A clinical study of ABSK021 in Patients with Tenosynovial Giant Cell Tumor to determine whether the study drug is safe and how well the antitumor activity of study drug

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Abbisko Therapeutics Co. Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

4 Oct 2023 → ongoing

Decision date (initial)

2023-09-04

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Abbisko Therapeutics Co., Ltd.

External identifiers

EU CT number

2023-503245-58-00

WHO UTN

U1111-1286-9453

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Therapy, Efficacy

To compare the Objective Response Rate (ORR) within 25
weeks after treatment with ABSK021 or placebo in TGCT
patients based on RECIST 1.1.

Secondary objectives 6

1. To compare the Objective Response Rate (ORR) within 25 weeks after treatment with ABSK021 or placebo in TGCT patients based on Tumor Volume Score (TVS)
2. To compare the effects of ABSK021 and placebo on the Range of Motion (ROM) in TGCT patients at Week 25
3. To compare the effects of ABSK021 and placebo on Patient Reported Outcome (PRO) in TGCT patients at Week 25.
4. To compare the Duration of Response (DOR) after treatment with ABSK021 or placebo in TGCT patients based on RECIST 1.1 and TVS, respectively.
5. To compare the safety of ABSK021 and placebo in TGCT patients
6. To evaluate the pharmacokinetic (PK) profile of oral ABSK021

Conditions and MedDRA coding

Tenosynovial Giant Cell Tumor

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Patients should understand the study procedures and sign the informed consent form prior to screening
2. Age ≥ 18 years
3. A histologically confirmed TGCT with not amenable to surgery prior to randomization.
4. Measurable disease as defined by RECIST 1.1, and with at least one lesion of ≥ 2 cm prior to randomization.
5. Stable prescription of analgesic regimen for patients with an analgesic need during the 2 weeks prior to randomization.
6. Participants should complete stiffness and pain scales during the screening period, and symptomatic disease because of active TGCT should meet minimum requirements as outlined in study protocol.
7. Adequate organ function and bone marrow function.

Exclusion criteria 7

1. Known allergy or hypersensitivity to any components of the investigational drug product
2. Previous treatment with highly selective inhibitors targeting CSF-1/CSF-1R prior to randomization. However, patients who have received prior treatment with multi-kinase inhibitors that include the CSF-1/CSF-1R pathway are allowed, such as Imatinib and Nilotinib
3. Known additional malignancy that required active treatment and may affect the patient’s participation in the study or affect the outcome of the study as assessed by the Investigator
4. Known metastatic TGCT
5. Major surgery or previous anti-tumor therapy for TGCT within 4 weeks prior to randomization, or unhealed, infected, or dehiscence of previous surgical wounds, or adverse events from prior therapies did not recover to ≤ Grade 1 (CTCAE 5.0).
6. Impaired cardiac function or clinically significant cardiac disease
7. Known active liver or biliary disease, or other diseases that may lead to abnormal liver function test results during the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 25-Week Objective Response Rate (ORR) by Blinded Independent Review Committee (BIRC) based on RECIST 1.1.

Secondary endpoints 8

1. 25-Week Objective Response Rate (ORR) by Blinded Independent Review Committee (BIRC) based on Tumor Volume Score (TVS)
2. Mean change from baseline in Range of Motion (ROM) of the affected joint at Week 25.
3. Mean change from baseline in the Worst Stiffness Numeric Rating Scale (NRS) score at Week 25
4. Mean change from baseline in the Worst Pain Numeric Rating Scale (NRS) score at Week 25
5. Mean change from baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Functioning score at Week 25
6. Mean change from baseline in EuroQol visual analogue scale (VAS) score at Week 25
7. Duration of Response (DOR) by Blinded Independent Review Committee based on RECIST 1.1.
8. Duration of Response (DOR) by Blinded Independent Review Committee based on Tumor Volume Score (TVS).

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Abbisko Therapeutics Co. Ltd.

Sponsor organisation

Abbisko Therapeutics Co. Ltd.

Address

No 3 898 Lane Halei Road, Trade Zone Trade Zone

City

China () Pilot Free

Postcode

201203

Country

China

Scientific contact point

Organisation

Abbisko Therapeutics Co. Ltd.

Contact name

Clinical Development Department

Public contact point

Organisation

Abbisko Therapeutics Co. Ltd.

Contact name

Clinical Development Department

Third parties 14

Locations

5 EU/EEA countries · 9 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 80 files

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

Application history

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