CLINICAL TRIALS PROFILE FOR CLADRIBINE

All Clinical Trials for CLADRIBINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002805 ↗	Combination Chemotherapy in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1997-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome in first relapse or who did not achieve first remission.
NCT00002805 ↗	Combination Chemotherapy in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome	Completed	Children's Oncology Group	Phase 2	1997-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome in first relapse or who did not achieve first remission.
NCT00002833 ↗	Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1994-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. Colony stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy. PURPOSE: Phase II trial to study the effectiveness of peripheral stem cell transplantation plus filgrastim in treating patients who have acute or chronic myelogenous leukemia.
NCT00002833 ↗	Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia	Completed	M.D. Anderson Cancer Center	Phase 2	1994-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. Colony stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy. PURPOSE: Phase II trial to study the effectiveness of peripheral stem cell transplantation plus filgrastim in treating patients who have acute or chronic myelogenous leukemia.

Clinical Trial Conditions for CLADRIBINE

Condition Name

Condition Name for CLADRIBINE
Intervention	Trials
Acute Myeloid Leukemia	29
Leukemia	19
Multiple Sclerosis	13
Hairy Cell Leukemia	9

Condition MeSH

Condition MeSH for CLADRIBINE
Intervention	Trials
Leukemia	78
Leukemia, Myeloid, Acute	59
Leukemia, Myeloid	52
Lymphoma	21

Clinical Trial Locations for CLADRIBINE

Trials by Country

Trials by Country for CLADRIBINE
Location	Trials
United States	255
China	47
Canada	18
Germany	17
Poland	15

Trials by US State

Trials by US State for CLADRIBINE
Location	Trials
Texas	25
Washington	16
California	11
New York	11
Maryland	10

Clinical Trial Progress for CLADRIBINE

Clinical Trial Phase

Clinical Trial Phase for CLADRIBINE
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	1
PHASE2	8

Clinical Trial Status

Clinical Trial Status for CLADRIBINE
Clinical Trial Phase	Trials
Recruiting	59
Completed	43
Unknown status	13

Clinical Trial Sponsors for CLADRIBINE

Sponsor Name

Sponsor Name for CLADRIBINE
Sponsor	Trials
National Cancer Institute (NCI)	34
M.D. Anderson Cancer Center	20
EMD Serono	8

Sponsor Type

Sponsor Type for CLADRIBINE
Sponsor	Trials
Other	241
Industry	54
NIH	35

Cladribine: Clinical Trials Update, Market Analysis, and Projections

Last updated: February 5, 2026

What is the current status of Cladribine clinical trials?

Cladribine is approved for multiple sclerosis (MS) under the brand names Mavenclad and Mavenclad tablets, with regulatory approval in the United States, Europe, and other regions. Ongoing and planned clinical research extend into other indications, including hematologic cancers like hairy cell leukemia and potential off-label uses.

Completed and Ongoing Trials

• Multiple Sclerosis (MS):
  The Cladribine Tablet (Mavenclad) was approved by FDA in 2019 for relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS). The approval was based on Phase III trials (Clarity and Clarity Extension), which demonstrated efficacy in reducing relapse rates and disability progression.

• Hematologic Cancers:
  The drug has demonstrated activity in clinical trials for hairy cell leukemia (HCL).

  • In a Phase II trial, Cladribine showed response rates exceeding 85%.
  • Preparatory studies explore its use in B-cell chronic lymphocytic leukemia (CLL) and lymphomas, with mixed results awaiting further phase III validation.

• Extended and Future Trials:
  Researchers are investigating Cladribine in combination with other therapies for multiple indications. For example, a Phase II trial (NCT04222798) assesses its efficacy alongside rituximab for B-cell malignancies.

Regulatory and Investigational Status

• FDA Approvals:
  Approved for MS since 2019; no current application for other indications approved.
• EMA Approvals:
  Approved for MS in Europe since 2017.
• ClinicalTrials.gov Registrations:
  Around 20 trials investigating Cladribine for MS, leukemia, and other B-cell neoplasms. These include Phase I to III studies, with some in recruitment or extended follow-up stages.

