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WithdrawnNCT05246384Updated Oct 31, 2022

Study to Evaluate the Safety, Pharmacokinetics and Clinical Activity of RP7214 in Combination With Azacitidine in Patients With Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Acute Myeloid Leukemia

A Phase 1/2 interventional study of RP7214 in Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Acute Myeloid Leukemia, sponsored by Rhizen Pharmaceuticals SA. Withdrawn. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-10-31.

Sponsored by Rhizen Pharmaceuticals SA · Phase 1/2, Interventional, and Treatment

Why this study was withdrawn
Change in development plan
Phase
Phase 1/2
Study type
Interventional
Enrollment
0
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

This is a multi-center, open-label, non-randomized, two-part Phase I/Ib study of RP7214 in combination with azacitidine in patients with AML, MDS and CMML. Part I is a 3+3 dose-escalation study to identify the MTD/RP2D of RP7214 and azacitidine combination in patients with AML, MDS, and CMML. Part II is a dose-expansion study to evaluate the clinical activity and safety of RP7214 and azacitidine combination in AML.

02

Conditions studied

  • Myelodysplastic Syndromes
  • Chronic Myelomonocytic Leukemia
  • Acute Myeloid Leukemia

Keywords

  • RP7214
  • Dihydro-orotate Dehydrogenase (DHODH)
03

In context

Myelodysplastic Syndromes

2,165 studies on the registry are indexed under Myelodysplastic Syndromes; 322 are open to participants now.

Browse Myelodysplastic Syndromes studies →

Lead sponsor

Rhizen Pharmaceuticals SA is the lead sponsor of 17 studies on the registry; 1 is open to participants now.

Of its 7 completed or terminated interventional studies of FDA-regulated products, 4 (57%) have results posted.

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

04

Who can participate

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

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Patient must sign informed consent.
  2. Patient should be ≥ 18 years of age.

  3. Patients who are candidates for treatment with azacitidine and present with one of the following:

    a. Part I: Dose Escalation study i. Patient with histologically or cytologically confirmed relapsed/refractory AML as per World Health Organization (WHO) classification, 2016 'OR' ii. Newly diagnosed AML patients who are ineligible for intensive induction chemotherapy due to co-morbidity or other factors 'OR' iii. Intermediate-2 or high-risk MDS according to the International Prognostic Scoring System (IPSS) 'OR' iv. Chronic Myelomonocytic Leukemia (CMML) b. Part II: Dose Expansion study i. Newly diagnosed AML patients who are ineligible for intensive induction chemotherapy due to co-morbidity or other factors.

  4. Patient should have an Eastern Cooperative Oncology Group (ECOG) Performance score of 0 to 2.
  5. Patients must be amenable to serial bone marrow biopsies/aspirates and peripheral blood sampling as required by the protocol.

Exclusion criteria

Exclusion Criteria:

  1. Any cancer-directed therapy taken (e.g., chemotherapy, immunotherapy, biologic therapy or an investigational drug) within 14 days or 5 half-lives, whichever is shorter, prior to C1D1. For radiation therapy, at least 60 days should elapse from prior Total Body Irradiation (TBI) and at least 14 days from local palliative radiation therapy.
  2. Patients with rapidly increasing peripheral blast counts (WBC count > 25,000/μL) while on hydroxyurea prior to C1D1.
  3. Patients with Acute Promyelocytic Leukemia (French American-British Class M3 AML).
  4. Patients on immunosuppressive therapy post autologous or allogeneic stem cell transplantation (ASCT or Allo-SCT) at the time of screening, or with clinically significant Graft-Versus-Host Disease (GVHD) in the opinion of the Investigator or has not recovered from transplant-associated toxicities prior to C1D1.
  5. Patient who discontinued prior therapy with DHODH inhibitors or azacitidine due to drug-related toxicity.
  6. Evidence of uncontrolled/progressing infection.
  7. Patients with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or Disseminated Intravascular Coagulation (DIC).
  8. Presence of isolated extramedullary relapse.
  9. Pregnant or lactating women
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    Part I (dose escalation) RP7214 + Azacitidine

    Participants will receive RP7214 orally in combination with Azacitidine in a 28-day cycle. The dose levels will be escalated until MTD/a recommended Phase 2 dose (RP2D) has been identified.

    Drug: RP7214

  • Experimental
    Part II (dose expansion) RP7214 + Azacitidine

    Participants will receive RP7214 orally at the MTD/RP2D in combination with Azacitidine in a 28-day cycle.

    Drug: RP7214

Interventions

  • DrugRP7214

    RP7214 will be administered daily twice a day orally; Azacitidine will be administered from Days 1 to 7 of each 28-day cycle

06

What researchers measure

Primary outcomes

  1. Maximum Tolerated Dose (MTD)/Recommended Phase 2 Dose (RP2D) of RP7214 in combination with azacitidine

    The maximum tolerated dose will be defined as the highest dose tested in which a DLT is experienced by 0 out of 3 or 1 out of 6 patients among the dose levels.

    Time frame: 28 days

Secondary outcomes

  1. Tmax

    Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of RP7214

    Time frame: 35 days

  2. Objective Response Rate (ORR)

    Defined as the percentage of patients who achieve Complete Remission (CR), Complete Remission with incomplete bone marrow recovery (CRi) and Partial Remission (PR)

    Time frame: 2 years

  3. Clinical Benefit Rate (CBR)

    Defined as the percentage of patients achieving a CR, CRi, PR and Stable Disease (SD) lasting for at least 8 weeks.

    Time frame: 2 years

  4. Duration of Remission

    Defined as the number of days from the date of first remission (CR, CRi, or PR) to the recurrence or Progressive Disease (PD)

    Time frame: 2 years

  5. Percentage of patients requiring blood and/or platelet transfusions

    Defined as number of patients requiring blood and/or platelet transfusions

    Time frame: 2 years

  6. Cmax

    Pharmacokinetics: Maximum Concentration (Cmax) of RP7214

    Time frame: 35 days

  7. AUC

    Pharmacokinetics: Area Under the Concentration Curve (AUC) of RP7214

    Time frame: 35 days

07

Study locations

No study locations are listed for this record.

08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Start is estimated for Jan 2023; primary completion for Dec 2024, 23 months later.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 29, 2022
First posted
Feb 18, 2022
StartEst.
Jan 2023
Primary completionEst.
Dec 2024
CompletionEst.
Nov 2025
Last update
Oct 31, 2022
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Oct 31, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05246384
Lead sponsor
Rhizen Pharmaceuticals SA
Responsible party
Sponsor
First posted
Feb 18, 2022
Start date
Jan 2023 (estimated)
Primary completion
Dec 2024 (estimated)
Completion
Nov 2025 (estimated)
Last update
Oct 31, 2022

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is withdrawn, as verified in Oct 2022. You cannot join it, but the record below documents what was studied.

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