AnemiaPhase 2SATISFY-CANCT07055243
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
A Phase 2 study testing Mitapivat in people with Anemia, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anemia
- What is being tested?
- Mitapivat
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteCanada
- Study dates
- 20 Oct 2025to Oct 2029
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female with RBC membranopathy or congenital dyserythropoietic anemia type II (CDAII). Diagnosis must be supported genetically by a ACMG class 3 (VUS), 4 or 5 variant.
- Age ≥18 years.
- Average hemoglobin (Hb) concentration (average of at least 2 Hb measurements separated by a minimum of 7 days the during screening period) must be less than 13.0 g/dL for males and 11.0 g/dL for females. Patients with average Hb >10.0 g/dL for males and females must meet at least one of the following additional criteria:
- Splenomegaly (length ≥12.5 cm)
- Fatigue attributed to hemolysis
- Active hemolysis as evaluated by one or more of the following: haptoglobin, bilirubin, LDH, reticulocytes
Show all 14Show fewer
- Subjects must start or continue taking at least the equivalent of daily 0.8 mg oral folic acid for the duration of the study.
- Have adequate organ function, as defined by:
- Serum aspartate aminotransferase (AST) ≤2.5 × ULN (unless the increased AST is assessed by the Investigator as due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase (ALT) ≤2.5 × ULN.
- Normal or elevated levels of serum bilirubin. In subjects with serum bilirubin > ULN, the elevation must be attributed to hemolysis with or without Gilbert's syndrome and must not be associated with choledocholithiasis, cholecystitis, biliary obstruction, or hepatocellular disease Elevated bilirubin attributed to hemolysis with or without Gilbert's syndrome is not exclusionary.
- Estimated glomerular filtration rate ≥45 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration creatinine
- Be willing and able to give written informed consent and to comply to all study procedures for the duration of the study.
- For women of reproductive potential, have a negative urine or serum pregnancy test during the Screening Period (Day -50 to Day -1). Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy or tubal occlusion; or who have not been naturally postmenopausal (i.e. who have not menstruated at all for at least the preceding 12 months prior to signing informed consent), or has a known diagnosis of hypogonadotropic hypogonadism.
- For women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use a highly effective method of contraception. A highly effective form of contraception is defined as combined (estrogen and progestin containing) hormonal contraceptives (oral, intravaginal, or transdermal) associated with inhibition of ovulation; progestin-only hormonal contraceptives (oral, injectable, or implantable) associated with inhibition of ovulation; intrauterine device; intrauterine hormone releasing system; bilateral tube occlusion; or vasectomized partner. Women of reproductive potential using hormonal contraception as a highly effective form of contraception must also utilize an acceptable barrier method while enrolled in the study and for at least 28 days after their last dose of study drug. An acceptable barrier method includes male or female condoms with or without spermicide, and cervical cap, diaphragm, or sponge with spermicide.
Who cannot join
- Known history of pyruvate kinase deficiency (decreased PK activity or two pathogenic PKLR alleles). PK activity and PKLR testing is not required.
- Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic or preventive transfusion), defined as more than 5 transfusion episodes in the 12-month period up to the first day of study treatment, and/or have received a transfusion within the past 3 months prior to the first day of study treatment.
- Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound interpretation of the study data. Such significant medical conditions include, but are not limited to:
- Poorly controlled hypertension (defined as systolic blood pressure >150 mm Hg or diastolic blood pressure >90 mm Hg) refractory to medical management.
- Any history of congestive heart failure; myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; or recent (< 6 months prior to signing informed consent) deep venous thrombosis; or pulmonary or arterial embolism.
- Cardiac dysrhythmias judged as clinically significant by the Investigator.
Show all 29Show fewer
- Clinically symptomatic cholelithiasis or cholecystitis. Prior cholecystectomy is not exclusionary. Subjects with symptomatic cholelithiasis or cholecystitis may be rescreened once the disorder has been treated and clinical symptoms have resolved.
- History of drug-induced cholestatic hepatitis.
- Severe iron overload as evaluated by the Investigator. This includes cardiac (eg, clinically significant impaired left ventricular ejection fraction) or hepatic (eg, fibrosis, cirrhosis) dysfunction.
- Have a diagnosis of any other congenital or acquired blood disorder or any other hemolytic process, except mild allo-immunization, as a consequence of transfusion therapy.
- Positive test for HBsAg or HCVAb with signs of active hepatitis B or C virus infection. Subjects with hepatitis C may be rescreened after receiving appropriate hepatitis C treatment.
- Positive test for HIV-1 or -2 antibodies. j. Active infection requiring the use of parenteral antimicrobial agents or Grade ≥3 in severity (per NCI CTCAE) within 2 months prior to the first dose of study treatment.
- Diabetes mellitus judged to be under poor control by the Investigator or requiring >3 antidiabetic agents, including insulin (all insulins are considered 1 agent); use of insulin per se is not exclusionary.
- History of any primary malignancy, with the exception of: curatively treated non-melanomatous skin cancer; curatively treated cervical or breast carcinoma in situ; or other primary tumor treated with curative intent, no known active disease present, and no treatment administered during the last 3 years.