How does Cladribine's market environment look?

Market size for Cladribine in MS

• Global MS drug market:
  Valued at approximately USD 22 billion in 2022 with an expected CAGR of 6.5% through 2030.
• Cladribine's market share:
  Dominated in MS by disease-modifying therapies (DMTs) including Ocrevus, Tecfidera, and Mavenclad. Mavenclad holds roughly 10-15% of the DMT market for relapsing MS in regions where it is approved.

Competitive landscape and market dynamics

• Key competitors:
  o Ocrevus (rituximab) - immunotherapy, extensive data, high market share
  o Tecfidera (dimethyl fumarate) - oral therapy with broad use
  o Aubagio (teriflunomide) - oral DMT, moderate market penetration

• Pricing and reimbursement:
  Mavenclad has a high price point (~USD 70,000 per year in the U.S.), with reimbursement in most major markets. Market penetration depends on local approval timing and formulary inclusion.

Market outlook for non-MS indications

• The hematologic oncology market for drugs treating hairy cell leukemia and other B-cell cancers is valued at about USD 1.8 billion globally.
• Cladribine-based treatments could capture a portion of this, contingent on further clinical validation and regulatory approval.

What are the future projections for Cladribine?

Market growth estimates

• MS segment:
  Projected to grow at a CAGR of 6-7% through 2030 due to increased diagnosis rates, expanding indications, and evolving treatment guidelines favoring oral, less frequent dosing strategies.
  Mavenclad is expected to maintain or slightly increase its market share within this segment.

• Hematologic oncology:
  Growth driven by ongoing trials and emerging data. If regulatory approvals are obtained for additional indications, sales could reach USD 500 million to USD 1 billion annually by 2030.

Regulatory and development prospects

• Additional indications:
  Trials for CLL, lymphomas, and other B-cell disorders could lead to expanded labeling over the next 3-5 years.
• Line of therapy shifts:
  Mavenclad's utility in early MS could increase with accumulating long-term data suggesting durable disease control with fewer doses.

Risks and challenges

• Clinical efficacy in new indications:
  Low response rates or safety concerns could delay or restrict approval.
• Competitive pressures:
  New oral agents and biologics with superior safety profiles or broader indications can erode market share.
• Regulatory hurdles:
  Differences in approval processes, especially for off-label uses, could impact timelines.

Key Takeaways

• Cladribine's most substantial current market is MS, where it has secured approval since 2017 (EMA) and 2019 (FDA). It commands a significant market share among oral DMTs.
• Clinical trials continue in oncology, with some promising data for hairy cell leukemia. Further validation and regulatory steps are required for expansion beyond MS.
• Market growth depends on approval of new indications, expanding its scope in B-cell neoplasms and potentially other autoimmune or lymphoid conditions.
• The drug faces competition from other oral DMTs and biologics in MS, with pricing and reimbursement policies influencing adoption.
• By 2030, Cladribine's revenue potential hinges on clinical development success, regulatory approvals, and competitive dynamics in both neuroimmunology and oncology segments.

5 FAQs

1. Is Cladribine approved for conditions other than MS?
   Yes. It is approved for hairy cell leukemia in some regions but remains investigational for many other B-cell cancers.

2. What are the safety concerns associated with Cladribine?
   Potential lymphopenia and increased infection risk are primary safety concerns, especially with repeated courses.

3. How does Cladribine compare cost-wise with other MS therapies?
   It is priced around USD 70,000 annually in the U.S., similar to other high-efficacy DMTs, but with less frequent dosing.

4. Are there ongoing trials for Cladribine in other autoimmune diseases?
   Few trials are exploring non-MS autoimmune indications, primarily focusing on lymphoid malignancies.

5. What is the timeframe for potential approval in new indications?
   Pending trial outcomes, regulatory review, and submission timelines, new approvals could occur within 2-5 years.

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Sources

[1] GlobalData. MS Market Report, 2022.
[2] ClinicalTrials.gov. Cladribine Trials Database.
[3] U.S. Food and Drug Administration. Mavenclad Approval Notice, 2019.
[4] European Medicines Agency. Mavenclad Product Information.