Day 1. AUC0-24 was normalized by dose.

Secondary Endpoints

Incidence and Severity of Acute Toxicities
baseline, Day 1, Day 2, Day 3
Adverse Effects
30 minutes, 60 minutes, 120 minutes, 240 minutes, 1 month, 6 months, 12 months post donation for each subject
Incidence of and Kinetics of Neutrophil and Platelet Recovery After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Day +1 through neutrophil recovery or Day 21 (whichever is first)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Related donors receiving plerixaforEXPERIMENTALCollection of sufficient CD34+ cells using plerixafor as the mobilizing agent. Eligible donors determined according to institutional standards * 18-65 years of age * 6/6 HLA-matched sibling * Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor * Serum creatinine \<1.5 x institution upper limit of normal (ULN) or estimated creatinine clearance (CLCR) \>50 mL/min Treatment Description: * Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later. * Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is \> 4.0 x 106/kg with a minimum of \> 2.0 x 106/kg.
Recipients, myeloablative regimenNO_INTERVENTIONPatients undergoing conditioning under a myeloablative regimen Myeloablative (one of four general regimens): Busulfan (\> 9 mg/kg po or iv total) with fludarabine Busulfan (\> 9 mg/kg po or iv total) with cyclophosphamide Total body irradiation (\> 1000 cGy) plus etoposide Total body irradiation (\> 500 cGy) plus cyclophosphamide
Recipients, reduced intensity conditioning regimen.NO_INTERVENTIONPatients undergoing conditioning using a reduced intensity conditioning regimen. Reduced Intensity (one of three general regimens): Busulfan (\< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (\> 2000 mg/m2 total)
G-CSF aloneACTIVE_COMPARATORPatients will receive G-CSF for 5 consecutive days
G-CSF plus plerixaforEXPERIMENTALPatients will receive G-CSF for 4 consecutive days, then receive plerixafor before the 5th dose of G-CSF
AMD3100 added to a G-CSF Mobilisation regimenEXPERIMENTALAMD3100 added to a G-CSF Mobilisation regimen
G-CSF plus placeboACTIVE_COMPARATORG-CSF plus placebo
Plerixafor (Mozobil)OTHERPlerixafor (Mozobil), continuous 7 day IV infusion. Starting at a dose of 20 ug/kg/hr, and subsequent dose levels of 40, 80 and 120 ug/kg/hr.
Plerixafor 160 μg/kgEXPERIMENTALPatients will receive subcutaneous (SC) injection of 160 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
Plerixafor 240 μg/kgEXPERIMENTALPatients will receive subcutaneous (SC) injection of 240 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
Plerixafor 320 μg/kgEXPERIMENTALPatients will receive subcutaneous (SC) injection of 320 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
Transplant RecipientsEXPERIMENTALTransplant recipients from matched sibling donors and dual cord donors. Subjects will receive plerixafor at 240 ug/kg subcutaneously every other day beginning at day +2 after transplant until day +21 or engraftment occurs.
plerixaforEXPERIMENTALSingle subcutaneous (SC) dose of plerixafor (160 μg/kg, 240 μg/kg, or 400 μg/kg)
PlaceboPLACEBO_COMPARATOR -
Normal renal functionACTIVE_COMPARATORParticipants with normal renal function (creatinine clearance (CLcr) \> 90 ml/min) who serve as the study control. Participants treated with one dose of plerixafor (240 µg/kg) administered by subcutaneous (SC) injection.
Mild renal impairmentEXPERIMENTALParticipants have mild renal impairment (creatinine clearance (CLcr) = 51 to 80 mL/min). Participants treated with one dose of plerixafor (240 µg/kg) administered by subcutaneous (SC) injection.
Moderate renal impairmentEXPERIMENTALParticipants have moderate renal impairment (creatinine clearance (CLcr) = 31 to 50 mL/min). Participants treated with one dose of plerixafor (240 µg/kg) administered by subcutaneous (SC) injection.
Severe renal impairmentEXPERIMENTALParticipants have severe renal impairment (creatinine clearance (CLcr) \< 31 mL/min, not requiring dialysis). Participants treated with one dose of plerixafor (240 µg/kg) administered by subcutaneous (SC) injection.

Interventions

NameTypeDescription
PlerixaforDRUG -
Granulocyte-colony stimulating factor (G-CSF)DRUG10 mcg/kg, solution, subcutaneous injection
Plerixafor (AMD3100)DRUG0.24mg/kg SC for 2 to 7 days.
Can be any registered nonpegylated form of G-CSFDRUG10mcg/kg SC for 4 days followed by an additional 2 to 7 days.
PlaceboDRUGSingle subcutaneous (SC) dose of placebo
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: Donor: * Donor eligibility will be determined according to applicable federal, state and local regulations and institutional standards * 18-65 years of age * 6/6 HLA-matched sibling * Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor * Serum...

