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CompletedNCT01613976Updated Dec 19, 2020

A Phase Ib Study of Panobinostat (LBH589) in Combination With 5-Azacitidine for Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML) Patients

A Phase 1 interventional study of Panobinostat in Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) and Acute Myeloid Leukemia (AML), sponsored by Novartis Pharmaceuticals. Completed at 6 sites in Japan. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2020-12-19.

Sponsored by Novartis Pharmaceuticals · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
20 Years and older
Sex
All
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Study summary

The purpose of this study is to confirm the safety and tolerability of oral panobinostat (PAN) in combination with a fixed dose of 5-Azacitidine (5-Aza) in adult Japanese patients with Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML).

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Conditions studied

  • Myelodysplastic Syndromes (MDS)
  • Chronic Myelomonocytic Leukemia (CMML)
  • Acute Myeloid Leukemia (AML)

Keywords

  • Myelodysplastic Syndromes
  • MDS
  • Chronic Myelomonocytic Leukemia
  • CMML
  • Acute Myeloid Leukemia
  • AML
  • Panobinostat
  • LBH589
  • 5-Aza
  • Azacitidine
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In context

Leukemia

5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.

This study's enrollment of 10 is below the median of 38 across 4,247 interventional studies indexed under Leukemia.

Browse Leukemia studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.

Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
20 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Japanese patients who are candidates for treatment with 5-Aza and present with one of the following:

    • intermediate-2 or high-risk MDS according to the International Prognostic Scoring System (IPSS). OR
    • AML with multilineage dysplasia and maximum of 30% blasts (former RAEB-T according to FAB) OR CMML
  2. Patient has an ECOG performance status of ≤ 2
  3. Patients must have the following laboratory values unless elevations are considered due to MDS or leukemia: AST/SGOT and/or ALT/SGPT ≤ 2.5 x ULN; serum creatinine ≤ 1.5 x ULN; serum bilirubin (total and direct) ≤ 2 x ULN; electrolyte panel without clinically relevant abnormalities

Exclusion criteria

Exclusion Criteria:

  1. Patient who is planned for or has history of hematopoietic stem-cell transplantation (HSCT)
  2. Patients with relapsed/refractory AML
  3. Patient is receiving concurrent anti-cancer therapy
  4. Patient has received prior treatment with deacetylase inhibitors (DACi)
  5. Patient has received prior treatment with 5-Aza or 6-aza-2'-deoxycytidine (decitabine)
  1. Patient has shown suspected hypersensitivity to 5-Aza or Mannitol 8. Patients with impaired cardiac function 9. Patient taking medications with relative risk of prolonging the QT interval or inducing Torsade de pontes if such treatment cannot be discontinued or switched to a different medication prior to starting study treatment 10. Patients with clinical evidence of relevant mucosal or internal bleeding 11. Patient has any other concurrent severe and/or uncontrolled medical conditions

Other protocol-defined inclusion/exclusion criteria may apply

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Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (actual)

Study arms

  • Experimental
    Panobinostat and Azacitidine

    combination regimen

    Drug: Panobinostat

Interventions

  • DrugPanobinostat

    Also known as: LBH589

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What researchers measure

Primary outcomes

  1. Incidence of Dose Limiting Toxicitiy(DLT)

    DLT will be assessed during PK run-in period (up to 7 days) and 1st cycle (28 days)

    Time frame: first 5 weeks of treatment period

Secondary outcomes

  1. PK parameter - Cmax

    Time frame: Day 1 to 3 of PK run-in period; pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 8, 24 and 48 hours

  2. PK parameter - Tmax

    Time frame: Day 1 to 3 of PK run-in period; pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 8, 24 and 48 hours

  3. PK parameter - AUC (AUC0-48, AUC0-tlast)

    Time frame: Day 1 to 3 of PK run-in period; pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 8, 24 and 48 hours

  4. PK parameter - T1/2 (apparent oral clearance, volume distribution)

    Time frame: Day 1 to 3 of PK run-in period; pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 8, 24 and 48 hours

  5. PK parameter - AUC0-inf

    Time frame: Day 1 to 3 of PK run-in period; pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 8, 24 and 48 hours

  6. Trough level of PAN in combination with 5-Aza

    Time frame: Day 4, 5, 8 of the 1st cycle; pre-dose (0 hour)

  7. Frequency and severity of Adverse Events (AEs)

    Safety will be measured in terms of type, frequency and severity of adverse events according to CTCAE v4.03.

    Time frame: Participants will be followed for the duration of treatment, an expected average of 6 months

  8. Laboratory abnormalities

    Time frame: duration of treatment, an expected average of 6 months

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Study locations

6 sites
  • Novartis Investigative Site
    Nagoya-city, Aichi 466-8650, Japan
  • Novartis Investigative Site
    Nagoya, Aichi 460-0001, Japan
  • Novartis Investigative Site
    Kobe-city, Hyogo 650-0017, Japan
  • Novartis Investigative Site
    Sendai-city, Miyagi 980-8574, Japan
  • Novartis Investigative Site
    Chuo-ku, Tokyo 104-0045, Japan
  • Novartis Investigative Site
    Kyoto, 602-8566, Japan
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References and documents

Publications

  • Kobayashi Y, Munakata W, Ogura M, Uchida T, Taniwaki M, Kobayashi T, Shimada F, Yonemura M, Matsuoka F, Tajima T, Yakushijin K, Minami H. Phase I study of panobinostat and 5-azacitidine in Japanese patients with myelodysplastic syndrome or chronic myelomonocytic leukemia. Int J Hematol. 2018 Jan;107(1):83-91. doi: 10.1007/s12185-017-2327-9. Epub 2017 Sep 13. PubMed 28905323 ↗
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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 19, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT01613976
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Jun 7, 2012
Start date
Aug 2012
Primary completion
May 2014
Completion
May 2014
Last update
Dec 19, 2020

Study contacts

Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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