The Holistic Study

NCT07051915 | Not Yet Recruiting Phase 3 DMC

• 1 other identifier: RGCH006
• Study Type: interventional
• Target: 220
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study is a phase 3 study where eligible patients will be randomized 1:1 to one of two treatment strategies: receiving a thrombopoietin receptor agonist (Avatrombopag), or anti-CD20 (Rituximab).

Conditions

Thrombocytopenia Purpura

Keywords

Avatrombopag; Rituximab; Thrombocytopenia; Immune thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• Efficacy of the oral TPO-RA, Avatrombopag, to Rituximab

  To compare the efficacy of the oral TPO-RA, Avatrombopag to Rituximab by measuring the rates of durable responses defined as achieving platelet counts ≥ 50 X109/L in ≥3 of the bi-weekly measurements between weeks 20 and 28 including the last count without having received any other platelet elevating agents after randomization apart from rescue therapy received before end of week 10.

  Assessed at week 28

Secondary Outcomes (3)

• The changes in the disease specific HRQoL

  Baseline to weeks 28 and weeks 78

• Changes in the level of fatigue

  Baseline to weeks 28 and weeks 78

• Rates of Sustained Response Off-Treatment (SROT)

  At 78 weeks

Study Arms (2)

Avatrombopag

EXPERIMENTAL

Open label, oral Avatrombopag 20 mg tablets taken daily during the first week. Dose tapering period commencing from week 28 for up to 8 weeks.

Drug: Avatrombopag; Drug: Rituximab (Arm B)

Rituximab

ACTIVE COMPARATOR

Open label, intravenous infusions of 1000mg Rituximab 2 weeks apart

Drug: Avatrombopag; Drug: Rituximab (Arm B)

Interventions

Avatrombopag DRUG

Daily tablets

Avatrombopag; Rituximab

Rituximab (Arm B) DRUG

I.V.

Avatrombopag; Rituximab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female aged ≥18 years.
• Clinical need for subsequent platelet elevating therapy assessed by the physician in charge.
• Signed and dated written informed consent.

You may not qualify if:

• Previous treatment for ITP with: Rituximab, other immune suppressants (including mycophenolate mofetil, azathioprine, cyclosporine), dapsone, danazol, chemotherapy (apart from vincristine as rescue therapy) or splenectomy. Short treatment with any thrombopoietic agent is allowed if given for a limited duration of a maximum of 2 weeks as rescue therapy for quick elevation of platelet count in emergency situations e.g. bleeding.
• Pregnancy or lactation.
• Females of child-bearing potential refusing to follow effective contraceptive methods for at least 12 months following the last administration of Rituximab or during treatment with Avatrombopag.
• Secondary ITP: ITP secondary to lymphoma or chronic lymphocytic leukemia; ITP secondary to the following autoimmune disorders: Systemic Lupus Erythematosus, Antiphospholipid Syndrome, or Common Variable Immune Deficiency; ITP secondary the following viral infections: Human Immunodeficiency Virus or Hepatitis C Virus.
• Concomitant autoimmune hemolytic anemia.
• Active hepatitis B virus (positive HBsAg). Patients with HBsAg negative and HBV core antigen antibody positive (HBcAb) should accept to receive entecavir (Baraclude) for 12 months if they will be allocated to Rituximab. Monthly HBV DNA monitoring will be required while on treatment and for the 6 months after the last dose of the study drug.
• Presence of any serious comorbidity where the condition may worsen by and of the study drugs.
• Known allergy, sensitivity or contraindication to Rituximab or Avatrombopag.
• Patients in a severely immune compromised state.
• 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).

Sponsors & Collaborators

• Ostfold Hospital Trust: lead
• Oslo University Hospital: collaborator
• University Hospital, Akershus: collaborator
• Sykehuset i Vestfold HF: collaborator
• St. Olavs Hospital: collaborator
• Helse Stavanger HF: collaborator
• Sorlandet Hospital HF: collaborator
• University Hospital of North Norway: collaborator
• Haukeland University Hospital: collaborator
• Helse Nord-Trøndelag HF: collaborator
• Helse Fonna: collaborator
• Nordlandssykehuset HF: collaborator

MeSH Terms

Conditions

Purpura, Thrombocytopenic; Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag; Rituximab

Condition Hierarchy (Ancestors)

Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Blood Platelet Disorders; Cytopenia; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Hemorrhagic Disorders; Autoimmune Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2025
• First Posted: July 4, 2025
• Study Start: September 15, 2025
• Primary Completion (Estimated): January 31, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: September 9, 2025

Record last verified: 2025-09