Countries:United StatesBelgiumEstoniaLithuaniaSwedenGermanyUnited KingdomCzechiaDenmarkFranceHungaryIsraelItalyNetherlandsPolandSpain
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Competitive Landscape -Lymphoma 338 trials

Top 20 of 66 competitors

CompanyTickerTrialsLead PhaseDrugs
Regeneron Pharmaceuticals, Inc.REGN7PHASE3Odronextamab, Rituximab, Cyclophosphamide, Doxorubicin, Vincristine
Eli Lilly and CompanyLLY12PHASE3Pirtobrutinib, Idelalisib, Bendamustine, Rituximab
AstraZeneca PLCAZN14PHASE3Surovatamig, R-CHOP, R-CVP, BR
Merck & Co., Inc.MRK16PHASE3Nemtabrutinib, Fludarabine, Cyclophosphamide, Bendamustine, Rituximab
AbbVie, Inc.ABBV11PHASE3Venetoclax
Novartis AG Sponsored ADRNVS8PHASE3Tisagenlecleucel, Lenalidomide and rituximab in 28-day cycles for up to 12 cycles, Rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone or prednisolone in 21-day cycles for 6 to 8 cycles, Lymphodepleting chemotherapy
Incyte CorporationINCY16PHASE3Tafasitamab, Lenalidomide
BeOne Medicines Ltd. Sponsored ADRONC20PHASE3BGB-16673, Bendamustine, Rituximab, Methylprednisolone, Chlorambucil
Gilead Sciences, Inc.GILD7PHASE3Axicabtagene Ciloleucel, Cyclophosphamide, Fludarabine, Lenalidomide, Rituximab
Genmab A/S Sponsored ADRGMAB18PHASE3Epcoritamab, Rituximab, Lenalidomide
Bristol-Myers Squibb CompanyBMY17PHASE3Golcadomide, Rituximab, Cyclophosphamide, Doxorubicin, Vincristine
Johnson & JohnsonJNJ11PHASE3Ibrutinib
Pfizer Inc.PFE4PHASE3Brentuximab vedotin, Rituximab, Lenalidomide
Nurix Therapeutics, Inc.NRIX6PHASE3NX-5948, Pirtobrutinib
ADC Therapeutics LtdADCT4PHASE3Loncastuximab Tesirine, Rituximab, Gemcitabine, Oxaliplatin
Corvus Pharmaceuticals, Inc.CRVS2PHASE3Soquelitinib, Belinostat, Pralatrexate
Grifols, S.A. Sponsored ADR Class BGRFS1PHASE3Xembify
HUTCHMED (China) Limited Sponsored ADRHCM2PHASE3HMPL-760, R-GemOx
Nuvalent, Inc. Class ANUVL1PHASE3Neladalkib, Alectinib
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK6PHASE2Brentuximab Vedotin, Cyclophosphamide, Doxorubicin, Prednisone
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Frequently asked questions about Plerixafor

What is Plerixafor used for?

Plerixafor is an investigational small molecule being studied for several oncology and transplant-related conditions, including metastatic pancreatic adenocarcinoma, Ewing's sarcoma/soft tissue sarcoma, acute myeloid leukemia, and failure of bone marrow graft. It is also studied in related donors donating peripheral blood stem cells to a family member and in healthy volunteers.

Who makes Plerixafor?

Plerixafor is being developed by Sanofi, a company traded under the ticker SNY. The drug is currently in Phase 1 clinical development for its studied indications.

What phase is Plerixafor in?

Plerixafor is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials have been completed, but the drug remains in early-stage development.

What clinical trials is Plerixafor in?

Plerixafor has been studied in several completed clinical trials, including NCT00445302, NCT01288573, NCT01696461, and NCT02179970. These trials investigated the drug in renal impairment, pediatric cancer patients, stem cell mobilization in related donors, and advanced pancreatic, ovarian, and colorectal cancers.

What does Plerixafor target?

Plerixafor is a small molecule that targets the CXCR4 receptor, a chemokine receptor involved in cell migration and retention of stem cells in the bone marrow. By blocking this receptor, it mobilizes stem cells into the bloodstream, which is relevant for stem cell collection and potential anti-cancer effects.

Is Plerixafor the same as AMD3100?

Plerixafor is also known as AMD3100, as indicated in the clinical trial title for NCT00445302. This alternative name is used in research contexts, and both names refer to the same investigational drug.