CLINICAL TRIALS PROFILE FOR PLERIXAFOR

505(b)(2) Clinical Trials for PLERIXAFOR

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Indication	NCT00901225 ↗	Study of Plerixafor for Rescue of Poor Mobilizers in Autologous Stem Cell Transplant	Completed	Genzyme, a Sanofi Company	Phase 2	2009-05-01	Plerixafor, administered at a dose of 240 ug/kg, potentiates the effect of granulocyte colony-stimulating factor (G-CSF) to increase peripheral blood progenitor cells in both healthy volunteers and cancer patients. Furthermore, in cancer patients, cells collected via apheresis using Plerixafor and G-CSF have been successfully transplanted. In December 2008, Plerixafor received approval from the Food and Drug administration for use in combination with G-CSF to aid in mobilization of progenitor cells for apheresis. The proposed study is not designed to support approval of a new indication or change in the advertising for Plerixafor. The route of administration and dosage level are identical to that which is listed on the package insert. Although Plerixafor is not approved for patients with Hodgkins Lymphoma, there is no known or theoretic increased risk of the use of this drug in this patient population. The study hypothesis for this study is that patients with a circulating CD34+ count < 20 cells/ul after 5 days of mobilization with G-CSF alone will achieve > or equal to 2 X 10(6)CD34+ cells/kg within 3 days of apheresis after receiving Plerixafor with G-CSF.
New Indication	NCT00901225 ↗	Study of Plerixafor for Rescue of Poor Mobilizers in Autologous Stem Cell Transplant	Completed	Duke University	Phase 2	2009-05-01	Plerixafor, administered at a dose of 240 ug/kg, potentiates the effect of granulocyte colony-stimulating factor (G-CSF) to increase peripheral blood progenitor cells in both healthy volunteers and cancer patients. Furthermore, in cancer patients, cells collected via apheresis using Plerixafor and G-CSF have been successfully transplanted. In December 2008, Plerixafor received approval from the Food and Drug administration for use in combination with G-CSF to aid in mobilization of progenitor cells for apheresis. The proposed study is not designed to support approval of a new indication or change in the advertising for Plerixafor. The route of administration and dosage level are identical to that which is listed on the package insert. Although Plerixafor is not approved for patients with Hodgkins Lymphoma, there is no known or theoretic increased risk of the use of this drug in this patient population. The study hypothesis for this study is that patients with a circulating CD34+ count < 20 cells/ul after 5 days of mobilization with G-CSF alone will achieve > or equal to 2 X 10(6)CD34+ cells/kg within 3 days of apheresis after receiving Plerixafor with G-CSF.

All Clinical Trials for PLERIXAFOR

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00075335 ↗	AMD 3100 (Mozobil Plerixafor) to Mobilize Stem Cells for Donation	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2004-01-01	Peripheral blood progenitor cells (PBPC) have become the preferred source of hematopoetic stem cells for allogeneic transplantation because of technical ease of collection and shorter time required for engraftment. Traditionally, granulocyte-colony stimulating factor (G-CSF) has been used to procure the peripheral blood stem cell graft. Although regimens using G-CSF usually succeed in collecting adequate numbers of PBPC from healthy donors, 5%-10% will mobilize stem cells poorly and may require multiple large volume apheresis or bone marrow harvesting. Although G-CSF is generally well tolerated in healthy donors, it may be associated with bone pain, headache, myalgia and rarely life threatening side effects like stroke, myocardial infarction and splenic rupture. AMD3100, is a bicyclam compound that inhibits the binding of stromal cell derived factor-1 (SDF-1) to its cognate receptor CXC- chemokine receptor 4 (CXCR4). CXCR4 is present on cluster of differentiation 34 (CD34)+ hematopoetic progenitor cells and its interaction with stromal cell derived factor 1 (SDF-1) plays a pivotal role in the homing of CD34+ cells in the bone marrow. Inhibition of the CXCR4-SDF1 axis by AMD3100 releases CD34+ cells into the circulation, which can then be collected easily by apheresis. Recently, a published report demonstrated that large numbers of CD34+ cells were rapidly mobilized in healthy volunteers following a single subcutaneous injection of AMD3100. Remarkably, the number of CD34+ cells collected by apheresis following a single injection of AMD3100 was comparable to the number of CD34+ cells collected from historical controls receiving 5 days of G-CSF prior to stem cell mobilization. In this study we will collect PBPCs following a single subcutaneous injection of AMD3100 from healthy donors who have previously had PBPC collected using standard G-CSF mobilization. The AMD3100 mobilized cells, G-CSF mobilized cells, and circulating cells prior to both AMD3100 and G-CSF mobilization will be analyzed in terms of cellular content and function of lymphocytes, natural killer (NK) cells, and antigen presenting cells. AMD3100 mobilized PBPC will be collected for the purpose of research studies and will not be used for therapeutic purposes.
NCT00082329 ↗	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2004-06-18	This 12-day study will test whether the combination of G-CSF (granulocyte-colony stimulating factor) and AMD3100 (Mozobil) is more efficient in mobilizing stem cells for collection than the use of G-CSF alone. Traditionally, the growth factor G-CSF has been given to stem cell donors to mobilize, or push, stem cells out of the bone marrow and into the blood circulation for collection for transplantation. Although a sufficient quantity of cells usually can be collected with G-CSF treatment, some donors do not respond well and may require multiple apheresis procedures (see below) to collect enough cells. Studies indicate that G-CSF used together with a drug called AMD3100 may be more effective in mobilizing stem cells for collection than G-CSF alone. The Food and Drug Administration has approved G-CSF for stem cell mobilization. AMD3100 is a new drug that also mobilizes stem cells in large numbers within a few hours. Normal healthy volunteers between 18 and 60 years of age may be eligible for this study.
NCT00082329 ↗	G-CSF and AMD3100 to Mobilize Stem Cells in Healthy Volunteers	Completed	Richard Childs, M.D.	Phase 2	2004-06-18	This 12-day study will test whether the combination of G-CSF (granulocyte-colony stimulating factor) and AMD3100 (Mozobil) is more efficient in mobilizing stem cells for collection than the use of G-CSF alone. Traditionally, the growth factor G-CSF has been given to stem cell donors to mobilize, or push, stem cells out of the bone marrow and into the blood circulation for collection for transplantation. Although a sufficient quantity of cells usually can be collected with G-CSF treatment, some donors do not respond well and may require multiple apheresis procedures (see below) to collect enough cells. Studies indicate that G-CSF used together with a drug called AMD3100 may be more effective in mobilizing stem cells for collection than G-CSF alone. The Food and Drug Administration has approved G-CSF for stem cell mobilization. AMD3100 is a new drug that also mobilizes stem cells in large numbers within a few hours. Normal healthy volunteers between 18 and 60 years of age may be eligible for this study.
NCT00103610 ↗	Mobilization of Stem Cells With AMD3100 (Plerixafor) in Non-Hodgkin's Lymphoma Patients	Completed	Genzyme, a Sanofi Company	Phase 3	2005-01-01	The purpose of this study is to determine whether the combination of AMD3100 (plerixafor) and granulocyte colony-stimulating factor (G-CSF or generic name filgrastim) is better than G-CSF alone to mobilize and collect the optimal number of stem cells in non-Hodgkin's lymphoma patients for autologous transplantation.

Clinical Trial Conditions for PLERIXAFOR

Condition Name

Condition Name for PLERIXAFOR
Intervention	Trials
Multiple Myeloma	35
Non-Hodgkin's Lymphoma	11
Lymphoma	11
Acute Myeloid Leukemia	10

Condition MeSH

Condition MeSH for PLERIXAFOR
Intervention	Trials
Multiple Myeloma	44
Lymphoma, Non-Hodgkin	36
Neoplasms, Plasma Cell	35
Lymphoma	33

Clinical Trial Locations for PLERIXAFOR

Trials by Country

Trials by Country for PLERIXAFOR
Location	Trials
United States	232
Spain	17
Canada	11
Italy	8
France	7

Trials by US State

Trials by US State for PLERIXAFOR
Location	Trials
California	24
Missouri	21
Maryland	18
New York	16
Texas	15

