A Phase I/IIa Study of ODX (OsteoDex) in Multiple Myeloma

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dextech Medical AB

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

16 Sep 2024 → 2 Mar 2026

Decision date (initial)

2024-09-16

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-516969-36-00

EudraCT number

2022-001635-91

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

• To determine the safety and tolerability of ODX in subjects with relapsed/refractory multiple myeloma.

Secondary objectives 1

1. • To evaluate the preliminary efficacy of ODX, as determined by the IMWG response criteria, in subjects with relapsed/refractory multiple myeloma. • To evaluate the efficacy of ODX on serum biomarkers (M-protein, FLC, CTX, osteocalcin, and bone-specific S-ALP) in subjects with relapsed/refractory multiple myeloma.

Conditions and MedDRA coding

Multiple Myeloma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. Subject (male or female) is ≥ 18 years of age at the time of signing the informed consent form (ICF). 2. Documented diagnosis av multiple myeloma according to the International Myeloma Working Group (IMWG) diagnostic criteria. 3. Measurable disease defined as either: • Serum monoclonal paraprotein (M-protein) level ≥ 0.5 g/dL or urine M-protein level ≥ 200 mg/24 hours; or • Light chain multiple myeloma without measurable disease in the serum or the urine: Serum immunoglobulin free light chain (FLC) ≥ 10 mg/dL and abnormal serum immunoglobulin kappa lambda FLC ratio. 4. Subjects must have received 1-5 prior lines of any therapy 5. Subjects must have documented evidence of progressive disease based on the IMWG criteria on or after their last line of therapy. 6. Performance status ECOG 0-2 7. Laboratory requirements: Haematology: Neutrophils ≥ 1.0 x 109/l Hemoglobin ≥ 80 g/l Platelets ≥ 50 x 109/l Hepatic function: Total S/P-bilirubin ≤ 1.5 times the upper limit of normal (ULN) AST (SGOT) / ALT (SGPT) ≤ 2.5 times ULN Renal function: S/P-creatinine ≤ 1.5 times ULN Electrolytes: S/P-sodium, S/P-potassium, S/P-calcium corrected for S/P albumin , S/P-phosphate, and S/P magnesium, all within normal ranges. At the discretion of the Investigator, supplements may be given to correct these values, in which case electrolytes must be shown to be within normal ranges before inclusion into the study. 8. No evidence (< 5 years) of prior malignancies (except successfully treated basal cell or squamous cell carcinoma of the skin). 9. Able to adhere to the study visit schedule and other protocol requirements.

Exclusion criteria 1

1. 1. Concurrent use of other anti-cancer agents/treatments. 2. Any treatment modalities involving chemotherapy, radiation, or major surgery within 4 weeks prior to treatment in this study. 3. Simultaneous participation in any other study involving investigational drugs or having participated in an investigational study less than 4 weeks prior to start of study treatment. 4. Any condition, including the presence of laboratory abnormalities, which confounds the ability to interpret data from the study or places the patient at unacceptable risk if he or she participates in the study. 5. Known active CNS involvement or exhibits clinical signs of meningeal involvement of multiple myeloma. 6. Plasma cell leukemia, Waldenstrom’s macroglobulinemia or POEMS syndrome. 7. Dental surgery (dental extraction), periodontal disease, local trauma including poorly fitting dentures within 6 months prior to the first dose of study drug. 8. Treatment with bisphosphonates or denosumab within 6 weeks prior to first dose of study medication. 9. Male subjects not willing to use condom to prevent pregnancy and drug exposure of a fertile female partner and refrain from donating sperm from the date of the first dose until the end of study treatment. 10. Pregnant or breastfeeding females. 11. Female subjects of childbearing potential* not willing to use a contraceptive method with a failure rate of < 1% to prevent pregnancy during study treatment. Highly effective birth control methods include: • combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: o oral o intravaginal o transdermal • progestogen-only hormonal contraception associated with inhibition of ovulation: o oral o injectable o implantable • intrauterine device • intrauterine hormone-releasing system (for example, progestin-releasing coil) • vasectomized male (with appropriate post vasectomy documentation of the absence of sperm in the ejaculate) • bilateral tubal occlusion or hysterectomy. *Female subjects are considered of non-childbearing potential if they are pre-menopausal females with a documented tubal ligation or hysterectomy or bilateral oophorectomy; or as post-menopausal females defined as at least 12 months of amenorrhea. 12. Subjects in which pre-medication with dexamethasone, antihistamine, and paracetamol would be contraindicated.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint parameters are occurrence of AEs/SAEs, vital signs, physical examination, ECG, and the results of urinalysis and safety laboratory tests (haematology, electrolytes, liver function, and biochemistry including S/P-Creatinine and S/P-Cystatin C). Note that the primary endpoints will be used to confirm the safety and tolerability of ODX in the patient population and no statistical testing will be performed for these endpoints.

Secondary endpoints 1

1. • Best overall response rate (partial response [PR] or better) reached during the 14-week treatment/follow-up period, as measured by the IMWG response criteria.• Change in the levels of the serum biomarkers M-protein, FLC, CTX, osteocalcin, and bone-specific S ALP from baseline to Weeks 2, 4, 6, 8, 10, 12, and 14.• Quality of Life (QoL) scores, as measured by the EQ-5D-5L assessment tool, from baseline to Weeks 2, 8, and 14

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dextech Medical AB

Sponsor organisation

Dextech Medical AB

Address

P. O. Box 389

City

Uppsala

Postcode

751 06

Country

Sweden

Scientific contact point

Organisation

Dextech Medical AB

Contact name

Anders Holmberg

Public contact point

Organisation

Dextech Medical AB

Contact name

Anders Holmberg

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications