Trials Sponsors Conditions Drugs Pricing

Conditions

Sphingomyelin Lipidosis

Outcome Measures

Primary Outcomes (6)

Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline
Percent predicted Hb and Altitude-adjusted DLco was calculated as: 100\*Adjusted DLco/Predicted DLco in unit of mL CO/min/mmHg where, adjusted DLco = Observed DLco (in mL CO/min/mmHg) times Hemoglobin-adjusted factor times Altitude-adjustment factor.
Baseline (Day 1)
Percent Change From Baseline in Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Week 52
Percent predicted Hb and Altitude-adjusted DLco was calculated as: 100\*Adjusted DLco/Predicted DLco in unit of mL CO/min/mmHg where, adjusted DLco = Observed DLco (in mL CO/min/mmHg) times Hemoglobin-adjusted factor times Altitude-adjustment factor.
Baseline, Week 52
Combination Spleen Endpoint: Component 1: Spleen Volume (in MN) at Baseline
Spleen volume was assessed by abdominal magnetic resonance imaging (MRI) to quantitate the degree of splenomegaly in multiples of normal (MN).
Baseline (Day 1)
Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52
Spleen volume was assessed by abdominal MRI to quantitate the degree of splenomegaly in MN.
Baseline, Week 52
Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline
The SRS rates 5 items: abdominal pain, abdominal discomfort, early satiety, dissatisfaction with abdominal body image, and difficulty to bend down using a numerical rating scale of 0 (absent) to 10 (worst imaginable). The total score of SRS ranges from 0 to 50, with higher scores (50) indicated worst imaginable rating. It was pre-specified as secondary endpoint for countries outside of US.
Baseline (Day 1)
Combination Spleen Endpoint (Primary for US Only): Component 2: Change From Baseline in Splenomegaly-Related Score (SRS) at Week 52
The SRS rates 5 items: abdominal pain, abdominal discomfort, early satiety, dissatisfaction with abdominal body image, and difficulty to bend down using a numerical rating scale of 0 (absent) to 10 (worst imaginable). The total score of SRS ranges from 0 to 50, with higher scores (50) indicated worst imaginable rating. It was pre-specified as secondary endpoint for countries outside of US.
Baseline, Week 52

Secondary Outcomes (9)

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) up to Week 52
From the first infusion of investigational medicinal product (IMP) up to 52 weeks
Number of Participants With Adverse Events of Special Interest (AESIs) up to Week 52
From the first infusion of IMP up to 52 weeks
Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Liver Function Tests up to Week 52
From Baseline (Day 1) up to 52 weeks
Number of Participants Who Developed Anti-Drug Antibodies (ADA) to Olipudase Alfa up to Week 52
Baseline (Day 1) and Week 52
Percent Change From Baseline in Liver Volume (in MN) at Week 52
Baseline, Week 52
+4 more secondary outcomes

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo (saline) administered intravenously once every 2 weeks during the 52 weeks of the primary analysis period for patients randomized to placebo.

Drug: placebo (saline)

Olipudase alfa

EXPERIMENTAL

Olipudase alfa dose (3 mg/kg body weight) in saline administered intravenously once every 2 weeks during the 52 weeks of the primary analysis period for patients randomized to olipudase alfa, and during the extension treatment period for all patients.

Drug: Olipudase alfa

Interventions

placebo (saline)Drug

Pharmaceutical form: solution administered once every two weeks during the 52 weeks of the primary analysis period for participants randomized to placebo. Route of administration: intravenous infusion

Placebo

Olipudase alfaDrug

Pharmaceutical form: Powder for concentrate for solution for infusion administered once every two weeks during the 52 weeks of the primary analysis period for participants randomized to olipudase alfa, and during the extension treatment period for all participants. Route of administration: intravenous infusion

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersYes

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

The participant was willing and able to provide signed written informed consent.
The participant was male or female aged 18 years or older.

