Primary - To evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.Secondary - To further evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.
ID
Source
Brief title
Condition
- Autoimmune disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
• Proportion of participants attaining a durable hemoglobin response, defined
as hemoglobin >= 10 g/dL with an increase from baseline of >= 2 g/dL not
attributed to rescue therapy at >= 3 of the 4 available visits at Week 12 and/or
later during the 24-week double-blind treatment period
Secondary outcome
• Proportion of participants with a >= 3-point increase from baseline in FACIT-F
score at Week 24.
Background summary
Autoimmune hemolytic anemia is a rare acquired disorder in which autoantibodies
directed against RBC membrane antigens lead to their accelerated destruction.
Currently, there is no approved and effective targeted therapy for treatment of
primary wAIHA. Despite current treatment algorithms, there is a significant
morbidity and mortality rate, and disease relapse remains an ongoing challenge
for a significant number of patients with wAIHA. Corticosteroids remain the
first-line therapy; however, high initial doses are required, responses are
often achieved slowly, and only a minority of patients achieve a lasting
response.Therefore, there remains an unmet need for new treatments for wAIHA.
In addition to showing
efficacy in a number of B-cell-related cancers, treatment with parsaclisib has
shown significant improvement in animal models of AIHA and lupus nephritis, as
well as other antibody-mediated diseases. Parsaclisib may represent an
alternative treatment for participants who have failed at
least 1 prior treatment
Study objective
Primary - To evaluate the efficacy of parsaclisib in the treatment of
participants with wAIHA.
Secondary - To further evaluate the efficacy of parsaclisib in the treatment of
participants with wAIHA.
Study design
Randomized, double-blind, placebo-controlled, multicenter study
Intervention
Participants will receive parsaclisib 2.5 mg or placebo QD for 24 weeks.
Participants who complete the double-blind treatment period, are tolerating
study treatment, and in the investigator's opinion will benefit from continued
treatment will continue into an open-label extension period for an additional
24 weeks of parsaclisib 2.5 mg QD. A 12-week post-treatment follow-up period
will include 3 visits every 4 weeks to assess safety and
persistence of effect. Participants who benefit from the treatment can
participate in the long-term extension study.
Study burden and risks
Adverse events
- Potential adverse events in relation to the study treatment (adverse events
are listed in the ICF attachment D)
-Mogelijke bijwerkingen van de behandeling (bijwerkingen worden beschreven in
Bijlage D van het ICF)
Procedures
-Blood draws/blood tests: Momentary discomfort, soreness, bruising, and in rare
cases, infection at the draw site or excess bleeding; rarely light headedness,
or fainting. Approximate total amount of blood drawn of the course of the
study: 343 mL, average amount for blood donation is 480 mL.
-Electrocardiogram (ECG): Rash or minor irritation of the skin may occur from
the sticky pads used.
-Six-minute walk test: get out of breath or become exhausted. However, the
participant may slow down, stop, or rest during the test if you need to.
The potential risks to study participants in association with parsaclisib
treatment in the study are mitigated by the planned safety measures detailed in
the protocol (Table 5) and are balanced by the potential improvement in
hemoglobin in a population who have failed at least 1 prior therapy, have
limited treatment options and supported by positive benefit/risk seen with
parsaclisib 2.5 mg dose in Study INCB 50465-206.
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Wilmington 19803
US
Augustine Cut-Off 1891 1891
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Listed location countries
Age
Inclusion criteria
1. Ability to comprehend and willingness to sign a written ICF for the study.
2. Men or women, age >= 18 years at the time of signing the ICF.
Note: For Japan, men and women, age >= 20 years or older.
3. Diagnosis of primary wAIHA based on the presence of hemolytic anemia and
serological evidence of anti-erythrocyte antibodies, detectable by a DAT
positive for IgG only or IgG plus C3d.
Note: Prior documentation of DAT testing is permitted.
4. Participants who were inadequately controlled with, were intolerant to, or
have a contraindication to other therapies. There is no limit to the number of
prior treatment regimens.
5. Hemoglobin >= 6.5 to < 10 g/dL with symptoms of anemia as assessed by the
investigator at screening (hemoglobin as determined by local laboratory).
6. FACIT-F score <= 43 at screening.
Exclusion criteria
1. Women currently pregnant or breastfeeding or participants expecting to
conceive or father children within the projected duration of the study,
starting with the screening visit through 90 days from the date of last dose of
study drug.
2. A diagnosis of other types of AIHA; CAD, cold agglutinin syndrome,
mixed-type AIHA or paroxysmal cold hemoglobinuria.
3. Warm AIHA suspected to be secondary to a lymphoproliferative malignancy or
secondary to an autoimmune disease (eg, systemic lupus erythematosus,
Castleman's disease, Sjögren's syndrome, or other autoimmune diseases) or
diagnosis of Evans syndrome.
4. A splenectomy less than 3 months before randomization
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2021-002844-66-NL |
| CCMO | NL78438.078.21 |