Exploring the Efficacy, Safety of a Modified Starting Dosage of Avatrombopag in Immune Throbocytopenia (ITP) - a Pilot Study

NCT ID: NCT07133659

Last Updated: 2025-08-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-08

Study Completion Date

2027-07-08

Brief Summary

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This is a single-center, open label pilot trial where patients with primary ITP who require second line treatment will be offered avatrombopag at a reduced starting dose, adjusted thereafter according to the response and continued for up to 24 weeks. The study aims to acquire experience on use of avatrombopag and explore the efficacy and safety of lower starting dose of avatrombopag

Detailed Description

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Avatrombopag is an oral thrombopoietin receptor agonist that is licensed for chronic ITP. Avatrombopag is administered at a starting dose of 20 mg daily. Overshooting of platelet count is a frequent problem that occurs in 20 to 40% of the patients after initiating avatrombopag as recommended. In this open label, single arm, pilot study, we will start avatrombopag at a reduced starting dose of 20 mg every other day. The dose will be adjusted thereafter according to the platelet response. The study consists of 3 phases: Dose adjustment phase, a maintenance phase, and dose tapering/disconsolation and follow-up phase. The study aims to acquire experience on use of avatrombopag and explore the efficacy and safety of lower starting avatrombopag-dose, and assess the rate of sustained response off-treatment. The duration of treatment with avatrombopag is 6 months.

The study is an investigator-initiated trial sponsored by Center for Transplantation and Blood Diseases. Medical City Complex, Baghdad Iraq.

Conditions

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ITP - Immune Thrombocytopenia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Avatrombopag

Description: Patients will start on avatrombopag 20 mg every other day for a week then the dose will be readjusted according to the platelet count.

Group Type EXPERIMENTAL

Avatrombopag 20 mg Oral Tablet

Intervention Type DRUG

Description: Patients will start on avatrombopag 20 mg every other day for a week then the dose will be readjusted according to the platelet count.

Interventions

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Avatrombopag 20 mg Oral Tablet

Description: Patients will start on avatrombopag 20 mg every other day for a week then the dose will be readjusted according to the platelet count.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Male or female aged ≥18 years.
2. Diagnosis of primary ITP and having a platelet count of \< 30 x109/L measured within two weeks prior to inclusion with failure to achieve response or relapse after at least one cycle of dexamethasone (20-40 mg daily for 4 days) or prednisone /prednisolone (1 mg/kg for at least two weeks). Shorter courses or lower doses are allowed if discontinued or modified due to side effects.
3. Clinical need for second (subsequent) line treatment with a platelet elevating therapy assessed by the physician in charge.
4. Signed and dated written informed consent.

Exclusion Criteria

1. Previous treatment with TPO-RA.
2. Pregnancy or lactation.
3. Patients with active serious bleeding or at high risk of bleeding as judged by physician in charge.
4. Females of child-bearing potential refusing to follow effective contraceptive methods (as described in SmPC) during treatment with Avatrombopag.
5. Secondary ITP defined as ITP secondary to lymphoma or chronic lymphocytic leukemia; ITP secondary to the following autoimmune disorders Systemic Lupus Erythematosus or Antiphospholipid Syndrome; ITP secondary to Common Variable Immune Deficiency; ITP secondary to the following viral infections eg Human Immunodeficiency Virus.
6. Concomitant autoimmune hemolytic anemia, Evans syndrome.
7. Presence of any serious comorbidity where the condition may worsen the study drugs.
8. Presence of active malignancy unless deemed cured by adequate treatment. Participants with the following neoplastic conditions can be included:

* Monoclonal gammopathy of undetermined significance (MGUS) or monoclonal B lymphocytosis of undetermined significance (MBUS)
* Basal/squamous cell carcinoma of the skin
* Carcinoma in situ of the cervix
* Carcinoma in situ of the breast
* Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
9. Patients with history of poor compliance or history of alcohol/drug abuse or excessive alcohol beverage consumption that would interfere with the ability to comply with the study protocol, or current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent.

\-
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Al-Mustansiriyah University

OTHER

Sponsor Role lead

Responsible Party

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Alaa Fadhil Alwan

consultant hematologist

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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hematology center / Medical City

Baghdad, , Iraq

Site Status RECRUITING

Countries

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Iraq

Central Contacts

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Alaa Alwan, MD

Role: CONTACT

770 274 3114 ext. 00964

Facility Contacts

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Alaa Alwan, MD

Role: primary

0770 274 3114 ext. 00964

References

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Guillet S, Crickx E, Azzaoui I, Chappert P, Boutin E, Viallard JF, Riviere E, Gobert D, Galicier L, Malphettes M, Cheze S, Lefrere F, Audia S, Bonnotte B, Lambotte O, Noel N, Fain O, Moulis G, Hamidou M, Gerfaud-Valentin M, Marolleau JP, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Robbins A, Lega JC, Puyade M, Comont T, Limal N, Languille L, Zarrour A, Luka M, Menager M, Belmondo T, Hue S, Canoui-Poitrine F, Michel M, Godeau B, Mahevas M. Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study. Blood. 2023 Jun 8;141(23):2867-2877. doi: 10.1182/blood.2022018665.

Reference Type BACKGROUND
PMID: 36893453 (View on PubMed)

Pascual-Izquierdo C, Sanchez-Gonzalez B, Canaro-Hirnyk MI, Garcia-Donas G, Menor-Gomez M, Gil-Fernandez JJ, Monsalvo-Saornil S, de-Laiglesia A, Alvarez-Roman MT, Jarque-Ramos I, Llacer MJ, Pedrote-Amador B, Zafra-Torres D, Caparros-Miranda I, Ortuzar-Pasalodos A, Revilla-Calvo N, Bastida JM, Chica-Gullon E, Alvarellos M, Jimenez-Barcenas R, Bernat S, Martinez-Carballeira D, Lakhwani S, Lopez-Ansoar E, Moreno-Beltran ME, Lorenzo-Vizcaya A, Aguirre MA, Lasa-Eguialde M, Canet M, Gonzalez-Gascon-Y-Marin IT, Caballero-Navarro G, Cuesta A, Diaz-Lopez M, Arquero T, Moreno-Carbonell M, Mingot-Castellano ME; Spanish ITP Group (GEPTI) of the Spanish Society of Hematology and Hemotherapy (SEHH). Avatrombopag in immune thrombocytopenia: A real-world study of the Spanish ITP Group (GEPTI). Am J Hematol. 2024 Dec;99(12):2328-2339. doi: 10.1002/ajh.27498. Epub 2024 Oct 12.

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Reference Type BACKGROUND
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Reference Type BACKGROUND
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PMID: 37700877 (View on PubMed)

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Reference Type BACKGROUND
PMID: 30191972 (View on PubMed)

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Reference Type BACKGROUND
PMID: 36787705 (View on PubMed)

Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966.

Reference Type BACKGROUND
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Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, Gonzalez-Lopez TJ, Grainger J, Hou M, Kruse C, McDonald V, Michel M, Newland AC, Pavord S, Rodeghiero F, Scully M, Tomiyama Y, Wong RS, Zaja F, Kuter DJ. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817. doi: 10.1182/bloodadvances.2019000812.

Reference Type BACKGROUND
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Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.

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Reference Type BACKGROUND
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Other Identifiers

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RGHC007

Identifier Type: -

Identifier Source: org_study_id

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