Clinical Trial Progress for PLERIXAFOR

Clinical Trial Phase

Clinical Trial Phase for PLERIXAFOR
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	2
PHASE1	6

Clinical Trial Status

Clinical Trial Status for PLERIXAFOR
Clinical Trial Phase	Trials
Completed	82
Recruiting	31
Terminated	23

Clinical Trial Sponsors for PLERIXAFOR

Sponsor Name

Sponsor Name for PLERIXAFOR
Sponsor	Trials
Genzyme, a Sanofi Company	45
National Cancer Institute (NCI)	16
Washington University School of Medicine	13

Sponsor Type

Sponsor Type for PLERIXAFOR
Sponsor	Trials
Other	218
Industry	90
NIH	33

Plerixafor Clinical Trials Update, Market Analysis, and Projection: What’s in the Pipeline, Who’s Running Studies, and When Revenue Returns Are Realistic

Last updated: May 23, 2026

Plerixafor is an investigational CXCR4 antagonist associated with hematopoietic stem cell mobilization and oncology-linked indications. Public, consistently citable trial and commercialization data is limited for plerixafor specifically, which prevents a complete, audit-grade build of a “current clinical trials update” plus quantified market forecasts tied to dosing schedules, recruitment status, and endpoints across geographies.

What clinical trials currently evaluate plerixafor, and what are the key endpoints?

Featured snippet: Plerixafor’s clinical development is centered on CXCR4 blockade for stem cell mobilization, with oncology-linked programs historically exploring tumor microenvironment trafficking and related mechanisms.

Which trial phases cover plerixafor stem cell mobilization?

Featured snippet: Stem cell mobilization programs are the most consistently referenced clinical use-case for plerixafor as a CXCR4 antagonist.

H3: Common mobilization trial design elements

• Comparison arms that include standard mobilization (often chemotherapy and/or G-CSF depending on protocol)
• Measurement endpoints that typically include CD34+ cell yield, time to collection, and collection efficiency
• Safety endpoints focused on adverse events related to mobilization and mobilizer-related physiology

Are there active oncology trials of plerixafor?

Featured snippet: Oncology trials for CXCR4 antagonists typically target mechanisms such as immune cell trafficking, tumor cell mobilization, or combination synergy with chemo, radiation, or immunotherapy. For plerixafor, publicly verifiable, up-to-date oncology trial status cannot be compiled into an accuracy-checked update without dependable source visibility.

What outcomes define success for plerixafor trials?

Featured snippet: In mobilization, success is usually collection yield and feasibility within target collection windows, not surrogate imaging endpoints.

H3: Mobilization endpoints used in CXCR4 antagonist development

• Peak CD34+ cells in peripheral blood
• Total CD34+ cells collected per kg
• Number of apheresis sessions required to reach a collection target
• Time to first collection and time to target yield
• Safety and tolerability including cardiopulmonary and hematologic events

Who is developing plerixafor, and what does the competitive landscape look like?

Featured snippet: The competitive landscape for plerixafor is defined by established stem cell mobilization standards and other CXCR4 antagonists where approved, plus biosimilar and generic dynamics in the background for conventional mobilizers.

How does plerixafor compete against G-CSF based mobilization?

Featured snippet: CXCR4 antagonists compete by improving yield and shortening collection time, reducing burden of repeated apheresis, and potentially improving patient logistics.

H3: Decision drivers in transplant mobilization

• Collection success rate and number of procedures
• Patient-specific constraints such as prior lines of therapy
• Hospital operational burden and scheduling
• Side-effect profile tolerability relative to existing standards

Are other CXCR4 antagonists a closer proxy for market uptake?

Featured snippet: If another CXCR4 antagonist has meaningful uptake, the adoption curve and payer coverage patterns can inform plerixafor’s likely ceiling, but that requires specific, citable analog commercialization data.

When does plerixafor lose exclusivity, and what patents protect commercialization?

Featured snippet: A complete “exclusivity timeline” and “Orange Book status” analysis for plerixafor cannot be produced in an audit-grade way from the information available here.

What is the Orange Book status of plerixafor?

Featured snippet: Orange Book status requires an approved NDA with listed patents and FDA listings. Without reliable, specific FDA listing identifiers for plerixafor, the Orange Book analysis cannot be completed.

What patents protect plerixafor (composition, formulation, and methods)?

Featured snippet: Patent estate analysis requires exact patent numbers, assignees, priority dates, and claim scope.

H3: Patent families typically relevant to CXCR4 antagonists

• Composition-of-matter for the active molecule
• Salt/crystal forms if any
• Pharmaceutical compositions and dosing unit claims
• Methods of mobilization and collection regimens
• Combination therapy claims with G-CSF or chemo

What is the market size and revenue projection for plerixafor?

Featured snippet: A quantitative market projection requires an approved indication, dosing economics, assumed penetration, and payer coverage inputs. For plerixafor, the data needed for a precise, defensible revenue model is not available in a citable form here.

How many transplant-eligible patients drive demand?

Featured snippet: Stem cell mobilization demand is driven by the incidence of hematologic malignancies requiring autologous or allogeneic transplant and by transplant utilization rates.

H3: Key demand components for a mobilization product forecast

• Eligible patient pool by geography
• Autologous transplant rate vs. allogeneous transplant
• Mobilization practice patterns (standard vs. adjunct mobilizer use)
• Adoption friction (hospital protocols, physician familiarity, inventory and training)

What adoption curve assumptions are required for a model?

Featured snippet: Adoption depends on yield superiority claims, safety profile, operational benefits, and payer coverage.

H3: Payer and formulary drivers

• Evidence strength relative to standard mobilization
• Cost per successful collection vs. per procedure
• Inclusion in transplant-center formularies
• Reimbursement rules for outpatient apheresis logistics

What generic or biosimilar entry risks exist for plerixafor?

Featured snippet: Generic risk depends on whether plerixafor is approved and whether patents have been cleared. Biosimilar risk depends on whether the product is biologic, which plerixafor is not in typical descriptions as a small molecule, but the approval status still must be verified.

Could Paragraph IV challenges arise?

Featured snippet: Paragraph IV challenges require an ANDA filing against an approved reference listed drug.

What manufacturing or IP barriers would delay entry?

Featured snippet: Even with regulatory clearance, barriers can include validated synthesis routes, formulation know-how, and method-of-use patent constraints.

What trial-to-market risks most affect plerixafor’s commercial outcomes?

Featured snippet: For mobilization agents, the principal risk is demonstrating collection superiority that is clinically meaningful and operationally actionable versus standard practice.

What are the biggest efficacy risks?

H3: Common efficacy risk points for mobilizers

• Insufficient CD34+ yield improvement in key subgroups
• Longer or variable time to target yield
• Higher adverse event rates that limit repeat use or broad adoption

What are the biggest safety risks?

H3: Safety risk points in CXCR4 antagonism

• Known class effects can include hematologic and vascular events, which require clean tolerability in real-world transplant workflows
• Risk signals that trigger protocol amendments can delay readouts or reduce adoption confidence

What does plerixafor’s clinical and commercial outlook look like by geography?

Featured snippet: Geographic uptake tends to follow reimbursement, transplant center adoption, and regulatory clearance. Without confirmed approved status and current trial progress, a reliable region-by-region outlook cannot be compiled here.

Key Takeaways

• Plerixafor is a CXCR4 antagonist historically linked to hematopoietic stem cell mobilization development.
• A complete clinical trials “update” and a quantified revenue projection require audit-grade, current trial status and commercialization inputs that are not available in a citable way here.
• A patent exclusivity, Orange Book, Paragraph IV, and litigation-style analysis cannot be completed without a verifiable approved product listing and named patent entries.

FAQs

1) Is plerixafor FDA-approved, and what indications does it have?
2) What is plerixafor’s mechanism of action as a CXCR4 antagonist?
3) What dosing and collection metrics do plerixafor trials typically report?
4) How do CXCR4 antagonist mobilization outcomes compare with standard G-CSF protocols?
5) What patent claim types most often block generic entry for small-molecule mobilizers?

References

1. (No citable sources were provided in the prompt, and no sourceable identifiers for FDA listings, trial registries, or patent publications are included here.)