A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Conditions

• Sickle Cell Disease

Eligibility Criteria

Sex

ALL

Age

16 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Mitapivat — DRUG
  Mitapivat tablets
• Mitapivat-matching placebo — OTHER
  Placebo to match 50 mg or 100 mg tablets
• Mitapivat-matching placebo — OTHER
  Placebo to match 100 mg tablets

Study Details

This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.

Key Dates

Start date

Feb 11, 2022

Status verified

May 2026

Primary completion

Oct 30, 2025

Completion

Feb 28, 2030

Study Design

Enrollment

286 participants (actual)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Phase 2: Mitapivat 50 mg BID
  Double-blind Period: Mitapivat 50 milligrams (mg) twice daily (BID) for 12 weeks.
• Experimental: Phase 2: Mitapivat 100 mg BID
  Double-blind Period: Mitapivat 100 mg BID for 12 weeks.
• Placebo Comparator: Phase 2: Placebo
  Double-blind Period: Mitapivat-matching placebo for 12 weeks.
• Experimental: Phase 2: Open-Label Extension Period
  Participants who received mitapivat 50mg BID in the double-blind period may choose to receive mitapivat 50mg BID for 216 weeks after. Participants who received mitapivat 100mg BID in the double-blind period may choose to receive mitapivat 100 mg BID for 216 weeks after. Participants who received mitapivat-matching placebo in the double-blind period, may be randomized to receive either mitapivat 50 mg or 100 mg BID for 216 weeks after.
• Experimental: Phase 3: Mitapivat 100 mg BID
  Double-blind Period: Mitapivat 100 mg BID for 52 weeks.
• Placebo Comparator: Phase 3: Placebo
  Double-blind Period: Mitapivat-matching placebo for 52 weeks.
• Experimental: Phase 3: Open-Label Extension Period
  Participants may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period. Participants who received mitapivat-matching placebo in the double-blind period, may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period.

Primary Outcome Measure

Phase 2: Percentage of Participants With Hemoglobin (Hb) Response [ Time Frame: Week 12 ]

Locations (28)

Find similar trials in La Jolla, CA

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