- Unstable extramedullary hematopoiesis that could pose a risk of imminent neurologic compromise.
- Severe hepatic issues such as liver fibrosis (F3 or worse), significant cirrhosis or non-alcoholic fatty liver disease (NASH).
- Current or recent history of psychiatric disorder that, in the opinion of the Investigator, could compromise the ability of the subject to cooperate with study visits and procedures.
- Alcohol use disorder.
- 4. Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo. Participation in registry studies is allowed.
- 5. Have exposure to any investigational drug, device, or procedure within 5 half-lives or 3 months (whichever is longer) to the first dose of study treatment.
- 6. Have had any prior treatment with a pyruvate kinase activator.
- 7. Have a prior bone marrow or stem cell transplant.
- 8. Are currently pregnant or breastfeeding or planning to become pregnant during the course of the study.
- 9. Have a history of major surgery within 6 months prior to signing informed consent. Note that procedures such as laparoscopic gallbladder surgery are not considered major in this context.
- 10. Are currently receiving medications that are strong inhibitors of CYP3A4 and strong inducers of CYP3A4 that have not been stopped for a duration of at least 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) prior to the first dose of study treatment.
- 11. Are currently receiving hematopoietic stimulating agents (eg, erythropoietins, granulocyte colony stimulating factors, thrombopoietins) that have not been stopped for a duration of at least 28 days prior to the first dose of study treatment.
- 12. Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, and mannitol) or history of acute allergic reaction to drugs characterized by acute hemolytic anemia, drug-induced liver injury, anaphylaxis, rash of erythema multiforme type or Stevens-Johnson syndrome, cholestatic hepatitis, or other serious clinical manifestations.
- 13. For men and women of reproductive potential: unwillingness to be abstinent or use double anticonception during the trial period.
- 14. Are currently receiving herbal or dietary supplements that have not been stable in dose and preparation for >8 weeks prior to randomization.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies - Canada (SATISFY-CA)
This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study. Patients will be in the trial for 57 weeks of treatment and a safety follow-up visit after 30 days from last dose. First 8 weeks will be dose escalating period followed by 48 weeks of fixed dose period. 57th week will be dose tapering week, unless patient is deemed eligible for the fixed dose extension periods, in which case patient will continue on treatment for additional 24 months in total. Dose taper period will then occur on week 161 and safety follow-up will occur 4 weeks after last dose. Data collected from Princess Margaret will be incorporated to the main study conducted in EU for analysis. Overall, approximately 25 patients are expected to be enrolled: Approximately 16 patients at sites in the EU and approximately 9 patients in Canada.
Read the detailed description
This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII. Global target is 25 patients. 9 patients from UHN, Canada will be enrolled to the study. All enrolled patients will receive the study drug Mitapivat. All eligible subjects who enter the Dose Escalation Period will receive an initial dose of 50 mg mitapivat BID. After 4 weeks dose will be increased from 50 mg BID to 100 mg BID unless dose limiting side effects have occurred or maximum allowed Hb levels have been reached. The maximum dosage is 100 mg BID, for patients in which no dosage limiting side effects have occurred and maximum allowed hemoglobin levels have not been reached.
Maximal study duration per subject is screening period (≤50 days) + 57 weeks on study drug + two 12 months fixed dose extension periods:
- 8 weeks Dose Escalation Period
- 24 weeks Fixed Dose Period 1
- 24 weeks Fixed Dose Period 2
- 12 months Fixed Dose Extension Period 1
- 12 months Fixed Dose Extension Period 2
- 1 week Dose Taper Dose reduction is allowed at any time during both the Dose Escalation Period and the Fixed Dose Extension Period. In cases, where 50 mg BID provides dose limiting side effect(s), subjects can be offered dose reduction to 20 mg BID. If 20 mg BID provides dose limiting side effect(s), subjects can be further dose reduced to 5 mg BID. Subjects receiving dosage of <50mg BID are allowed to continue in the study.
Physical exam, ECG, vital signs, DEXA scan, MRI scan, safety blood work will be carried out thorough out the study. Patients will be asked to complete 2 questionnaires in a online web browser during the study visits at the clinic.
- Conditions
- Anemia
- Being tested
- Mitapivat
- Medical subject headings
- Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MitapivatDrug
Mitapivat will be given to adult patients with erythrocyte membranopathies
| Study group | Role | What they receive |
|---|---|---|
| Mitapivat | Experimental | All patients will receive the drug Mitapivat Receives: Drug: Mitapivat |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate safety of mitapivat
Type, incidence, severity and relationship of mitapivat to AE and SAE
Measured: 61 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2025
- Study start20 Oct 2025
- Primary completionOct 2029 (estimated)
- Study completionOct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AnemiaStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Agios Pharmaceuticals, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07055243
- Sponsor's protocol ID
- OZUHN-025
- Acronym
- SATISFY-CA
- Status last verified
- Oct 2026
- First submitted
- 17 Apr 2025
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided