NCT00109538

A Pivotal Randomized Study of Lonafarnib Versus Placebo in the Treatment of Subjects With Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) Who Are Platelet Transfusion Dependent With or Without Anemia

Terminated Phase 3 Last updated 9 April 2015

What this trial tests

Phase 3 trial testing Lonafarnib in Myelodysplastic Syndromes in 47 participants. Terminated before completion.

Timeline

1 May 2005

Primary endpoint
1 August 2008

1 August 2008

Quick facts

Lead sponsor	Merck Sharp & Dohme LLC
Phase	Phase 3
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	47
Start date	1 May 2005
Primary completion	1 August 2008
Estimated completion	1 August 2008

Drugs / interventions tested

• Lonafarnib (LONAFARNIB) — full drug profile →
• Placebo

Conditions studied

• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →
• Leukemia, Myelomonocytic, Chronic — all drugs for Leukemia, Myelomonocytic, Chronic →
• Myelodysplasia — all drugs for Myelodysplasia →
• Myelomonocytic — all drugs for Myelomonocytic →

Sponsor

Merck Sharp & Dohme LLC — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndromes or Leukemia, Myelomonocytic, Chronic. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Proportion of subjects who achieved platelet transfusion independence for any 8-consecutive week period after randomization without worsening of RBC transfusion requirements or hemoglobin (untransfused) during the same 8-consecutive-week period.
  Time frame: Any 8-consecutive week period after randomization

Sponsor's own description

The purpose of this study is to assess the benefit of lonafarnib (versus placebo) in patients with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). Benefit will be measured by achievement of platelet transfusion independence for at least 8-consecutive weeks, and without simultaneous worsening of hemoglobin and/or need for red blood cell (RBC) transfusion. Additional endpoints will be hematologic response (which includes complete remission, partial remission, hematologic improvement), number of RBC transfusions, bleeding events, infections and safety.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Tumor biomarkers for diagnosis, prognosis and targeted therapy.
   Zhou Y, Tao L, Qiu J, Xu J, et al · · 2024 · cited 379× · PMID 38763973 · DOI 10.1038/s41392-024-01823-2
2. Protein lipidation in health and disease: molecular basis, physiological function and pathological implication.
   Yuan Y, Li P, Li J, Zhao Q, et al · · 2024 · cited 56× · PMID 38485938 · DOI 10.1038/s41392-024-01759-7
3. A metabolic modeling approach reveals promising therapeutic targets and antiviral drugs to combat COVID-19.
   Santos-Beneit F, Raškevičius V, Skeberdis VA, Bordel S. · · 2021 · cited 33× · PMID 34099831 · DOI 10.1038/s41598-021-91526-3
4. Protein lipidation in the tumor microenvironment: enzymology, signaling pathways, and therapeutics.
   Xu M, Xu B. · · 2025 · cited 3× · PMID 40335986 · DOI 10.1186/s12943-025-02309-7