Phase 3 Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in Achondroplasia

Browse trials for

• Achondroplasia 47

Achondroplasia

47 studies on the registry are indexed under Achondroplasia; 13 are open to participants now.

This study's planned enrollment of 144 is above the median of 74 across 28 interventional studies indexed under Achondroplasia.

Lead sponsor

Ascendis Pharma A/S is the lead sponsor of 17 studies on the registry; 7 are open to participants now.

Study arms

• Experimental
  Once weekly navepegritide SC and lonapegsomatropin SC

  Drug: Dual therapy with navepegritide and lonapegsomatropin

• Experimental
  Once weekly navepegritide SC and placebo for lonapegsomatropin SC

  Drug: Dual therapy with navepegritide and placebo for lonapegsomatropin

• Experimental
  Once weekly lonapegsomatropin SC and placebo for navepegritide SC

  Drug: Dual therapy with lonapegsomatropin and placebo for navepegritide

Interventions

• DrugDual therapy with navepegritide and lonapegsomatropin

  Administered weekly as two separate SC injections. Treatment duration of up to 104 weeks

• DrugDual therapy with navepegritide and placebo for lonapegsomatropin

  Administered as two separate SC injections. Treatment duration of up to 52 weeks of navepegritide and placebo for lonapegsomatropin followed by 52 weeks of navepegritide and lonapegsomatropin.

• DrugDual therapy with lonapegsomatropin and placebo for navepegritide

  Administered as two separate SC injections. Treatment duration of up to 52 weeks of lonapegsomatropin and placebo for navepegritide followed by 52 weeks of navepegritide and lonapegsomatropin.

Primary outcomes

1. Annualized Growth Velocity (AGV)

   To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to navepegritide alone

   Time frame: 52 weeks

Secondary outcomes

1. Annualized Growth Velocity (AGV)

   To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to lonapegsomatroptin alone

   Time frame: 52 weeks

2. Annualized Growth Velocity (AGV) change from baseline

   To evaluate the change from baseline in AGV, cm per year

   Time frame: 52 weeks

3. ACH-specific height Z-score

   To evaluate the change from baseline in ACH-specific height Z-score

   Time frame: 52 weeks

4. CDC-based height Z-score

   To evaluate the change from baseline in CDC-based height Z-score

   Time frame: 52 weeks

5. Treatment-emergent Adverse Events (AE)

   Number of events

   Time frame: Throughout trial duration 104 weeks

6. Bone safety

   Qualitative assessment with radiology

   Time frame: Throughout trial duration 104 weeks

7. Arm span Z-score

   Change from baseline in arm span Z-score.

   Time frame: 52 weeks

8. Upper to lower body segment ratio

   Change from baseline in upper to lower body segment ratio.

   Time frame: 52 weeks

9. Arm span

   Change from baseline in arm span, cm.

   Time frame: 52 weeks

10. Interpedicular distance (IDP)

    Change from baseline in IDP (L1-L4 average), mm

    Time frame: 52 weeks