A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

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• Atypical Hemolytic Uremic Syndrome 42

Atypical Hemolytic Uremic Syndrome

42 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 12 are open to participants now.

This study's enrollment of 83 is above the median of 34 across 23 interventional studies indexed under Atypical Hemolytic Uremic Syndrome.

Lead sponsor

Hoffmann-La Roche is the lead sponsor of 2,061 studies on the registry; 85 are open to participants now.

Of its 319 completed or terminated interventional studies of FDA-regulated products, 239 (75%) have results posted.

Study arms

• Experimental
  Crovalimab

  Participants will be enrolled in three cohorts: \[1\] Naive Cohort - participants who have not been previously treated with complement inhibitor therapy; \[2\] Switch Cohort - participants who switch to crovalimab from another Complement Component 5 (C5) inhibitor and \[3\] C5 Single Nucleotide Polymorphism (C5 inhibitor) Cohort - participants with documented C5 polymorphism.

  Drug: Crovalimab

Interventions

• DrugCrovalimab

  Crovalimab will be administered at a dose of 1000 milligrams (mg) intravenous (IV) (for participants with body weight at least 40 (\>=) and up to 100 kilograms (kg) or 1500 mg IV (for participants with body weight \>=100kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg subcutaneously (SC). On Week 5 and every 4 weeks (Q4W) thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight \>= 40kg to \<100kg) or 1020 mg SC (for participants with body weight \>=100kg).

Primary outcomes

1. Percentage of Participants with Complete Thormbotic Microangiopathy Response (cTMAr)

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

Secondary outcomes

1. Change from Baseline in Dialysis Status

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

2. Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

3. Percentage of Participants with Change from Baseline in Chronic Kidney Disease (CKD) Stage

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

4. Observed Value in Platelet Count

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

5. Observed Value in Lactate Dehydrogenase (LDH)

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

6. Observed Value in Hemoglobin

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

7. Change from Baseline in Platelet Count

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

8. Change from Baseline in Lactate Dehydrogenase (LDH)

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

9. Change from Baseline in Hemoglobin

   Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

10. Mean Change From Baseline in Fatigue (in Adult Participants only)

    Assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Questionnaire. The FACIT-Fatigue (version 4) assesses self-reported fatigue and its impact upon daily activities and function. It consists of 13 items that assess fatigue using a 7-day recall period. Items are scored on a 0 (not at all) to 4 (very much) response scale. Relevant items are reverse scored and all items are summed to create total scores ranging from 0 \[worse score\] to 52 \[better score\].

    Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

11. Percentage of Participants with Platelet Count >= Lower Limits of Normal (LLN) (Naive Cohort only)

    Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

12. Percentage of Participants with Normalization of LDH (i.e. =< Upper Limit of Normal (ULN)) (Naive Cohort only)

    Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

13. Percentage of Participants with >=25% Decrease in Serum Creatinine (Naive Cohort only)

    Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

14. Time to cTMAr (Naive Cohort only)

    Time frame: Up to 8 years

15. Duration of cTMAr (Naive Cohort only)

    Time frame: Up to 8 years

16. Percentage of Participants with Ongoing cTMAr (Naive Cohort only)

    Time frame: At Week 25

17. Percentage of Participants with Maintained Thrombotic Microangiopathy Control (mTMAc) (Switch Cohort only)

    Time frame: Baseline up to Week 25 (after 24 weeks on treatment)

18. Percentage of Participants with Adverse Events (AEs)

    Time frame: Up to 8 years

19. Percentage of Participants with Injection-Site Reactions, Infusion-Related Reactions, Hypersensitivity, Malignant Hypertension (Including Malignant Renal Hypertension) and Infections (Including Meningococcal Meningitis)

    Time frame: Up to 8 years

20. Number of Participants with AEs Leading to Study Drug Discontinuation

    Time frame: Up to 8 years

21. Percentage of Participants with Clinical Manifestations of Drug-Target-Drug Complex (DTDC) Formation Amongst Those Participants who Switched to Crovalimab Treatment From Eculizumab Treatment or Ravulizumab Treatment

    Time frame: Up to Week 25

22. Serum Concentrations of Crovalimab Over Time

    Time frame: Up to 8 years

23. Prevalence of Anti-Crovalimab Antibodies at Baseline

    Time frame: Baseline

24. Percentage of Participants with Anti-Crovalimab Antibodies

    Time frame: Up to 8 years

25. Observed value of Pharmacodynamic Markers (CH50, Free/Total C5)

    Time frame: Up to 8 years

Individual participant data

Plan to share: Yes — For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing