A Phase 3 Study of Rusfertide (PTG-300) in Patients with Polycythemia Vera

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Protagonist Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

21 Sep 2022 → ongoing

Decision date (initial)

2024-04-15

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Protagonist Therapeutics, Inc.

External identifiers

EU CT number

2023-509750-58-00

EudraCT number

2021-004732-29

WHO UTN

U1111-1300-1551

ClinicalTrials.gov

NCT05210790

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety, Pharmacokinetic, Pharmacodynamic

To evaluate the safety and efficacy of rusfertide in subjects with polycythemia vera in maintaining hematocrit control.

Conditions and MedDRA coding

Polycythemia Vera

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 1 - Male and female subjects aged 18 (or the minimum country specific age of consent if >18) years or older.
2. 2 - Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera
3. 3 - Phlebotomy requiring defined as ALL of the following: a. At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization, and b. Last phlebotomy due to inadequate hematocrit control within 3 months before randomization, and c. No phlebotomy within 6 days prior to randomization (do not include day of phlebotomy and day of randomization in the 6-day count). Note: Phlebotomies performed within an 8-day period will be counted as a single phlebotomy
4. 4 - CBC values immediately prior to randomization: a. Hematocrit <45%, b. WBC 4000/µL to 20,000/µL (inclusive) and c. Platelets 100,000/µL to 1,000,000/µL (inclusive).
5. 5 - Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen
6. 6 - Subjects treated with phlebotomy alone at randomization must have stopped cytoreductive therapy 2 to 6 months before screening.

Exclusion criteria 7

1. 1 - Clinically meaningful laboratory abnormalities at Screening
2. 2 - Subjects who require phlebotomy at hematocrit levels lower than 45%
3. 3 - Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.
4. 4 - Active or chronic bleeding within 2 months prior to randomization.
5. 5 - History of invasive malignancies within the last 5 years, except a) localized cured cancer (e.g. prostate cancer and cervical cancer). b) localized cured in situ or stage 1 squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or in situ melanoma of the skin.
6. 6 - Subjects with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during screening unless the cancer is adequately treated before randomization.
7. 7 - Received busulfan, pipobroman or 32Phosphorus within 7 months prior to screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of subjects achieving a response starting at Week 20 through Week 32 (inclusive) who receive rusfertide compared to placebo. A response is defined as absence of phlebotomy eligibility. Phlebotomy eligibility is defined as either: • a confirmed hematocrit ≥45% and that is at least 3% higher than the baseline hematocrit (value immediately prior to randomization at Week 0); or • a hematocrit ≥48%.

Secondary endpoints 5

1. 1 - Mean number of phlebotomies between Week 0 through Week 32 (inclusive).
2. 2 - Proportion of subjects with all hematocrit values <45% between Week 0 through Week 32 (inclusive).
3. 3 - Mean change from baseline at end of Part 1a (Week 32) in the Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 8a total T-score
4. 4 - Mean change from baseline at end of Part 1a (Week 32) in the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF)
5. 5 - Mean change from baseline at end of Part 1a (Week 32) in the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF)

Investigational products

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

Test 5

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Protagonist Therapeutics Inc.

Sponsor organisation

Protagonist Therapeutics Inc.

Address

7707 Gateway Boulevard

City

Newark

Postcode

94560-1160

Country

United States

Scientific contact point

Organisation

Protagonist Therapeutics Inc.

Contact name

Medpace Regulatory Submissions

Public contact point

Organisation

Protagonist Therapeutics Inc.

Contact name

Medpace Regulatory Submissions

Third parties 6

Locations

11 EU/EEA countries · 52 investigational sites

By country

Investigational sites

Country notifications

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Unexpected events 1 · Art. 53 CTR

Note: SUSARs are reported via EudraVigilance, not CTIS — events shown here are CTIS-public notifications only.

Results and documents

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

Documents 99 files

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

Application history

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