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ALN-CC5

Phase 1

Paroxysmal Nocturnal Hemoglobinuria (PNH) | Small molecule | Hematology |Alnylam Pharmaceuticals, Inc.|Last Updated: Mar 30, 2020

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment62

FDA Designations

No designations recorded

Clinical trial landscape

ALN-CC5 · 1 trial · 1 indication

Phase 1 1
NCT02352493A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHParoxysmal Nocturnal Hemoglobinuria (PNH)
COMPLETED62 Analytics
PHASE1COMPLETED
A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
Paroxysmal Nocturnal Hemoglobinuria (PNH)Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Adverse Events
Part A: through day 658; Part B: through day 532; Part C: through day 280

Adverse events were reported for single-ascending doses (SAD) or multiple ascending doses (MAD) of ALN-CC5 when administered to healthy adult subjects and of multiple doses (MD) in patients with paroxysmal nocturnal hemoglobinuria (PNH)

Secondary Endpoints

Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP)
Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP)
Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels
Part A: through day 70; Part B: through day 140; Part C: through day 140
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALN-CC5ACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -

Interventions

NameTypeDescription
ALN-CC5DRUGSingle or multiple doses of ALN-CC5 by subcutaneous (sc) injection
Sterile Normal Saline (0.9% NaCl)DRUGcalculated volume to match active comparator
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Eligibility Criteria

Age Range18 Years to 45 Years
SexALL
Healthy VolunteersYes
Study Sites4

Inclusion Criteria: * Adequate complete blood counts, liver and renal function * 12-lead electrocardiogram (ECG) within normal limits * Female subjects of child bearing potential agreeing to use a protocol specified method of contraception * Male subjects agreeing to use protocol specified methods ...

Countries:SpainUnited Kingdom
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Frequently asked questions about ALN-CC5

What is ALN-CC5 used for?

ALN-CC5 is an investigational drug being studied for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who is developing ALN-CC5?

ALN-CC5 is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker ALNY. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria.

What phase is ALN-CC5 in?

ALN-CC5 is in Phase 1 clinical development. A Phase 1/2 study has been completed, involving healthy volunteers and patients with Paroxysmal Nocturnal Hemoglobinuria. The drug remains investigational and is not yet approved for any use.

What clinical trials is ALN-CC5 in?

ALN-CC5 has been studied in a completed Phase 1/2 trial with the identifier NCT02352493. This randomized, double-blind, placebo-controlled study enrolled 62 participants, including healthy volunteers and patients with Paroxysmal Nocturnal Hemoglobinuria, at sites in Spain and the United Kingdom.

Is ALN-CC5 the same as any other drug?

ALN-CC5 is the investigational name for this drug candidate. No alternative names have been disclosed in the clinical trial information. It is being studied specifically for Paroxysmal Nocturnal Hemoglobinuria and is not known to be marketed under any other name.