Sponsors & Collaborators

Genzyme, a Sanofi Company
Industrylead

Study Sites (24)

UCSF Medical Center Site Number : 840005

San Francisco, California, 94143, United States

Emory University Site Number : 840003

Decatur, Georgia, 30033, United States

Montefiore Medical Center Site Number : 840006

The Bronx, New York, 10467, United States

Investigational Site Number : 032001

Córdoba, X5004FHP, Argentina

Investigational Site Number : 036001

Westmead, New South Wales, 2145, Australia

Investigational Site Number : 056001

Leuven, 3000, Belgium

Hospital De Clinicas De Porto Alegre Site Number : 076001

Related Publications (2)

Mistry PK, Cassiman D, Jones SA, Lachmann R, Lukina E, Prada CE, Wasserstein MP, Thurberg BL, Foster MC, Patel RM, Underhill LH, Peterschmitt MJ. Acid sphingomyelinase deficiency and Gaucher disease: Underdiagnosed and often treatable causes of hepatomegaly, splenomegaly, and low HDL cholesterol in lean individuals. Hepatol Commun. 2025 Jan 7;9(1):e0621. doi: 10.1097/HC9.0000000000000621. eCollection 2025 Jan 1.
PMID: 39774103DERIVED
Wasserstein MP, Lachmann R, Hollak C, Barbato A, Gallagher RC, Giugliani R, Guelbert NB, Hennermann JB, Ikezoe T, Lidove O, Mabe P, Mengel E, Scarpa M, Senates E, Tchan M, Villarrubia J, Thurberg BL, Yarramaneni A, Armstrong NM, Kim Y, Kumar M. Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial. Orphanet J Rare Dis. 2023 Dec 2;18(1):378. doi: 10.1186/s13023-023-02983-0.
PMID: 38042851

MeSH Terms

Conditions

Niemann-Pick Disease, Type A

Interventions

Sodium Chlorideolipudase alfa

Condition Hierarchy (Ancestors)

Niemann-Pick DiseasesSphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHistiocytosis, Non-Langerhans-CellHistiocytosisLymphatic Diseases

Results Point of Contact

Title: Trial Transparency Team
Organization: Sanofi-Aventis Recherche & Développement

Study Officials

Clinical Sciences & Operations
Sanofi
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

36 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2 3
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR
Expanded Access: Yes

Study Record Dates

First Submitted

November 26, 2013

First Posted

December 9, 2013

Study Start

December 18, 2015

Actual (reported)

You may not qualify if:

The participant had received an investigational drug within 30 days before study enrollment.
The participant had a medical condition, including significant intercurrent illness; significant cardiac disease (e.g., clinically significant arrhythmia, moderate or severe pulmonary hypertension or clinically significant valve dysfunction, or less than or equal to (\<=)40% left ventricular ejection fraction by echocardiogram); active hepatitis B or hepatitis C, or infection with human immunodeficiency virus (HIV); malignancy diagnosed within the past 5 years (other than non-melanoma skin cancer), or any other serious medical condition that might have precluded participation in the study.
The participant had a platelet count less than (\<)60,000/microliters based on the average of 2 samples.
The participant had an international normalized ratio (INR) greater than (\>)1.5.
The participant had alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>250 IU/L or total bilirubin \>1.5 mg/dL (except for participant with Gilbert's syndrome).
The participant had a major organ transplant (eg, bone marrow or liver).
The participant was scheduled during the study for in-patient hospitalization including elective surgery and excluding the liver biopsies required per protocol.
The participant, in the opinion of the investigator, was unable to adhere to the requirements of the study.
The participant was unwilling or unable to abstain from the use of alcohol for 1 day before and 3 days after each study drug infusion. Testing for blood alcohol levels was not required.
The participant was unwilling or unable to avoid 10 days before and 3 days after the protocol scheduled liver biopsies the use of medications or herbal supplements that were potentially hepatotoxic (e.g., 3-hydroxy-3-methyl glutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, anti-depressants, kava, echinacea) and/or might have caused or prolonged bleeding (e.g., anti-coagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng).
The participant required medications that might have decreased olipudase alfa activity (e.g., fluoxetine, chlorpromazine, tricyclic antidepressants \[e.g., imipramine, or desipramine\]).
The participant required use of invasive ventilatory support.
The participant required use of noninvasive ventilator support while awake for longer than 12 hours daily.
The participant was breast-feeding.
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Results Posted

Study Completion

Conditions

Outcome Measures

Primary Outcomes (6)

Secondary Outcomes (9)

Study Arms (2)

Placebo

Olipudase alfa

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (24)

Related Publications (2)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Intervention Hierarchy (Ancestors)

Data Sharing

Source record

Documents

Locations