Refractory or Relapsed acute leukaemia (AL) MedDRA version: 16.1 Level: LLT Classification code 10000835 Term: Acute leukemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 16.1 Level: LLT Classification code 10024330 Term: Leukemia acute System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 16.1 Level: LLT Classification code 10024329 Term: Leukemia System Organ Cl
Conditions
Interventions
Product Name: ORY-1001
Product Code: ORY-1001
Pharmaceutical Form: Concentrate for oral solution
INN or Proposed INN: ORY-1001
Current Sponsor code: ORY-1001
Other descriptive name: OG-RR
Concentratio
Sponsors
Oryzon Genomics S.A.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients aged 16 and above. 2. Patients must have relapsed or refractory AL (exluding promyelocytic leukaemia) considered by the investigator ineligible for intensive chemotherapy regimen at that time 3. Patients must have ECOG Performance Status (PS) of 0-2 4. Women of child-bearing potential must have negative serum or urine pregnancy test within two weeks prior treatment start 5. Fertile male or female patients must use highly efficient contraception for the duration of the study and 6 months after the last ORY-1001 dose. 6. Male patients must use condoms to avoid drug exposure of their partner. 7. Patients must be capable of understanding and complying with protocol requirements, and they must be able and willing to sign a written informed consent 8. Life expectancy of at least 2 months Are the trial subjects under 18? yes Number of subjects for this age range: 2 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1. Cancer history that according to the investigator might confound the assessment of the study endpoints 2. Patients with uncontrolled hypertension or diabetes, hepatitis or HIV. 3. Inter-current illness or social situation that will limit compliance with study requirements 4. Pregnancy or lactating / breast feeding 5. Any medical condition which in the opinion of the investigator places the patient at an unacceptably high risk for toxicities if entered into the clinical study 6. Acute myeloid leukaemia treatment within the previous 14 days. Hydroxyurea or 6-mercaptopurine are allowed until 12 hours prior study treatment start and after the first treatment block (day 1-5) in case of hyperleucocytosis. 7. Patients medicated with anti-depressants reported to have KDM1A/LSD1 inhibitory activity: Tranylcypromine or Phenelzine. 8. Radiotherapy less than 2 weeks prior to the start of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the safety (hematological and non-hematological toxicities) and tolerability of ORY-1001;Secondary Objective: - Characterize the PK of orally administered ORY-1001 in patients with relapsed / refractory AL - Assess responses (CR/CRi/PR) with ORY-1001 in patients with relapsed or refractory AL, particularly in those with rMLL gene - Evaluate surrogate PD markers for target engagement;Primary end point(s): The PK of ORY-1001 in patients with relapsed /refractory AL The PD of ORY-1001 in patients with relapsed / refractory AL Assess maximum tolerated dose of ORY-1001 in humans as a monotherapy Adverse events assessed by patient reporting Clinical observations and vital signs ECG Laboratory data;Timepoint(s) of evaluation of this end point: Patients are to attend study centre visits during baseline (within 14 days before the first dose) and at defined days after start of treatment. Assessments will be made at baseline, day 1, 2, 3, 4, 5, 6, 7 of the first treatment block and on the first, third and fifth day of administration for the 3 other treatment blocks in the 28 day cycle. Additional visits may be made for safety reasons. Hematology samples (5ml) will be obtained on the first, third and fifth day of each treatment block. Samples for the analysis of blood chemistry (5ml) will be obtained weekly on the first day of each treatment block. A follow up visit will be performed at a minimum of 30 days after the last ORY-1001 dose. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Remission rate (CR + CRi) Gene expression changes in blood cells;Timepoint(s) of evaluation of this end point: DAY1: 0h (pre-dosing)2h, 4h, 6h, 8h, 12h, 18h DAY2: 24h, 28h, DAY3: 48h, 52h, DAY4: 72h, 76h, DAY5: 96h,100h, During the next 3 treatment blocks in the first cycle, samples will be taken only on the first day and the fifth day, just prior to administration | — |
Countries
Spain, United Kingdom
Contacts
Public ContactTamara Maes
Oryzon Genomics S.A.
Outcome results
None listed